Patient needs and concerns play a growing role in life science companies’ drug development and evaluation. To prioritize patient-centered healthcare, life science companies are working toward improving patients’ experiences during the drug development process to make clinical trial protocols more productive and less stressful.
This goal has now been federally legislated as PFDD (patient-focused drug development), developed by the U.S. Food and Drug Administration (FDA) as part of the 21st Century Cures Act.
Listening to the patient’s voice through PFDD meetings
PFDD is designed to give the patient and their caregiver a voice during drug development and drug evaluation. The FDA established the PFDD initiative in 2012 to systematically obtain patient perspectives on specific diseases and their treatments. From 2012 to 2017, the FDA conducted 24 PFDD meetings with key stakeholders (HCPs, drug developers, researchers, patient advocates, etc.) to listen to patients’ concerns about their diseases. (External PFDD meetings on specific disease areas and concerns continue, led by patient organizations, with FDA help.)
Certainly, physicians can share what their patients tell them. Yet only patients can directly share their lived experiences. People who live with a medical condition are the best authorities on everyday life with a disease. Their real-world perspective is critical to the evaluation process, giving context to the effects of therapies on their lives. Patient feedback can spur considerations that may have been overlooked earlier in the shaping of a study protocol.
For example, in the case of study-required blood draws, rather than asking patients to travel to a faraway official trial site for the draw, study leaders employ a local lab or have a nurse or technician come to the patient’s home. Cancer and other ongoing disease treatments can be done in specified sites closer to patients’ homes. Data can be collected conveniently through patient-centered technology on personal devices, home PCs, or online through a virtual engagement platform.
What is FDA guidance for PFDD?
As pharmaceutical companies work to live up to their patient-centric promises, they’ll look to FDA guidance to update and refine their own patient-focused processes.
In 2020 the FDA presented the first final guidance for PFDD, with three more guidance documents to come. Here are highlights of the four-part guidance series designed for life science companies planning clinical trials:
Guidance 1 includes:
- Help for stakeholders to define target groups of patients and caregivers who can provide robust, meaningful data and other relevant information that can consistently help inform drug development and regulatory decision making
- Approaches for stakeholders to use to better collect comprehensive and representative patient and caregiver input
Guidance 2 includes:
- Methods to gain information from these identified groups, collecting data about their symptoms, impacts of their disease, and other issues that are important to patients.
- Best practices in qualitative research include conducting interviews, developing interview guides, selecting appropriate survey questions, and other considerations in collecting data
- Approaches to facilitate patient enrollment and ease the burden of patient participation in clinical trials
Guidance 3 includes:
- Guidance on the appropriate use of methods to capture information on patient preferences
- Methods to help identify and develop fit-for-purpose COAs (clinical outcome assessments) of importance to patients
Guidance 4 includes:
- Methods, standards, and technologies to use for collection, capture, storage, and analysis of COA data
- Ways to better incorporate COAs into clinical study endpoints that are more useful in regulatory decision making
- Information on format and content required for regulatory submission that includes patient experience (particularly COA data)
The way forward with PFDD
A major aim of the PFDD approach is to raise the dismal completion rate of many clinical trials, often due to untenable disruptions to patients’ lives and their frustrations in communicating the patient perspective. Patient engagement in healthcare and input in drug development and evaluation is an untapped resource that life science companies can no longer afford to ignore. To improve PFDD, pharma and medical device teams should develop and strengthen patient engagement strategies.
PFFD provides an outlet for patients and supplies life science companies with valuable insights that help shape product development. The company and patient insight – paired with that of physicians and other HCPs – create a more complete picture of specific disease communities. Patients are a key part of the scientific narrative that develops over a product’s lifecycle. Focusing on patient needs isn’t just the right thing — it’s smart business.
Advances in communication tools help make patient experience data collection easier before, during, and after clinical trials. To learn more, download our white paper Best Practices Guide: Insights Management and Virtual Patient Engagement.