A glimpse into the future of CGTs with ARM’s Rita Johnson-Greene

Cencora would like to thank Mrs. Johnson-Greene for joining the summit and providing her insights and thoughts on where the industry is headed. 

Lung-I: I think we can confidently say that 2023 was a banner year for gene therapy. As we look further ahead in terms of genetic medicine and personalized treatments, what are you most excited about? 

Rita: I’m excited about the diversity of approvals. If you think about the eight anticipated approvals in the US in 2024, four are gene therapies, three are cell therapies and one is a tissue therapy¹. This speaks to the maturing of the regenerative medicine field. Another sign that the field is maturing is competition in one disease state. It started with two sickle cell products that were approved in December 2023 – from Vertex Pharmaceuticals and CRISPR Therapeutics (CASGEVY™) and from bluebird bio (LYFGENIA™). In January this year, CASGEVY™ was also approved for beta thalassemia. Pfizer's gene therapy for hemophilia B, BEQVEZ™, was approved in April, becoming the second hemophilia B gene therapy product to be approved in the US (after CSL Behring’s Hemgenix). There’s potential for Pfizer to get approval for a hemophilia A product, which would also make it the second, sometime in 2025. And there's a potential for Abeona Therapeutics to get the second recessive dystrophic epidermolysis bullosa (DEB) product approved next year (the first being Vyjuvek from Krystal Biotech). That competition excites me because competition fuels product differentiation, which promotes innovation. 

Next is globalization. The CRISPR/Vertex sickle cell product has been approved in the EU, the UK, the US, Saudi Arabia, and Bahrain, which is remarkable when you think about the short amount of time that it has been on the market. Ultimately, our responsibility is to treat all patients and that requires us to strive for globalization. 

Lung-I: Building off your point about globalization, one of the challenges we have been discussing is demonstrating the value for these products. How do we as an industry ensure that value is recognized with the right effort so that we can improve access for patients? 

Rita: Science is continually advancing, and we need to continually improve how we communicate that transformational value to patients and society. The first step is education. The pool of stakeholders that have questions about cell and gene therapy includes not only patients, caregivers and regulators, but also payers, religious leaders, bioethicists, academia, the media and investors. So, we need to spearhead the conversation and anticipate the questions they’re likely to have. As an example, ARM recently held a meeting with religious leaders at the Vatican to discuss gene editing.  

We also need to make a clear and factual case for value. We use our sector analysis and data to support the value narrative. For example, the average life expectancy of patients with rare diseases that are targeted by gene therapies currently approved in the US is less than 40 years old. That's half the normal average life expectancy, and it speaks to the devastating nature of these diseases. These are also expensive to treat. The lifetime cost of hemophilia B is $21 million², and severe sickle cell is $4 million to $6 million³.  

We do believe the US healthcare system can absorb these costs. If we consider gene therapies are expected to cost us about $7.5 billion in 2030, that represents 1/10th of a percent of the projected healthcare spend in the US in 2030⁴.  

Finally, as a sector we need to continually increase engagement with patient groups so that we can understand the burden of the diseases and the benefits of cell and gene therapies. We aim to do that by bringing patient groups to our conferences to speak and to bolster the patient voice. 

Lung-I: That’s the best data argument I have heard for cell and gene therapy. You touched on payers. As we know, in Europe, the Joint Clinical Assessment (JCA) is due to come into effect in January 2025, starting with oncology products and CGTs, or advanced therapy medicinal products (ATMPs). Does ARM have a view on the JCA as it is right now and how should biopharma companies think about preparing for this change? 

Rita: The JCA will impact pricing and reimbursement policy at national levels by assessing the relative clinical effectiveness of therapeutic products. The assessment will inform how member states will reimburse, but, if applied without exception, the draft JCA guidelines will not recognize the full therapeutic value and benefit of cell and gene therapies. The draft guidelines state that nonrandomized control trials, including single arm trials, may be insufficient to prove relative effectiveness, which may require member states to perform their own clinical assessment, and that creates duplication. We believe there are three options. One is to modify the guidelines so as not to discredit single arm trials in certain cases, such as cell and gene therapies for rare disease. Another would be for the JCA Coordination Group and key member state Health Technology Assessments (HTAs) to accept real-world evidence to address gaps in data. A third would be for EU member states to be flexible with resolving uncertainties. 

Lung-I: If we think about the broader cell and gene community, how can we collaborate to advance patient empowerment? 

Rita: Multi-stakeholder input ensures that ARM's policy recommendations are workable and supported by all parties. One example of our efforts to support a broader advocacy coalition is our Congressional Fly-in, which is scheduled for July⁵. This is an opportunity to amplify our presence on Capitol Hill by bringing members from across the country and industry together to champion pivotal matters on regenerative medicine. These stakeholders include developers, service providers, academic institutions, and patient groups. 

Lung-I: Can you share some of the other initiatives that ARM is supporting or working on from a policy perspective? 

Rita: Given that some rare diseases, such as sickle cell, disproportionately impact communities of color, ensuring access is essential to advancing equality. So, we advocate for policies that ensure that all patients, regardless of their healthcare insurance or their geography, have timely access to treatment. We support enabling value-based payment arrangements and support cross-border provider arrangements for those patients who have to cross state lines to access treatment. We also support legislation that allows manufacturers to provide fertility preservation support for gene therapy patients without violating the Anti-kickback Statute⁶. ARM recently held a congressional briefing with the Institute for Gene Therapies on Capitol Hill focused on providing patients with access to CGTs. The briefing included speakers from the patient advocacy community. Multi-stakeholder input is critical to implementing change.  

Lung-I: In my final question to you, as you look ahead, what strategies or innovations do you believe will be instrumental in expanding patient access to cell and gene therapies? 

Rita: As I said earlier, I believe competition in the field will drive innovation that will result in lower drug pricing and also expanded patient access. The other is globalization. If you think about Zolgensma (for spinal muscular atrophy), it is now approved in more than 50 countries⁷. So, some larger companies are making strides across the globe. A third priority will be industrialization to expand access. Some drivers for industrialization are artificial intelligence, digitization, workforce development, as well as improved automation. Lastly, the ability to manufacture at scale. We’re seeing flexible manufacturing platforms that allow for seamless translation from Research and Development (R&D) to Good Manufacturing Practice (GMP), and this will enable widespread patient access and deliver products that are affordable. Overall, innovative technologies will help scientists to optimize their processes and deliver these lifesaving and life-changing medicines, at scale. 

Cencora encourages readers to review the references provided herein and all available information related to the topics mentioned herein and to rely on their own experience and expertise in making decisions related thereto as the article may contain marketing statements and does not constitute legal advice.

Resources

¹ Alliance for Regenerative Medicine. Sector Snapshot. April 2024. https://alliancerm.org/wp-content/uploads/2024/05/Sector-Snapshot-4.30.2024.pdf 

² Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model, Journal of Medical Economics, March 2021. https://www.tandfonline.com/doi/full/10.1080/13696998.2021.1891088 

³ Gene Therapy Approvals Expected to Ramp Up in 2024 Amid Manufacturing, Cost Challenges, Jan 2024. https://www.biospace.com/article/gene-therapy-approvals-to-ramp-up-in-2024-amid-manufacturing-cost-challenges/ 

⁴ Centers for Medicare & Medicaid Services. CMS Office of the Actuary Releases 2021-2030 Projections of National Health Expenditures. https://www.cms.gov/newsroom/press-releases/cms-office-actuary-releases-2021-2030-projections-national-health-expenditures 

⁵ Cell & Gene Congressional Fly-In, ARM. https://alliancerm.org/arm-event/dcforum/#:~:text=The%20ARM%20Congressional%20Fly%2DIn,to%20cell%20and%20gene%20therapies. 

⁶ Fraud & Abuse Laws, Office of Inspector General, HHS. https://oig.hhs.gov/compliance/physician-education/fraud-abuse-laws 

⁷ Novartis presents new data on safety and efficacy of Zolgensma, including maintained and improved motor milestones in older and heavier children with SMA, Novartis, March 2024. https://www.novartis.com/news/media-releases/novartis-presents-new-data-safety-and-efficacy-zolgensma-including-maintained-and-improved-motor-milestones-older-and-heavier-children-sma