Rare Disease Health Technology Assessment Working Group – 2022

OBJECTIVE:

Provide guidance, identify best practices, and set policy on coverage decisions for rare disease treatments to ensure patients can easily access and afford approved treatments.

SCOPE:

• Review the challenges of health technology assessment (HTA) in Europe for rare disease treatments and the impact of those challenges on patients.
• Understand the history and effectiveness of HTA efforts in the United States.
• Assess the current U.S. access landscape for rare disease patients.
• Outline strengths and weaknesses of existing HTA programs and insurance coverage for rare disease patients.
• Evaluate strategies and develop guidelines to improve coverage and affordability of rare disease treatments.

BACKGROUND:

Heath technology assessment (HTA) is used to evaluate health technologies and guide coverage and pricing decisions in many developed economies, including Australia, Canada, France, Germany, Japan, and the United Kingdom. Recognizing the inherent challenges in research and development of treatments for rare diseases, regulatory bodies in those countries often use more flexible approval processes to approve such treatments. 

Reimbursement policy, however, rarely follows suit. Standard HTA methods require robust information on comparable efficacy, effectiveness, and associated costs of new healthcare interventions that can be difficult or impossible to generate for small patient populations.  Even if sufficient data can be collected for a particular rare disease therapy, it still unlikely to meet the arbitrary cost-effectiveness ratios needed for reimbursement. Standard HTA methods simply cannot assess the “value” of rare disease treatments to patients, caregivers, their families, and society – because they are not designed to do so. 

While some federal programs and state Medicaid agencies use HTA in limited circumstances, the United States has historically preferred a market-oriented approach and a decentralized insurance system, not a national HTA program. The last few years, however, have seen increasing attempts to more broadly apply HTA systems in U.S. healthcare. In the absence of a national HTA program, private entities like the Institute for Clinical and Economic Review (ICER) have attempted to take on the role of evaluating and determining the “value” of new therapies. 

ICER and groups like it have a troubling pattern of ignoring the needs of patients living with rare diseases, resulting in decisions that would limit access and affordability for approved therapies. We must find policy solutions that do not erect additional barriers to care for patients with rare diseases.