Thought Leadership

“One-Size-Fits-All” HTA would be a Major Setback for Rare Disease Patients

By: Dr. Jason Shafrin of FTI Consulting. This op-ed was written in response to a new white paper published by Alexion, AstraZeneca Rare Disease, a company member of the Rare Disease Company Coalition.

Although individual rare diseases have a low prevalence, one in 10 Americans is affected by one of 7,000 known rare conditions. Approximately 37 percent of rare disease patients have reduced life expectancies due to a lack of treatment options for conditions, and currently, 95 percent of rare diseases have no treatment option.[i]

Legislation, such as the Orphan Drug Act, helped increase the number of rare disease treatment options available; however, the steadily rising costs for these treatments are a cause of concern for lawmakers. As a result, policy experts are calling for a health technology assessment (HTA) framework that would determine whether the value of a pharmaceutical warrants its price.[ii]

Despite intentions to curb rising health care costs, the implementation of an HTA framework could stifle the balance of innovation in and access to orphan drugs. Research from FTI Consulting’s Center for Healthcare Economics and Policy on how orphan drugs would be affected under an HTA framework finds that the HTA framework could negatively impact innovation, development and access to orphan drugs. This is due to a tendency for many HTAs to apply a one-size-fits-all framework to orphan drugs for rare diseases, which inherently have unique needs and are high-risk endeavors.

The research finds that adopting a national HTA body based on the practices of the Institute for Clinical and Economic Review (ICER) could severely curtail access to orphan drugs for rare diseases.[iii] The millions of patients in the United States who already struggle to gain access to treatment for rare conditions cannot afford to lose even more access to treatment.  For example, a  recently approved therapy for spinal muscular atrophy underwent significant approval delays in the United Kingdom (among other countries) because the UK’s HTA body—known as NICE—determined that the price was too high to justify the treatment gains, despite robust clinical evidence that the drug showing a “substantial benefit.”[iv]  Even for those on treatment, rare disease patients can benefit from improved innovations that help enable them to live longer, fulfilling lives.

Many of the value assessments conducted by current HTA bodies fail to account for broader societal benefits orphan drugs provide nor do they take into account how orphan drugs could reduce health disparities.  Few HTA bodies value assessments framework incorporate value from allowing patients to return to work or school, or whether new treatment reduce the burden on parents caring for children with rare diseases. Impacts on health disparities are also largely ignored within traditional value assessment and patient advocates are concerned that negative HTA assessments will impact access.  Regarding ICER’s negative assessment of new myasthenia gravis treatments, one patient advocate group stated that any barriers to care, such as ICER’s negative evaluation, “… will likely further disproportionately impact Black patients and exacerbate health inequities”.[v]

Looking ahead, applying an HTA framework to orphan drugs for rare diseases could not only hamper access to existing rare disease treatments, but also it could also decrease the likelihood that new orphan drugs will be introduced to the market.  Such as the Congressional Budget Office have found that government price controls—whether due to HTA value assessment or other sources—would significantly reduce the number of new treatments that are brought to market.[vi]

As Congress is assessing current health care initiatives and programs for the future, now is an important opportunity to examine how access to rare orphan drugs are impacted through assessment under the national health technology assessment framework.

As the research points out, a one-size-fits-all model has potential negative consequences for access to and development of orphan drugs. Industry leaders, policymakers, and patient advocates can use this research as a starting point for a timely discussion on how to preserve access and innovation for orphan drugs. In order to properly address potential negative outcomes of all frameworks, a discussion is the first step. Now more than ever, it is imperative that we convene all stakeholders to build on this research. Millions of Americans living with rare diseases are depending on it.

The views expressed herein are those of the author(s) and not necessarily the views of FTI Consulting, Inc., its management, its subsidiaries, its affiliates, or its other professionals.

 FTI Consulting, Inc., including its subsidiaries and affiliates, is a consulting firm and is not a certified public accounting firm or a law firm.


[i] Stoller, James K. “The Challenge of Rare Diseases.” ScienceDirect, June 2018.

[ii] Lakdawalla, Darius et al., “Health Technology Assessment in the U.S.-A Vision for the Future.” USC Schaeffer. February 9, 2021.

[iii] FTI Consulting, “Challenges in Preserving Access to Orphan Drugs Under an HTA Framework.” December 2, 2021.

[iv] Taylor, Phil. “Shock as NICE Turns Down Biogen’s SMA Therapy.” PMLive, August 14, 2018.

[v] “ICER Myasthenia Gravis Public Comment.” ICER. ICER, April 2, 2021.

[vi] Congressional Budget Office. CBO’s Simulation Model of New Drug Development. August 2021.