When it comes to the patient voice (or any voice), the plural of anecdote isn't data. But the plural of data is science.
One of the hallmark pieces of FDASIA enshrines the concept of patient-focused drug development. Per the FDA’s own website:
Patients who live with a disease have a direct stake in the outcomes of the drug review process and are in a unique position to contribute to the entire medical product development enterprise. Under FDASIA, the FDA will increase patient participation in medical product regulation.
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Patient Participation in Medical Product Discussions under FDASIA. Sec. 1137 of the new law will assist the agency in developing and implementing strategies to solicit the views of patients during the medical product development process and consider their perspectives during regulatory discussions. This will include:
- Fostering participation of FDA Patient Representatives as Special Government Employees in appropriate agency meetings with medical product sponsors and investigators; and,
- Exploring means to provide for identification of potential FDA Patient Representatives who do not have any, or have minimal, financial interest in the medical products industry. - Patient-Focused Drug Development under PDUFA V. The PDUFA V agreement provides for a new process enhancement under a commitment that will provide a more systematic and expansive approach to obtaining the patient perspective on disease severity or the unmet medical need in a therapeutic area to benefit the drug review process. In other words, the patient perspective will provide context in which regulatory decision-making is made, specifically the analysis of the severity of the condition treatment and the current state of the treatment armamentarium for a given disease.
But patient input needs to be more than anecdotal – it needs to be data-driven so that divisional decisions can be more scientifically-driven by the patient community. And nowhere is that more urgently needed than in discussions over risk/benefit calculations.
Steve Usdin’s cover story in BioCentury, “Calming the Pendulum,” makes the case in the first sentence, Regulators and drug developers have converged on the idea that enhancing and broadening patient engagement is a key to improving drug development and adjudicating controversies over benefit-risk decisions and the value of medicines.
Absolutely right. But that’s not patient-focused drug development (after all, isn’t all drug development patient-focused), it’s patient-driven drug development. PD3. It’s time to change the name to properly fit the task. PD3 places the patient voice squarely in the middle of the drug development ecosystem.
Usdin quotes Dr. John Bridges (senior fellow at the Center for Medicine in the Public Interest and Associate Professor at the Johns Hopkins Bloomberg School of Public Health) who says that the FDA’s use of decision-based preference data “changed the world.” If only it were that easy.
Usdin writes, “Patient engagement provisions in the 21st Century Cures discussion draft were written based on input from patient groups, industry and FDA. They are intended to create regulatory certainty around the use of patient perspectives, including formal pathways for patient groups and companies to submit information about patient preferences, as well as defining how FDA will incorporate these submissions into approval and other decisions.
In Bridges’ new paper, Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study (The Patient, DOI 10.1007/s-40271-014-0081-0) he provides important qualitative evidence on stakeholders’ views as to important issues associated with emerging therapies for idiopathic pulmonary fibrosis.
Bridges, et al., identifies multiple issues were identified spanning the impact of emerging therapies, including the need to document the patient experience with treatment, and factors associated with disease progression.
The paper discusses the value of qualitative research both in understanding the benefits and risks of emerging therapies and in promoting patient-centered drug development.
Patient passion is important to share. When combined with data and a more dispassionate understanding of regulatory paradigms, a patient-driven pathway can is, and must evolve into a tool used to impact regulatory decision-making.
