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The National Health Council and the entire patient community embrace the Food and Drug Administration’s forward-thinking efforts to engage patients early and often to make drug development truly patient-focused.
In 2015, the NHC and the Genetic Alliance hosted a multi-stakeholder dialogue, “Advancing Meaningful Patient Engagement in Research, Development, and Review of Drugs,” which was in response to stakeholders’ increasing demands that, in addition to demonstrating product safety and efficacy, new product sponsors engage patients and ensure that products enhance the outcomes patients care most about. Our dialogue identified critical barriers hindering advancement of patient-focused drug development, which fell into three major categories: culture, communication and the need for regulatory guardrails. Participants proposed actionable steps to overcome these barriers, including creating regulatory guidance. At the time, we suggested the patient community and regulators work collaboratively to set a path forward.
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With the release of the FDA’s much anticipated, first draft guidance on patient-focused drug development, “Collecting Comprehensive and Representative Input,” the NHC is pleased to see concrete steps toward overcoming these three critical barriers. Through the FDA’s “Voice of the Patient” open meetings, reports, public comments and presentations over the last several years, we have witnessed the FDA’s support of patient engagement in medical product development. We now have a document that publicly expresses and formally recognizes the FDA’s acknowledgement of the importance of patient engagement.
This first draft guidance represents a collaborative effort, responsive to input and feedback from the NHC and its membership, and reflects many topics of interest to the patient community. It clarifies that the patient’s journey should be defined from the patient perspective — a significant advancement from historic norms. It reinforces the notion that “patients should be meaningfully involved throughout the medical product development process — not only as study subjects, but as partners.”
The draft guidance expressly states, “FDA values the use of patient input to help foster the development and availability of safe and effective medical products.” These statements provide legitimacy to patient-focused drug development and provide sponsors with a green light to take action and meaningfully engage patients.
The guidance reflects a shared and flexible — but rigorous — approach to patient-focused drug development. It provides a common nomenclature that can be adopted for consistency and standardization across stakeholders. It recognizes that the science of collecting patient input is constantly evolving, that flexibility in methods and approaches is needed. As technology evolves and we gather more experience, the good practices of today may or may not become the best practices of tomorrow. It approaches the topic of representativeness from both a statistical and context-specific perspective.
There are still areas that will need to be addressed, tested and improved. Policy is never perfect. These guidances alone will not bring us overnight into the era of truly patient-focused drug development. However, the quality of the work and time dedicated to this first draft guidance to get it right, combined with the direction from the FDA leadership, has created a momentum that is pivotal and an important milestone for all stakeholders, especially those in the patient community.
To promote widespread, rigorous drug development that is truly patient-focused, the NHC and its members stand ready to work with regulators and all stakeholders to build a solid platform of guidance documents over the coming years to ensure that patient-focused drug development becomes routine.
Marc Boutin, JD, is chief executive officer of the National Health Council.
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