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Expanding the Role of Patients in Drug Development

June 17, 2015
Working alongside scientists, universities, and biotech companies, patients and patient organizations have the ability to play a critical role in the search for treatments and cures for chronic and deadly diseases.  As part of the 5th authorization of the Prescription Drug User Fee Act (PDUFA V), the Food and Drug Administration made a commitment to more systematically gather patients’ perspectives on their condition and the available therapies in order to incorporate these findings into their regulatory decision-making processes.  The more patients become engaged in the process, the better off we all are.

Since medicines have benefits and risks that must be taken into consideration by the FDA before they receive the agency’s stamp of approval, it is critical that the patient voice is heard throughout the drug development process.  During that consideration process, it is vital that the FDA take patient perspectives and feedback into account.   Experiential knowledge can provide regulators with a deeper understanding of the personal side effects and benefits involved in taking a specific medicine or treatment.

Just as the FDA has started incorporating patient perspectives into their regulatory processes, biotechnology companies are seeking to incorporate these perspectives early in the research continuum. Seeking patient input for endpoint selection and clinical trial design holds much promise to ensure that medicines best meet the needs of patients who need them.

But this input can’t be integrated in an ad-hoc or anecdotal manner – just as we take a scientific approach to drug development, we must take a scientific approach when gathering and evaluating patient input.  With patient advocates, innovators, and regulators working together, we must adopt a structured benefit-risk framework that standardizes a holistic assessment of benefits and risks and incorporates the patient perspective throughout the drug development continuum.

Moving forward, biotech innovators need to shift their thinking in order to involve patients in every phase of drug development.  But, this needs to be a two-way conversation, with patients serving as advocates for their diseases and providing the research and scientific community with feedback and input.

BIO has laid out recommendations in a white paper “A lifecycle Approach to FDA’s Structured Benefit-Risk Assessment Framework,” which has the potential to serve as a bridge between patient-focused drug development and individual product approvals so that regulatory decisions could be informed by – and grounded by – patient preference data and other data-driven perspectives on balancing benefits and risks.

As we work to transform the biotech industry and the role patients play, events like the upcoming 2015 BIO International Convention’s “Putting Patients in the Center: Advancing the Science of Patient Preference Assessment” provide a platform for patient organizations to connect with biotech executives, government officials and researchers and discuss how we can all work together.

Moreover, advances like the 21st Century Cures initiative further bolster efforts made by patients and the broader health community to generate patient preference data and utilize structured benefit-risk approaches that will continue to help ensure that the patient voice is captured when making important regulatory approval decisions.