PFDD: BIO Comments on FDA Draft Guidance Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data

March 21, 2019

Re: Docket No. FDA-2018-D-4455: FDA Draft Guidance, Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data

Dear Sir/Madam:

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA or Agency) for the opportunity to submit comments regarding the Draft Guidance on Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data.

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 are involved in the research and development of innovative healthcare, agricultural, industrial, and environmental biotechnology products.

BIO thanks the Agency for promoting partnership between patients, industry, and the FDA by providing patient groups and other stakeholders with a pathway to submit proposed draft guidance and other resources related to patient experience data to the FDA. We welcome the opportunity to integrate information generated by patient groups and other external stakeholders into our drug development programs, and also seek to contribute to the open exchange of information regarding patient’s experiences. We encourage FDA to continue to facilitate the open exchange of patient experience data with all stakeholders such that clinical science is able to advance towards the development of outcomes that are the most meaningful to patients.

BIO believes that strong trilateral communication between the FDA, patients and patient organizations, and Sponsors around patient experience data will result in incorporation of patient experience data throughout the product lifecycle, including drug development and review. BIO appreciates that the Agency included Table 1 (beginning on line 423) in the Draft Guidance as it provides examples for all stakeholders regarding how patient experience data may play a role in the drug development lifecycle; however, as presented, the key stakeholders appear to function in isolation of each other rather than showing how the trilateral communication between patient stakeholders, medical product developers/researchers, and regulators results in effective collection and use of patient experience data. To better support trilateral communication, BIO requests that the FDA clearly outline a process by which the FDA will communicate and provide feedback to stakeholders that submit draft guidance or other patient experience data. Draft guidance development by an external stakeholder (including patients and patient organizations) is time and resource intensive and, for example, if a patient organization submits a draft guidance and the FDA makes the determination to only use some information from the submitted draft guidance, it would be helpful for the FDA share the rationale as to why only portions of the submitted draft guidance will be supported by the Agency. To further support trilateral communication, BIO requests that the Agency communicate how proposed draft guidance submitted by external stakeholders is leveraged to influence thinking related to development of draft guidance by the Agency. We acknowledge that this level of communication is time-consuming, but it will help inform efforts by all stakeholders if the Agency provides feedback around why specific data were or were not utilized.

The Draft Guidance also indicates that if a draft guidance is submitted to the Agency, the FDA will open a docket for stakeholder’s comments. To support trilateral communication, BIO also requests that when such a docket is opened, the FDA also post notification of that docket in the Federal Register.

Finally, BIO also appreciates that the Draft Guidance continues to emphasize that the FDA encourages stakeholders to contact FDA staff early to discuss patient experience data that may be useful to collect and submit to the FDA; however we note that the FDA has not articulated a process by which Sponsors and patients and patient organizations may engage with the Agency around patient experience data. BIO requests that the Draft Guidance provide details regarding who and when Sponsors’ and other stakeholders may engage around patient experience data for the purposes of developing a draft guidance but for other purposes as well (e.g., patient experience data for regulatory decision-making). Along these lines, it would also be helpful for the Agency to clearly indicate how CDER, CBER, and FDA Patient Affairs Staff will be engaged in various interactions with stakeholders around patient experience data. One way that the FDA may clearly provide this information is through a chart or a decision tree based upon the types of stakeholder (e.g., patient, patient organization, or Sponsor), the purpose for which the patient experience data will be used, as well as the time point in the drug development/review lifecycle during which the patient experience data will be collected, analysed, used and submitted.

BIO appreciates this opportunity to submit comments regarding FDA’s Draft Guidance, Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data. We would be pleased to provide further input or clarification of our comments, as needed.