Policy Summit Speakers Discuss Access to Testing and Treatment

On the final day of the ASGCT Policy Summit, speakers focused on payment and access issues related to gene and cell therapies as well as newborn screening policies.

The morning opened with remarks from U.S. Senator Tim Scott of South Carolina, who is co-chair of the bicameral and bipartisan Congressional Personalized Medicine Caucus. Scott indicated his support of getting approved treatments to patients through his role on the caucus, and mentioned his work on legislation that would unleash innovative value-based payment models that address the unique nature of gene therapies.

Following Scott was keynote speaker Joe Grogan (former director, U.S. Domestic Policy Council), who  addressed the importance of active industry involvement in the next iteration of the Prescription Drug User Fee Act (PDUFA), which will be reauthorized in 2022. He advised industry to submit fully baked proposals to FDA on issues such as attracting the best scientists as reviewers and getting the fellowships it needs.

“I believe in this industry. I believe in this technology,” Grogan said, but added there are profound challenges to get this into the healthcare system.

He posited that the most likely scenario to improve patient access to an approved gene therapy would be a disease-specific program, such as that which exists for HIV, with the potential for providing creative funding mechanisms. When asked about the role of the federal government in payment policy, he said he would like to see the federal government become a backstop so that when there is a market failure, the federal government could step in.

The first session on real world experiences of gene therapy access brought together payment experts, industry, and a patient perspective. Dan Symes, and his wife, Jodi, talked about their treatment journey after Dan was diagnosed with B-cell non-Hodgkin’s lymphoma. Despite chemotherapy treatments, Dan was told his cancer was progressing and he only had a few months to live. His oncologist referred him to a CAR T-cell therapy clinical trial. In 2016, Dan got his CART cell infusion. By the time he left the hospital 10 days after treatment, he was able to walk again. Now, Dan is coming up on four years in remission.

Since the treatment, “we’ve been privileged to have this time together and to enjoy our friends and our family and each other,” Jodi said.

Although Dan’s immune system is weakened, he said it has been improving. After retiring in 2018, Dan and Jodi were able to take the 6,000-mile boat trip that he has always wanted to take. They’re currently planning their next trip and living every day to the fullest—something they recognize would not be possible without the treatment Dan received.

The session continued with Adam Hutchings (Dolon) discussing patient and market access to approved therapies in the EU. Hutchings gave an overview of the patient access pathway to advanced therapy medicinal products (ATMPs) in Europe, which includes health technology assessments followed by price negotiations with public payers. He observed that quite a bit of political pressure currently exists for some governmental flexibilities in assessing gene therapies because of the highly positive outcomes, but it is questionable whether such flexibilities will be sustainable as more products come to market. He noted that the price of ATMPs has been quite similar between the U.S. and Europe, but price increases are rare in the EU, though there are some examples for orphan products.

Andrew Walker, Ph.D. (Salus Alba) addressed cost effectiveness for gene therapies in the pipeline in the EU. Walker has worked for health technology assessment bodies (HTAs) including the UK’s National Institute for Health and Care Excellence (NICE). He explained HTA consideration in making cost-effectiveness determinations. Payers can, do and will pay for cell and gene therapies, Walker said, but they will not pay just because it is a cell or gene therapy, and they may not pay the list price.

He provided advice for product developers in providing evidence of value, such as choosing the right comparator treatment to demonstrate savings; measuring quality of life; and taking early scientific advice from an HTA.

The last speaker in the session was Nicola Heffron (bluebird bio) who talked about her company’s value-based payment over time approach, noting the difficulty of adoption by payment systems with annual budgets. The good news, she said, is that progress is being made in addressing these challenges through the efforts of various stakeholders in the system.

The last session focused on newborn screening policies in the U.S. Diane Berry, Ph.D. (Sarepta Therapeutics) talked about the importance of screening all newborns for genetic, metabolic, hormonal, and functional conditions not otherwise apparent at birth, noting that 35% of deaths in the first year of life are due to rare diseases. In the U.S., HHS recommends that states screen for a list of disorders on the Recommended Uniform Screening Panel (RUSP). Rare disease leaders have been collaborating to modernize newborn screening, Berry said, as diagnostic advances offer promise for screening for many disorders simultaneously. Annie Kennedy (EveryLife Foundation for Rare Diseases) emphasized that screening panels are specific to each state, so some states screen for fewer than 30 diseases while others screen for as many as 60. States need infrastructure and resources to keep up with the pace at which gene therapies become available, Berry added.

With recent approvals of gene therapies, Kennedy said, the rare disease community is cautiously optimistic. Still, many babies are born eligible for therapies and are not being identified.

“We can do better,” she said.

Kennedy also discussed her involvement in a newborn screening pilot program in New York for Duchenne muscular dystrophy. Pilot programs are necessary for nomination to RUSP, but cost millions of dollars, Kennedy said, and patient advocacy organizations can’t fund that cost alone. She and Berry agreed it’s critical for advocacy groups to have industry partners.

Thank you for joining us at the 2020 Policy Summit! If you missed anything this week, all sessions are free for registered attendees to watch on the meeting website for 30 days. You can read the recaps of the first two days on our blog.

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