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“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…

Comments submitted to the agencies reveal a growing divide between public health groups that urge FDA and USDA to move quickly and industry organizations that argue existing frameworks are too broad