Quintiles recently provided input to the U.S. Food and Drug Administration regarding the issue of patient engagement in drug development. The FDA requested comments as part of their ongoing efforts to ‘develop and implement strategies to solicit patient perspectives during the medical product development process and in regulatory discussions‘, as directed by the Section 1137 of the 2012 FDA Safety and Innovation Act (FDASIA).
We were excited to share our thoughts on this important topic. Engaging patient feedback in the drug development process has long been a priority for Quintiles, and we have derived extensive insight from the more than 100,000 successful trial designs and 110 product launches we’ve supported. Working with experts from multiple perspectives, we made the following three recommendations to FDA:
- Development Planning - Seek early patient input on what outcomes clinical trials should measure. Gathering direct patient input on what types of outcomes are most meaningful to patients can be invaluable in shaping the products that are developed, what outcomes are most important to achieve and how they are measured in clinical trials (i.e., the use of PROs/patient-reported outcomes instruments). To ensure alignment among the sponsor, FDA and the intended population on what outcomes are most important to measure efficacy, we recommended that FDA provide a mechanism for interaction with a sponsor to discuss patient input on what measures matter to them before the Pre-IND milestone. We believe that these early meetings between FDA and sponsors would greatly increase the effect of patient input to clinical development planning for product.
- Protocol Design - Gather patient input on study design/protocols. Such input will provide sponsors with a unique perspective on how to measure the agreed-upon patient outcomes, including whether patients would be willing to participate in a particular type of study, and what procedures, including frequency and duration, would be tolerable for that population. In our experience, such input can improve study design, and positively impact enrollment and retention. We recommended that FDA provide guidance encouraging sponsors to obtain input from patients on protocol feasibility as part of their clinical development plan. FDA should also develop a way to ensure this input factors into FDA decision-making and recommendations when evaluating or commenting on new approaches, such as frequency of visits or use of more convenient technologies to obtain patient data. Input should be collected from potential study participants, as well as patient advocates for specific diseases.
- Patient input on inclusion of compassionate use programs as part of the study design/protocol. This is an important topic for seriously ill patients and their families, particularly those who have exhausted other options. Seeking feedback from relevant patients for the offering and design of compassionate use programs for potentially life-saving therapies would make such programs more widely available to those who could benefit from them. We recommended that FDA consider providing guidance to allow for contributions from relevant patients in order for encouragement of inclusion of such programs in the study design and protocol, and we welcome the agency's draft guidance on this topic.
The FDA’s efforts to effectively solicit and incorporate patient input will benefit all stakeholders in the drug development and approval process. We believe these are important practical steps that would contribute to the overall effort to make drug development and regulatory decisions more patient-centric. Gaining early agreement between FDA and sponsor companies on what clinical trials should measure and how, informed directly by patient perspectives, would improve the probability of clinical trial success and accelerate the delivery of much-needed new or better options to patients.