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Article: Engaging patient advocacy groups - The key to conducting effective rare disease research

Harrison Gaiger, September 2019

Published in Pharma Times

Until recently, the size of the market for specific rare diseases had made it difficult for pharmaceutical companies to justify research and development costs. The introduction of regulatory benefits such as longer market exclusivity, breakthrough designations, reduced fees and tax incentives have encouraged more investment. 

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Nevertheless, where funding has been allocated for rare diseases, the scarcity and geographical dispersal of patients can make it difficult to conduct effective market research. Identifying and recruiting respondents is one of the core challenges when conducting research in this space. It is therefore crucial that researchers make methodology decisions carefully and place a special focus on maximising the efficiency of recruitment measures.

An effective research programme should aim to generate insights from a range of stakeholders, including patients and caregivers, physicians and key opinion leaders.

Engaging with Patient Advocacy Groups (PAGs) makes real sense as they not only provide an informed and representative embodiment of the patient condition, but are also key gatekeepers to recruiting individual patients into the research. While their members face many obstacles to getting help for their condition, PAGs help to build strong, unified communities that work together to develop and advocate for policies that improve patients’ lives and access to new treatments. In addition to their advocacy work, PAGs are a key source of information for patients throughout their healthcare journey. They offer practical advice, emotional support and a wide range of services for people living with a rare disease. It is because of these close interactions that they have a deep understanding of the unmet needs and key challenges faced by patients and their caregivers, making their holistic perspective a critical one for any piece of rare disease research.

PAGs are commonly run by groups of people that are directly affected by the rare disease themselves and can therefore provide real insight into the world of the patient. Fortunately, PAGs are often keen to take part in research because it gives them the opportunity to influence the rare disease landscape and ensures that the voice of the patient is heard when addressing significant issues such as access to care, treatment innovation, and healthcare policies. Furthermore, PAGs are regularly involved in helping to enrol patients in to clinical trials and market research projects, and will offer support to the patient throughout the process. By including PAGs as research partners, the feasibility and success of market research studies can be greatly enhanced by providing access to a much wider group of respondents.

While market research can help bridge the gap between patients and manufacturers, willingness to participate in research can vary widely depending on the therapy area and market. PAGs can augment access, but rightly remain protective of the people whose interests they represent. They also curate varying types of relationship with different companies active in the disease area. Careful consideration needs to be given as to how PAGs will be approached for market research purposes. Building trust and communicating the concept of mutual benefit is the key to engagement; by offering to support PAG initiatives, upholding ethical research practices, and demonstrating that patient welfare and interests are paramount to your approach, a strong relationship can begin to flourish.

For pharmaceutical companies looking to conduct rare disease research, patient advocacy groups are the key to success. Working with an agency that not only has the research experience, but an extensive history of collaborating with PAGs is therefore invaluable.

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