Survey: Payers are Creating New Reimbursement Solutions for Gene Therapies


Most of the solutions have established systems to track patient outcomes over time, and several include performance guarantees as part of their solution.

Payers recognize that cell and gene therapies provide great innovation, and the market is starting to adapt, creating new solutions and reimbursement methods, according to a recent survey of managed care organizations and PBMs. The findings were presented at the Academy of Managed Care Pharmacy (AMCP) Nexus 2021 meeting in Denver, Oct. 18-21, 2021.

The survey was conducting by FoCUS Project, which is part of MIT Center for Biomedical Innovation. FoCUS was launched in 2016 to develop new models and solutions for the reimbursement of cell and gene therapies.

Jane F. Barlow, M.D.

Jane F. Barlow, M.D.

“The purpose FoCUS is to ensure that there’s sustainable access for patients to these new one-time, durable therapies,” Jane F. Barlow, M.D., senior advisor, FoCUS Project, MIT Center for Biomedical Innovation, said in an interview with FormularyWatch. She is also executive VP and chief clinical officer at Real Endpoints.

FoCUS conducted a request for information survey with 12 health plans and PBMs that have been known to cover cell and gene therapies. The purpose was to understand how the market is adopting new solutions that are being offered to different stakeholders to help support these emerging products.

The survey found that payers used four broad categories for covering cell and gene therapies. These include payer reinsurance/stop-loss/risk carve out solutions; contract negotiation and data management services for payers and pharmaceutical companies; provider contract negotiation services; or financial and pharmaceutical company warranty services.

“We're seeing the market respond to the challenges of selling gene therapy, with new products and solutions. But there's really no one solution that’s going to address all of the stakeholder concerns. As in most things, there's no magic bullet,” Barlow said.

Most market solutions have established methodologies or systems to track patient outcomes over time. Five organizations included performance guarantees as part of their solution. None of the solutions required additional payment from the patient over and above their usual plan benefit cost share. Several did require data sharing or clinical follow up to assess outcomes.

Payers employ creative solutions

BCS Financial, CVS Health, and OptumRx are using stop-loss solutions for self-insured employers in exchange for a fixed per member, per month fee. BCS’s solution is for very large employer who generally self-insure without stop-loss coverage. CVS Health solution is for Caremark or Aetna self-insured companies without stop-loss coverage and there are separate eligibility requirements. OptumRx provides cost and quality management services including outcomes-based contracts.

MedImpact Healthcare Systems and OutcomeRx offer specialty carve-out reinsurance with a per member, per month fee to take on the risk of high-cost therapy claims. Evernorth’s Embarc Benefit Protection Plan brings together health services, medical management, and specialty pharmacy expertise in a carve-out designed to shield health plans and customers from high costs. PayRx offers a plan that expands traditional pharmaceutical payment assistance programs via a proprietary analytics model.

Two payers offer contract management and data management services for payers and pharmaceutical companies. Audaire’s Gene and Cell Therapy Outcomes Management Service offers a range of services, including negotiation of performance-based contracts on a plan’s behalf; automatic data capture of patients and providers; data aggregation; contract administration on behalf of the plan; patient tracking, and patient portability.

Real Endpoints’ Marketplace is a forum payers and pharmaceutical companies to efficiently develop and implement performance-based contracts. Real Endpoints acts as a third party to assess and manage performance of these contracts.

Two companies offer contract negotiation services: Blue Cross Blue Shield Association and Emerging Therapy Solutions.

Three companies offer financial and warranty services. August Care Health offers an Outcomes-based Financial Solutions platform that provides the opportunity to move from high upfront costs to paying for achieved performance. CVS Health offers an installment payment plan for gene therapies that allows health plans to pay over several years, smoothing the impact of an immediate large, incurred cost.

OutcomeRx’s Specialty Therapy Warranty is an outcomes-based contract mechanism that serves as a risk sharing mechanism between payers and pharmaceutical companies by guaranteeing the efficacy/durability of cell and gene therapies.

Barlow said plans may have different risks depending on their size and who their beneficiaries are, and going forward, it will be important for payers to assess the risks and challenges that are most important and develop a strategy for those risks.

“There really are not a lot of cell and gene therapies on the market right now. But when you look at the pipeline, over time, there are expected to be quite a few products on the market. By 2030, we're projecting somewhere between 54 and 74 cell and gene therapy approvals, so there will be like more of a cumulative effect,” Barlow said.

Gene therapies on the market

So far there have been eight such approvals, although seven were available when the FoCUS survey was conducted.

Last week, the FDA has approved Enzyvant Therapeutics’ Rethymic (allogeneic processed thymus tissue-agdc), a one-time therapy for immune reconstitution in pediatric patients with congenital athymia. Rethymic is engineered human thymus tissue designed to regenerate the thymic function.

Read more about Rethymic

Available cell and gene therapies include:

  • Abecma (idecabtagene vicleucel) was approved March 26, 2021, for the treatment of adult patients with relapsed or refractory multiple myeloma. This is a B-cell maturation antigen-directed genetically modified autologous chimeric antigen receptor T-cell therapy. This is the first cell-based gene therapy for multiple myeloma. It was developed by Bristol Myers Squibb and bluebird bio.
  • Breyanzi (lisocabtagene maraleucel) was approved Feb. 5, 2021, for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy. It consists of autologous T cells that are genetically modified to produce a CAR protein, allowing the T cells to identify and eliminate CD19-expressing normal and malignant cells. It was developed by Bristol Myers Squibb.
  • Kymriah (tisagenlecleucel) was approved in August 2017 to treat pediatric and young adult patients with a form of acute lymphoblastic leukemia and approved in May 2018 to treat adults with relapsed, large B-cell lymphoma. It is an autologous CAR T cell therapy directed at CD19. It was developed by Novartis.
  • Luxturna (voretigene neparvovec-rzyl) was approved in December 2017 to treat children and adults with an inherited form of vision loss that leads to blindness. Luxturna delivers a normal copy of the RPE65 gene directly to the retinal cells. It was developed by Spark Therapeutics.
  • Tecartus (brexucabtagene autoleucel) was approved in July 2020 to treat adult patients diagnosed with mantle cell lymphoma, and it was approved Oct. 1, 2021 to treat adult patients with B-cell precursor acute lymphoblastic leukemia. It was developed by Kite, a Gilead company.
  • Yescarta (axicabtagene ciloleucel) was approved in March 2018 to treat large B-cell lymphoma, including non-Hodgkin lymphoma, and it was approved March 8, 2021, to treat adult patients with relapsed refractory follicular lymphoma. it is an autologous, CD-19 directed CAR T. It was developed by Kite, a Gilead company.
  • Zolgensma (onasemnogene abeparvoved-xioi) was approved in May 2019 to treat children with spinal muscular atrophy. It is an adeno-associated virus vector gene therapy. It was developed by Novartis.

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