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How Proposed Regulatory Reforms Will Affect Your Clinical Trial

21st Century Cures (also called Cures2015) is a bipartisan initiative undertaken by the Committee on Energy and Commerce of the US House of Representatives. Amongst the many factors motivating this landmark legislation is the concern  that regulatory procedures must keep up to date with innovations in clinical development. Cures2015 aims to reform the regulation of drugs, biologics and medical devices while increasing patient access to safe and effective drugs across therapeutic areas.

21st Century Cures presented a draft bill in January 2015 that would affect several regulatory procedures at agencies like the FDA. Included in the various proposals are reforms to the regulation of adaptive clinical trials and the use of Bayesian methods, and expedited approval processes for breakthrough therapies and medical devices. The landmark legislation also incentives improvements to Phase 2 trial design for certain therapeutic areas.   

Adaptive Clinical Trials & Bayesian Methods

The provisions of the draft bill which relate to adaptive clinical trials and the use of Bayesian methodology are largely found in Title III, Subtitle B, aptly named ‘Broader Application of Bayesian Statistics and Adaptive Trial Designs.’ The following proposals are included in this section:   

  • The Secretary of Health and Human Services should finalize the draft guidance documents on Adaptive Design Clinical Trials for Drugs and Biologics, and on the Guidance for the use of Bayesian Statistics in Medical Device Clinical Trials (p. 233).
  • The Secretary of Health and Human Services should revise the list of acceptable statistical standards within four years of signing the bill into law, in order to reflect advances in statistical methods (p. 235).
  • The Secretary of Health and Human Services ought to hold a public meeting with all stakeholders (including trial sponsors, academics, patient advocates and members of the FDA) before updating the list of appropriate statistical methods, and should do so within a year of the bill being signed into law (p. 234).  

Breakthrough Therapies

Title I Subtitle 3 is entitled ‘Approval of Breakthrough Therapies.' Proposals in this section include requirements that:   

  • There should be guidelines for the approval of breakthrough therapies for serious or life-threatening diseases or conditions based on satisfactory completion of early stage studies (e.g. one or more Phase 2 studies) when those studies provide ‘sufficient evidence for approval of the drug as safe and effective…’ (p. 29 – 30).
  • The Secretary of Health and Human Services may approve breakthrough therapies based on Phase 2 studies conditional on post-market review (p. 30). 
  • The publication of guidance for approval of such breakthrough therapies should be available within twelve months of the bill being signed into law (p. 33-34).

Medical Devices

Title I Subtitle E is entitled ‘Priority Review for Breakthrough Devices,’ while Title I Subtitle F is entitled ‘Accelerated Approval for Breakthrough Devices.’ These sections of the draft bill propose that:

  • Breakthrough devices which offer ‘significant advantages over existing approved or cleared alternatives’  should be candidates for priority review (p. 72-73).
  • Breakthrough devices whose availability can provide a variety of benefits to patients (e.g. improve quality of life, or empower patients to manage their own care,) should be viewed as candidates for priority review (p. 73).
  • Breakthrough devices which are in the best interest of patients should be considered candidates for priority review (p. 73).
  • Accelerated approval should be available for certain medical devices where an effect on a surrogate endpoint is ‘reasonably likely to predict clinical benefit, or on a clinical endpoint that can be measured earlier than irreversible morbidity or mortality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit.’ However, such approval may also be conditional on post-market review. (p. 81).

Related Items of Interest

21st Century Cures Act (Draft Bill, January 2015)

21st Century Cures Discussion Document

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