How Can US FDA Bolster Drug Development? The Agency Wants Your Advice

Public meeting scheduled for November to seek recommendations for policies that FDA could implement short-term to promote drug development programs.

The US Food and Drug Administration is holding a public meeting in November to get stakeholder suggestions on how it can promote innovative and effective drug development programs across multiple therapeutic areas.

The purpose of the 7 November meeting is to "solicit specific, actionable policy suggestions" that could be implemented in the near-term by the review staff of the Center for Drug Evaluation and Research's Office of New Drugs, the agency said in a Federal Register notice to be published on 12 August.

The format of the meeting will differ from that of other gatherings as the agency will not be inviting specific presenters. Instead, it is asking interested stakeholders to make presentations to a panel of leaders from the OND review divisions.

FDA does not say what prompted the meeting, entitled "Promoting Effective Drug Development Programs: Opportunities and Priorities for FDA's Office of New Drugs." But the agency has long been addressing ways it might improve drug development.

The agency notes that it is engaged in multiple, high-priority policy initiatives to facilitate effective drug development, including the potential use of real-world evidence in regulatory decision-making and the inclusion of the patient perspective in drug development and review. The agency said the meeting will not focus on these topics since it has already released comprehensive plans to advance those efforts.

FDA specifies that it would like input from stakeholders about where OND can provide additional guidance or prioritize additional scientific discussion in the near-term to improve clarity and encourage effective drug development.

"Given that OND's portfolio includes a diverse spectrum of drugs and diseases, such input should focus on specific policy need for various clinical areas linked by a shared therapeutic context (e.g., drugs intended to treat serious, life-threatening rare diseases; non-serious, self-limited conditions, etc.), rather than focusing on any specific disease or condition," the notice states.

Extending Novel Trial Designs

The agency notes that over the past decade drugs have been targeted to the underlying genetic or molecular pathophysiology of a disease. However, FDA says precise targeting is not possible for many diseases. It asks for suggestions for guidance or policy development that OND could pursue to facilitate drug development for diseases not currently amenable to targeted therapies.

FDA also asks for stakeholders' views on the advantages and disadvantages of extending novel trial designs, such as the use of master protocols, to additional therapeutic areas and what guidance development it could provide. Novel trial designs have been used for serious and life-threatening diseases to study multiple therapies and multiple diseases under a common infrastructure.

CDER Director Janet Woodcock noted FDA's embrace of innovation in clinical trial design and the use of master protocols in a 2017 New England Journal of Medicine article she co-authored. (Also see "Master Protocols Are Both Welcome And Inevitable – US FDA's Woodcock" - Pink Sheet, 6 Jul, 2017.)

And last year, Woodcock highlighted the agency's desire to see sponsors use adaptive clinical trial designs more frequently. (Also see "Woodcock Hopes I-SPY 2 Trial Is 'Blazing The Trail' For Future Adaptive Design Uptake" - Pink Sheet, 12 Sep, 2018.)

The agency notes that sponsors may be reluctant to use innovative approaches to drug development given their uncertainty.

"Sometimes, a well-understood development pathway may be chosen solely because of existing precedents in the therapeutic area.  We would like to hear how OND can promote effective drug development when this tension exists," FDA says.

FDA also wants recommendations as to the topics it should address in future guidance documents. It says that if stakeholders believe that OND review divisions are implementing existing guidances in different ways that are not explained by case-specific features, this may indicate a need for the agency to revise guidance or develop additional policy.

The agency is also accepting written comments, which can be submitted until 7 January.