The Now and Later: Maximizing Your Rare Disease Development Program Now While Driving Your Market Success Later

rare disease research, rare disease clinical trials

A strong rare disease development strategy considers the end goal of approval and adoption at the very beginning of the program by incorporating key stakeholders along the development journey. Patient advocacy groups, medicine agencies, insurers, and key opinion leaders all hold insights which can point your development program on the path to success. By thinking strategically about how you work with these stakeholders, you can uncover the insights you need to direct your program as it is developing and build the foundation for their support when you enter the market later. Here’s how to start building those partnerships:

Create Mutually Beneficial Relationships with Patient Advocacy Groups Early

Regular patient and patient advocacy group (PAG) interaction provides you with the information you need to design a program and product that work for your patients now – while also providing you an avenue for early awareness, interest, and buy-in on your product once it enters the market.

Mutually beneficial relationships result in greater support networks for patients and access to patients for participation in your study. Many PAGs, especially for rare diseases, are early in development and started by patients or families of patients. Providing insight to these groups is invaluable to their growth in support of their patients and a great foundation to build a relationship that leads to patient referrals to your study. In research-heavy indications, these relationships can provide the leg up you need to beat out competition for enrolling the same patients.

Beyond the study benefits, good relationships with patient advocacy groups leads to market potential later. PAGs want the best for their community, and if you’ve built a close partnership, it is likely they believe your product to be a strong option for their patients. Their promotion and support of your treatment post approval can lay the groundwork for a successful entry into the market. Learn more about how to work with PAGs and patients.

Build Relationships with Leading Clinicians

Clinicians that have reached key opinion leader status (KOL) in their indications are knowledge powerhouses. Tapping into them can provide benefits both in the short-term during study development and the long-term for market success.

KOLs provide access to patients and can help you to evaluate the clinical meaningfulness of the product and feasibility of your study design in that indication. As KOLs, if previous trials have taken place in your indication, they likely participated and have seen the successes and failures of those programs. Their insights will be valuable to help you set up your program for success with their patients. Ultimately their involvement early on will more likely lead to their interest in being an investigator and enrolling their patients in your study. And, if you have developed your product in partnership with them, they are more likely to recommend and promote your product to patients and their circles of influence once your product is approved.

Flexibility is Key

Working with these stakeholders, if done well, optimizes your program’s chances for success. You must be able and willing to be flexible and shift your program based on the insights you gather along the way. At Worldwide, our quick ability to pivot and respond sets our rare disease team apart. For insight on how to work with medicines agencies and insurers and how to reach market success read our e-book or contact us directly.

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