Meagan Spychala, Ph.D., Assistant Vice President of Patient Engagement and Program Strategy at Rho, serves as Rho’s expert on patient-focused clinical research activities and incorporates the patient and caregiver voice and experience into the clinical development program.

Patient-centricity and family-centricity has been a hot topic for the past few years in clinical research, as more groups in pharmaceutical companies and CROs are working on patient engagement and patient-centric approaches to clinical research programs. Recruitment and retention are one part of patient-centricity, but the more important up-front and overarching aspect of patient-centric research comes from Patient-Focused Drug Development (PFDD). PFDD is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into the development and evaluation of medical products throughout the product life-cycle. Patients and caregivers know the disease and understand what would have the biggest impact to their lives; this wisdom can and should significantly inform the clinical research being conducted. If done correctly, the research will collect data on outcomes that matter most to the patient community. Using a PFDD approach to engage patients and caregivers throughout the product development process can lead to better study designs, more relevant data, better trial enrollment, and ultimately better products.

Since 2012, several key pieces of legislation have been signed into law to support performing more patient-focused research. The Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, the 21st Century Cures Act of 2016, and the FDA Reauthorization Act of 2017 collectively position FDA to learn about, promote, and encourage the integration of patient perspectives into the development of drugs, biologics, and devices. FDA has been pursuing a number of initiatives under the auspices of this legislation (see links and references below); one being conducting PFDD meetings, of which there have now been 24. In a parallel development, in 2018 the FDA entered into an understanding (MOU) with the National Organization for Rare Disorders (NORD) to conduct pilot listening sessions to enhance the incorporation of the patient experience into regulatory discussions. Twelve listening sessions have been conducted under the MOU since October 2018 and more are currently being scheduled for 2020.

The FDA is working to collate the principles and insights gleaned across these PFDD meetings into 4 formal guidance documents. Collectively, these guidances will describe the steps, processes, and considerations to bear in mind when collecting information from patients and caregivers. This includes discerning what is most salient, important, and impactful to them; using this information to create or use COA or PRO tools; and potentially validating new tools to be used as endpoints for clinical research. The first draft guidance was issued mid-2018 and focused on who to get input from and how to collect the information needed. This is intentionally an initial overview; therefore, it is a high-level view of key philosophies about patient involvement via providing patient experience data to guide a product development program into using the most sensitive and clinically meaningful endpoints. The second draft guidance, issued October 2019, provided methods for eliciting information from the patient population. Three main methods of research for obtaining information to understand what matters most to patients living with a disease and their caregivers were detailed: qualitative research, quantitative research and mixed-methods research. Each type has strengths and limitations, so deciding on which method to use to support a research program depends on the patient population and research objectives. No matter what type of research method used, a research protocol, interview or survey guide, training materials, glossary, data management plan, and data analysis plan are needed before proceeding with collecting information and the protocol should be reviewed in a meeting with the FDA.

While it will likely take some time until all four guidance documents are complete, we eagerly await the third and fourth draft guidances, as patient advocacy groups and product development organizations are already moving forward with PFDD and need additional FDA guidance and advice on how to incorporate the patient perspective into clinical research. Even though these documents are not completed, ongoing development programs should still strive to be patient-focused. We have seen from having conducted natural history studies and other patient-centric work how important it can be to have a clear understanding of the disease and patients’ (and caregivers’) perspectives; absent such understanding, it is hard to plan and execute sound development programs that result in products the patients need and benefit from.

Want advice on how to proceed with a PFDD approach? Rho has the scientific, operational, and regulatory strategy experience needed to support your PFDD program.

References:

Guidances:

• G1 June 2018: https://www.fda.gov/media/113653/download
• G2 Oct 2019: https://www.fda.gov/media/131230/download
• Also — CDRH guidance on patient engagement: https://www.fda.gov/media/130917/download. This guidance is brief, covers a few basic topics at high level, similar to the tenor and level of specific guidance in the 1st 2 PFDD guidances discussed here.

Other references:

• FDA patient engagement: https://www.fda.gov/patients/learn-about-fda-patient-engagement
•  CDER-wide PFDD: https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development
• Oncology Center of Excellence PFDD: https://www.fda.gov/about-fda/oncology-center-excellence/patient-focused-drug-development
• CDRH Patient Engagement: https://www.fda.gov/about-fda/center-devices-and-radiological-health/cdrh-patient-engagement
• CDRH Patient and Care-partner Connection (P&CC): https://www.federalregister.gov/documents/2016/11/07/2016-26784/establishment-of-the-patient-and-care-partner-connection-establishment-of-a-public-docket-request
• CDRH Patient Preference page — https://www.fda.gov/about-fda/cdrh-patient-engagement/patient-preference-information-ppi-medical-device-decision-making