FDA Approvals: Zepatier®, Halaven®

FDA Brief: Week of Jan 25, 2016

FDA approved


merck 1.JPGmerck 2.JPG

ZEPATIER, (fixed dose combination product of elbasvi and grazoprevir) tablets

Merck , Whitehouse Station, New jersey, USA

Indication : With or without ribavirin for treatment of chronic HCV genotypes 1 or 4 infection in adults

Unmet Need :

  • Hepatitis C can lead to diminished liver function or liver failure
  • Most infected people asymptomatic until liver damage becomes apparent
  • 3 million Americans are infected with HCV, of which genotype 1 is the most common and genotype 4 is one of the least common
  • The approval provides another oral treatment option for patients with genotypes 1 and 4 HCV infections without requiring use of interferon.

Reg Pathway : Breakthrough Therapy designation, Priority Review

Mechanism of Action: Direct-acting antiviral agents against the hepatitis C virus


  • Six studies(n=1,373), open label and double-blind, with or without ribavirin, with or without cirrhosis, 12-16 weeks
  • Sustained virologic response (SVR) 12 weeks post-treatment : 94-97 % (genotype-1) and 97-100 % (genotype-4)
  • To maximize SVR rates :  Label provides recommendations regarding length of treatment with or without ribavirin specifically tailored to the characteristics of the patient and their virus


  • Warning : Elevations of liver enzymes
  • Most common side effects: Fatigue, headache, nausea. With ribavirin, anemia and headache.



HALAVEN  ( eribulin mesylate ) injection

Eisai, Woodcliff Lake, New Jersey, USA


Indication : Unresectable or metastatic liposarcoma who have received a prior anthracycline-containing regimen.

Unmet Need:

  • Liposarcoma is a  Soft Tissue Sarcoma(STS) in fat cells
  • STS most common in the head, neck, arms, legs, trunk and abdomen
  •  12,000 cases of STS diagnosed in US
  • Need for therapy with survival benefit

Reg Pathway : sNDA, Orphan Drug designation, Priority Review

Mechanism of Action : Inhibits growth phase of leading to G2/M cell-cycle block, disruption of mitotic spindles, and, ultimately, apoptotic cell death after prolonged mitotic blockage.


  • Single open-label, randomized, active-controlled trial (n=446.), patients with unresectable, locally advanced or metastatic liposarcoma or leiomyosarcoma, at least two prior systemic chemotherapies (containing anthracyclis) , Halaven vs dacarbazine
  • Overall Survival : Statistically significant improvement (p=0.011)
  • Progression-free survival: No significant difference in overall population
  • No evidence of with advanced or metastatic leiomyosarcoma


Safety :

  • Most common Serious Adverse Reactions: Neutropenia, Pyrexia
  • Most frequent Adverse Reactions leading to Discontinuation: Fatigue, thrombocytopenia.
  • Most common Adverse Reactions: Fatigue, nausea, alopecia, constipation, peripheral neuropathy, abdominal pain, and pyrexia

FDA Voice on Clinical Trial Diversity

FDA Brief: Week of Jan 25, 2016

FDA Voice

2016: The Year of Diversity in Clinical Trials

By: Robert M. Califf, M.D., FDA’s Deputy Commissioner for Medical Products and Tobacco

  • Increasing diversity in clinical trials is a priority for FDA
  • Research participants need to be representative of patients who will use product
  • FDASIA Section 907 :
    • evaluate underrepresented groups e.g. elderly, women, racial/ethnic
  • FDA Activities:
    • Drug Trial Snapsots  publication on clinical trial demographic information
    • Office of Minority Health tools for clinical trial participation
    • Office of Women’s Health’s Diverse Women in Clinical Trials initiative
    • Better estimates of treatment effects for diverse populations
    • Obtain patient’s perspective
    • Office of External Affairs plans to publish a consumer update



Women’s Health Research Roadmap

FDA Brief: Week of Jan 25, 2016

OWH Vision

A Strategy for Science and Innovation to Improve The Health of Women

By FDA Office of Women’s Health (OWH)

Research Priority Areas

  1. Advance Safety and Efficacy: Advance the safety and efficacy and reduce the toxicity of FDA-regulated products used by women
  2. Improve Clinical Study Design and Analyses: Improve clinical study design and conduct to better identify and evaluate possible sex differences related to FDA-regulated products
  3. Novel Modeling and Simulation Approaches: Evaluate and promote the adoption of novel modeling and simulation approaches that can aid in regulatory evaluation of FDA-regulated products
  4. Advances in Biomarker Science: Develop tools and methods that can help identify, evaluate, and qualify predictive or prognostic clinical and non-clinical biomarkers and surrogate endpoints
  5. Expand Data Sources and Analysis: Identify, develop, and evaluate data sources and efficient techniques for data mining, data linkage, and large data set analysis that can be used to assess the postmarket toxicity or the safety and effectiveness of FDA-regulated products
  6. Improve Health Communications: Develop, evaluate, and use tools and methods to foster the creation and easy availability of clear and useful information about FDA-regulated products used by women to help women and their health care professionals make informed health-related decisions
  7. Emerging Technologies: Support the identification of sex differences related to the use of emerging technologies




FDA focus on French Biotrial Phase 1 Tragedy

FDA Brief : Week of Jan 25, 2016


Drug Information Update-FDA works with regulatory partners to understand French-based Biotrial phase 1 clinical study

  • Conferring with European regulators regarding the tragic phase 1 clinical study, conducted by Biotrial that resulted in 1 death and 4 neurological injuries
  • BIA 10-2474 is inhibitor of fatty acid amide hydrolase (FAAH), an enzyme involved in cell function in the nervous system
  • FDA is collecting and reviewing safety information pertinent to FAAH inhibitors in US and will work with sponsors, patients, investigators to ensure safety




2016 CDER Guidances, CDRH Interoperable Devices, Neurological Devices Reclassification

FDA Brief: Week of Jan 25, 2016

fda guidances


New & Revised Draft Guidances CDER is Planning to Publish During Calendar Year 2016


Advertising, Biopharmaceutics, Clinical/Antimicrobial,  Clinical/Medical, Clinical Pharmacology,  Clinical/Statistical,  Electronic Submissions,  Labeling, Pharmaceutical Quality/CMC,  Pharmaceutical Quality/Manufacturing Standards (CGMP), Pharmacology/Toxicology,  Procedural,


  • Health Care Economic Information in Promotional Labeling and Advertising
  • Treatment/Prevention of Anthrax, Attachment to HIV-1 Infection, Bacterial Vaginosis, Chronic Hepatitis C Virus Infection, Recurrent Herpes Labialis, Vulvovaginal Candidiasis, Allergic Rhinitis,  Exocrine Pancreatic Insufficiency,  Rare Diseases,  Nonallergic Rhinitis, Ulcerative Colitis
  • Patient Reported Outcomes for Varicose Vein burden, COPD
  • Adaptive Clinical Trial Design, Meta-Analysis for Safety
  • Post-marketing Safety reporting



Design Considerations and Pre-market Submission for Interoperable Medical Devices

  • Interconnectivity of  medical devices characterized as  “interoperability.”
  • Interoperability – Ability of two or more products, technologies or systems to exchange information and use of exchanged information
  • Highlights:
    •  designing systems
    •  performance testing and risk management activities
    • specifying functional, performance, and interface characteristics in labeling


Neurological Devices; Reclassification of Cranial Electrotherapy Stimulator  (CES)

Intended To Treat Insomnia and/or Anxiety : Class III to II – 510(k)

Intended To Treat Depression : PMA

Recalssification based on deliberations of  2012 Panel meeting,  reclassification petitions  and  new information that there is sufficient information to establish special controls to mitigate risks.





NINLARO® (ixazomib) Capsule for Patients with Multiple Myeloma

A reversible proteasome inhibitor, preferentially binds and inhibits the chymotrypsin-like activity of the beta 5 subunit of the 20S proteasome.

Pharmacodynamics (PD) Did not prolong the QTc interval at clinically relevant exposures
Pharmacokinetics (PK) Approximately 58% absolute bioavailability (median Tmax of 1 hour) in patients.

A 28% and 69% decreased in ixazomib AUC and Cmax, respectively, following the administration of NINLARO with a high-fat meal

AUC increased in a dose proportional manner over a dose range of 0.2 to 10.6 mg (i.e., 0.05 to 2.65 times the approved recommended dosage)

Accumulation approximately 2-fold

Systemic clearance was approximately 1.9 L/h and terminal half-life (t1/2) was 9.5 days

Extensive metabolism with both non-CYP and CYP enzymes contributing to ixazomib metabolism. At higher than clinical concentrations (in vitro), CYP3A4 is predominant (42%)

Plasma protein binding was approximately 99%.

Approximately 62% of the administered radioactivity was excreted in the urine (< 3.5% as unchanged drug) and 22% in the feces

PK-PD Analysis Did not prolong the QTc interval at clinically relevant exposures based on pharmacokinetic-pharmacodynamic analysis of data from 245 patients
Population PK There was no clinically meaningful effect of age (range 23-91 years), sex, body surface area (range 1.2-2.7 m2), or race on the clearance of ixazomib
Special Populations PK of ixazomib was similar in patients with normal hepatic function and in patients with mild hepatic impairment (total bilirubin ≤ ULN and AST > ULN or total bilirubin > 1-1.5 x ULN and any AST)


PK of ixazomib was similar in patients with normal renal function and in patients with mild or moderate renal impairment (creatinine clearance ≥ 30 mL/min). Mean AUC was 39% higher in patients with severe renal impairment or ESRD requiring dialysis as compared to patients with normal renal function.

Drug Interactions Co-administration of ixazomib with rifampin decreased ixazomib Cmax by 54% and AUC by 74%


Co-administration of ixazomib with clarithromycin did not result in a clinically meaningful change in the systemic exposure of ixazomib


Ixazomib is a low affinity substrate of P-gp, but is not expected to cause transporter-mediated drug-drug interactions.

Source : http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/208462lbl.pdf


NUCALA Clinical Pharmacology Card


NUCALA® (Mepolizumab) for subcutaneous (SC) injection for Severe Asthma Aged 12 Years and Older


Mechanism of Action Interleukin-5 (IL-5) antagonist monoclonal antibody (IgG1 kappa), responsible for the growth and differentiation, recruitment, activation, and survival of eosinophils
Pharmacodynamics (PD) Blood eosinophils decreased in a dose-dependent manner for doses 12.5 mg SC, 125 mg SC, 250 mg SC
Pharmacokinetics (PK) Exhibited approximately dose-proportional pharmacokinetics over a dose range of 12.5 to 250 mg. in subjects with asthma

 Accumulation approximately 2-fold at steady state

 Approximately 80% is absorbed following SC administration in the arm

 Central volume of distribution of mepolizumab in patients with asthma is estimated to be 3.6 L for a 70-kg individual

 Mean terminal half-life ranged from 16 to 22 days

Proteolytic enzymatic degradation that is widely distributed in the body

PK-PD Analysis Have not been reported
Population PK There was no significant effect of race and gender on mepolizumab clearance

There was no significant effect of age on mepolizumab clearance, ranging in age from 12 to 82 years

Special Populations No clinical trials have been conducted to investigate the effect of renal impairment or hepatic impairment on the pharmacokinetics of mepolizumab
Drug Interactions No formal drug interaction studies have been conducted

Source : http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/125526Orig1s000Lbl.pdf


FDA Brief, Week of Jan 18, 2016


Oncology Drug Approvals: Year in Review

2015 Office of Hematology and Oncology Products (OHOP) approvals & expedited review programs.

Richard Pazdur, M.D., Director of the Office of Hematology and Oncology Products


  • 16 new molecular entities (NMEs): non-small-cell lung cancer, colorectal cancer, breast cancer, melanoma, renal cancer, pancreatic cancer
  • Multiple Myeloma advances: Darzalex (daratumumab), Empliciti (elotuzumab), Ninlaro (ixazomib), and Farydak (panobinostat)
  • Biosimilar products: Zarxio (filgrastim-sndz) a bone marrow stimulant, Unituxin dinutuximab), for pediatric high-risk neuroblastoma


  • Impressive metric: 6 approvals using expedited review programs in Nov. 2015
  • Accelerated Approval: ‘Frequently’ used based upon a surrogate endpoint reasonably likely to predict a clinical benefit, e.g. overall survival
  • Priority Review:  Granted after NDA submission For serious and life-threatening diseases and provides significant improvement in safety or effectiveness over available therapy. Reduces NDA review times from 12 mo (standard) to 8 mo
  • Breakthrough therapy: Granted at IND stage. For expediting development of drugs for serious and life-threatening with preliminary clinical evidence. More dynamic interaction with sponsors to expedite development


  • Development of “targeted agents” – Greater effectiveness in a specific population and may generally have a more favorable benefit-risk profile
  • Breakthrough therapy designations – High response rates substantially better than available therapy


  • Review team and high standards of review quality
    • OHOP’s oncologists practicing physicians : medical, pediatric oncologists, radiation oncologists, oncology nurses, physician assistants, and oncology pharmacists
    • In addition, statisticians, basic scientists examining clinical pharmacology and toxicology, chemists reviewing manufacturing
  • External Engagement:
    • Advocacy groups and professional organizations
    • Other Agency centers, offices and National Cancer Institute
    • Ex-US regulators to share ideas and concerns
    • Expansion of “patient voice” initiative



FDA clarification of Flibanserin approval – after 2 rejections !

  • Careful evaluation of efficacy endpoints and clinical meaningful improvement, safety concerns and benefit/risk framework
  • Required additional Phase 3 studies as well as Clin Pharm/ Drug interaction studies
  • Considered generalizability of Ph 3 outcomes to all women likely to be prescribed
  • Factored in external feedback on HSDD’s on women’s sense of identity, emotional well-being, relationships
  • Risk evaluation and mitigation strategy with “elements to assure safe use” (REMS-ETASU) to ensure benefits outweigh increased risk of hypotension and syncope with alcohol
  • Risk management with labeling – Warning and Medication Guide
  • Required post-approval trials with enhanced pharmacovigilance to take further actions if needed



FDA approved

novartisArzerra (ofatumumab)  


Indication: Extended treatment of patients who are in complete or partial response after at least two lines of therapy for recurrent or progressive chronic lymphocytic leukemia (CLL).

Reg Pathway: sBLA, Priority Review


  • Single randomized, open-label  (n=474), patients with  complete or partial response after at least two lines of prior therapy, ofatumumab vs . observation
  • Event rate (progressed or died) : 33% vs. 51%
  • Investigator-assessed median Progression Free Survival : 29.4 mo. vs. 15.2 mo. (p<0.0001)



  • Most common adverse reactions: infusion reactions, neutropenia and upper respiratory tract infection
  • Most common serious adverse reactions: Pneumonia, pyrexia and neutropenia (including febrile neutropenia)


evis exera

EVIS EXERA II TJF-Q180V duodenoscope

with design modifications intended to reduce infection risk

Olympus medical Systems, Center Valley, PA

Intended Use:  Olympus TJF-Q180V duodenoscope with modifications to the device’s design and labeling intended to help reduce the risk of bacterial infections

Unmet Need:

  • Duodenoscopes  have been associated with the transmission of infectious agents, including antibiotic-resistant infections
  • Need to improve safety of duodenoscopes by reducing  risk of fluid leakage into the elevator channel, which in turn can reduce patient exposure to bacteria and other potential infections

Design Modifications:

  • Elevator channel sealing mechanism to create a tighter seal and reduce the potential for leakage of patient fluids and tissue into the closed elevator channel
  • Conduct annual inspections of each scope in use by facilities to identify any wear and tear on the elevator channel sealing mechanism or the presence of attached debris at the scope’s tip, which would require replacement of the potentially contaminated parts.

Reg Pathway:  510(k) for design modifications

  • 2014 FDA letter : Need for remarket notification for the “closed” elevator channel model which had never been cleared for marketing
  • 2015 FDA Safety Communication : Validated instructions for cleaning and disinfecting of TJF-Q180V while the 510(k) under review
  • Voluntary recall of the original TJF-Q180V model from health care facilities and make needed repair
  • Post market surveillance and Annual reporting

fda guidances


Implanted Blood Access Devices for Hemodialysis

  • Reclassifying implanted blood access devices for hemodialysis, which were preamendments Class III devices, into Class II (special controls) and subject to premarket notification.- requiring 510(k) Submission





Sterility Information for 510(k) Submissions for ‘Sterile’ Devices

  • Clarification of information regarding sterilization processes for 510(k)s for devices labeled as ‘sterile’
  • ‘Sterile’ –  subject to industrial terminal sterilization processes based on microbial inactivation
  • Describes ESTABLISHED and NOVEL methods





Director’s Corner Podcast

WHO: Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research.

WHAT: Discussion of major events of the past year and priorities for 2016.


LINK to Podcast



SBIA Chronicles

WHO: CDER Small Business and Industry Assistance

WHAT:  Best Communications Practices with FDA

WHY: Communication between FDA and sponsors during product development and at critical junctures ultimately facilitate earlier availability of safe and effective drugs to the American public.





WHO:  AIDSinfo is releasing HIV/AIDS Guidelines app for iOS and Android devices.

WHAT: Provides mobile access to the  federally approved HIV/AIDS medical practice treatment guidelines.

WHY:  To provide Health Care Providers mobile access to up-to-date HIV information at the point of care—even when Internet connection is unavailable

HOW: AIDSinfo to download the free Guidelines app to your device

2017-2018 FDA CALENDAR



 Newly added 


Jan 12: FDA Grand Rounds- Developing Regulatory Methods for Characterizing Nanomaterials in FDA-Regulated Products Describe methodology for characterizing inorganic and organic nanoparticles, using various microscopic techniques, light scattering, size-based separation methodology, elemental analysis, and mass spectrometry. INFORMATION

Jan 24: Guidance Webinar: Collection of Race and Ethnicity Data in Clinical Trials To  learn more about the guidance document. INFORMATION


Feb 2:  Ninth Annual Sentinel Initiative Public Workshop . Duke-Margolis Center for Health Policy and FDA  forum to bring together leading experts and interested stakeholders to discuss the ongoing development of the Sentinel Initiative. INFORMATION

Feb 2: Webinar – Final Guidance on Medical Device Accessories: Describing Accessories and Classification Pathway for New Accessory Types. Discuss final guidance, provide clarification on definition of medical device accessory and recommendations for classifying accessories, de novo process for  risk-based classification. INFORMATION

Feb 2: Public Workshop – Coordinated Registry Network (CRN) for Devices Used for Acute Ischemic Stroke Intervention (DAISI) Obtain stakeholders’ input on the coordination of registries for DAISI. INFORMATION

Feb 3:  Webinar – Final Rule on Postmarketing Safety Reporting (PMSR) for Combination Products. Discuss final rule.INFORMATION

Feb 7: Webinar -Collaborating with FDA: Get Involved with FDA’s MedWatch Adverse Event Reporting Program How to utilize FDA’s drug information, medication safety resources, and regulatory guidance, to improve delivery of patient care and optimize outcomes.INFORMATION

Feb 7: National Black HIV/AIDS Awareness Day Twitter Chat. FDA hosting a Twitter chat with BlackDoctor.org in recognition of National Black HIV/AIDS Awareness Day. Use to chat. INFORMATION

Feb 9: Webinar – Final Guidance on Factors to Consider Regarding Benefit-Risk in Medical Device Product Availability, Compliance, and Enforcement Decisions. Discuss final guidance document and clarify FDA’s approach to weighing benefits and risks for medical device product availability, compliance, and enforcement decisions. INFORMATION 

Feb 23: Circulatory System Devices Panel: Discuss de novo request for SENTINEL® Cerebral Protection System, a first of a kind embolic protection device to be used with transcatheter aortic valve replacement (TAVR) procedures. INFORMATION

Feb 23: Webinar – Factors to Consider When Making Benefit-Risk Determinations for Medical Device Investigational Device Exemptions Final Guidance. Help manufacturers and other interested stakeholders understand the information provided in this final guidance document. INFORMATION

Feb 23: Precision Cardiovascular Medicine Webinar Discuss current research on genetic misdiagnoses and cardiomyopathy and precision cardiovascular medicine for multiethnic populations INFORMATION

Feb 27-28: FDA-USP Workshop on Standards for Pharmaceutical ProductsCritical Importance of Excipients in Product Development – Why Excipients are Important Now and In the Future. INFORMATION


Mar 1:  Current State and Further Development of Animal Models of Serious Infections Caused by Acinetobacter baumannii and Pseudomonas aeruginosa. Public workshop on the current state and further development of animal models for serious infections caused by Acinetobacter baumanii and Pseudomonas aeruginosa. INFORMATION

Mar 6-7:  Pediatric Advisory Committee Meeting. Safety reviews for drugs – NITROPRESS, KUVAN, TRUVADA, EXJADE and devices EPICEL, IMPELLA RP SYSTEM, LIPOSORBER LA-15 SYSTEM, MEDTRONIC ACTIVA DYSTONIA THERAPY. Also discuss safety reviews for NOVOEIGHT  and RIXUBIS Coagulation Factor IX. INFORMATION

Mar 9: Vaccines and Related Biological Products Advisory Committee Meeting.  Discuss and make recommendations on the selection of strains to be included in the influenza virus vaccines for the 2017-2018 influenza season INFORMATION

Mar 13-14: Joint Meeting of the Drug Safety and Risk Management Advisory Committee and the Anesthetic and Analgesic Drug Products Advisory Committee. Safety issues for OPANA ER (oxymorphone hydrochloride) NDA – pre- and post-marketing data about the abuse and the overall risk-benefit of product. INFORMATION

Mar 15: Advisory Committee Meeting for Pharmaceutical Science and Clinical Pharmacology.  Physiologically-based pharmacokinetic modeling and simulation throughout a drug’s lifecycle & mechanistic model-informed safety evaluation with a focus on arrhythmias.  INFORMATION

Mar 17:  Joint Meeting of the Ophthalmic Devices Panel of the Medical Devices Advisory Committee and the Risk Communication Advisory Committee.  Potential risks of misuse of PEROXIDE-BASED CONTACT LENS PRODUCTS, adequate labeling and packaging of these over-the-counter products. INFORMATION

Mar 19-21: FDA/PhUSE Computational Science Symposium. Review progress on data standards, best-practices-driven analytical tool development, business processes that are driving development of information systems, experiences and evaluation of current tools by users themselvesINFORMATION

Mar 22: FDA Webinar Regulatory Overview for Developers and Sponsors of Neurological Devices: An Introduction to the De Novo Pathway . For developers and sponsors of neurological devices on the De Novo pathway, an alternate pathway to classify novel devices INFORMATION

Mar 30: Grand Rounds: Vaccine adjuvants -New ways to evaluate their safety and effectiveness.  Adjuvants and immune responses, safety concerns and appropriate studies. INFORMATION


Apr 4-5: Blood Products Advisory Committee Meeting. Discuss Recombinant Human Coagulation Factor IX, GlycoPEGylated, responses to Blood Donor Deferral Policy for Reducing the Risk of HIV  Transmission by Blood and Blood Products, research programs in Laboratory of Emerging Pathogens, Division of Emerging Transfusion-Transmitted Diseases. INFORMATION

Apr 5: Joint Meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and the Drug Safety and Risk Management Advisory Committee Meeting.  Discuss NDA for oxycodone hydrochloride immediate-release oral tablets with the proposed indication of management of moderate to severe pain where the use of an opioid analgesic is appropriate. Product formulated with properties intended to deter abuse. INFORMATION

Apr 6: Public Workshop: Emerging Tick-Borne Diseases and Blood Safety. Discuss tick-borne pathogens, effectiveness of mitigation strategies, blood safety interventions.INFORMATION

Apr 6: Public Meeting: Patient-Focused Drug Development Meeting for Sarcopenia. Perspectives of patients with sarcopenia on i) symptoms and the daily impacts of their condition ii) current approaches to treatment, and iii) decision factors taken into account when selecting a treatment.INFORMATION

Apr 12-13: Antibody Mediated Rejection (AMR) in Kidney Transplantation Workshop. Regarding new developments and scientific issues related to antibody mediated rejection (AMR) in kidney transplantation. INFORMATION

Apr 13: FDA CERSI Lecture on Zika Virus Vaccines and Therapeutics. Dr. Monica McArthur, MD. PhD.  will discuss the anti-Zika virus vaccine candidates now in the pipeline and efforts to identify effective therapeutics.  INFORMATION

Apr 19-20: Public Workshop: Battery Safety Concerns in Electronic Nicotine Delivery Systems. Hazards and risks associated with use of batteries in ENDS, including e-cigarettes. INFORMATION

Apr 24: International Council for Harmonization Regional Public Consultation. Co-hosting with Health Canada to solicit public input prior to next ICH meeting. Topics relate to harmonization of various manufacturing guidelines. INFORMATION

Apr 24-26: 11th Annual FDA/DIA Statistics Forum. Discuss relevant statistical issues associated with the development and review of therapeutic drugs and biologics. INFORMATION

Apr 25; FDA/C-Path PRO Consortium: Second Annual Workshop on Clinical Outcome Assessments (COAs) in Cancer Clinical Trials. Engage with leaders in academia, industry, international regulatory and health technology assessment (HTA) bodies, and patient groups to thoughtfully discuss how to integrate PRO measures and other COAs into cancer clinical trials.INFORMATION


May 3:  FY 2017 Generic Drug Research Public Workshop. Obtain input from industry and other interested stakeholders on regulatory science priorities. INFORMATION

May 3-5: FDA/Xavier University Medical Device Conference (MedCon). Public conference includes presentations from FDA officials, industry experts.  INFORMATION

May 4: Patient-Focused Drug Development for Autism. Gather patients’ perspectives on symptoms and daily impacts and discuss current treatment optionsINFORMATION

May 4: Reagan Udall Foundation for the FDA Public Meeting.  To receive public input on Foundation’s efforts to  catalyze programs and projects by bringing together FDA, patient/consumer groups, academia, other government entities, and industry to work together transparently in developing cutting edge regulatory scienceINFORMATION

May 8-9:  Meeting of the Pharmacy Compounding Advisory Committee Meeting. Discuss quality standards and conditions at certain compounding facilities. INFORMATION

May 9-10: Regulatory Education for Industry (REdI) Spring Conference. Conference Topics include: CDER: Types of INDs; Chemistry, Manufacturing and Controls (CMC); Pharmacology/Toxicology; Drug Inspections CDRH: 510(k); Biocompatibility in Premarket Submissions; Nonconforming Product; Device Inspections. INFORMATION

May 9-10: Training Health Care Providers on Pain Management and Safe Use of Opioid Analgesics – Exploring the Path Forward. Public meeting on issues and challenges with Federal efforts to support training on pain management and the safe prescribing, dispensing, and patient use of opioids.  INFORMATION

May 10: Meeting of the Medical Imaging Drugs Advisory Committee. Discuss NDA for 5-Aminolevulinic Acid Hydrochloride [5-ALA HCl], Powder, for oral solution, for the proposed indication as an imaging agent to facilitate the real time detection and visualization of malignant tissue during glioma surgery. INFORMATION

May 11: FDA Grand Rounds. FDA Research: an Alternative Approach to Presenting Risk Information in Prescription Drug TV Ads. Discuss “major statement” in direct-to-consumer TV ads INFORMATION

May 12: Roadmap for Engaging With the FDA/CDER. Public Workshop for patient advocacy groups. Enhance understanding of approval of safe and effective drug therapies for patient advocacy.  INFORMATION

May 17: Gastroenterology and Urology Devices Panel of the Medical Devices Advisory Committee Meeting. Discuss PMA for TRANSMEDICS ORGAN CARE SYSTEM (OCS)–Lung System, by TransMedics, Inc.  The proposed Indication for Use -Portable organ perfusion, ventilation, and monitoring medical device intended to preserve donor lungs in a near physiologic, ventilated, perfused state for transplantation  INFORMATION

May 18-19: Public Workshop – Cybersecurity of Medical Devices: A Regulatory Science Gap Analysis. Examine opportunities for FDA engagement with new and ongoing research, catalyze collaboration among Health Care and Public Health stakeholders to strengthen medical device cybersecurity. INFORMATION

May 24: Meeting of the Oncologic Drugs Advisory Committee. Discuss  NDA for neratinib maleate, for proposed indication for the extended adjuvant treatment of adult patients with early-stage HER2-overexpressed/amplified breast cancer who have received prior adjuvant traustuzumab-based therapy (morning session). Discuss NDA for L-glutamine powder (oral solution), for the proposed indication of treatment of sickle cell disease (afternoon session) INFORMATION

May 25: Meeting of the Oncologic Drugs Advisory CommitteeDiscuss BLA  for  proposed biosimilar to Epogen/Procrit (epoetin alfa) INFORMATION

May 31-Jun 1: 2017 FDA Science Forum. Highlight breadth and depth of cutting-edge science FDA conducts and demonstrate how FDA’s scientific research informs regulatory decision-making. INFORMATION


Jun 6: DDI Webinar Series: Ongoing Role of FDA in Medication Error Prevention. Information on the Division of Medication Error Prevention and Analysis (DMEPA)’s role in preventing and addressing medication errors, as well as the role of pharmacists in identifying, preventing and mitigating medication errors. INFORMATION

Jun 15: Risk Evaluation and Mitigation Strategies (REMS): A Deeper Dive.  Purpose, Process, and Challenges and Structured Product Labeling Format. INFORMATION

Jun 15: Safe Use Symposium: A Focus on Reducing Preventable Harm from Drugs in the Outpatient Setting. Discuss sources of preventable harm from drugs in outpatient setting, interventions to reduce preventable harms and how these interventions can be studiedINFORMATION

 Jun 19: The Ins and Outs of Presenting Clinical Pharmacology Information in Prescription Drug Labeling. Highlights from the FDA guidance, Clinical Pharmacology Section of Labeling for Human Prescription Drug and Biological Products—Content and Format, and key prescription drug labeling regulations. INFORMATION

Jun 20: Endocrinologic and Metabolic Drugs Advisory Committee Meeting.  Discuss sNDA for VICTOZA (liraglutide) injection (sNDA 022341), for proposed additional indication to reduce the risk of major adverse cardiovascular events in adults with type 2 diabetes mellitus and high cardiovascular risk. INFORMATION

Jun 21-22: Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee Meeting  Gauge investigator interest in potential pediatric development plans for several products in various stages of development for adult cancer indications. INFORMATION


Jul 10: Public Workshop – Sentinel Training at the Food and Drug. Provide training on using health care claims data within the FDA Sentinel System and understanding of system capabilities INFORMATION  

Jul 11: Oncology Drugs Advisory Committee Meeting. Discuss BLA for MYLOTARG for proposed indication (use) as combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of adult patients with previously untreated, de novo acute myeloid leukemia (AML). INFORMATION

Jul 12: Oncology Drugs Advisory Committee Meeting.  Discuss BLA for tisagenlecleucel-T suspension for proposed indication (use) for treatment of pediatric and young adult patients 3 to 25 years of age with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL). INFORMATION

 Jul 13: Oncology Drugs Advisory Committee Meeting.  Discuss BLA for ABP 215, a proposed biosimilar to Genentech/Roche’s AVASTIN (bevacizumab) and BLA for MYL-1401O, a proposed biosimilar to Genentech Inc.’s HERCEPTIN. INFORMATION

Jul 13: Optimizing Your Study Data Submissions to FDA – Updates from CDER and CBER. Recent updates Study Data Technical Conformance Guide and recommendations to optimize your study data submission to improve efficiency of review process and avoid technical rejection. INFORMATION

Jul 17: Public Workshop: Developing Rabies Monoclonal Antibody Products as a Component of Rabies Post-Exposure Prophylaxis. Provide information for, and gain perspective from stakeholders on various aspects of development efforts pertaining to animal models, laboratory assays, and clinical trials. INFORMATION

 Jul 18: Public Meeting: Administering the Hatch-Waxman Amendments. Ensuring a Balance Between Innovation and Access. Submit comments on Federal Food, Drug, and Cosmetic Act (FD&C Act) to balance drug innovation and accelerating availability to lower cost alternatives. INFORMATION

 Jul 19: Public Workshop: Development of New Tuberculosis Treatment Regimens-Scientific and Clinical Trial Design Considerations. Provide information for and gain perspective from stakeholders regarding scientific and clinical trial design considerations related to the development of new TB regimens. INFORMATION

Jul 20: FDA/MDIC Forum on Case for Quality. The CDRH Case for Quality team will lead a discussion on ongoing preparations for the proposed third party voluntary quality programINFORMATION

Jul 26:  Webinar- Regulatory Overview for Developers and Sponsors of Neurological Devices: An Introduction to Premarket Approvals. Overview of PMA process, non-clinical testing,  manufacturing, post-approval studies for neurological devices.  INFORMATION

 Jul 28: Biological Advisory Committee Meeting Discuss safety and efficacy of a Hepatitis B Vaccine manufactured by Dynavax. INFORMATION


Aug 1:  Digital Health Software Precertification (PreCert) Pilot Program. Discuss and answer questions about this precertification pilot program.   INFORMATION

Aug 2:  Arthritis Advisory Committee Meeting. Discuss BLA for PLIVENSIA (sirukumab) injection for treatment of adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or are intolerant to one or more disease modifying anti-rheumatic drugs. INFORMATION

Aug 3:  Arthritis Advisory Committee Meeting. Discuss sNDA for XELJANZ (tofacitinib) and for XELJANZ XR extended release tablets for the treatment of adult patients with active psoriatic arthritis. INFORMATION

Aug 24:  Webinar – CDRH Final Guidance: Qualification of Medical Device Development ToolsMDDT program is a voluntary process intended to reduce regulatory burden for tool developers and FDA reviewers through the qualification of tools that can aid in the development and evaluation of medical devices. INFORMATION

Aug 31: FDA/CDRH – RSNA SIG Joint Meeting on 3D Printed Patient-specific Anatomic Models. FDA-CDRHand Radiological Society of North America 3D printing Special Interest Group (RSNA SIG) to discuss clinical use of 3D printed patient-specific anatomic models. INFORMATION


Sep 8:  Pediatric Trial Design and Modeling: Moving into the next decade. Review current best practices in designing trials, discuss problems and potential solutions, optimize trials to achieve labeling. INFORMATION

Sep 8: Meeting of the Medical Imaging Drugs Advisory Committee. Discuss the potential risk of gadolinium retention in the brain and other body organs in patients receiving gadolinium-based contrast agents for magnetic resonance clinical imaging procedures. INFORMATION

Sep 11: Patient-Focused Drug Development Public Meeting for Alopecia Areata. To obtain patient input on the impact of alopecia areata, including on daily life, patient views on treatment approaches, and decision factors taken into account when selecting a treatment. INFORMATION

Sep 12: Public Workshop: Reducing the Risk of Preventable Adverse Drug Events associated with Hypoglycemia in the Older Population Discuss importance of individualized glycemic control targets for older patients with diabetes. INFORMATION

Sep 13: Public Workshop – Antimicrobial Susceptibility and Resistance: Addressing Challenges of Diagnostic Devices. Discuss scientific and regulatory challenges of development of traditional devices for antimicrobial susceptibility testing (AST), novel diagnostic technologies for detection of antimicrobial resistance markers, and new regulatory solutions per 21st Century Cures Act. INFORMATION

Sep 13: Vaccines and Related Biological Products Advisory Committee Meeting Announcement. Discuss safety and effectiveness of Zoster Vaccine Recombinant (Adjuvanted) [Shingrix], manufactured by GlaxoSmithKline. INFORMATION

Sep 14: FDA Grand Rounds- Developing a Mechanistic Model-Based Approach to Assess Cardiac Safety of New Drugs. A new model, the Comprehensive in vitro Proarrhythmia Assay (CiPA) initiative, aims to be a more accurate and comprehensive, mechanistic-based assessment of drug safety. INFORMATION

Sep 14: Joint Meeting of the Anesthetic and Analgesic Drug Products Advisory Committee and the Drug Safety and Risk Management Advisory Committee. Discuss sNDA for BUTRANS (buprenorphine) transdermal system in pediatric patients ages 7 through 16 years for management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. INFORMATION

Sep 15: FDA-ASCO: Hematology and Oncology Fellows Day Workshop. Provide current hematology and oncology fellows with a solid foundation in the process of oncology drug development and regulatory science. INFORMATION

Sep 18: Public Workshop on Benefit-Risk Assessments in Drug Regulatory Decision-Making. Discuss structured assessment of benefits and risks in drug regulatory decision-making, incorporating patient perspectives, methods to advance structured benefit-risk assessment. INFORMATION

Sep 18-19: Public Workshop- Advancing the Development of Pediatric Therapeutics (ADEPT): Application of “Big Data” to Pediatric Safety Studies. To understand (1) how to access and analyze “Big Data” associated with safety information in the healthcare setting and (2) utility and challenges associated with the use of “Big Data” to study the safety of therapeutics in children. INFORMATION

Sep 18-20: FDA-Agência Nacional de Vigilância Sanitária (ANVISA), Brazil Health Authority, Global Summit on Regulatory Science Emerging Technologies for Drug and Food Safety. Held at ANVISA Brasilia, DF BRAZIL. INFORMATION

Sep 19:  Meeting of the Oncologic Drugs Advisory Committee. Discuss sNDA for SUTENT (sunitinib malate) oral capsules for the adjuvant treatment of adult patients at high risk of recurrent renal cell carcinoma (RCC) following nephrectomy. INFORMATION

Sep 19: Webinar – Enhancements to CDRH Export Certification and Tracking System (CECATS). Highlight enhancements to  CECATS, a web-based, voluntary electronic system for requesting export documents for international tradeINFORMATION

Sep 21: Yale-CERSI Webinar.“Real World” Evidence: Beyond Randomized Controlled Trial. Concept of real world evidence, policy implications, available approaches and frameworks, observational evidence. INFORMATION

Sep 25: Primary Pediatric Hypertension Workshop: Epidemiology and Treatment Gaps. To discuss the current state of hypertension epidemiology, pathophysiology,  diagnosis, and treatment in the pediatric population. INFORMATION

Sep 27: Public Meeting on Patients Who Have Received an Organ Transplant. To obtain patient input on the impact of receiving an organ transplant on daily life and patients’ views on currently available therapies to manage organ transplantation.  INFORMATION

Sep 27-28: FDA Small Business Regulatory Education for Industry (REdI): Fall Conference. Provide direct, relevant, and helpful information on the key aspects of drug and device regulations. INFORMATION

Sep 28: Peripheral and Central Nervous System Drugs Advisory Committee. Discuss NDA for ataluren for oral suspension, sponsored by PTC Therapeutics, Inc., for the treatment of patients with dystrophinopathy due to a nonsense mutation in the dystrophin gene. INFORMATION


Oct 2-3: Leveraging Quantitative Methods and Modeling to Modernize Generic Drug Development and Review. Discuss current and emerging scientific approaches and applications for quantitative modeling and simulations in generic drug development. INFORMATION

Oct 5: Electronic Drug Registration and Listing Using CDER Direct  INFORMATION

Oct 6: Demonstrating Equivalence of Generic Complex Drug Substances and Formulations: Advances in Characterization and In Vitro Testing. Discuss use of in vitro testing to demonstrate bioequivalence of complex generic drugs.  INFORMATION

Oct 10:  Voluntary Medical Device Manufacturing and Product Quality Program.  Announce the proposed framework and preliminary outline of a voluntary pilot program that recognizes an independent assessment of manufacturing and product quality.  INFORMATION

Oct 10: Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices. Overview of real-world data and real-world evidence and FDA evaluation of dataINFORMATION

Oct 11, 12: Inaugural meeting of Patient Engagement Advisory Committee (PEAC).  Provide advice and recommendations to FDA on complex issues relating to medical devices, the regulation of devices, and their use by patients. INFORMATION

Oct 12: Biologics Advisory Committee Meeting: Cellular, Tissue, and Gene Therapies:  Discuss BLA for voretigene neparvovec, submitted by Spark Therapeutics, for treatment of vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy.

Oct 17:  An Overview of FDA Resources. Resources to respond to questions, and to promote safe use of drug products. Topics will include drug shortages, recalls, adverse events, and safety labeling updatesINFORMATION

Oct 19: Health Canada and FDA Joint Public Consultation on ICH Technical Requirements for Pharmaceuticals for Human Use. Solicit public input prior to the next ICH Assembly meeting and the expert working groups meetings in Geneva, Switzerland. INFORMATION

Oct 20: Overcoming Barriers to Product Development, Regulatory Approval and Commercialization of Affordable, High Quality, Generic Topical Dermatological Drug Products. Discuss current barriers in development of topical dermatological generic drug products and ways to improve patient accessINFORMATION

Oct 26:  Design Considerations and Premarket Submission Recommendations for Interoperable Medical Devices Final Guidance. Discuss Agency’s recommendations for developing safe and effective devices that exchange and use patient information  electronically. INFORMATION

Oct 30: CDER Rare Diseases Public Workshop: Strategies, Tools and Best Practices for Effective Advocacy in Rare Diseases Drug Development.Primarily for rare disease community members to help understand FDA’s needs, case studies on beneficial overlap of effective advocacy techniques and FDA regulations.  INFORMATION

Oct 31: Webinar – Final Guidance on “Evaluation and Reporting of Age, Race, and Ethnicity Data in Medical Device Clinical Studies” Recommendations to improve the quality, consistency and transparency of data on how medical devices perform within specific age, race, and ethnic groups. Proper evaluation and reporting of this data can benefit patients, clinicians, researchers and regulators INFORMATION


Nov 1: Joint Meeting of the Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee. Discuss new drug application (NDA) 210136, buprenorphine subcutaneous injection, submitted by Braeburn Pharmaceuticals, Inc., for treatment of opioid dependence. INFORMATION

Nov 6:  Public Workshop: Geriatric Oncology by FDA and the American Society of Clinical Oncology (ASCO) Discuss improving the evidence base for treating the geriatric oncology population.  INFORMATION

Nov 6-7: National Center for Toxicological Research Science Advisory Board Meeting. Center-wide update on scientific initiatives and accomplishments, discuss  research strategic needs and potential areas collaboration.  INFORMATION

Nov 7: Vaccines and Related Biological Products Advisory Committee Meeting
Discuss clinical development plan for Pfizer’s investigational Staphylococcus aureus vaccine intended for pre-surgical prophylaxis in elective orthopedic surgical populations. INFORMATION

Nov 7:Industry Basics Workshop – Nonconforming Product and Complaint Files Presentation and Q/A session with Office of Compliance and Office of Communication and Education INFORMATION

Nov 9: FDA Grand Rounds: Assessing the safety and effectiveness of new and emerging therapeutic ultrasound technologies. Research into therapeutic ultrasound’s bioeffects, many of which remain poorly understood. INFORMATION

Nov 8-9: Immune Globulin Potency in the 21st Century. Public workshop to discuss new challenges to meet U.S. potency requirements for Immune Globulin (IG) products and to identify measures to address challenges.  INFORMATION

Nov 13: Partners in Progress: Cancer Patient Advocates and FDA. Provide basic training on the role of the FDA and cancer patient advocates in oncology product development. Supported by American Association for Cancer Research (AACR), American Society of Clinical Oncology (ASCO), and American Society of Hematology (ASH).INFORMATION

Nov 15-16: FDA Clinical Trial Requirements, Regulations, Compliance, and Good Clinical Practice. Co-sponsored with Society of Clinical Research Associates (SOCRA).to aid the clinical research professional’s understanding of the mission and authority of FDA and to facilitate interaction with FDA representatives. INFORMATION

Nov 16: Devices Referencing Drugs; Public Hearing. Potential approach for certain device sponsors to seek marketing authorization for a device labeled for use with a drug that is already approved and on the market, when the drug sponsor does not wish to pursue the new use. Called devices referencing drugs (DRDs), these products have the potential to advance public health by offering new uses with approved, marketed drugs that might not otherwise be availableINFORMATION 

NOV 16: Antimicrobial Drugs Advisory Committee.Discuss NDA for ciprofloxacin inhalation powder, sponsored by Bayer HealthCare Pharmaceuticals, Inc., for the proposed indication of reduction of exacerbations in non-cystic fibrosis bronchiectasis (NCFB) adult patients with respiratory bacterial pathogensINFORMATION

Nov 16:  Webinar on Final Guidances on “Deciding When to Submit a 510(k) for a Change to an Existing Device” and “Deciding When to Submit a 510(k) for a Software Change to an Existing Device”.  For medical device changes and software-specific changes INFORMATION

Nov 17: Webinar – PreCertification (Pre-Cert) Pilot Update First in series on Digital Health PreCertification (Pre-Cert) Pilot Program; keep efforts transparent, share updates throughout pilot process INFORMATION

Nov 21: Webinar – De Novo Classification Process (Evaluation of Automatic Class III Designation) Provide updated recommendations for interacting with the FDA, including what information to submit when seeking a path to market via the De Novo classification process. INFORMATION

Nov 28: Public Workshop – Cardiac Troponin Assays. Discuss development of innovative troponin assays designed to aid in the diagnosis of Myocardial Infarction (MI) and additional clinical uses of these assays. INFORMATION

Nov 28FDA-NCI Public Workshop: Defining Disease Recurrence and Harmonizing Conduct in Adjuvant Bladder and Kidney Cancer Trials. Joint FDA, NCI, and  Society of Urologic Oncology (SUO) workshop to improve conduct of adjuvant clinical trials in bladder cancer and kidney cancer by harmonizing definitions and management of disease recurrence.  INFORMATION

Nov 30:  Webinar – Unique Device Identification: Direct Marking of Devices Final Guidance. Discuss direct marking requirements including how FDA interprets the term “intended to be reprocessed,” further explaining other aspects of UDI direct marking. INFORMATION


Dec 5:  DDI Webinar Series: Biosimilars. Educational webinars targeting the needs of all health care professionals and students, including physicians, physician assistants, nurses, pharmacists, and pharmacy technicians INFORMATION

Dec 7: Advisory Committee Meeting: Bone, Reproductive and Urologic Discuss appropriate patient selection criteria and clinical trial design features, for drugs intended to treat interstitial cystitis and bladder pain syndrome. INFORMATION

Dec 11-12: Packaging, Storage, and Disposal Options to Enhance Opioid Safety-Exploring the Path Forward. Role of packaging, storage, and disposal options within the larger landscape of activities aimed at addressing abuse, misuse, or inappropriate access of prescription opioids INFORMATION

Dec 12: Orthopaedic and Rehabilitation Devices Panel of the Medical Devices Advisory Committee Meeting. Discuss PMA for Barricaid Anular Closure Device by Intrinsic Therapeutics, intended to be implanted following a limited discectomy, to prevent reherniation and the recurrence of pain or dysfunction INFORMATION

Dec 15: Public Workshop: Medical Gas Regulation.  Opportunity for medical gas
manufacturers to provide input on potential areas of Federal drug regulation that should be revised with respect to medical gases.  INFORMATION

Dec 18: Public Workshop: Patient-Focused Drug Development – Guidance 1, Collecting Comprehensive and Representative Input. Discussion on methodological approaches that a person seeking to collect patient experience data for submission to FDA to inform regulatory decision-making may use. INFORMATION

Dec 18: Webinar – Regulatory Overview for Developers and Sponsors of Neurological Devices. An Introduction to Humanitarian Device Exemptions (HDEs).  Overview of HDE regulatory pathway, and humanitarian use devices (HUDs) INFORMATION


⊗ Jan 8: CLIA Waiver Applications Draft Guidance Documents. Discuss draft guidances on CLIA waiver applications and Dual 510(k) and CLIA waivers INFORMATION

Jan 11: Public Workshop – Self-Collection Devices for Pap Test. Obtain feedback on feasibility, benefits, risks for self-collection cervical sampling devices for cervical cancer screening by Pap testing INFORMATION

⊗ Jan 11:  Safety Assessment for IND Safety Reporting. Convened by the Duke-Robert J. Margolis Center for Health Policy at Duke University and FDA; to bring stakeholder community together to discuss IND safety topics INFORMATION

 Jan 29: Weighing the Evidence: Variant Classification and Interpretation in Precision Oncology. To discuss how genetic sequencing data is best implemented in patient management to advance innovative regulatory strategies to support development of safe and effective precision-based drugs and devices. INFORMATION

 Jan 30-31: Fostering Digital Health Innovation. Developing the Software Precertification Program. Discuss progress of pilot precertification program and seek input on ongoing development of the Software Precertification Program. INFORMATION

FDA Brief, Week of Jan 11, 2016

CDRH Priorities


  • Establish a National Evaluation System for medical devices : To successfully harness from the diverse set of real-world evidence in an efficient manner
    • Increase access to real-world evidence to support regulatory decision-making
    • Increase use of real-world evidence to support regulatory decision-making
  • Partner with Patients; interact with patients as partners and work together to advance the development and evaluation of innovative devices, and monitor the performance of marketed devices
    • Promote a culture of meaningful patient engagement by facilitating CDRH interaction with patients
    • Increase use and transparency of patient input as evidence in decision-making
  • Promote a culture of quality and organizational excellence; manufacturer’s ability to design and make high-quality, safe and effective devices and CDRH’s ability to provide the necessary oversight to assure devices on the market are high-quality, safe and effective 
    • Strengthen culture of quality within CDRH
    • Strengthen product and manufacturing quality within medical device ecosystem



FDA Voice

Progress and Collaboration on Clinical Trials

Barbara D. Buch, M.D, . Chair of the 907 Steering committee and the Associate Director for Medicine in FDA’s Center for Biologics Evaluation and Research

Based on Congress’s directive in Section 907 of FDASIA, FDA looking more closely at sex, age, and race/ethnicity data collected in clinical trials.

Three priorities:

  • improving the quality and comprehensiveness of demographic subgroup data collection, reporting and analysis
  • identifying and eliminating barriers for increased participation in clinical trials
  • improving the transparency of subgroup data.


  • Public meeting on this topic on February 29.
  • Guidances : Integrated Summary of Effectiveness: Guidance for Industry & Evaluation of Sex-Specific Data in Medical Device Clinical Studies
  • Supportive Programs across FDA’s Divisions and Offices : Office of Minority Health (OMH), CDER, CDRH
  • Updated Medwatch Forms to standardize collection of demographic information


  • OWH and NIH collaborative workshop on importance of diversity
  • Availability of demographic data through ‘Drug Trials Snapshots’
  • FDA and Johns Hopkins University co-sponsored clinical trials workshop on Safety and Efficacy for a Diverse Population


  • Language Access Plan Working Group to address needs of under-represented subpopulations
  • CBER transparency pilot program for open access to demographic information from BLAs
  • CDRH modified templates for certain medical devices to ensure inclusion of demographic information


CDER conversations

Compendium of CLINICAL OUTCOME ASSESSMENTS (COAs) to promote the use of patient-focused outcome measurement in drug development

Elektra Papadopoulos, M.D., MPH, Acting Associate Director, Clinical Outcome Assessments Staff, Office of New Drugs, CDER, FDA


What is patient-focused outcome measurement about?

Understanding of the impact of a disease on the people who have it, and what they value most in terms of alleviating symptoms

How does patient input influence the choice of outcome measures in drug development?

Patient input into the selection of outcomes that are meaningful to them can profoundly influence drug development by ensuring the patient voice is captured.

What is a clinical outcome assessment and how are COAs captured in drug development?

COAs are captured using four types of COA measures: patient-reported, clinician-reported, observer-reported, and performance outcome measures.

What are patient-reported outcomes?

Patient-reported outcomes (PROs) are outcomes that are based on reports coming directly from the patient about how he or she feels or functions as a result of treatment. PROs may not always be feasible or appropriate depending on the particular context, and other types of COAs may be utilized.

What is the COA Compendium? And what is its purpose?

Table describing use of COAs in drug development related symptoms) and support labeling claims

Designed to be used as a starting point when considering the use of COAs in clinical trials

Why is this COA Compendium important and what does it mean for patients?

Tool to foster patient-focused drug development mission and  to see knowledge gaps

Is FDA encouraging drug companies to consider using PROs in trials that support approval of a drug?


Will this compendium be an all-conclusive list of COAs?

No – this is a pilot. Hope to get public comment to expand scope.

Where can people find the COA Compendium?

Federal Register notice seeking review and comment on the content and format of the compendium

What type of information are you hoping to obtain?

Seeking public feedback on the utility, approach for development, suggestions for future approaches to expand scope of COA Compendium




Clinical Outcome Assessment (COA) Compendium


  • Describes clinical outcome assessments to support labeling claims
  • Identifies clinical outcome assessments qualified for potential use
  • Recognizes ongoing qualification projects


  • Not a comprehensive list
  • Sponsors strongly encouraged to seek advice from the relevant Office of New Drug (OND) review division early in drug development
  • Inclusion in COA Compendium does not equate to an FDA endorsement


  • CDER’s DDT COA Qualification Program
  • Drug Labeling Approved From 2003 to 2014 (New Molecular Entity (NME) labeling)


  • Importance of collaborative development of COAs in response to unmet measurement needs
  • Provides regulatory conclusion on specific COA, specific interpretation and application in regulatory review
  • Inclusion of labeled COA serve as additional resource


  • Table alphabetically listing conditions or diseases
  • Six Columns :
    • Disease Condition
    • Indication and/or Claim(s) Description
    • Outcome of Interest
    • COA (COA Type)
    • COA Context of Use
    • COA Qualification Information


  • Not a comprehensive list
  • Not a replacement for FDA interactions or supersede existing guidances

FDA seeking public feedback 
FDA will host Webinar


fda guidances


CDRH Draft Guidance on Cybersecurity

  • Recommendations for monitoring, identifying and addressing cybersecurity vulnerabilities and exploits as part of medical device management
  • General principles for Pre-Market and Post-Market considerations, essential clinical performance
  • Risk management based on exploitability of the cybersecurity vulnerability, and health impact severity if the vulnerability were to be exploited
  • Remediating and Reporting vulnerabilities including controlled and uncontrolled risks to essential clinical management
  • Content for PMA Periodic Reports including description of vulnerability, sponsor conclusion and description of changes made
  • Elements of an Effective Postmarket Cybersecurity Program : Identify, Protect/Detect, Protect/Respond/Recover
  • To be discussed at the Jan 2021 FDA Workshop on Cybersecurity





POCA – Phonetic and Orthographic Computer Analysis

  • Part of CDER, Office of Surveillance and Epidemiology (OSE), Division of Medication Errors Prevention and Analysis (DMEPA)’s mission to prevent medication errors due to proprietary name confusion
  • POCA is a web application to determine written and phonetic similarities between proposed drug names using advanced algorithms
  • Compares a drug name against multiple drug names found in several different “data sources” contained in the software
  • FDA is providing zip files to download and install to help Sponsor evaluate potemtial proprietary names vs FDA database. – ‘Drugs @ FDA’ and ‘RxNorm’





drug links

WHAT : Drug Industry -specific resources pages focused on information that serves as a guide to key information




WHAT : CELP serves as a liaison between the FDA and the cardiovascular and endocrine health professional and patient communities. CELP encourages and supports active participation in forming FDA regulatory policies that promote healthy dietary and nutrition practices and advance the safety and effectiveness of human medical products that treat diabetes, hypertension, heart disease, and obesity.

WHO : Your organization can collaborate with FDA

HOW : Phone: 301-796-8460, Cardio.Endo@fda.hhs.gov



Mechanism of Action

Fixed-dose combination of antiretroviral drugs elvitegravir (EVG), boosted by the CYP3A inhibitor cobicistat (COBI), emtricitabine (FTC), and tenofovir alafenamide(TAF)

Pharmacodynamics (PD)

Effect on QT interval is not known

Pharmacokinetics (PK)

Median Tmax: EVG: 4 hours; COBI: 3 hours; FTC: 3 hours; TAF: 1 hour

Food Effect: EVG AUC was increased by 34% and 87% when GENVOYA was co-administered with a light meal and high fat meal, respectively. No clinically meaningful food effect on COBI, FTC or TAF was observed.

Median Terminal half-lives: EVG: 12.9 hours; COBI: 3.5 hours ; FTC: 10 hours; TAF: 0.51 hours

Metabolism: EVG: CYP3A (major) and UGT1A1/3 (minor): COBI: CYP3A (major) and CYP2D6 (minor): FTC: Not significantly metabolized; TAF: Cathepsin A (major), carboxylesterase 1 (minor), and CYP3A (minimal)

Excretion: FTC is excreted renally; TFV (the primary metabolite of TAF) is excreted renally; EVG and COBI metabolites are primarily eliminated in feces

Plasma protein binding: EVG: ~99%;  COBI: ~98% ; FTC: <4%; TAF: ~80%

PK-PD Analysis

No exposure-response relationships for safety or efficacy were identified for any of the components of GENVOYA at the approved recommended dosage.

Population PK

No dosage adjustment is recommended based on race or gender

Special Populations

No dosage adjustment is recommended based on creatinine clearance ≥30 mL/min, mild (Child-Pugh Class A) or moderate (Child-Pugh Class B) hepatic impairment, or age ≥12 to ≤75 years.

Renal Impairment: GENVOYA is not recommended in patients with estimated creatinine clearance below 30 mL/min.

Hepatic Impairment: GENVOYA is not recommended in patients with severe hepatic impairment (Child-Pugh Class C).

Drug Interactions

GENVOYA can alter the concentration of drugs metabolized by CYP3A or CYP2D6.

Coadministration of GENVOYA is contraindicated with drugs that are highly dependent on CYP3A for clearance and for which elevated plasma concentrations are associated with serious and/or life-threatening events, and drugs that may lead to reduced efficacy of GENVOYA and possible resistance, such as potent CYP450 inducers.

Coadministration of GENVOYA is not recommended with rifabutin, rifapentine, salmeterol, and/or colchicine in patients in patients with renal or hepatic impairment.

Consider alternatives to the coadministration of GENVOYA with oxcarbazepine, systemic dexamethasone, and/or inhaled/nasal fluticasone.

Consider alternatives to the coadministration of GENVOYA with contraceptive patch, vaginal ring, injectable contraceptives, and oral contraceptives containing progestogens other than norgestimate.

Dose reduction may be necessary for sedative/hypnotics, neuroleptics, midazolam, and beta blockers when coadministered with GENVOYA.

Specific dosage regimens are required for phosphodiesterase-5 inhibitors, colchicine, and bosentan when coadministered with GENVOYA.

Initiate with the lowest starting dose of atorvastatin when coadministered with GENVOYA and titrate carefully while monitoring for safety.

Separate GENVOYA and antacid administration by at least 2 hours.

The maximum daily dosage of ketoconazole or itraconazole should not exceed 200 mg per day when coadministered with GENVOYA. Assessment of benefit/risk is recommended to justify use with voriconazole.



FDA Brief, Week of Jan 4th, 2016

FDA Voice

2015: Another Strong Year for Patients in Need of New Drug Therapies

By: John K. Jenkins, M.D. Director of the Office of New Drugs, CDER


Strong year for novel new drug approvals:  45 novel new therapies vs. average of 28 over the previous nine years

  • Cancers : Multiple myeloma (4), lung, skin, breast, brain, colorectal, and other cancers.
  • Heart failure, high cholesterol, cystic fibrosis, irritable bowel syndrome
  • Rare Diseases

Approval Highlights

  • 36% : “First-in-class,” : Addyi, Bridion, Corlanor, Cosentyx, Darzalex, Empliciti, Entresto, Ibrance, Kanuma, Nucala, Orkambi, Praluent, Praxbind, Strensiq, Unituxin, Xuriden
  • 40% : rare or “orphan” diseases: Alecensa, Cholbam, Cotellic, Cresemba, Darzalex, Empliciti, Farydak, Kanuma, Lenvima, Natpara, Ninlaro, Orkambi, Portrazza, Praxbind, Repatha*, Strensiq, Tagrisso, Unituxin, Uptravi, Xuriden, Yondelis
  • First reversal agent for a blood thinner: Praxbind
  • 60% : Designated in one or more categories for Expedited Development and Review methods
    • Fast Track 31%
    • Breakthrough 22%
    • Priority Review 53%
    • Accelerated Approval 13%
  • 64% approved first in the US
  • 96% PDUFA goal dates met
  • 87% “first cycle” approval
  • Qualified Infectious Disease Product (QIDP, GAIN Act): 2


FDA 2015: A Look Back (and Ahead) – Part 2: Medical Product Safety and Oversight

By: Stephen M. Ostroff, M.D., Acting Commissioner of Food and Drugs

Reflection on FDA’s impact on medical product safety and oversight.

Responding to Ebola : Expediting availability of diagnostic tests, investigational therapeutics, vaccines;  investigating fraudulent products

Addressing Transmission of Infections from Duodenoscopes : Safety communication, public Advisory Committee meeting, Warning letters,  Postmarket surveillance studies on reprocessing in real-world clinical settings.

Compounding:  Policy documents, meetings with stakeholders, including pharmacy, physician, and consumer groups, and collaborations with state governments.

Addressing the Opioid Abuse Crisis:  Multipronged approach to balance availability of medical treatments vs tragic consequences of abuse and misuse. Guidance for development of abuse-deterrent opioid formulations, options for medication-assisted treatment of opioid-dependence, treat consequences of overdoses.


FDA approved


Integra Omnigraft Dermal Regeneration Matrix to treat diabetic foot ulcers

Integra LifeSciences Corporation of Plainsboro, New Jersey

Indication for Use : Treat certain diabetic foot ulcers that last for longer than six weeks and do not involve exposure of the joint capsule, tendon or bone, when used in conjunction with standard diabetic ulcer care.

Unmet need :

  • 29 million people in US diagnosed with diabetes; 25% will experience foot ulcer during their lifetime
  • Chronic diabetic foot ulcers associated with tissue and bone infections and result in 50,000 amputations each year.
  • Need for new innovation in diabetes care with the potential to improve the number of foot ulcers that heal

Reg Pathway : PMA Supplement

– PMA (1996) : Treatment of life threatening burn injuries when the use of a patient’s own skin for a graft was not possible

– PMA Supplement (2002) : Treat patients undergoing reconstructive surgery for burn scars when they cannot have skin grafts

Effectiveness :

  • Study, a multi-center, randomized, controlled, parallel group clinical trial conducted under an Investigational Device Exemption (IDE). Omnigraft  vs. standard of care (includes cleaning and covering the wound with a surgical bandage and keeping weight off of the foot with the ulcer)
  • 51 percent of patients treated with Omnigraft had healed ulcers after 16 weeks compared to 32 percent of patients treated with standard diabetic foot ulcer care alone

Safety :  Adverse events included infections, increased pain, swelling, nausea, and new or worsening ulcers


fda guidances


Draft Guidance : Hearing Aid Devices and Personal Sound Amplification Products

  • New efforts to balance patient safety vs encouraging advancements in hearing aid technology and patient access
  • Hearing aids often underutilized medical device
  •  Most hearing aids are Class I (low-risk);  possible modifications to regulation to a “decrease in cost and improvement in capability, convenience and use of assistive hearing devices.”
  • Public workshop :  “Streamlining Good Manufacturing Practices (GMPs) for Hearing Aids,” April 21, 2016 : Alternative regulatory models to accelerate innovation while enabling quality design and manufacturing
  • Re-opened Public Comment period on draft guidance: Through May 19, 2016





Noxafil (posaconazole)

  • Dosing errors when switching between different oral formulations of antifungal
  • Drug labels revised to indicate that the two oral formulations cannot be directly substituted for each other but require a change in doseP
    • Prescribers – Specify the dosage form, strength, and frequency
    • Pharmacists – Request clarification from prescribers when the dosage form, strength, or frequency not specified
    • Patients – Talk to their health care professional before switching


Surgical Mesh for the Transvaginal Repair of Pelvic Organ Prolapse

  • Strengthened data requirements for  to address safety risks
  • Two final orders to manufacturers and the public
    • Reclassify from class II (moderate-risk) to class III (high-risk)
    • Require manufacturers to submit PMA application for safety and effectiveness




  • Transparency – provide interested parties an unprecedented look into how FDA performs its work.
  • Results – highlights performance measures and results with relevance to the agency’s public health mission.
  • Accountability – requires senior managers to develop, track, and report performance measures that will improve the agency’s accountability to the public; holds the program offices accountable for their priorities, plans and results.
  • Credibility – encourages sharing of information about FDA performance which is essential for the agency’s credibility; provides the opportunity to submit suggestions which will be considered as part of the continuous improvement efforts.
  • Knowledge-sharing – enables the identification of common issues and interdependencies among program offices to improve FDA’s operational effectiveness through better collaboration and sharing of ideas.



Nanoparticles in Drug Development


Nanoparticle technologies have revolutionized the drug development process and change the landscape of the drug industry. Nanoparticles (NP) could improve the aqueous solubility, dissolution rate, and absorption of poorly water-soluble substances.

Addressing Pharmaceutical Unmet Need : Major issues associated with poorly water-soluble compounds are poor bioavailability, fed/fasted variation in bioavailability, lack of dose-response proportionality and that leads to suboptimal dosing, need a pharmacokinetic (PK) booster to enhance bioavailability, and noncompliance by the patient, i.e., inconvenience dosing schedules.

Characteristics: NP can be defined as solid particles with a size in the range of 10-1000nm. The increased surface area of NP will enhance the dissolution of poorly water soluble drug substances. The chance gravitational settling is less for NP  because of their small size and can be suspended easily in liquid formulation. The NP approach is suitable for highly potent compounds (low dose) but it is not practical when the dose requirement may be big. In recent years nanotechnology is gaining popularity by formulators for reformulating the old formulation of poorly soluble compounds. For marketed products requiring lifecycle extension opportunities, NP formulation strategies provide a means to develop a new drug-delivery platform with improved therapeutic outcome incorporating the existing drug, thus creating new avenues for addressing unmet medical needs.

Pharmaceutical Application : An increasing number of the drug candidates synthesized each year by pharmaceutical companies which may have poor water solubility and many of these promising compounds are rejected from development because traditional formulation approaches will not be able to improve bioavailability. Nanotechnology could rescue these compounds. For new chemical entities development, the nanotechnology can be of great value in preclinical pharmacokinetics and efficacy/ safety assessment studies in the early development phase. Drug formulation with NP could reduce or eliminate food-effect related variability resulting in no dosing restriction since NP improve the solubility of drug substances. There are several techniques to make drug NP out in the literature, e.g bottom-up (Precipitation methods), top-down etc.

Disposition : Enhancing solubility and dissolution rate of poorly soluble compounds correlates with improved PK profile since dissolution kinetics is the primary driving force behind the improved PK/ bioavailability of NP formulations of poorly water soluble compounds. The enhanced bioavailability should be translated to faster onset of action that may translate into improved therapeutic outcome.

Marketed products: Oral products developed using nanoparticle technology are in the market and listed below: Rapamune (Sirolimus); Emend (Aprepitant), TriCor (Fenofibrate), MegaceES (Megestrol). Avinaza (Morphine sulphate),  Focalin (Dexmethyl-phenidate HCl),  and Zanaflex (Trizanidine HCl) and the intravenous products: intravenous nutritional fat emulsion (Intralipid) and liposomal products (Doxil, AmBisome).

FDA BRIEF, Weeks of Dec 21st and Dec 28th, 2015



FDA Voice



FDA 2015: A Look Back (and Ahead) – Part 1: Medical Product Innovation

By: Stephen M. Ostroff, M.D.

 Reflection of  FDA’s  2015 accomplishments by  Acting Commissioner

Medical Product Approvals

  • > 40 approvals including 4 new treatments for multiple myeloma, 2 new drugs for heart failure, and several drugs for  rare or “orphan” diseases
  • Several important vaccines, including meningococcal disease, seasonal influenza vaccine for elderly, anthrax
  • Several innovative devices  including brain cancer, congenital heart disease
  • Speed and efficiency in both drug and device approvals

Amplifying the Patient Voice

  • Patient-Focused Drug Development initiative
  • Voice of the Patient report (drug)
  • Patient Preference Initiative (device)
  • Draft guidance, Patient Engagement Advisory Committee


  • First biosimilar approved
  • Several Guidances

Next Generation Sequencing Tests and Strengthening Clinical Trials

  • Growing ability to apply technologies of next generation sequencing and precision medicine
  • Precision Medicine Initiative, precisionFDA web platform
  • Refine clinical trial design and statistical methods of analysis to create more efficient studies with smaller patient populations, more focused therapies, and better outcomes


FDA Invites Patient Organizations to Take a Place at the Podium

By: Theresa M. Mullin, Ph.D.

Reflection on Patient-Focused Drug Development (PFDD) program by is Director of FDA’s Office of Strategic Programs

  • More first-hand knowledge from those most affected by the diseases
  • Better understand context for regulatory decision making
  • Committed to hold meetings for at least 20 disease areas & 24 disease-focused meetings 2017
  • Encourage patient organizations in conducting an externally-led PFDD meeting and keeping FDA informed



FDA approved


ardea    ZURAMPIC  (lesinurad)


Indication: In combination with a xanthine oxidase inhibitor for the treatment of hyperuricemia associated with gout in patients who have not achieved target serum uric acid levels with a xanthine oxidase (XO)  inhibitor alone

 Unmet need:

  • Gout is a painful form of arthritis caused by uric acid buildup
  • Need for new treatment option for controlling hyperuricemia for the long-term treatment of gout

Reg pathway: NDA Standard Review

Mechanism of Action: Inhibits transporter proteins functions involved in uric acid reabsorption in the kidney and reduces serum uric acid levels


  • 3 multicenter, randomized, double-blind, placebo-controlled studies, Patients with hyperuricemia and gout
  • Zurampic + XO inh (allopurinol or febuxostat) vs XO inh
  • N=511, 510, 516, Duration :  12 mo.
  • Superior lowering of serum uric acid to less than 6 mg/dL at 6 mo.


  • Boxed Warning : Risk for acute kidney (renal) failure
  • Common Adverse Reaction : Headache, influenza, increased blood creatinine, and gastroesophageal reflux disease
  • Postmarketing Study : Renal and Cardiovascular safety



UPTRAVI (selexipag)


Indication : Treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression and reduce the risk of hospitalization for PAH

Unmet Need:

  • PAH is high blood pressure in arteries connecting heart to lungs; can lead to limitations on exercise ability and shortness of breath
  • Need for additional treatment option for PAH patients

Reg Pathway : NDA – Standard review, Orphan Drug Designation

Mechanism of  Action:  Oral prostacyclin receptor agonist


  • Single multi-center, double-blind parallel group, N=1156, Uptravi vs Placebo, 1.4 yrs
  • Primary endpoint : Time to first occurrence of : death, hospitalization for PAH, PAH worsening,  initiation of parenteral prostanoid therapy or chronic oxygen therapy, other disease progression
  • 40% reduction (99% CI: p-value < 0.0001) of primary endpoint events vs placebo  – primarily due to reduction in hospitalization for PAH and reduction  disease progression events


  • Common Adverse Reaction : Headache, diarrhea, jaw pain, nausea, muscle pain (myalgia), vomiting, pain in an extremity, and flushing



FENIX  Continence Restoration System

Torax Medical Inc, Shoreview, Minnesota

Indication for Use: Treat fecal incontinence in patients who are not candidates for, or have previously failed, medical or other surgical options

Unmet Need:

  • Fecal incontinence caused by damage anal sphincter muscles; frequently underreported
  • Need for viable surgical option other non-invasive methods have failed

Reg Pathway: PMA, Humanitarian Device Exemption (HDE) process


  • 3 components: Implant, Anal Sphincter Sizing Tool, Introducer Tool
    • Implant : Titanium beads with magnetic cores; minimizes involuntary opening of anal canal
    • Sizing Tool : Associates anal sphincter size to appropriate implant
    • Introducer Tool : Guides sizing tool and implant into position


  • Study (n=35), evaluations before and after procedure and at 6 wks, 3 mo, 6 mo, 12 mo
  • Bowel diary to track fecal incontinence events & validated questionnaire (Fecal Incontinence Quality of Life Scales)
  • 50% reduction after 12 mo: 9 %  (fecal incontinence episodes), 54.3 % (fecal incontinence days), 37.1 % (urgent episodes)
  • Improvements in Quality of Life measures : depression, self-perception, embarrassment


  • Adverse Events : Pain, infection, impaction or defecatory disorder, device erosion, device removal/re-operation, and bleeding
  • Patients required register with MedicAlert Foundation


fda guidances.JPG


emerging signals

  • Need for FDA to notify the public about emerging signals that the Agency is monitoring or analyzing
  • Proposes criteria, timeframes, communication, follow-up for emerging signals that may impact benefit/risk
  • Emerging Signals : New adverse event, increase in severity/frequency,  new product-product interactions, device malfunctions, patient injuries,  reduction in benefit
  • FDA will assess of need for communication : Within 30 days of receipt of information
  • If yes, FDA will communicate using standardized format and provide updates


  • Electroconvulsive Therapy (ECT) Devices for Severe Major Depressive Episode associated with Major Depressive Disorder or Bipolar Disorder reclassified  into class II from class III
  • Guidance provides recommendations for complying with the special controls and for 510(k) information for special controls


  • Specific emerging technology review assigned to a group within CDER (Emerging Technology Team – ETT) to collaborate with sponsor to implement emerging manufacturing technology
  • IND, NDA, BLA, ANDA implementation– specific discussion meetings to present proposal and align on requirements


CDRH Fiscal Year 2016 (FY 2016) Proposed Guidance Development and Focused Retrospective Review of Final Guidance


A List

Final Guidance Topics

  • General Wellness Products
  • Medical Device Accessories
  • Benefit-Risk Factors to Consider when Reviewing IDE Submissions
  • UDI Direct Marking
  • Adaptive Design for Medical Device Clinical Studies
  • Incorporating Patient Preferences into Medical Devices Premarket Approvals, Humanitarian Device Exemptions, and De Novo Classifications
  • Applying Human Factors & Usability Engineering to Optimize Medical Device Design
  • Policy for Regulatory Oversight of Laboratory Developed Tests (LDTs)
  • Submission and Review of Sterility Information for Devices Labeled as Sterile
  • Use of ISO 10993-1, Biological Evaluation of Medical Devices Part I: Evaluation and Testing
  • Postmarket Surveillance Studies
  • Medical Device Reporting (MDR) for Manufacturers

Draft Guidance Topics

  • Medical Device Decision Support Software
  • Use of Symbols in Labeling
  • 510(k) Modifications
  • Software Modifications
  • 510(k) Third Party Review Program
  • Companion Diagnostics Co-Development
  • Use of Real-World Observational Patient Data to Support Decision Making for Medical Devices
  • UDI Convenience Kit
  • Public Notification of Emerging Postmarket Medical Device Signals

There is also a B List


HELPFUL LINKS : Drug Approvals and Databases



Clinical Investigator Inspection List (CLIIL)

WHAT : Names, addresses, pertinent information FDA inspections of clinical investigators

WHEN : August 1977-present,  updated quarterly





Bioresearch Monitoring Information System (BMIS)

WHAT : Clinical investigators, contract research organizations, and institutional review boards involved in IND studies, Based on information from 1572

WHEN : Updated quarterly





NEW OFFICE: Office of Dietary Supplement Programs (ODSP)

WHY : Need to take action against fraudulent supplements that can cause serious risk to consumer (e.g.  egregious benefit claims in treating serious diseases) or widespread economic fraud.

WHAT : Newly created; to enhance effectiveness of dietary supplement regulation

WHO : Under CFSAN, will work closely with CDER to help remove falsely labeled supplements