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.

FDASIA reflects the FDA’s position that involving patients or their caregivers in drug development can produce better outcomes. Specifically, it requires the agency to develop and implement strategies to solicit patient viewpoints and to consider patient perspectives during regulatory deliberations, and it authorizes the FDA to collect industry user fees to fund review of new products, promotes innovation to speed access to safe and effective products, and seeks to enhance the safety of the drug supply chain.

The EMA, through a 2014 revision of its framework on stakeholder interaction, aims to better understand the real-life experiences of patients and their use of medicines, more efficiently communicate with patients to support safe and rational use of treatments, and improve patient advocates’ understanding of the role of EU drug regulators.

Potential benefits of joining a patient advocacy group include:

  • Access to the available research on a disease or condition.
  • Updates on medical and regulatory developments.
  • Help dealing with insurance companies.
  • Identification of fundraising opportunities.
  • Support for patients and families as they confront the daily manifestations of a life-limiting condition.
  • In pediatric conditions, strategies for working with schools, some of which can be less than accommodating.

Why the Reluctance?

For all the potential advantages of joining a patient advocacy group, some patients and families cite equally compelling reasons to stay clear. Foremost among these is privacy. While some families are motivated by the challenge of facing the gauntlet a diagnosis often presents, many more feel uncomfortable sharing details about their private lives with strangers.

In truth, patients and families have few legitimate privacy worries when they engage with an advocacy group. Membership rolls are not made public, and most of these organizations do not share information externally and certainly do not do so without obtaining permission from the patient or the patient’s legal representative. But the internet and high-profile hacking cases have made us generally more concerned about privacy than ever before, and online security breaches breed general suspicion and distrust.

The advocacy movement’s very expansion and success is, ironically, yet another reason some patients and families choose not to engage. Some indications have spawned a dozen or more advocacy groups, sowing confusion that can discourage would-be participants who are overwhelmed by the abundance of seemingly redundant options.

Finally, not everyone is wired for high levels of engagement. Some demonstrate heroic levels of commitment—for example, Sharon King, who founded Taylor’s Tale after her daughter was diagnosed in 2006 with CLN1, a sub-type of Batten disease, one of a group of rare and always fatal nervous system disorders. Taylor died last year, but her mother continues to run the organization in support of the overall rare disease community. But for every Sharon King, many more patients and family members are working hard just to get through life.

Why Get Involved?

Beyond information access, fundraising mechanisms, and other typical reasons people join patient groups, it’s important to recognize the important work these organizations do to advance the availability of groundbreaking new treatments. As noted, regulators are serious about expanding patients’ role in the process. Through the FDASIA legislation, the agency mandated a five-year Patient-Focused Drug Development program to learn from patients about the impact of diseases on their daily lives, and it convened numerous public meetings on specific disease areas. It also set up a public-private working group to advise the FDA on creating a risk-based regulatory framework on health information technology.

Patient input is also essential to ensuring that researchers are pursuing the right goals. The patient voice can mean the difference between developing therapies that change a value in a lab and those that result in improved medical or functional outcomes. To recognize what is really significant, we need to understand the burden of the disease and what living with the condition is really like. This knowledge is critical to our understanding how therapeutic changes affect patients in ways that are meaningful to them.

Yet another practical need for engaged patients is the process that yields insurance reimbursement decisions. If drug developers do not work with patients and families, they miss the opportunity to understand a potential therapy’s value to the community for which it is being developed. And if they don’t understand what the value is, they can’t make their best case to a reimbursement body. That might lead to reimbursement being denied, delayed, or granted conditionally, pending further patient input.

The earlier you start engaging the patient community, the better. By talking with patients, their caregivers, and their advocates early, you can design and execute studies that close the gap between marketing authorization and the reimbursement decision while reducing the burden on patients and their families.

  • Juliet Moritz

    Juliet Moritz is Vice President, Patient and Stakeholder Engagement, Rare Disease & Pediatrics at Premier Research. Juliet has been involved in clinical research for 29 years, having started her career as a study coordinator at an academic research hospital. Since that time she has held positions of increasing responsibility and scope for small, medium, and large biotech and pharma companies and CROs.


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