In one of the first sessions of AMCP Nexus 2021, Jane F Barlow, MD, MPH, MBA senior advisor, MIT Center for Biomedical Innovation, chief clinical officer, Real Endpoints, and Michelle Harika, PharmD, senior advisor, MIT Center for Biomedical Innovation, chief clinical officer, Equity Healthcare, discussed the latest financial and market challenges associated with emerging gene therapies, as well as offered potential next steps stakeholders can take to better navigate this rapidly evolving space.

According to their presentation, the first genetically engineered cell therapy was approved in the United States in 2017 and the market has exploded since; however, the growing landscape creates unique challenges for payer financing and reimbursement.

Tisagenlecleucel, axicabtagene ciloleucel,voretigene neparvovec-rzyl, and lisocabtagene maraleucel are all examples of approvals since 2017 but “what’s more important than what is already approved is what the pipeline looks like,” stressed Dr Barlow.

Dr Barlow went to share that between 54 and 74 therapies are projected to be approved for the US market by 2030, prompting the need to address financial challenges like payment timing risks, actuarial risk, productive performance risks, and mobility.

Some precision financial solutions that Dr Barlow and the MIT team proposed included:

• Orphan Reinsurer and Benefit Manager (ORBM) and Risk Pools
• Short-term milestone-based contracts
• Multi-year performance-based annuities
• Warranty Model
• A Netflix-like subscription model

Dr Barlow noted, “Not all payers are positioned to manage gene therapies on their own,” referring to the smaller commercial payers, self-insured employers, among others who will need to evolve to meet the emerging need of the market.

Dr Harika explained the project conducted by MIT included conducting a 45-question RFI survey via Qualtrics to gather information and identify other potential dissemination pathways in the following major categories:

• Responding organization information
• Product history and description (therapies included, targeted customers, and eligibility requirements)
• Financing challenges addressed by product
• Impacts to patients and ability to address patient mobility
• Data tracking: financial and clinical
• Quality Assurance
• Physician or provider requirements

Survey respondents comprised a total of 12 solicited organizations from personal networks and MIT FoCUS work groups.

Per the results of the MIT survey, which had a 100% response rate, Drs Barlow and Harika and coresearchers, identified 16 new products or services. Those products were categorized into one of four categories: (1) reinsurance/stop loss/risk carve out; (2) contract negotiation and data management; (3) provider contract negotiations/including COE; and (4) financial and warranty services.

Similar themes among responders included launch time frames, overcoming financial barriers, and that coverage will expand with new approvals.

Dr Harika pointed out the differences among survey responders which included varied products and services, different customer targets, coverage of therapies, and quality of care.

When Dr Harika surveyed the attendees in the room with the following question, “Outside of product cost, what challenge do you think is most important for these products to address?” more than 60% of the room responded that value-based reimbursement and paying for what works is the most important.

From the MIT survey, reinsurance/stop loss/risk carve out products— which carve out of financial responsibility or risk for specified cell and/or gene therapies, or other specified high-cost therapies—do not offer performance guarantees but assume all actuarial/financial risk, which makes them suitable for commercial/government health plans and PBMs, though they may not benefit financially. These products may not be suitable for providers due to the potential for increased utilization because of lower cost burden to the payer though. These products have the potential to increase access to therapy for patients but may prohibit moving employers or changing employment status to avoid changes in benefit.

Contract negotiation and data management products can reduce administrative, negotiation, and data-tracking burden from the payer as they act as a third party to negotiate gene-therapy contracts. These products also present an opportunity to care management and advocacy groups to partner with provider or manufacturer to supply clinical data.

Overall, results of the survey revealed that products in each of the main four categories take different approaches to address different stakeholder needs, and there is not one single solution to effectively managing gene therapies, as well as all stakeholders’ concerns and needs. However, the market is responding appropriately to the challenges and making innovative solutions.

To read the full white paper published from the results of the MIT study, visit payingforcures.mit.edu/.