COVID-19 News: VEKLURY for COVID treatment, Advisory committee for vaccines, Evidence Accelerator for real world data, New NextGen portal for manufacturing capacity, New Sentinel website for pandemic activities

VEKLURY (remdesivir) injection

Gilead

INDICATION: For adults and pediatric patients (12 years of age and older and weighing at least 40 kg) for the treatment of coronavirus disease 2019 (COVID-19) requiring hospitalization. VEKLURY should only be administered in a hospital or in a healthcare setting capable of providing acute care comparable to inpatient hospital care

ADDRESSING UNMET NEED: First treatment for COVID-19

MECHANISM OF ACTION: Inhibitor of the SARS-CoV-2 RNA-dependent RNA polymerase (RdRp), which is essential for viral replication

EFFICACY: Three randomized, controlled clinical trials that included patients hospitalized with mild-to-severe COVID-19. Recovery was defined as either being discharged from the hospital or being hospitalized but not requiring supplemental oxygen and no longer requiring ongoing medical care

  • Median time to recovery was significantly lower with Veklury vs. placebo
  • Odds of clinical improvement were also statistically significantly higher or numerically better in the Veklury group when compared to the placebo group. 

SAFETY: Possible side effects include: increased levels of liver enzymes, which may be a sign of liver injury; and allergic reactions, which may include changes in blood pressure and heart rate, low blood oxygen level, fever, shortness of breath, wheezing, swelling (e.g., lips, around eyes, under the skin), rash, nausea, sweating or shivering

REGULATORY PATHWAY: NDA

  • Prior EUA approval
  • Fast Track and Priority Review designations
  • Material Threat Medical Countermeasure Priority Review Voucher
  • Postmarketing pediatric study requirement (birth to <18 years, including neonates)
  • Postmarketing study in or remdesivir resistant SARS-CoV-2 variants  

LABEL , FDA Review Summary

COVID-19 Vaccines and FDA Advisory Committee

FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) met on 10/22 to discuss development, authorization and/or licensure of COVID-19 vaccines

  • Committee members are external scientific and public health experts from around the country, specializing in fields such as immunology, virology, infectious diseases, pediatrics, vaccine development, and vaccine safety.
  • Presentations from the experts as well as FDA scientists on COVID-19 disease and vaccine development
  • Thorough discussion on studies needed to support authorization or approval, post-marketing safety studies, ongoing safety monitoring following issuance of an emergency use authorization
  • Members of the public speak and provide input
  • Overall, to help ensure clear public understanding of the data needed for FDA authorization or approval of COVID-19 vaccines, as well as plans for their continued evaluation following authorization or approval  

Read , Event materials , To view the webcast replay, visit FDA’s YouTube Channel.

COVID-19 Evidence Accelerator: An initiative launched by the Reagan-Udall Foundation for the FDA

To gather Real world data (RWD) to help advance our nation’s ability to respond to the pandemic

  • Informing and safely speeding diagnostic and therapeutic processes
  • Provide a unique venue for major data organizations, government and academic researchers, and health systems to gather and design quick-turn-around queries and share their results
  • Bring together leading experts in health data aggregation and analytics to share insights, compare results, and answer key questions about COVID-19 treatment and response as quickly as possible

READ

CDER NextGen portal includes manufacturing capacity data for COVID-19 drugs

CDER is gathering more information about manufacturing capacity for investigational COVID-19 products

  • Portal captures information about the specific IND, manufacturer, production rates, treatment courses, and allocations 
  • To analyze and support manufacturing efforts for potential vaccines and therapeutics such that there is a capability for mass production to meet the population need

Portal

New Sentinel website for the pandemic

Launch of newly redesigned Sentinel Initiative website to increase organization and usability and assist the Sentinel community

  • Coronavirus (COVID-19) activities, to keep up with how FDA is using Sentinel activities to protect and promote public health during the COVID-19 pandemic
  • Completed ARIA assessments & impact, for a list of safety-related outcomes, results, and regulatory impacts
  • Drug assessments, where you can search materials based on medical product, safety outcome, and study type
  • Publications & presentations, so you can read up on Sentinel activities in journals and conference materials

Website


Image credit: FDA

News and Views: Orphan products grants, Cybersecurity, Opioid disposal toolkit, Opioid Use Disorder guidance, Breast implants patient labeling, Flu vaccine

Funding to advance medical products for the treatment of rare diseases

 Orphan Drugs are for rare diseases/disorders that affect fewer than 200,000 people in the US

Six new clinical trial research grants to principal investigators from academia and industry totaling over $16 million over the next four years 

  • Stargardt disease
  • Prevention of graft versus host disease
  • Cystic fibrosis patients colonized with nontuberculosis mycobacterium
  • CD4 positive T cell neoplasms
  • Pancreatic cancer
  • Peripheral T-cell lymphoma

READ


FDA and Cybersecurity

Medical devices are increasingly connected to the Internet, hospital networks, and other medical devices to improve health care

  • Increase the risk of potential cybersecurity threats and impact device safety and effectiveness
  • Reducing cybersecurity risks is important but challenging

Fact Sheet on FDA’s role in medical device cybersecurity

  • FDA works closely with several federal government agencies including the U.S. Department of Homeland Security
  • Manufacturers must address pre-market and post-market cybersecurity risks; make updates to strengthen cybersecurity
  • Manufacturer is responsible for software validation to address cybersecurity vulnerabilities, conduct pre-market testing including the use of OTS software

Fact sheet


Safe Opioid Disposal ‘Remove the Risk’ Outreach Toolkit

Remove the Risk raises awareness of the serious dangers of keeping unused opioid pain medicines in the home and provides information about safe disposal of these medicines

  • Free toolkit materials—public service announcements (PSAs), social media images and posts, fact sheets
  • Designed to help groups speak with others about safe opioid disposal

Remove the Risk


Guidance to Encourage Development of Novel Medicines to Treat Opioid Use Disorder (OUD)

Guidance addresses acceptable clinical endpoints for demonstrating effectiveness to treat OUD and to predict a sustained clinical benefit

  • Reductions in adverse outcomes related to OUD (mortality, emergency medical interventions, Hep C infections)
  • Change in disease status using OUD diagnostic criteria
  • Change in drug use pattern
  • Patient Reported Outcomes (e.g., improvement in sleep or mood)
  • Other outcome measures (e.g. ability to resume work, school, or other productive activity)

Guidance


Breast Implants Labeling Recommendations to Improve Patient Communication

FDA guidances to help ensure that a patient receives and understands the benefits and risks of breast implants

  • Labeling for saline/silicone breast implants for breast augmentation or breast reconstruction
  • Important for patients to have benefit and risk information for a balanced discussion with their physicians
  • Includes: Boxed Warning, Patient Decision checklist, Rupture Screening recommendation, Material/Device description, Patient Device card

Guidance


FDA approved Flu vaccines provide important benefits such as preventing illnesses, hospitalizations and deaths

  • Likely that flu viruses and COVID-19 will circulate together this fall and winter; flu shot will not prevent COVID-19 but help to keep you healthy and preserve health care resources for COVID-19
  • Annual vaccination to prevent flu is the best way to reduce the risk of getting the flu and spreading it to others
  • Annual shots are needed because flu viruses can change from year to year, so the vaccine is updated; FDA, WHO, CDC, and public health experts collaborate to identify likely strains
  • Typically, children and older people are most at risk of getting sick with influenza
  • FDA ensures flu vaccine is safe, effective, and of high quality

READ


Image credits: FDA, DHS

COVID-19: CDRH response to date, Patient outreach toolkit, Laboratory safety, Vaccine EUA guidance, Medical countermeasures R&D

Center for Devices and Radiological Health’s (CDRH) Response to Coronavirus (COVID-19)

Timeline: January 1 – September 18, 2020

  • Regulatory Flexibility: EUA templates, guidance documents, policies
  • Emergency Use Authorizations: 10x medical devices the number authorized pre-2020, tests
  • Shortage Mitigation Activities: Outreach to 1,000+ manufacturing sites across 12 countries
  • Public Health Service Corps Deployment: 58 CDRH Commissioned Corps officers
  • Engagement with Stakeholders: Emails, phine queries, webinars, townhalls

fda.gov/medical-devices


Patient Outreach Toolkit

Useful COVID-19 resources for healthcare professionals to share with patients

Fraudulent Products Factsheet
(English / Español – PDF, 1.2MB)

FDA Response to COVID-19: What You Need to Know Infographic
(English / Español – PDF, 4.4MB)

Approved COVID-19 Tweets
(English / Español – PDF, 1.6MB)


A Culture of Responsibility: Laboratory Safety at the FDA

In observance on NIH’s Biosafety & Biosecurity Month, FDA is implementing the folloiwng in its labs

  • Proper use of PPE (masks, face shields, gloves, gowns, shoe covers)
  • Engineering controls (biosafety cabinets, secure rooms)
  • Administrative controls (risk assessment, pre-work approval, medical surveillance, respiratory protection programs)
  • Best practices for decontaminating surfaces and proper procedures for packing and transporting any hazardous biomaterials, including SARS-CoV-2

READ


Industry Guidance: Emergency Use Authorization for
Vaccines to Prevent COVID-19

Data and information needed for Emergency Use Authorization (EUA) for an investigational COVID-19 vaccine that could be potentially administered to millions of individuals

  • Chemistry, manufacturing, and controls information (CMC); nonclinical data and information; and clinical data and information, as well as administrative and regulatory information

Safety and Effectiveness Information for evaluation of benefit-risk profile

  • Bioassays for assessment of clinical endpoints
  • Supportive nonclinical studies e.g., characterization of markers associated with enhanced disease, biodistribution, shedding, and attenuation)
  • Clinical
    a. Prespecified success criteria for primary efficacy endpoint (previous guidance), placebo controlled efficacy trial of at least 50%
    b. All safety with focus on serious adverse events, adverse events of special interest, and cases of severe COVID-19 among study subject
    c. Data from Phase 3 studies should include a median follow-up duration of at least two months after completion of the full vaccination regimen
  • In addition
    a. Local and systemic solicited adverse reactions for reactogenicity
    b. All safety data from database lack, n> 3,000
    c. Sufficient cases of severe COVID-19 among study subjects
    d. Sufficient cases of individuals with prior SARS-CoV-2 infection

READ


Cellular signaling and immune correlates for SARS-CoV-2 infection

Research award to Stanford University to help inform the development and FDA review of COVID-19 medical countermeasures

  • Explain host factors contributing to coronavirus immune responses to more rapidly predict patient outcomes
  • Innovative in-depth analysis of existing tissue samples from previous clinical and nonclinical studies
  • Profile circulating immune signatures of coronavirus infection, pathology, tissue imaging, tissue viral reservoirs, how different systems in the body are affected

READ


Image credit: FDA, Stanford University