Patient and Stakeholder Engagement

PM360 – Working with Advocates: Understanding Patient Concerns

Patient advocacy groups have exploded in number and scope in recent years and in many ways are reshaping the drug development landscape, from trial design to recruitment support to participation in the regulatory approval process. Thousands of organizations now advocate for millions of patients, and while many patients and families eagerly engage with these groups, others are more guarded in approaching them—if they entertain the idea at all.

The CRO where I am head of patient and stakeholder engagement works extensively with many advocacy groups, and while we generally see a number of advantages for patients engaging with these organizations, we never overtly recommend that patients or their families get involved with advocacy organizations. That’s because it’s a highly personal decision that patients and their families need to make, weighing their questions about privacy, being stigmatized socially as a result of the condition in question, and a number of other complex factors that vary from family to family.

A better approach for sponsors and their partners is to understand patients’ concerns about getting involved in advocacy and to support opportunities to show how engaging with an advocacy organization can benefit patients and the overall drug development process—from providing disease specific information and helping with fundraising to supporting new compounds as they move toward marketing authorization and increasing the likelihood that they qualify for insurance reimbursement.

A Diverse, Expanding Crowd

“Patient advocacy group” and “patient advocacy organization” are broad terms that describe everything from organizations that raise millions of dollars and heavily influence government policy to worried parents hunched over a kitchen table raising awareness of a condition that has suddenly upended their lives. U.S. and European regulators have encouraged proliferation of these groups: The U.S. Food and Drug Administration (FDA), through a 2012 law known as FDASIA, the FDA Safety and Innovation Act, and the European Medicines Agency via its framework for interaction among the EMA, patients, and consumers.

Read more at PM360.