Thought partnership: How rare disease drug developers can engage with patient advocacy groups

Patient advocacy groups (PAGs) play a vital role in helping companies design rare disease studies that are convenient, transparent, and humane for patients, caregivers, and families. However, companies must engage with PAGs as thought partners from the earliest stage of development to achieve the optimal clinical trial design. Too often, companies turn to PAGs as a last resort when trial recruitment fails rather than approaching them as partners from the early study concept stage. When PAGs, clinical research organizations (CROs), and biopharmaceutical companies engage in a sustained and meaningful dialogue, they create opportunities for patients to participate all along the drug development journey. We asked Parexel experts Pam Rattananont and Julie Shutt to share their thoughts and experiences on how companies can form effective long-term partnerships with rare disease PAGs.

What do PAGs want from drug developers?

Kistler: PAG organizations in the rare disease space are extremely savvy and want partners with the same sense of urgency to provide treatments for their patient communities. They know how drug development works. At annual industry and patient advocacy meetings, educational sessions teach PAGs how to form biotech firms and fund their research. Patients and caregivers want to work with sponsors. Still, they get frustrated when they provide data and money, and the sponsor decides to change direction or rebalance their portfolio for business reasons. That can be challenging for patients and parents, especially when dealing with rapidly progressive diseases. Sometimes, PAGs go directly to researchers, bypassing companies, and fund the research themselves. They are learning how to attract investors and drive clinical development programs.

How can PAGs help drug developers?

Kistler: PAGs can give sponsors insights into endpoints that reflect patients’ quality of life. Many patients are looking for therapies that will reduce the burden of their disease: If they don’t have to go to the emergency room as often, or if their child has 50 seizures a month versus 100, that’s a win for them. 

One recent example is an emerging biotech company developing a drug to treat a rare neurological disorder. They used real-world data gathered from a patient-consented medical records platform to bolster an investigational new drug (IND) filing. Based on these data, the FDA removed an initial clinical hold on the IND. That’s how valuable patient data and collaboration can be for developers.

What are some best practices for collaborating with PAGs?

Kistler: If you’re going to work with rare disease patients, invest in being a good thought partner. Take the time to do it right and understand the needs of the patient population in terms of daily logistics, dietary requirements, and coordinating care for not only the rare disease patient but for their whole family. Often these patients have rapidly advancing diseases and very little time. I remember one trial where the sponsor did not do proper due diligence and didn’t understand that the target patient population was on a special diet. They developed a drug formulation that infringed on dietary restrictions. So, after enrolling patients and raising their hopes of an effective treatment, the sponsor had to halt the trial and start over with a new formulation.

What are common mistakes to avoid when working with PAGs?

Kistler: Don’t overstate what an experimental agent can do or downplay the risks, of which there are many. For example, gene therapies only work on a small subset of a rare disease population. Even if patients have the relevant mutation, they may have an additional mutation that excludes them from a clinical trial. Patients’ emotions and hopes are sky-high, so the messaging needs to be accurate, science-based, and comprehensive, not aspirational.

Also, cell and gene therapies (CGTs) are still so new that many patients don’t understand the treatments or follow-up requirements. Even physicians may not understand CGTs, and the level of awareness and access can vary tremendously by country. At Parexel, we have a team of in-house CGT experts who support our work with sponsors, PAGs, and the healthcare community to help them with appropriate information and manage expectations.

If you’re going to work with rare disease patients, invest in being a good thought partner - take the time to do it right and understand the needs of the patient population in terms of daily logistics, dietary requirements, and coordinating care for not only the rare disease patient but for their whole family.

Jamie L. Kistler, Ph.D.
Senior Director, Customer Strategy, Medical Communications, 
Parexel International