Why Cell & Gene Therapy Companies Should Talk with RFK Jr.

Tim Hunt, CEO, Alliance for Regenerative Medicine

As the CEO of the cell and gene therapy advocacy organization the Alliance for Regenerative Medicine (ARM), I’ve been asked one question more than any other amid the constant barrage of news headlines the past two months.

How are you approaching the Trump Administration?

I hear it from across our community, including biotechnology companies, academic and medical research institutions, patient advocacy groups, investors, and our Board of Directors.

Here is how I explain it, starting with important context.  For many of our major stakeholders, cell and gene therapy (CGT) represents highly disruptive technology, much more akin to AI than small molecules. It elicits tremendous hope but also raises justifiable questions and concerns — from patients, payors, policy leaders, and religious leaders. So, we engage with everyone to share our views on the technology and meet people where they are. Everyone gets our respect and our time.

We are treating the new Administration and Congress the exact same way.

I first came to Washington, DC, as a young man during the George H.W. Bush Administration and I’ve seen challenges and opportunities associated with every administration since. At ARM, it’s our duty to recognize — and operate within — this reality and advocate for the patients we serve. 

During the first Trump Administration, the FDA approved the first gene therapies for rare genetic diseases and the first CAR-Ts for blood cancers and proposed a rule that encouraged the use of outcomes-based payment arrangements in Medicaid. But the same administration proposed tying the prices of physician-administered drugs in Medicare Part B to the prices paid in foreign countries, which would have dramatically reduced prices and discouraged further investment in cell and gene therapy startups. The Trump Administration also implemented an immigration ban that harmed the biotechnology workforce.

The Biden Administration took similar positions with regard to our industry. It started the Centers for Medicare and Medicaid Services’ Cell and Gene Therapy Access Model, a welcome effort to modernize Medicaid by facilitating the use of outcomes-based agreements for the FDA-approved gene therapies for sickle cell disease. But skyrocketing inflation — and the rise in interest rates that came with it — depressed investment in development-stage biotechnology companies that create many of the most innovative therapies. And the Inflation Reduction Act, which gave Medicare the ability to drive down prices it pays for a certain number of medicines each year, hit the large biopharmaceutical companies that are often the engines of commercialization for cell and gene therapies through partnerships and acquisitions.

So, with that understanding, when the opportunity arose last week to meet with US Health and Human Services Secretary Robert F. Kennedy, Jr., with a small group of healthcare stakeholders on regenerative medicine, I jumped at the opportunity.

While it wouldn’t be appropriate for me to share specific details about an off-the-record meeting, I can say Secretary Kennedy and his team were highly engaged, asked good questions, and appeared eager to support the field on behalf of the patients we serve. I left the meeting thinking there is important work we can do together to fine-tune the federal regulatory and reimbursement approaches to CGTs to balance safety, efficacy, and speed, and to break down barriers to access.

Beyond that particular meeting, we generally think there is a philosophical alignment between CGTs and aspects of the Make America Healthy Again (MAHA) movement. CGTs address the root cause of disease and can reduce the need for ongoing chronic interventions. Indeed, when the Trump Administration announced the MAHA Commission in February, it cited the work it did during the first term to accelerate advancements in genetic treatments for sickle cell disease.

For CGTs to flourish and reach patients in need, and for the US to outcompete rising powers like China, we need an ecosystem that combines trailblazing science, flexible regulation, modernized healthcare systems, and robust capital markets.

There are three priority areas that we’re discussing with the Administration and Congress:

• A strong and innovative regulatory framework: The Center for Biologics Evaluation and Research (CBER) has modernized its regulatory approach, harnessed expertise, and hired critical staff in the Office of Therapeutic Products to prepare for the coming wave of CGTs. CBER and OTP have demonstrated an outstanding commitment to reform and improvement in line with scientific advancement. The changes have been particularly notable over the past 18 months – and our community wants to see this progress continue through appropriate resourcing and continuity of the strong CBER/OTP leadership.
• Patient access to CGTs: Patients who need CGTs should be able to access them, regardless of whether they are on private or public insurance. The Administration has correctly noted that our current healthcare system is designed for chronic medical interventions rather than preventive treatment. We must better align financial incentives with health outcomes and promote the use of value-based payment models in Medicaid in particular. There is a lot that CMS can do under its existing statutory authority to support payment innovation and alleviate structural and administrative barriers to care, including the lack of uniformity in physician credentialing standards that make it difficult for Medicaid patients to travel across state lines to receive specialized care. Secretary Kennedy has expressed an eagerness to leverage existing rules and regulations to make changes that don’t require action from Congress.
• Investment in biomedical research: Maintaining robust public and private investment in scientific research is a cornerstone of CGT innovation. The NIH has been instrumental in advancing our understanding of genetic diseases and cancers and supports research on ultra-rare conditions for which treatments may lack a viable commercialization pathway. Without ongoing support for scientific discovery, we risk falling behind in the global race for the next generation of treatments.

We strive to find common ground with this, and any other, administration because our patients for the most part do not have other treatment options they can turn to. As the father of a young man with a rare brain condition (Dandy-Walker Syndrome), I am aware of the joys, but also the many juggles and struggles that take place in our community. I’ve met countless CGT patients who have benefitted from our advanced therapies, but also many families who have lost loved ones to cancer and rare genetic diseases.  

When it’s about the future of CGT and our patients, not engaging is never an option.

Tim Hunt is CEO of the Alliance for Regenerative Medicine