Challenges in Preserving Access to Orphan Drugs Under an HTA Framework

That is a title of a new white paper from FTI Consulting and Alexion. The key findings from the article are:

HTA practices are designed to be used for medical technologies and pharmaceuticals that treat commonly-occurring diseases, and are not fit to determine the value of orphan drugs for rare disease, which face unique challenges.
Estimating the value or clinical improvement for orphan drugs is complicated by small patient groups, making it difficult for HTA bodies to precisely estimate clinical and economic value and set a value-based price.
Standard HTA that uses value-based price evaluation does not properly account for the value of orphan drugs for rare disease patients, especially when they are the only treatment for a given disease. Instead, health gains are treated the same for all patients regardless of disease severity.
Standard European HTAs have effectively restricted access to orphan drugs, delaying or denying access to treatment for rare disease. While many countries with HTA bodies offer orphan drugs special considerations in the assessment process, these accommodations are often insufficient to overcome the challenges of applying the HTA framework to orphan drugs.
Quality-Adjusted Life Years (QALYs), the most commonly used tool to assess a drug’s value through the HTA process, fail to measure many of the life-altering benefits that orphan drugs bring.5 As a result, HTA bodies are more likely to deem orphan drugs unworthy of their price and reliance on QALYs could restrict the number of orphan drugs that come to market.
Cost-effectiveness considerations may result in low reimbursement rates for orphan drugs. The use of price thresholds in any capacity creates a disincentive for rare disease research by cutting into manufacturers’ return on investment.
If the U.S. were to model a national HTA body based on the practices of the Institute for Clinical and Economic Review (ICER), access to orphan drugs could be severely curtailed. As of 2020, all orphan drugs in the U.S. received a negative ICER assessment.
The value assessment frameworks that many HTA bodies employ do not take into account the benefits new medical technologies may provide in terms of reducing health disparities among racial and ethnic minority groups.
The HTA value assessment framework may inadvertently perpetuate health disparities by undervaluing certain benefits, such as the diminution of burdensome side effects of existing treatment alternatives, that improve both quality of life and health outcomes – particularly in Black patients.

For more details, do read the whole report here. The white paper was also discussed as part of a forum on value assessment in rare diseases with The Hill last Friday.

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