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Zhaohui Su: Rare Disease Trials Face Ongoing Challenges in Demonstrating Therapeutic Value
Zhaohui Su, VP, Strategic Consulting at Veristat, shared on LinkedIn:
“Obtaining regulatory approval is one part of the challenge for rare disease therapies. Following approval, HTA agencies and payers evaluate the clinical, economic, and patient-centered value of a therapy to inform pricing, reimbursement, and patient access decisions. Historically, rare disease trials have often relied on small patient populations, single-arm or open-label designs, surrogate endpoints, and limited patient-reported outcomes(PROs). While these approaches are frequently necessary due to the realities of studying rare diseases, they can introduce uncertainty when assessing the magnitude, durability, and treatment benefit.
A recent study reviewing pivotal trials for rare disease drugs expected to enter the market showed encouraging trends, such as an increased use of randomized controlled trials (RCT), active comparators, and double blind designs. However, challenges remain, particularly the ongoing reliance on surrogate endpoints and the limited incorporation of PROs
The key question is whether a trial generates the evidence needed to demonstrate value to HTA bodies, payers, clinicians, patients, and other stakeholders. As reimbursement decisions increasingly rely on comparative effectiveness, patient-centered outcomes, and real-world value, evidence generation strategies must be designed with these requirements in mind from the outset
This underscores the importance of integrating clinical development, regulatory strategy, market access planning, and evidence generation early in the development process. A well-designed program can reduce uncertainty at launch, strengthen the value proposition, and enhance the likelihood that innovative therapies reach the patients who need them most
If you’re evaluating evidence-generation strategies for a rare disease program, contact Veristat’s Strategic Consulting team for a conversation.”
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cancerCancer researchOncoDailyOncologypatient reported outcomesRandomized Controlled Trialsrare diseasesVeristat Strategic ConsultingZhaohui Su
