Zhaohui Su highlights ongoing challenges in rare disease trials, particularly in demonstrating therapeutic value for pricing and reimbursement decisions. The reliance on small patient populations and limited patient-reported outcomes complicates evaluations by HTA agencies and payers.
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, ... 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). Zhaohui Su: Rare Disease Trials Face Ongoing Challenges in Demonstrating Therapeutic Value / cancer, Cancer research, OncoDaily, Oncology, patient reported 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.” 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.
Original title: “Zhaohui Su: Rare Disease Trials Face Ongoing Challenges in Demonstrating Therapeutic Value - OncoDaily”
