Challenges in Preserving Access to Orphan Drugs Under an HTA Framework

Executive Summary

Under pressure to address rising health care costs for American families, policymakers in the U.S. are increasingly looking overseas for potential policy remedies, particularly with respect to drug pricing. Advocates for establishing a national health technology assessment (HTA) body in the U.S. argue that such systems enable payers to set coverage and reimbursement policies based on a treatment’s value to patients and society. While placing a dollar value on life across diverse patient populations remains controversial generally, HTA’s one-size-fitsall approach may be particularly problematic when it comes to orphan drugs developed to treat rare disease.

Assessment practices by HTA bodies around the world were created to evaluate widely used medical technologies and do not fully account for the unique value proposition of orphan drugs, which provide significant clinical benefits to the millions of Americans afflicted with rare disease. For this reason, few HTA bodies use standard assessment practices for orphan drugs. Instead, for orphan drug evaluations, most HTA bodies provide targeted exceptions to their value assessment policies. These exceptions may include:

Assumed benefit thresholds – Allow approved orphan drugs that remain below a certain cost threshold to be automatically deemed beneficial to patients—meaning that HTA bodies cannot issue a negative reimbursement decision.
Specialized HTA pathways – Offer a more flexible approach to evaluating pharmaceuticals for rare disease and increase the willingness to pay threshold to accommodate increased costs.
Higher willingness to pay thresholds – Place a higher willingness to pay threshold for health gains accruing to patients with rare diseases.
Pre-authorization programs – Allow certain orphan drugs to receive reimbursement decisions prior to market authorization.

Even with these exceptions, in practice, patient access to treatments is restricted under HTA. Based on the Center for Healthcare Economics and Policy’s analysis of access and outcomes in countries with centralized, government-run HTA bodies, we find the process has the potential to delay or deny access to treatment for those living with rare disease through inadequate reimbursement and pricing recommendations. HTAs may not only limit access to existing treatments, but could also significantly decrease the likelihood of new orphan drugs coming to market, denying patients with rare disease the hope of future treatment.

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