As healthcare costs in the United States have continued to grow, there has been an increasing desire to move towards “value-based” care in which patients and payers would be paying for the “value” of the treatment patients receive. This idea has been gaining particular momentum as policymakers look for strategies to curtail spending on pharmaceuticals. While there is merit in paying for services and treatments that work and eliminating wasteful spending, it is important to step back and consider to whom “value” is being provided. As the ultimate beneficiary, we would advocate that the measure of “value” in a healthcare setting should focus on value to the patient, but currently, we are concerned that the trend is to look towards “value” to the payer by prioritizing reduced cost over care outcomes for patients in the form of cost-effectiveness analyses. 

​Cell and gene therapies (C&GTs) are revolutionary advancements that offer potentially life-altering therapies – and in some cases even cures – for patients with rare and severe diseases. Around the globe, various health technology assessment (HTA) and value assessment organizations are attempting to estimate the health and economic value of new C&GTs to better inform healthcare decision making. These organizations most commonly employ cost-effectiveness analysis (CEA), which seeks to determine whether the costs of a given therapy are justified by its benefits; that is, whether it is a “good buy.”

Tony Coelho sent a personal letter to the National Academies of Sciences, Engineering, and Medicine to amplify the comments submitted by the disability community in response to the Discussion Draft of the Preliminary Framework for Equitable Allocation of COVID-19 Vaccine, commissioned by the Centers for Disease Control and the National Institutes of Health. He stated, "In my experience as a longtime disability rights advocate and an original of the Americans with Disabilities Act, I view this as a process that is aligned with our mantra of 'Nothing About Us Without Us' and therefore urge you to outreach directly to our disability community as you wrestle with difficult questions related to vaccine allocation even after the comment deadline.” He highlighted letters submitted by the Consortium for Citizens with Disabilities, the Association of University Centers on Disabilities, the American Association of People with Disabilities, and Not Dead Yet.