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Navigating reimbursement and access challenges for novel cell and gene therapies

By Amanda OHora

Amanda O’Hora
Senior Director, Reimbursement & Policy Insights

Pavel Lavitas, PharmD, BCPS
Clinical Team Lead, Clinical Pharmacy Services
University of Massachusetts Chan Medical School

Cell and gene-based therapies (CGTs) have captured our imagination for their potential to cure disease after a single course of therapy but have also raised eyebrows for their astronomical price tags.

Since August 2017, eight CGTs have been approved by the Food and Drug Administration (FDA), including six oncology chimeric antigen receptor T-cell (CAR-T) therapies and two gene therapies for ultra-rare diseases.1 The approved CGTs are: Abecma, Breyanzi®, Carvykti, Kymriah®, Tecartus®, Yescarta®, Luxturna®, and Zolgensma® (see Table 1).

Therapy Name Drug Class Indications*
(idecabtagene vicleucel)
CAR-T cell therapy Relapsed or refractory multiple myeloma
(lisocabtagene maraleucel)
CAR-T cell therapy Relapsed or refractory large B-cell lymphoma
(ciltacabtagene autoleucel)
CAR-T cell therapy Relapsed or refractory multiple myeloma
CAR-T cell therapy Relapsed or refractory B-cell acute lymphoblastic leukemia
Relapsed or refractory large B-cell lymphoma
Relapsed or refractory follicular lymphoma
(voretigene neparvovec-rzyl)
Gene therapy Biallelic RPE65 mutation-associated retinal dystrophy
(brexucabtagene autoleucel)
CAR-T cell therapy Relapsed or refractory mantle cell lymphoma
Relapsed or refractory B-cell precursor acute lymphoblastic leukemia
(axicabtagene ciloleucel)
CAR-T cell therapy Relapsed or refractory large B-cell lymphoma
Relapsed or refractory follicular lymphoma
(onasemnogene abeparvovec-xioi)
Gene therapy Spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 gene

With over 780 CGTs in clinical development for conditions with unmet medical needs, such as hemophilia or sickle cell disease, the availability of these treatments is expected to accelerate dramatically.2 By 2030, it is estimated that 54 to 74 gene therapies may be launched, with approximately 57 percent for oncology patients; 36 percent for orphan, non-oncology indications, and 7 percent for common diseases.

While these novel treatments may promise to cure or achieve sustained remission, small patient populations, limited long-term data, and high costs (ranging from $373,000 to $2.1 million per patient) have raised concerns about sufficient reimbursement to ensure patient access.

Reimbursement and patient access challenges in CGTs

Existing CGTs have faced reimbursement and access challenges over the past several years. Stakeholders can learn from these challenges and apply this knowledge to the numerous therapies that are coming to market – and presenting their own unique issues. Payers and providers may struggle with identifying the right patients due to diagnosis codes that are not specific enough and a lack of information to determine eligibility for treatment. Payment remains difficult for the entire market because many existing payment methodologies are not set up to accommodate the high-cost therapies. 

A case study conducted by AmerisourceBergen/Xcenda shows some challenges CAR-T therapies have faced since launch relating to Medicare coverage. Over time, Medicare has reassigned these therapies to different Medicare Severity-Diagnosis Related Groups (MS-DRGs), updated the coding, and revalued the relative weights for the MS-DRGs. 

As demonstrated in the case study noted above, a large variance exists in how hospitals are reporting the therapies and their charges, resulting in significant differences in payment. There are actions that life sciences manufacturers can take to help minimize some of these concerns through evidence generation, customer mapping, education, and support programs. 

To access the webinar recording on this topic, please click here.

Clinical Pharmacy Services and Xcenda: Providing valuable insights into promising medications 

The Clinical Pharmacy Services team at Commonwealth Medicine creates Product Snapshots available via Xcenda’s FormularyDecisions®,  a secure online platform that facilitates credible information exchange between life sciences manufacturers and health care decision makers. 

The Product Snapshots offer:
  • Important background information, clinical trial summaries, and formulary management considerations for each product. 
  • A valuable, high-quality tool for FormularyDecisions® users seeking insights into promising medications in late stages of the pharmaceutical pipeline and FDA-approved medications being investigated for new indications. 

FormularyDecisions—which was acquired by Xcenda, a part of AmerisourceBergen, in 2019—provides its community of health care decision makers with access to clinical evidence and health economic information on thousands of pharmaceutical products.


  1. Gene therapy's next installment. Nat Biotechnol. 2019;37(7):697.
  2. Paying for Cures Toolkit. Massachusetts Institute of Technology Center for Biomedical Innovation’s New Drug Development Paradigms (NEWDIGS) Initiative. 2022. Available from:
  3. U.S. Food & Drug Administration. Approved cellular and gene therapy products. Mar 2022. Available from:
  4. Sinclair A, Islam S, Jones S. Gene Therapy: An Overview of Approved and Pipeline Technologies. 2018 Mar 1. In: CADTH Issues in Emerging Health Technologies. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2016-. 171.
  5. Terry M. FDA Greenlights Gilead/Kite’s Tecartus for Mantle Cell Lymphoma. BioSpace. 2020 July 24. Available from:
  6. Xcenda. Analysis of the 2019 and 2020 Medicare Inpatient (Fee-For-Service) 100% claims database. June 2022. Data on file.