PFDD: FDA finalizes guidance on identifying what’s important to patients

Regulatory NewsRegulatory News | 28 February 2022 |  By 

The US Food and Drug Administration (FDA) on Friday finalized guidance to assist sponsors collect and submit “important” information from patients and caregivers used for medical product development under the agency’s patient-focused drug development (PFDD) initiative. The final guidance provides greater clarification on how industry can interact early with the agency to obtain feedback on patient experience data.
 
The guidance contains minor revisions from a draft issued in October 2019. (RELATED: Patient-Focused Drug Development: New FDA Draft Guidance Delves Into What’s Important, Regulatory Focus 30 September 2019). Overall, major industry groups supported the guidance while the reaction among patient advocacy groups was mixed.
 
The final guidance is the second in a series of four PFDD documents and is required under the 21st Century Cures Act.
 
The purpose of the guidance is to “present a range of methods and established best research practices to identify what is important to patients with respect to burden of disease, burden of treatment, and the benefits and risks in the management of the patient diseases,” FDA said in a Federal Register notice.
 
The final version reorders some of the sections and replaces Table 1 on interview types with a new Table 1 on the potential strengths and limitations of interview administration methods such as in-person, telephone, and online and virtual video conferences. It also renames the title of the section on “special considerations when obtaining data from specific populations” to “managing barriers to self-report” and revises the text in the section.
 
Industry applauds guidance
 
In comments on the draft, the agency received 84 comments from industry groups, patient advocacy groups and individuals. Major pharmaceutical groups and one patient group applauded the guidance while another said it did not go far enough in meeting the needs of patients.
 
Pfizer wrote that it “applauds the FDA’s commitment to better incorporate the patient’s voice in drug development and evaluation, and we support the Agency’s efforts to clarify the methods to identify what is important to patients and to advance the use of patient experience data (PED) to inform regulatory decision making.”
 
The Pharmaceutical Research and Manufacturers of America (PhRMA) concurred. The group said that it “supports FDA’s efforts to systematically incorporate the patient voice in drug development and regulatory decision-making and applauds the Agency on the issuance of the draft for the second of four guidances under the PDUFA VI Goals Letter. The biopharmaceutical industry places great importance on the participation of patients, family members and caregivers, as important stakeholders in the drug development process. Perspectives from such stakeholders may support the assessment of benefit and risk factors, aid in the evaluation of treatment and what would be a clinically meaningful outcome, and strengthen regulatory decision-making.”
 
The Biotechnology Industry Association (BIO) wrote that it “appreciates the agency’s use of multiple examples throughout the document. The examples are plentiful, easy to understand and largely represent realistic scenarios. We believe that the guidance is clear and useful, detail key considerations and methods for identifying what is most important to patients.”
 
Along the same lines, the National Organization for Rare Disorders (NORD) said that it “supports FDA’s efforts to incorporate the patient perspective in the development of medical products and regulatory product review. Toward that end, we appreciate that the emphasis of this draft guidance is on identifying what matters most to patients in terms of the disease ad treatments.”
 
Yet one patient group, the Cancer Support Community, said that the guideline “fell short” in assessing what is important to patients. They wrote that “we recognize that the research methodologies outlined in this Guidance are not exhaustive, however we ask the FDA to revise this Guidance to better serve the purpose of guiding stakeholders to determine what is most important to patients.”
 
Early interaction clarified
 
Yet one criticism was the guidance lacked clarity on the methods for interacting early with the agency on patient experience data.
 
The draft states that “FDA encourages stakeholders to have early interactions with FDA and obtain feedback from the relevant FDA review division when considering collection of patient experience data related to the burden of disease and burden of treatment.”
 
Both Pfizer and Biotechnology Industry Organization (BIO) called for more guidance on the correct pathways for early interaction meetings, while the National Organization for Rare Disorders (NORD) said that additional guidance is needed on whether these early engagements would be considered formal or informal meetings.
 
In response, the agency added a footnote stating that “in addition to the general considerations discussed in this guidance, a study may need to meet specific statutory and regulatory standards governing the collection, processing, retention, and submission of data to the FDA to support regulatory decisions regarding a marketed or proposed medical products. This guidance focuses on more general considerations that apply to many types of studies, and you should consult with the review division and applicable guidance regarding any other applicable requirements.”
 
Final guidance
 
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Tags: FDA, PFDD

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