Mapping the Unknown: Measuring Cell and Gene Therapy Adoption Using Real-World Evidence

Published August 9, 2022

It’s been 10 years since the first pediatric patient was treated with chimeric antigen receptor T-cell (CAR-T) therapy for her acute lymphoblastic leukemia. At the time, the approach to engineer immune cells was a novel, ground-breaking concept. In the subsequent decade, research into this space had continued to advance. Equally importantly, the patient has remained cancer-free, serving as one of the most visible examples of the impact this type of therapeutic may have for patients with complex or difficult to treat diseases. 

Cell and gene therapies (CGTs) are cutting-edge therapeutics with the potential to cure and treat a wide range of conditions, some of which have no other treatment options. Although the impacts can be profound, healthcare systems have been slower to adopt this potentially lifesaving treatment protocol. They are fundamentally different from medications and surgical procedures: they come with logistical complexities of sourcing, developing and culturing cells, and they must be administered in-person by a medical professional (rather than being provided in a formulation that allows patients to self-administer at home). 

The recent emergence of new, FDA-approved therapies in this space, small eligible patient populations, and challenges in data capturing make it difficult for Life Sciences teams to get a clear picture of current CGT utilization, which is needed to accurately assess the market, evaluate CGT uptake, and measure the impact of these therapies.

That’s where real-world evidence (RWE) can play a role.

Trinity Life Sciences conducted a first-of-its-kind study of CGTs using patient-level claims data in the Komodo Healthcare Map^TM  to build a baseline understanding of how, to whom and where the healthcare system is adopting and delivering these new therapies. Presented at ISPOR 2022, the study used longitudinal medical and prescription data encompassing adjudicated claims of >150 million unique enrollees from the U.S. commercial, Medicare, and Medicaid insured population. To identify this cohort of patients, we started with the current list of all approved CGTs, then removed those that weren’t approved or wouldn’t have any data at the time of the analysis (e.g., recently approved therapies). The list was further refined to remove products without adequate claims data coverage. Data on 7 of the 22 FDA-approved CGTs from January 1, 2015 to December 14, 2021 was included: PROVENGE^®, MACI^®, IMLYGIC^®, KYMRIAH^®, YESCARTA^®, ZOLGENSMA^®, and LUXTURNA^®. 

Overall, the analysis found that both available therapies and CGT utilization has increased over time. Although we saw slight declines in 2020-2021, this effect may have been due to pandemic impacts on medical care, incomplete data toward the end of 2021, patient warehousing in select cases, and potentially competitive pressures.

We also identified key treaters for these therapies. Consider Zolgensma, a one-time injection CGT used to treat spinal muscular atrophy. It is administered overwhelmingly by pediatric neurologist specialists. This subset of pediatric neurologists – those on the front lines of the healthcare system’s acquisition, implementation, and administration of these complex therapeutics — are educated and empowered users of CGTs. 

This work only scratches the surface of what RWE can unlock for Life Sciences. Equipped with this level of patient-centric insights, Commercial teams can use these data to support market sizing and assess therapeutic adoption of CGTs to understand how a complex therapy is being deployed and adopted in the market. Market Access teams can apply these insights to other complex drug launches, and to future plans to ensure equitable and complete coverage for patients who need it most. Health Economic and Outcomes Research teams can utilize this information to understand  holistic evidence generation strategies, in modeling and other research, and how those can complement pricing, contracting and reimbursement strategies. 

The analysis demonstrates the market’s demand for CGTs — and shows that the healthcare system is responding and adapting. Looking at the evidence, it’s clear that manufacturers are successfully delivering these therapies, payers are successfully covering, and providers are administering them to give more patients access. Understanding the evolving landscape is essential for manufacturers who want their future launches to be successful and optimized. And those types of insights are exactly the basis for the Komodo Health and Trinity Life Sciences partnership. Our joint work is focused on unlocking insights to drive smarter business strategies for pharma, biotech, and med-tech companies and improving outcomes for patients.  

Read more about how Trinity EvidenceFirst is applying RWE to unlock novel insights for life sciences teams.

CO-AUTHORS: Kevin Francis, Partner, Real World Evidence & Abby Silber, Associate Principal, Evidence Strategy (Trinity Life Sciences); Vicki Guan, Senior Customer Success Associate (Komodo Health)