Pam Cusick, senior vice president at Rare Patient Voice (RPV) shares why embedding the voices of patients and caregivers in research remains as important as ever

Written by Pam Cusick, senior vice president, Rare Patient Voice

In the rare disease community, the insights of patients and caregivers are invaluable. At Rare Patient Voice (RPV), we believe that their voices are critical to advancing research, improving products, and ensuring that solutions truly meet the needs of those they serve. Over the past decade, we’ve championed this philosophy, enabling meaningful engagement and results in over 10,000 studies. I’d like to share why participating in research matters and how it’s shaping the future of healthcare for rare diseases.

Why participate in research?

Patients and caregivers are the frontline experts in living with rare diseases. Their lived experiences offer researchers an authentic understanding of what works and what doesn’t. When patients share their needs, preferences, and challenges, they guide pharmaceutical companies, device manufacturers, and policymakers to develop practical and beneficial solutions. Their feedback can help improve the lives of others living with their rare disease as well as future patients and families, representing a true legacy of making a difference.

Participation also provides a tangible benefit: honoraria for their time. At RPV, we compensate participants, recognising their contributions as critical expertise. Just as professionals are remunerated for their insights, so too should patients and family caregivers.

Are companies really listening?

Yes, they are. Companies increasingly recognise the value of patient input, involving them earlier in product development. For example, we’ve seen patient feedback reshape clinical trial consent forms, making them more comprehensible and user-friendly. These changes reflect a broader trend: designing solutions with the patient in mind from the outset, not as an afterthought.

One memorable story involves a usability study for canes. A team member shared their feedback during the research, and those insights directly influenced the final product. Patients frequently report seeing the outcomes of their input, whether it’s a new device on the market or an advert featuring a product they helped refine.

Evolving types of research opportunities

Research participation has become more accessible and diverse. Many studies are now remote, utilising online surveys, video interviews, and virtual focus groups. These formats allow patients and caregivers to contribute from the comfort of their homes, making participation less burdensome.

Clinical trials, while more intensive, also benefit from evolving models like decentralised trials, where mobile nursing units bring the study to the patient. Such innovations reduce logistical challenges and make participation more inclusive.

Addressing barriers to participation

Despite the benefits, patients and family caregivers often have valid concerns about joining research studies. Time constraints, questions about data security, and uncertainty about the impact of their participation are common hesitations. Transparency and communication are essential in overcoming these barriers. At Rare Patient Voice, we strive to provide clear information upfront about time commitments, the nature of the study, and how their data will be used. Our goal is to ensure participants feel informed and valued every step of the way.

How to get involved

Signing up with Rare Patient Voice is simple and open to patients and family caregivers across various conditions and geographies. Once registered, participants receive tailored opportunities that match their experiences and availability. Even if a study isn’t immediately available, registering ensures individuals can be contacted for relevant opportunities in the future.

Participation isn’t limited to disease-specific research. Many studies focus on broader issues, like mobility challenges or hearing impairments, opening doors for more people to contribute their expertise.

Closing thoughts

The rare disease community’s participation in research is a vital driver of progress towards innovation, improvement, and cures. By sharing their voices, patients and family caregivers help shape a future where products and services are truly tailored to their needs. At Rare Patient Voice, we’re proud to be part of this journey, empowering the community to make an impact, one study at a time.

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About the author
Pam Cusick is the senior vice president at Rare Patient Voice, where she leads initiatives to amplify patient and family caregiver voices in rare disease research. She is passionate about connecting the rare disease community with opportunities to shape the future of healthcare. Connect with Pam here.

To find out more about participating in research opportunities via RPV please visit https://rarepatientvoice.com/  or to register visit https://rarepatientvoice.com/for-patients/patients/

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