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Experts Pitch FDA on Policies to Improve Drug Development

Posted 08 November 2019 | By Michael Mezher 

Experts Pitch FDA on Policies to Improve Drug Development

The US Food and Drug Administration (FDA) on Thursday heard from more than two dozen stakeholders on opportunities for the agency to promote more effective drug development programs at a public workshop at its headquarters in Silver Spring, Maryland.
“Effective drug development programs leverage the best available scientific knowledge to characterize the benefits and risks of a potential product and generate the data necessary to support product approval,” FDA says, noting its recent efforts to advance the use of real-world evidence and patient preference information.
However, FDA says it is looking for recommendations on new policies that it could implement in the near-term that would have an impact on drug development across multiple therapeutic areas.
Policy Suggestions
During the meeting, speakers representing drugmakers, patient groups, research institutions and consulting firms suggested policies and approaches FDA could pursue to promote more effective drug development.
Jennifer Hamilton, head of precision medicine at Regeneron Pharmaceuticals, suggested that FDA and industry could make drug development “smarter” by collaborating on regulatory standards and guidance for using genomic data to support submissions and labeling changes.
“It’s important for the industry to have a formal guidance from the FDA on how we can apply genomics data, and it’s also important that we harmonize these approaches with global agencies,” Hamilton said.
Both Judith Prescott, executive director for safety assessment at Merck, and Andrew Emmett, head of US regulatory policy at Pfizer, suggested that FDA develop cross-divisional guidance for developing drugs to treat severely-debilitating or life-threatening (SDLT) diseases with high unmet medical need, such as amyloidosis and advanced heart failure.
Prior to developing such guidance, Emmett called on FDA to host a public workshop with the goal of developing a set of objective criteria to define SDLTs across therapeutic areas.
“The rationale for the development of a cross-divisional guidance relates to the benefits of an agreed approach across the divisions and the need for a clear and clearly defined development path for SDLT therapeutics,” Prescott said.
According to Prescott, case-by-case consideration for individual development programs in this area lead to delays and result in sponsors defaulting to a more conservative approach than necessary.
Both Prescott and Emmett pointed to the International Council for Harmonisation (ICH) S9 guideline on nonclinical evaluation of anticancer pharmaceuticals as a template for guidance that allows for expedited drug development for life-threatening diseases.
Emmett also said that Pfizer would like to see greater acceptance for novel trial designs to satisfy postmarketing commitments and postmarketing requirements, and applauded the agency’s recently revised guidance on postmarketing studies that explains whether postmarketing reports and the agency’s active postmarket risk identification and analysis (ARIA) system are sufficient to assess a product’s risks in the postmarket setting.
Paul Melmeyer, director of regulatory affairs for the Muscular Dystrophy Association, said that his organization hopes for “a more structured, transparent and extensive disease specific guidance development process,” noting that FDA had only developed a few disease specific guidances for neuromuscular diseases so far.
Calling for greater transparency from FDA, Andrew Gustafson, senior director for US regulatory policy and advocacy at GlaxoSmithKline, said that sponsors would benefit if FDA released review summaries for new indication efficacy supplements as it does for newly approved drugs and biologics.
Gustafson said the summaries that could inform development programs and lead to more focused meetings with FDA, and that submitting Freedom of Information Act (FOIA) requests for the summaries is inefficient.


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