Rare Disease Patient Voice
PRA Health Sciences
PRA Health Sciences

The rare disease landscape is growing and changing rapidly. Not only are more drugs and therapies being approved for these diseases and conditions, but rare diseases are being discovered more frequently and there is an increase in the amount of research being done. More companies are investing in this research and in these disease communities.

PRA will be at the 2018 RARE Patient Advocacy Summit in Irvine, California, the largest gathering of rare disease patients, advocates, and thought leaders worldwide.

Uniquely, there is also an incredible amount of research being driven by patients and patient organizations. As more rare diseases are being discovered and researched, more individuals impacted by these diseases are coming together to form organizations to help drive change. Rare disease patients and patient organizations are increasingly taking a more active role in the research that is being done in their space. They are doing everything from creating elaborate support networks, finding researchers, funding research grants and projects, funding animal models, creating natural history studies and registries, and even starting biotech companies. They are learning and evolving at an accelerated rate.

This increase in knowledge, activity and progress is benefiting companies working in the rare disease space. Rare disease patients (and caregivers) offer a unique perspective and have tremendous amounts of knowledge about their diseases and the diseases impacting their loved ones. Even with the technological advances and increase in the amount of research being done in the rare disease space, there are still a lot of unknows and aspects of these diseases that are not well understood.

Patients can provide expert level knowledge about their diseases, detailed natural history and registry data, they can help identify endpoints, aid in protocol design, help with site selection, assist in clinical trial awareness and even advocate during the regulatory review process. They can also help drug developers understand the dynamics of the community and ensure that any research study material is patient/caregiver facing. From pre-clinical to market access, rare disease patients and advocacy groups can help inform, educate, and guide drug developers.

The PRA rare disease team believes that patients, caregivers and patient organizations are vital partners throughout the entire clinical research lifecycle as well as critical stakeholders in any clinical development program. They provide vital pieces to these very complex puzzles. Partnering with patients and patient advocacy organizations to ensure that we understand these communities, their unique challenges and needs, and working with them as partners in everything we do, is the heart of PRA Health Sciences’ Center for Rare Diseases approach to clinical research. The immense contributions these individual’s groups can provide are an essential component of every step of the team’s process. They work to engage with patient groups as early, often and intentionally as possible in order to begin to work toward a shift in the way that rare disease clinical trials are done.

With patients/patient organizations as our partners, we can shift the aspects of traditional clinical research that may not make sense for rare disease studies by ensuring patients are at the center of each study design, as well as an active partner throughout the process. We can work to design studies, so they are more attuned to patients’ lifestyles and schedules and what will work best for them.

The industry is encouraging drug developers to think about patients as partners in the drug development process. Ultimately, they are the ones who are using their products and new medicines and who have the most valuable opinions and insights into what they want and need as well as what will be realistic for them to participate in clinical research.

By thinking of patients as those who will be using these therapies/medicines and considering their behaviors and their preferences, we know that advancing new rare disease therapies through primary care physicians and traditional approaches will become increasingly difficult. We can benefit from understanding and adapting to the preferences of patients by using technologies to communicate and reshape the rare disease space by accelerating the development of new rare disease therapies through a connected data-driven, patient (consumer)-focused approach to rare disease clinical development.

When we begin by thinking about and engaging with the patients we are working for, and understanding their challenges, needs, wants and limitations, we set the stage for creating a clinical trial paradigm that is accessible to these patients and makes sense to them. When patients and patient organizations are partners from the beginning and continue to be active partners throughout the process, we set ourselves, and the rare disease community, up for success.

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