The Evolution and Future of Cell & Gene Therapy | Deloitte US has been saved
By Jennifer Rabin specialist master, Deloitte Consulting LLP
Cell and gene therapies (CGT) hold the promise of treating or even curing some cancers and rare diseases.1 These emerging therapies are also among the world’s most expensive treatments—often between $400,000 and $2 million per dose, and sometimes much more.2
A potential wave of new gene therapies is building on the horizon. The CGT market is expected to nearly quadruple over the next five years—from $5.3 billion in 2022 to $19.9 billion by 2027.3 In 2022, three CGTs were approved to treat rare diseases, another was approved for the treatment of bladder cancer.4 As we noted in our blog last October, 19 gene therapies and 10 chimeric antigen receptor T cell therapies (CAR-T) could be approved this year (see Multi-million-dollar therapies may alter payment models). However, neither commercial nor government payment models are set up in a way that ensures all patients have access to these potentially life-changing therapies.
A few months ago, Deloitte’s NextGen hosted an Industry Working Group meeting with some of our biopharmaceutical company clients. One panel focused on how CGT manufacturers can work more closely with health systems that administer the therapies. I recently had an opportunity to follow up with two of the panelists: David Porter, M.D., director of Cell Therapy and Transplant at University of Pennsylvania School of Medicine and Abramson Cancer Center, and Peter Olagunju, a CGT executive, who helped drive five CGT products through clinical development to commercialization. Here is an excerpt from our conversation:
Jennifer: Penn has a large program in both bone-marrow transplant and cell therapy, and it is site-approved for CAR-T cell products.5 Some of the earliest CAR-T clinical trials were conducted at Penn more than a dozen years ago. How rapidly has the CGT field progressed since then?
David: CGT is advancing at an incredible pace. It has only been six years since the Food and Drug Administration (FDA) approved the first CAR-T therapy. Now there are six FDA-approved CAR-T products. In the short term, I expect CAR-T products will become more effective and more potent. Most importantly, we are learning more about how to administer them safely. CAR-T therapies are potentially dangerous and could have life-threatening toxicities. The most unique toxicities are around cytokine release syndrome (CRS) and neurologic toxicity. Over the last several years, it has become easier to manage these toxicities. CGT is much safer than it was five to 10 years ago. And as more knowledge is gained about CGT, the therapies might be administered in more locations, which could make treatment more accessible.
Peter: A few companies have been successful in commercializing these therapies domestically. They have run into some challenges around accessing enough key materials and scaling up. And it can be a challenging environment from a capital perspective. Commercializing some of these therapies has taken longer than some investors expected. There are about 600 CGT companies globally. Many of these companies raised capital during the pandemic.
Jennifer: As the number and type of CGTs grow, clinical treatment centers face the burden of duplicative documentation around initial qualification, ongoing auditing, and reporting, with overlapping requirements from each manufacturer to ensure safe use of their specific product. The American Society for Transplantation and Cellular Therapy (ASTCT) convened the 80/20 Task Force to consider challenges and potential solutions to these issues.6 Could this help to make CGTs more accessible and equitable?
David: There is an enormous amount of redundancy on the on the part of manufacturers and sponsors when dealing with treatment sites. We estimate that about 80% of what they all do is redundant. If each manufacturer could focus on the other 20% that's unique to a manufacturer or product, it would go a long way toward making CGT implementation more efficient and could free up the treatment centers. It could also make treatment more cost effective.
Peter: Five or six years ago, the thought of these CGT being made available in the community would not have been possible. Today, as many as 20% of CGT patients are being treated outside of academic medical facilities. That speaks to the evolution of these therapies. Some large health systems, including Penn, have taken the initiative to invest in infrastructure and capabilities within their network of hospitals to expand access to CGT.
David: Penn has an affiliated health system in a suburban/rural area about 90 miles from Philadelphia. Many patients are unwilling or unable to drive to the big city for care. Yet they are close enough that if they need a certain level of care they can get here. It has expertise and highly trained clinicians in cancer and other diseases. It is an appropriate place to model this program. I believe strongly that cell therapy should be available in communities outside of academic centers. The academic centers will have less and less capacity as more products are approved. Academic medical facilities are best positioned to develop research-based treatments, but then can disseminate them to communities that don't have access to the major academic centers.
Jennifer: What are the challenges in making CGTs available outside of academic medical centers?
David: We are not suggesting that these therapies can go to just any community practice anywhere. It requires meticulous oversight and care, and the same level of management that would exist at a major academic center. A lot goes into choosing those community sites and they have to be accredited just like the major university hospitals. They undergo the same quality control and the same oversight. And staff have to be trained very specifically to be able to [administer these therapies] in the safest possible way. A typical community practice is not going to be able to do this. But there are community settings where this absolutely can be done.
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Endnotes:
1 How gene therapy can cure or treat diseases, FDA, July 28, 2022
2 $2M gene therapies could become the new norm in the US, November 3, 2022
3 The global bioinformatics market size is expected to reach $19.9 billion by 2028, GlobeNewswire, January 27, 2023
4 FDA approves first gene therapy for the treatment of high-risk, non-muscle-invasive bladder cancer, December 16, 2022
5 Bone marrow transplant, Penn Medicine
6 ASTCT Learning Center: 80/20 update and next steps, November 17, 2022
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