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MINNESOTA LIVING WITH HEART FAILURE QUESTIONNAIRE (MLHFQ)

University of Minnesota

QUALIFIED CONTEXT OF USE: The paper self-administered version of the MLHFQ can be used to determine whether a device treatment is effective for improving patients’ quality of life by reducing the adverse impact of heart failure.

The instrument can be used as a secondary endpoint in feasibility and pivotal studies of outpatients with symptomatic (NYHA class II and III) heart failure. The 21-item instrument is completed by patients after they have been properly instructed by study staff. Study staff should be properly trained to instruct the patient and if needed, administer the questionnaire, according to pre-set administration instructions.

The MLHFQ instrument may be used by medical device companies and sponsor-investigators in controlled clinical trials designed to test superiority or non-inferiority of medical devices in support of regulatory submissions.

TOOL DESCRIPTION AND PRINCIPLE OF OPERATION:

  • 21-item paper self-administered questionnaire as a measure of heart failure
  • Quantifies overall score as measure of impact of heart failure on patient’s life
    • Physical symptoms and signs of heart failure
    • Common physical/social functions
    • Psychosocial and cognitive function
    • Adverse impact on quality of life

SUMMARY OF EVIDENCE TO SUPPORT QUALIFICATION:

  • Long history of use in medical device and pharmaceutical clinical studies
  • Additional publications evaluating the psychometric and statistical properties of the score
  • Publications support reliability and validity of the MLHFQ total score

REGULATORY PATHWAY: Medical Device Development Tool Qualification

  • Clinical Outcome Assessment

Decision Summary


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ANDEXXA (coagulation factor Xa (recombinant), inactivated-zhzo) 

Lyophilized Powder for Solution For Intravenous Injection

Portola

INDICATION: For patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding

MECHANISM OF ACTION: Exerts procoagulant effect by binding and sequestering the FXa inhibitors, rivaroxaban and apixaban. Also binds and inhibits activity of Tissue Factor Pathway Inhibitor (TFPI)

EFFICACY:

Two prospective, randomized, placebo-controlled studies, healthy volunteers; examine percent change in anti-FXa activity, from baseline to nadir

  • Apixaban reversal: At 3 hours after the last apixaban dose (~ Cmax), ANDEXXA or placebo was administered, n=8
  • Rivaroxaban reversal: At 4 hours after the last rivaroxaban dose (~ Cmax), ANDEXXA or placebo was administered, n=13
  • Reduction in Anti-FXa Activity : Percent change from baseline in anti-FXa activity at its nadir was statistically significant (p < 0.0001) in favor of ANDEXXA

Ongoing multinational, prospective, single-arm, open-label study

  • ANDEXXA administered to patients taking FXa inhibitors who presented with acute major bleeding, n=185
  • Interim results : Median decrease from baseline to nadir was -93% for  apixaban and -90% for rivaroxaban

SAFETY:

  • Boxed Warning: Arterial and venous thromboembolic events, ischemic events, including myocardial infarction and ischemic stroke, cardiac arrest, sudden deaths
  • Most common adverse reactions: Urinary tract infections, pneumonia, infusion-related reactions

REGULATORY PATHWAY: BLA

  • Accelerated Approval based on change from baseline in anti-FXa activity in healthy volunteers; improvement in hemostasis has not been established
  • Continued approval may be contingent upon results of studies to demonstrate an improvement in hemostasis in patients

LABEL


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KYMRIAH (tisagenlecleucel)

Novartis

SUPPLEMENTAL INDICATION: For adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma

EFFICACY:

  • Single-arm, open-label, multi-center, phase 2 trial, adults with relapsed or refractory DLBCL and DLBCL after transformation from follicular lymphoma
  • Treated with at least two prior lines of therapy, including an anthracycline and rituximab, or relapsed following autologous hematopoietic stem cell transplant
  • Single infusion of tisagenlecleucel following completion of lymphodepleting chemotherapy, n=68
  • Overall response rate (ORR):  50% (95% CI: 37.6, 62.4)
  • Complete response (CR) rate: 32% (95% CI: 21.5, 44.8)
  • Estimated median response duration among patients in PR: 3.4 months

SAFETY:

  •  Most common adverse reactions: Cytokine release syndrome (CRS), infections-pathogen unspecified, pyrexia, diarrhea, nausea, fatigue, hypotension, edema, and headache. Because of the serious risks of CRS and neurologic toxicities
  • Has a Risk Evaluation and Mitigation Strategy (REMS)

REGULATORY PATHWAY: sBLA

  •  Priority review, Breakthrough Therapy designation, Orphan Product designation

LABEL


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AQUABEAM System 

Procept BioRobotics Corporation

INDICATION FOR USE: For the resection and removal of prostate tissue in males suffering from lower urinary tract symptoms (LUTS) due to benign prostatic hyperplasia

DESCRIPTION:

Personalized image-guided prostate tissue removal system that uses a high-velocity water jet to resect and remove a predetermined volume of tissue. Comprised of nine main components along with accessories

  • Conformal Planning Unit, Console, Motorpack, Roll Stand, Foot Pedal, Handpiece Articulating Arms, Articulating Arm, Handpiece, Scope

GENERIC DEVICE TYPE: Fluid jet system for prostate tissue removal.

Prescription device intended for the resection and removal of prostatic tissue for the treatment of benign prostatic hyperplasia (BPH). The device cuts tissue by using a pressurized jet of fluid delivered to the prostatic urethra. The device is able to image treatment area, or pairs with an imaging modality, to monitor treatment progress.

 EFFECTIVENESS & SAFETY:

  • Prospective, multicenter, international double-blinded randomized clinical trial,   AQUABEAM vs. standard transurethral resection ofthe prostate (TURP)
  • Primary efficacy endpoint: Change in International Prostate Symptom Score (IPSS) at 6 months
  • Primary safety endpoint: Occunence of Clavien-Dindo persistent grade 1 or grade 2 or higher perioperative complications at 3 months
  • Mean IPSS scores: Decreased from 22.9 at baseline to 5.9 at 6 months with AQUABEAM vs. from 22.2 at baseline to 6.8 in TURP group
  • Clavien-Dindo grade 1 persistent or grade 2 or higher event: 25.0% in AQUABEAM vs, 40.0% in TURP

IDENTIFED RISKS & MITIGATIONS:

  • Injury from device operation: Clinical performance testing, Animal testing, Labeling, Training
  • Adverse tissue reaction: Biocompatibility evaluation
  • Infection: Sterilization validation, Reprocessing validation, Shelf life testing
    Labeling
  • Failure to remove target tissue or removal of nontarget tissue: Clinical performance testing, Animal testing, Software verification, validation, and hazard analysis, Non-clinical performance testing, Labeling
  • Electrical shock or electromagnetic interference: Electrical safety testing, Electromagnetic compatibility testing, Labeling

REGULATORY PATHWAY; De Novo request

  • 21 CFR 876.4350
  • Regulation Name: Fluid jet system for prostate tissue removal
  • Regulatory Class: Class II
  • Product Code: PZP

CLASSIFICATION ORDER


Image credits: Unv. of Minnesota, Portola, Novartis, Procept

 

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