Addressing cell and gene treatment affordability

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A panel discussion at the Reuters Events' Cell & Gene Therapy Conference USA 2020 looked at the key challenges in the cell and gene therapy (CGT) space, namely affordability and access, and how to circumvent those.

Don Creighton, managing director, Huron Consulting Group, summarized the issues at the outset.

Looking at the CGT area, he said it has been a disruptive space in terms of typical pharma business and healthcare delivery models, with challenges and opportunities that are unique.

The industry has been very active, taking a hands-on approach in relation to healthcare delivery around CGTs, he said.

CGTs have a very significant price point and, particularly when you are talking about a one-time treatment, there has to be a level of certainty about the value you are bringing to a patient, to a plan, to a health system, to a society, and we are not quite there yet in terms of gene therapies on an individual basis, in terms of their efficacy.

“We are seeing a lot of interest in payment models than help address that uncertainty, particularly on the clinical side, and also helping, in some regards, with the payments that can be upfront, that can be very difficult for payers’ health systems, helping to try and absorb some of those costs,” said Creighton.

As the CGT area does not fit in the traditional space as it relates to affordability, delivery and provider economics or even capturing data, there are elements that need to be considered as it relates to systemic solutions, he said.

Along with financial risk sharing mechanisms, if the industry incorporates health systems that enable outcome-based contracts, treatment delivery standardization as well as long-term patient follow-up and mechanisms to track those patients, then companies may have successful ventures in this space.

All stakeholders from government to providers to payers to patients share the common goal of finding curative treatments through CGTs, but the affordability issue remains prominent, said the consultant.

One of the ways to address that creatively and proactively is at the product or portfolio level, said Creighton.

He outlined different contracting options such as a portfolio outcome guarantee whereby the payment would be made if the patient met the desired health outcome, using any combination or products offered in a portfolio. Other options could include a subscription model that involves a fixed payment for each patient to access the portfolio, with no health outcomes guaranteed.

Another idea is a warranty model whereby payment is made if the patient meets the desired health outcome using a particular product. A population warranty model was also proposed – this involves payment being made if the eligible pool of payments meets a desired health outcome, calculated by taking an average of health outcomes across the population. And a lease model would see a payer make a fixed payment each month for all patients to access the therapy.

“But one payer may prefer one type of solution, while another payer would prefer a different one.”

In a poll to the audience, as part of the panel discussion, asking them to choose between reimbursement policy, portability due to the movement of beneficiaries from one plan to another, performance metrics, or payer-manufacturer trust as the key affordability challenge in relation to CGTs, some 43% of attendees identified reimbursement policy as the most significant hurdle.