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