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Patient Access to Gene Therapies

Gene therapies have the potential to transform a patient’s quality of life. But how will a complex network of stakeholders figure out the question of access?

DNA strand and other medical icons

As more gene and cell therapies become available, it’s natural that all stakeholders, especially patients, want to be know how they can best access these potentially game-changing treatments.

It was a topic on the agenda at the 2022 National Organization for Rare Disorders (NORD) Breakthrough Summit. The question is essential because gene therapies aren’t like other kinds of medicine. When successful, they could eliminate the need for a lifetime of regular treatments and ultimately save the health care system millions of dollars.

“We need to see innovation in the way that we think about coverage and reimbursement that mirrors the innovation we are seeing on the clinical side,” Jack Rollins, Director of Federal Policy for the National Association of Medicaid Directors said at the NORD summit in October. Medicaid is a government-funded health care program that assists some lower-income U.S. citizens.

“To get the financing right is imperative if we are going to see access and achieve clinical promises that we are so hopeful we can all realize,” Rollins said.

Value-based contracts are one strategy being discussed because they could be a way to give patients access while spreading the costs and the benefits over time. A “portability mechanism” could spread costs across insurance providers as a U.S. patient moves, for instance, from Medicaid to commercial coverage and into Medicare later in life, Rollins said.

Mary-Lacey Reuther, who heads North American Policy, Advocacy and Government Affairs at CSL, said the key will be collaboration among various stakeholders, such as patients, governments, payors, policymakers and the companies, like CSL Behring, that are developing the new treatments. The entire health system must derive value, which will ultimately enable access, so it’s “really, really important that we get this right,” she said.

“This is a partnership – that’s how I view it,” Reuther said. “Industry is partnering with governments, partnering with patients, partnering with all policy makers.”

The Institute for Clinical and Economic Review (ICER) is an independent health technology assessment body that uses evidence to determine the value of a new therapy after considering factors like durability of benefit, long-term safety and incentive for future innovation, ICER’s Vice President of Research Foluso Agboola said at the NORD Summit. One way to calculate value is to compare a new therapy’s cost to the cost of a lifetime of treatments on the current regimen, Agboola said. In a recent analysis of gene therapies for people living with bleeding disorders, ICER found that gene therapies would represent a “large cost savings.”

Though health care is funded differently in Europe, some of these same questions are being asked there. Since 2021, CSL Behring has been recording a podcast series about cell and gene therapy, including an episode shared last year on access, featuring Yann Le Cam, CEO of EURORDIS and Elisabetta Zanon, Director, EU Public Affairs & Advocacy, Alliance for Regenerative Medicine.

On the podcast, they said that giving patients access to gene therapy treatments will require collaboration and exploration of outcomes-based payment models – alongside quality evidence on long-term effectiveness.

“In terms of speeding up access to innovative therapies, innovative payment models should be considered,” Zanon said.

Listen to the full podcast episode: Advancing Europe’s leadership in rare disease innovation