Interview With Liz Arnold and Betty Tran
Q1. What is your background and experience in rare disease commercialization, including key lessons from bringing therapies to rare disease patient populations?
Liz: My background is in global commercial strategy, with a focus on rare disease. I’ve worked across multiple disease areas helping companies decide which markets to prioritize and how to position therapies for launch and sustained growth. A key lesson has been the importance of evaluating commercial viability and unmet need early in development to ensure long-term value and uptake. I’ve also seen how essential cross-functional collaboration is especially in rare disease, where patient populations are small, and every strategic decision carries significant weight.
Betty: My background is in patient support services and have launched several commercial products in rare diseases. Some key lessons in the rare disease patient population are the flexibility and adaptability based on advancement in sciences and in patient’s care
Q2. How do you foresee the rare disease market evolving over the next 5–10 years, particularly in terms of pricing, access, and innovative commercial models?
Liz: I envision better genetic testing and screening will uncover more patients earlier, driving demand for earlier intervention. But with more therapies entering the market, payers will likely push harder on value. I think we’ll see more creative payment models to balance innovation with affordability
Betty: Patient’s voice and power of choice will play a dominate role in how we commercialize rare disease products. As new treatment comes to the market, pulling in the patient’s voice and incorporating their needs early in the process will ensure the UX is optimal.
Q3. What are the biggest hurdles in achieving commercial success for rare disease therapies, from reimbursement challenges to patient services and what strategies can overcome them?Â
Liz: Finding the right patients early enough is still the biggest barrier. Many are misdiagnosed or stuck in the system for years. Investing in diagnostic partnerships, real-world data, and strong KOL networks can have a meaningful impact. Ensuring the therapy fits into existing care pathways; clinically and logistically is also key to driving uptake.
Betty: Some of the biggest hurdles in rare disease are finding these patients early in the commercialization process to gain insights and watchouts. Patient support programs should be designed around the patient’s needs to get started on a treatment and maintain on therapy. Some strategies to overcome these hurdles are to do early market research and participate in advocacy groups to fully understand the patient’s experience.
Q4. What are you most looking forward to at the Commercial Excellence in Rare Disease Summit? Are there any sessions or discussions you think will be particularly impactful?
Liz: I’m looking forward to connecting with peers who are passionate about advancing rare disease care and sharing ideas on how we can move beyond traditional models to better serve patients.
Betty: I look forward to meeting passionate people who desire to deliver the best patient model to the rare disease community.
