12:00 AM
Feb 23, 2015
 |  BioCentury  |  Politics, Policy & Law

Calming the pendulum

What industry, FDA must do to realize the potential of patient engagement

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.

While the idea of patient engagement dates back to AIDS activists in the 1980s, the current iteration emphasizes collaboration over confrontation, and is moving away from protest and toward quantifiable metrics and peer-reviewed social science methods.

The goal is to move beyond sporadic involvement at specific points on the product development continuum and to systematically integrate distilled insights from patient experiences and perceptions throughout the process, including the target product profile and endpoints used in clinical development.

Making patients true partners thus would help create better medicines and reduce skepticism about regulators and the animus that has destroyed public trust of the pharmaceutical industry.

For companies, engaging with patients can help determine which products to develop, how to study them and how they could be used. Patients can also help define the value of medical interventions for payers in ways that are far more compelling than drug developers can.

By quantifying risk-tolerance, patient engagement could also help calm the pendulum that has been swinging for 20 years between permissive policies designed to speed new therapies to the market and stricter requirements intended to minimize safety risks.

For regulators, patients and their advocates can help inform and legitimize regulatory decisions by showing how those decisions reflect the interests and preferences of people living with diseases.

And patients can multiply the effectiveness of regulators and investment in medical research, for example by drafting FDA guidance documents and by directing money and organizational efforts toward targeted R&D.

To realize this potential, patient engagement must move beyond current approaches - which rely too much on anecdotes and personal testimony - to develop and validate methods that produce reliable, scientifically rigorous data.

To do so, the key challenges include reaching agreement on best practices for patient engagement, and providing advocacy organizations the financial and technical resources they need without imperiling their independence and credibility.

Patient advocates, FDA and drug companies are starting to create rules of the road. Patient engagement is industry's top priority for PDUFA reauthorization, the first topic addressed in the House Energy & Commerce Committee's 21st Century Cures legislative discussion draft, and a high priority for outgoing FDA Commissioner Margaret Hamburg and the directors of the agency's centers for drugs and devices.

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.

The agency already has established proof of principle for the process by issuing its first approval based on patient-centered criteria. In January, the Center for Devices and Radiological Health (CDRH) approved a new weight loss device, the Maestro Rechargeable System from EnteroMedics Inc., based on the division's Patient Preference Initiative.

From town halls to templates

Patient engagement was a major focus of the 2012 PDUFA V reauthorization, which mandated the creation of a Patient Focused Drug Development program at FDA.

The public face of the program has been a series of meetings to solicit patient perspectives about specific diseases. But it isn't possible to organize town hall meetings for every disease and condition, and there are limits to the utility of information that can be collected through public testimony.

FDA and the public have little if any information about how well the experience of the patients who testify at meetings reflects the experiences of other patients. Selection bias is a concern because patients who are too ill, lack financial resources or fear stigmatization may not attend the meetings.

Even if the testimony is broadly representative, there is no obvious way to structure the information so it can be used by regulators. FDA's reports about patient meetings are not stored in searchable databases, and the information collected would not meet inclusion criteria for evidence-based reviews.

"There is a lot of value in hearing directly from patients what their experience is to get a sense of what it means to live with a disease, the...

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