PFDD: BIO Comments on Patient-Focused Drug Development Guidance: Methods to Identify What Is Important to Patients and Select, Develop, or Modify Fit-for-Purpose Clinical Outcome Assessments

December 14, 2019

Re: Docket No. FDA–2018-N-2455: FDA’s open docket entitled “Patient-Focused Drug Development Guidance: Methods to Identify What Is Important to Patients and Select, Develop, or Modify Fit-for-Purpose Clinical Outcome Assessments”.

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments following the public meeting on Patient-Focused Drug Development (PFDD) Guidance: Methods To Identify What Is Important to Patients and Select, Develop, or Modify Fit-for-Purpose Clinical Outcome Assessments.

BIO is the world's largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members develop medical products and technologies to treat people afflicted with serious diseases, to delay the onset of these diseases, or to prevent them in the first place.

BIO commends the FDA for the tremendous work that the Agency has done in order to better ensure that patient experiences are more systematically collected and used to inform the development and review of new therapies. BIO particularly appreciates the Agency’s work to develop guidance documents and discussion guides prior to public meetings. It is through public discussion and the development of guidance documents and the collaboration of all stakeholders, including industry Sponsors, FDA reviewers, and patients and patient organizations, that will allow for patient experience data to be collected and used more widely in drug development and review.

BIO strongly believes that in order to truly support patient-focused drug development, patient experience data should be considered for use throughout the drug development and review lifecycle. Appropriate fit-for-purpose tools for collecting patient experience data have the potential to inform protocol design, endpoint development, benefit-risk assessments, and labeling, among other aspects of drug development and assessment. To encourage stakeholders to collect fit-for-purpose patient experience data, we request that the FDA more clearly indicate the breadth of regulatory decisions for which they will consider different types of patient experience data. To this end, BIO suggests that the Agency consider including in guidance documents or on the FDA website, External Resources or Information Related to Patient Experiences, in the form of a chart, the different drug development (e.g., internal decisions such as clinical trial design, and for which data to may not be submitted to the FDA) and regulatory decisions (e.g., benefit risk) that the FDA believes can be informed by patient experience data. This chart could be accompanied by case examples highlighting types of patient experience data and how the data was used to inform different decisions in the drug development and review lifecycle. To support these efforts, BIO developed, a chart (see page 38 of this letter) that the FDA may consider adopting or adapting for this purpose as well as case examples (see pages 39-43). While some case examples are included in this letter, BIO will continue to collect and develop additional case study examples to share with the FDA at a future date to help inform collection and utilization of patient experience data by all stakeholders.

While BIO also agrees that FDA’s upcoming PFDD guidance should be complementary to the Patient Reported Outcomes (PRO) Guidance[1], we believe that the latter is too restrictive and has as a result, limited the development and use of PRO tools/instruments to inform product labeling. In light of implementation challenges that make the PRO guidance more restrictive and to maximize utility of new PFDD guidance documents, BIO requests that FDA keep the scope broadly applicable and policy flexible. For example, the Discussion Guides refer to instrument changes that ‘may alter the way respondents respond to the same set of items’, including changing the timing of or procedures for instrument administration in a clinic visit, changing the application to a different setting, population or condition, changing the order of items, item wording, response options or recall period or adding to or deleting portions of an instrument, changing the instructions or placement of instructions and changing an instrument from paper to electronic format. Similar statements were included in the 2009 PRO Guidance and, and as a result made it very difficult for Sponsors to develop, validate, or repurpose existing Clinical Outcome Assessments (COAs). Given that successful repurposing of existing measures is part of the rationale for the new guidances, extra care should be taken in wording so as to avoid overly rigid interpretation.

In addition to the general comments above, we have also included the following specific responses to the questions posed by the FDA in the open docket as well as specific line edits to the Discussion Documents in tabular form.