Developing Long-Term and Effective Collaborations with Patient Advocacy Groups

The role of patient advocacy groups (PAGs) in drug development has been expanding in recent years, especially in rare diseases where awareness and understanding of both the burden of disease and patient needs are often limited.

Industry now clearly recognizes the importance of a patient-centric approach in both building awareness and advancing innovative clinical research programs. Increasingly, companies are building strategic plans that involve early and regular collaboration with PAGs. They are also working to focus more on the needs and goals of the patient advocates, which can often lead to more impactful collaborations.

When successful, these relationships provide valuable insights to support strategies in many areas, including planning for clinical research, patient recruitment and education, reimbursement, access, compliance, and other areas.

Successful collaboration with PAGs can support and potentially expedite regulatory review for new therapies to treat rare diseases and can also help to explain often unavoidable limitations on efficacy and safety data. For example, the advocacy group CureDuchenne worked aggressively to present perspectives from the patient community to the US Food and Drug Administration (FDA) to support regulatory review of Exondys-51 for the treatment of Duchenne muscular dystrophy. The FDA Advisory Committee initially recommended against approval of Exondys-51 due to perceived limitations on clinical trial design and lack of robust data. Efforts from the patient advocacy community are believed to have played an important role in building support for approval.

There are also several examples of PAGs working to highlight the experience of living with a rare disease to support reimbursement. In Belgium, letters from PAGs and individual patients have historically been included in reimbursement submissions. Examples include a letter in support of Tobi® Podhaler^® to suppress chronic lung infection in people living with cystic fibrosis. Another letter highlighted positive experiences with Exjade^® for the treatment of chronic iron overload. In both cases, therapies were approved for reimbursement.

In recent years, PAGs have also had a considerable impact on the policy landscape, especially in many developing countries where a lack of related national health care policies and dedicated funding for rare diseases can impede patient access to new treatments. Efforts by organizations such as the Geiser Foundation in Argentina and the Colombian Federation for Rare Diseases have contributed to the development of national plans for rare diseases with goals to improve research, care, and support.

As the role of patient advocates in drug development continues to expand, industry continually looks for new approaches that can improve these collaborations in various ways. Drug developers now consider patients as much more than potential customers; they consider them as partners who can provide important and often essential input related to development programs. As a result, many companies establish special teams to manage advocacy relationships to ensure that both parties are positioned to engage early, actively, and effectively. In many cases, these relationships are established as early as preclinical stage research, where development teams often need to understand and assess disease characteristics and burden to plan for later stage clinical programs.

Early engagement can also help drug developers understand both the capacity and mission of advocacy organizations to make sure mutual goals are in sync from the outset and at every stage of a collaboration. Very early on, companies need to have a clear sense of many factors, including unmet need, disease burden, and challenges in the standard of care. They must also carefully assess the resources and capabilities of advocacy partners and consider options to help them build capacity and enhance their influence and reach. They also often need to outline the development and regulatory review process to help advocates understand both their opportunities to participate and any limitations on their roles. All of this reinforces the benefits of both early and tailored engagement and ongoing partnership.

With these sorts of relationships in place, there can be multiple opportunities for mutual benefit. For example, advocacy leaders can provide important support for pharmaceutical companies’ needs including patient recruitment in clinical research—often a major challenge in rare diseases—and efforts to build disease awareness. Concurrently, industry can take steps to make sure advocacy groups are regularly updated on progress in clinical trials and other advances in research that they can share with their constituents, helping the entire community of patients to be better informed.

Open communication, transparency, and, as-needed, compromise, are essential to optimize results for both parties. At every stage in the relationship, the objectives, roles and responsibilities, timelines, and anticipated results should be clearly outlined. Ultimately, the end goal for everyone involved is that rare disease patients have increased access to the potentially life-saving treatments that they need.

Andrew Butcher is a vice president in the Life Sciences Practice at CRA. He regularly advises company senior leadership and brand teams on how to maximize the impact of stakeholder relations and communications to drive business strategy and internal change.

Cécile Matthews is a principal in the Life Sciences Practice at CRA. She has 20 years of experience in strategy consulting for the life sciences industry. Her areas of expertise include pricing, reimbursement, and market access globally.

Bhavesh Patel is a senior associate in the Life Sciences Practice at CRA. He has experience in a range of therapy areas including oncology, cardiovascular disease, diabetes, and rare diseases.

The views expressed herein are the authors’ and not those of Charles River Associates (CRA) or any of the organizations with which the authors are affiliated.