The Food and Drug Administration (FDA) Public Workshop on Clinical Outcomes Assessment Development and Implementation: Opportunities and Challenges, held earlier this week, was an important contribution to the evolution of understanding and emergence of a multi-stakeholder vision of how to enhance the patient-centeredness of the drug development process.
The workshop focused on how to identify and measure outcomes that are meaningful to patients. If clinical outcome assessments (e.g., patient-reported outcomes) can evolve to better capture outcomes that are meaningful to patients, we will make significant progress towards a patient-centered approach to drug development.
As the ultimate stakeholders in the development and availability of new medicines, the views of the people using, or hoping for, a medicine to make their life longer, healthier, and happier should inform the drug development and regulatory review processes. This requires the development and implementation of policies that connect a robust, science-based understanding of patients’ perspective to decisions that promote innovation and expedite drug development.
Theresa Mullin, Director of the Office of Strategic Programs of the Center for Drug Evaluation and Research at FDA, proposed important next steps as we continue along the path to patient-centered drug development: 1) advance the science of patient input and 2) provide FDA guidance to patient advocates and drug developers. Both elements are key to the development of a robust, predictable and reliable pathway for patient reports, assessments and preferences to be understood in a science-based way that informs both drug development and the benefit-risk assessments that are at the core of regulatory decision-making.
Paul Kluetz, Acting Deputy Office Director of the FDA Office of Hematology and Oncology Products, clarified that the Agency’s Patient-Reported Outcome (PRO) guidance was very much needed in 2009 as many innovators were trying to develop instruments in the absence of FDA recommendations of a systematic approach. As FDA pursues an approach that is more flexible and conducive to innovation, it is important that the Agency continue to make progress to provide timely and robust feedback to those seeking to develop and use clinical outcome assessments, and continue to consider ways to improve communication to stakeholders of complex regulatory decisions. In doing this, a structured approach to benefit-risk assessment may be a viable method to bring consistency to the communication of decisions that require flexibility and expert judgment.
FDA’s proposal of a compendium of clinical outcomes assessments organized by therapeutic area is a positive development. While not intended to substitute for dialogue between innovators and FDA on specific drug development programs and plans, the proposed compendium will improve public visibility into the Agency’s opinion of available tools, specifically those that have been included in the labels of approved medicines or are in the process of qualification. An additional goal of the compendium is to foster collaboration among stakeholders to address gaps in assessments in disease areas that are in need of outcome measures to move drug development programs forward.
In order to move closer to achieving our shared goal of truly patient-centered drug development, we must establish a clear path for patient advocacy organizations, FDA, consortia, and the biopharmaceutical industry to work collaboratively to advance the science of patient input and to translate this input into concrete tools such as clinical outcome assessments.
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