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FDA Invites Draft Guidance on Patient-Focused Drug Development

Posted 20 March 2018 | By Ana Mulero 

FDA Invites Draft Guidance on Patient-Focused Drug Development

The US Food and Drug Administration (FDA) is pushing stakeholders to help in developing patient-focused drug development (PFDD) guidance.

The push comes as officials from FDA’s Center for Drug Evaluation and Research (CDER), patient advocacy groups, and research organizations at a public workshop on Monday pointed to several existing barriers in collecting and incorporating patient experience data for purposes of drug development, such as a lack of a methodological framework for identifying what measures are the most important to patients.

FDA directives created by 21st Century Cures Act of 2016 led to the formulation of a plan last year for implementing provisions that related to PFDD. The plan consists of four guidances on “approaches and methods to bridge from initial patient-focused drug development meetings to fit-for-purpose tools to collect meaningful patient and caregiver input for ultimate use in regulatory decision-making.”

The latest reauthorization of the Prescription Drug User Fee Act (PDUFA) also included FDA commitments to better incorporate patients’ voice in drug development.

The planned guidances are aimed at addressing areas of needed policy clarifications, such as methods for collecting comprehensive patient input, and measures for identifying specific set of impacts (e.g. burden of disease) that can be incorporated into clinical trials and regulatory decision-making.

Last month, Pfizer, Roche and Allergan offered their perspectives on what such guidance should look like.

Industry Contribution

Another window of opportunity emerged for industry to contribute to the success of the PFDD objectives – opening the floor for debate on proposing draft guidance related to patient experience data, which is informally the “fifth guidance.”

FDA officials described potential areas for industry contribution through developing and submitting draft guidance for agency consideration. Such contributions could include the areas of natural history development to inform future research and serve as a basis for clinical trial recruitment, forming Centers of Excellence in study and treatment of diseases, and venture philanthropy to ensure access to the best treatments.

Keith Flanagan, director at the Office of Generic Drug Policy, said that those who are interested in proposing drafts should suggest specific issues and explain their reasoning behind why the guidance is needed by providing relevant information for its development.

“We should not and will not rubberstamp” a draft guidance submitted for FDA consideration, Keith clarified. “If we find that the proposed guidance context is right, the FDA might develop it into a guidance of our own.”

For instance, the ALS Association helped FDA in drafting its draft guidance on developing treatments for ALS.

Still, many at the workshop pointed to certain flaws with the current approach for informing industry on FDA’s current thinking with regard to drug development policies, as it typically takes the agency at least several months to fully develop its guidance documents.

To address these concerns, Flanagan pointed to a pilot project already underway at FDA’s Office of New Drugs to develop and issue “bulleted” guidance at a faster pace. Bullet points on critical elements of drug development “means focusing on a need to know rather than nice to know stuff,” he said.

Instructions for Submitting Drafts of Proposed Guidance Documents Electronically


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