Curative Gene Therapy Coverage May Need Government-Funded Risk Pools
Executive Summary
Former US FDA commissioner Scott Gottlieb makes a case for regional or national risk pools to ensure pediatric patients get timely access to gene therapy like the upcoming treatments for sickle cell disease.
Implementation of government-supported risk pools across US payers on a regional or state basis should be considered as a solution to paying for specific types of gene therapy, former FDA Commissioner Scott Gottlieb suggested at a meeting sponsored by the Duke Margolis Center for Health Policy on 11 September.
Many policy discussions about paying for curative therapies have focused on annuity models and value-based contracts, he noted. But “do we need to perhaps consider mechanisms where you just pool the risk, either regionally or even nationally?” That “hasn’t entered the discussion yet.”
Gottlieb, who is now a resident fellow at the American Enterprise Institute, spoke at a conference on payment reform to advance patient access to transformative treatments.
He argued the need for risk pooling will become more pressing if gene therapies for sickle cell disease enter the market within the next couple of years, as expected.
“I think our attention to this is going to be galvanized if we do truly develop sort of a cure, if you will, for sickle cell disease that is sufficiently non-toxic that it could be widely distributed to patients at an early age, because that’s not something that can be funded by individual plans,” he said.
“It’s going to require appropriations from Congress if we truly want to deploy something like that on a wide basis,” Gottlieb maintained.
Passing legislation to fund such a program would be challenging in the current environment on Capitol Hill. Nevertheless, the likely difficulties in paying for coverage among smaller payers or Medicaid agencies will prompt a public outcry and demands for new approaches to payment, he predicted. (Also see "Paying For Gene Therapy: Medicaid Will Be ‘Canary In Coal Mine’ " - Pink Sheet, 5 May, 2019.)
“For all of the discussions we’ve been having around these different payer models with private plans, we haven’t solved the Medicaid problem and I think we need to solve the Medicaid problem.”
“We’re going to need to think about different constructs because as it is, if you look at a lot of these risks with these pediatric diseases, they’re not evenly distributed across insurance pools” or Medicaid programs, nor are they evenly distributed geographically, he said.
That means with treatments for diseases like hemophilia or sickle cell anemia, “you will see some payers and certain payer types disproportionately impacted by the costs and the burden of trying to deliver these opportunities to patients,” Gottlieb noted. Cigna Corp.'s Express Scripts Holding Co. unit recently announced a novel program for covering gene therapy that is aimed primarily at smaller plans. (Also see "Cigna/Express Scripts Gene Therapy Solution Involves Plans ‘Pre-Paying’ For Coverage " - Scrip, 10 Sep, 2019.)
Access To Cures Based On ‘Socioeconomic Status’
The disparity in access between Medicaid and private plans could be particularly problematic, Gottlieb pointed out. For example, “when you’re dealing with diseases where six months to a year can make a big difference...in terms of reducing disability, if a Medicaid patient takes 12 months to get access to that and a privately insured patient gets it in three months, that’s significant,” he pointed out.
“You have differential access based on payer type and you have literally the specter of people’s destiny altered by their socioeconomic status. It’s not a sustainable place to be and it’s not going to be tolerable.”
So “for all of the discussions we’ve been having around these different payer models with private plans, we haven’t solved the Medicaid problem and I think we need to solve the Medicaid problem” with gene therapy, Gottlieb emphasized.
Patrick Conway, president and CEO of Blue Cross of North Carolina, echoed Gottlieb’s concerns. “Risk pooling is critical…I completely agree with Scott. We’re going to have to think more creatively on the payment mechanisms,” he said.
Conway was previously deputy administrator of the Centers for Medicare and Medicaid Services and chief medical officer and director of the Center for Medicare and Medicaid Innovation under President Obama.
“I couldn’t agree more that you’re going to see Medicaid-to-private payer differentials, which you're already seeing today and which I think are going to get worse," he said. And “you’re going to see Medicaid program differentials” across states.
“I think people underestimate what Scott just said," that differences in payers’ ability to absorb risk "actually can have major effects,” Conway warned.