shutterstock_577842559.jpgPatient communities are a valuable asset within the life sciences industry. In the rare disease space, these groups are a vital resource for families battling disease, and serve as much-needed patient representatives to help spur funding, research and scientific advancement. ZS’s Rachael Pius weighs in on why pharmaceutical companies should view these patient communities as partners in clinical development. 

Q: What role are patient communities playing in the rare disease space?

A: While emotions run high in rare disease patient communities, they need to have an incredibly strong focus on organizing, building out a business plan with clearly defined goals, and pulling the best scientific minds into advisory committees to shape the research that’s being conducted for any given disease. Because resources are limited, there’s a strong focus on making every second count and advocating to not only have new medications approved that cure, but also to identify key scientific advancements that might extend life or improve symptoms. And because the options are limited, there’s also a push to identify analogue medications that may already be approved for use in other spaces to speed up the process of having something available. In some cases, patients are actively contributing data and donating samples to document their own journey, educate the medical community, and develop end points that are more meaningful for families. However, there’s a large degree of variance in how well organized and structured these patient communities actually are. Some organizations struggle to take a business-minded approach and let emotions dictate their actions while others are learning that a more structured approach yields results. 

In addition, these communities proactively organize annual congresses that offer families and their patients a rare opportunity to connect in person with other individuals who are going through the same lived experiences. The power of the conversations that occur at these meetings has been used to not only motivate families to continue to stay strong, but also has been the nexus to bring new evidence to the FDA and pharmaceutical companies on important patient-reported outcomes and how to better capture these novel end points.

Q: How has the role of the patient community evolved as the healthcare ecosystem changes?

A: Online social presence in the rare disease space has evolved from being a unique differentiator for a community toward being a cost of entry to connect patients and researchers across the globe. Patients utilize their social media presence, for example, to discuss experiences with misdiagnosis, openly solicit feedback on different medications and actively seek emotional support. These conversations are often used as an early pulse check for the community on the impact of new interventions as they are being tested in clinical trials. 

Today, there are more patient key opinion leaders (KOLs) engaging with the FDA and pharmaceutical manufacturers than ever before. Rare disease patient communities are actively participating in public meetings and sharing information with the FDA on unmet community needs, the burden of disease and day-to-day management. Changes in the regulatory environment—including the formation of the Office of Professional Affairs and Stakeholder Engagement and the Patient-Focused Drug Development Program—have further enabled these groups to be part of the conversation. The insights shared by these patients have helped the agency prioritize initiatives based on meaningful patient benefit, and there’s an effort under way in many therapeutic areas to document how patient input is solicited as new drugs are approved. 

There’s also an increased focus within these communities to advocate for clinical trial outcomes that may not be accurately captured in the clinic, and patients are partnering closely with the life sciences industry and regulators to co-create clinical trial protocols that capture these alternative patient-reported outcomes. For example, the six-minute walk test—used to test a patient’s ability to handle sustained physical activity—can be difficult for a child who has had a busy day and a long walk to the hospital from the parking lot. Instead, patient communities are beginning to advise on alternative measures that more systematically capture meaningful milestones in the lives of their patients. For example, families have recommended measuring a child’s ability to use a computer, brush her teeth or feed herself. Patient communities are actively thinking through how some of these proxy measures might demonstrate decreases in time to disease progression as patients participate in clinical trials and are actively seeking opportunities to share their insights with regulators.


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Q: How can life sciences companies partner with patient communities, especially those in the rare disease space?

A: While the life sciences industry has worked for years to channel funding to programs focused on supporting rare disease communities, we now see the industry working toward partnerships with these communities, and it’s clear why. Companies need access to these patients, their experiences and their input to build effective clinical and commercial interventions that support patients in their disease journey.

With very high personal and business stakes at play, it’s imperative that rare disease patient communities and pharmaceutical manufacturers align on “how” they can best interact—the set of guidelines that enable all parties to maximize the engagement. This will ensure that both parties are responsive, transparent and accurately setting expectations surrounding what information is valued and how data and funding will be used. Similarly, it’s important that patient communities are clear about their mission, maintain independence, and are open about what lines they aren’t willing to cross. These groups should be prepared to proactively reach out to more than one manufacturer with a concrete business model and a clear description of need. In return, manufacturers should not only share funding but also be held responsible for sharing insights and lessons learned with the community.

The relationship between patient communities and the life sciences industry remains both critical and complicated. We’re seeing more and more cases of patient communities taking a structured and business-like approach to these partnerships, and this resonates in a much more effective way with manufacturers. These types of partnerships also require alignment between the missions of both the patient community and the pharmaceutical organization, as well as a mutual respect for one another’s mission. For example, patient communities aren’t working to advocate for any one particular drug, despite partnering with manufacturers. The patient community’s goal is to advance the science in meaningful ways toward improvements in their condition. On the other side, the pharmaceutical organization has specific needs for engaging patients, understanding their challenges, obtaining samples, securing participants for trials and soliciting ongoing feedback. Effective partnerships recognize the needs on both sides of the table and seek to achieve mutual benefit.

Many patient advocacy groups are educated entities and have a lot to offer, and many individuals in these groups are deep KOLs in their space. Treating them as a partner in clinical development and not a resource will continue to bridge the challenges, and sometimes skepticism, that may exist about what can be accomplished together.


Topics: Pharma, rare diseases, clinical trials, patient communities, rachael pius, RWE