Patient-Focused Drug Development Guidance Comments

Several stakeholders are asking FDA to clarify elements in its draft guidance on methods to identify what is important to patients that is part of the agency’s series of guidances on patient-focused drug development. The National Organization for Rare Disorders says it appreciates that the emphasis of the draft guidance is on identifying what matters most to patients in terms of the burden of their disease and treatments. The organization provides general and specific comments on areas of concern.

The Critical Path Institute says the draft is “thoughtful and well-written overall as it strikes a difficult balance of maintaining rigor with regard to expectations for drug developers while also indicating areas of flexibility.” It provides specific comments and recommendations.

Pharmaceutical Research and Manufacturers of America recommends that FDA include comments, examples, and references suggested by the association in the comments to the draft guidance discussion guides. It also says there are areas in which industry sponsors could benefit from further clarification and elaboration either in this draft guidance or by signaling whether the topics will be covered in future guidances.

Allergan says it appreciates the guidance format with comprehensive coverage of the various methodologies within the body of the document, while detailed information is included in the appendices and references are provided throughout the document for additional information. It recommends that FDA consider replacing the term “study subject” with “study participant,” which the company says it more humanizing. It also provides specific comments.

Pfizer identifies “several areas where further clarification in this guidance or signaling that these items will be covered in one of the two remaining planned guidance documents in this series would be beneficial. Addressing these topics will increase the guidance’s utility and provide more efficiency and certainty for sponsors as they integrate their use of patient experience data into their development programs.”

The Biotechnology Innovation Organization says the guidance would be strengthened by addressing how sponsors can engage “early and often” with review divisions and by addressing evidentiary standards for patient experience data to be considered for regulatory decision making, including benefit-risk decisions and labeling.