With the biopharma industry’s looming wave of gene therapy submissions and potential approvals, the senior senator is laying the groundwork for a legislative initiative to improve access to these expensive treatments.
Pictured: The U.S. Capitol building/iStock, Mikhail Makarov
Sen. Bill Cassidy (R-La.), ranking member of the Senate Health, Education, Labor, and Pensions Committee, is asking stakeholders for feedback regarding gene therapies, with an eye toward crafting legislation that could make these expensive treatments more accessible for patients.
“Policymakers have a responsibility to evaluate the market structure and identify where targeted changes can be made to ensure market access for patients with ultra-rare diseases,” Cassidy wrote in Tuesday’s request for information. “Any proposal put forward by Congress should be informed by the current work of states and the private market.”
Cassidy is looking for guidance on defining ultra-rare diseases and identifying patients who should be eligible for new coverage schemes. The Republican senator is also looking into the current payment and access practices for patients with ultra-rare diseases: how they pay for their therapy, how physicians provide access to these treatments and what programs—state-sponsored or otherwise—are there for them to leverage.
In addition, Cassidy is asking stakeholders—including health insurers and manufacturers—how they decide on a list price for gene therapies and how they manage the financial risk of covering these expensive treatments.
Responses to Cassidy’s request for information are due by Jan. 22, 2024, and will help lawmakers determine the appropriate role of the federal government in maximizing access to gene therapies without inadvertently hurting innovation, according to the announcement.
“Given the current limited number of approved gene therapies, manufacturers and commercial health plans alike are currently able to absorb costs in order to provide patient access,” but this current market environment is unsustainable, according to Cassidy, particularly as approvals in this space “are expected to grow dramatically over the next decade.”
Cassidy warned that “artificially low prices could promote patient access, but eliminate the incentive to bring these treatments to market.” At the same time, without pricing safeguards, “the small patient population runs the risk of bearing the full weight of the high price tag,” he noted.
The initiative in the U.S. Senate to draft legislation tackling the access and affordability of gene therapies comes as the industry and the FDA anticipate a looming boom not only in cell and gene therapy applications, but also in approvals.
On Friday, the FDA is set to release its verdict on yet another gene therapy, Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel), for the treatment of sickle cell disease. Neither company has indicated how much they plan to market the therapy, if approved, but it is likely to cost well above $1 million for a single dose.
In November 2022, the FDA approved CSL’s Hemgenix (etranacogene dezaparvovec-drlb) as the first gene therapy to treat adults with hemophilia B. The treatment costs $3.5 million per dose, a price tag that has won it the title of being the most expensive drug in the world.
Bluebird bio, also focused on cell and gene therapies, owns Zynteglo (betibeglogene autotemcel), an ex-vivo lentiviral treatment indicated for beta-thalassemia, and Skysona (elivaldogene autotemcel), indicated for active cerebral adrenoleukodystrophy. The therapies cost $2.8 million and $3 million, respectively.
Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.
