Pharma Firms Will ‘Walk Away’ From Gene Therapies Unless Payers Share Risk

Risk is inherent from both a financial and a clinical perspective when developing therapies for rare diseases – particularly if these are gene therapies. One way to bring these products to market is by sharing the risk across stakeholders, according to Bart Leroy, an ophthalmologist and clinical geneticist at the Ghent University Hospital in Belgium.

“I think that societies in modern countries like the EU and the US ought to also bear or take responsibility for the financial risk and the health risks that are implicated in getting a drug to market with limited data,” he said during a 26 February webinar.

Leroy has experience in the field of advanced therapy medicinal products , having worked on the team that brought Spark Therapeutics/Novartis’ vision loss gene therapy Luxturna (voretigene neparvovec) to the market.

“There are certain risks that can only be mitigated by saying ‘okay, let’s take the risk together,’” Leroy explained, warning that if national reimbursement authorities were not willing to do this, then drug companies “are simply going to walk away, because these developments cost a lot of money, and they need to at least get some return on investment.”

“I personally have seen some of the drug companies walk out of gene therapies, and I think that is something that we just cannot afford,” he stated.

During the webinar, which was organized by The Economist, experts discussed whether health technology assessment (HTA) bodies in the EU and UK were fit to judge the value of cell and gene therapies.

Lessons Learned From Luxturna

Leroy spoke about his experience with Luxturna, which was approved in the EU in 2018 for retinal dystrophy and has received positive HTA decisions in England, Scotland, France, Germany and Italy among others.

“I learned many [lessons],” but the most important ones were to interact early with regulators and HTA representatives, and to get all stakeholders on board,” he said.

Leroy contended that the methods used for both HTA and “earlier on in the [product evaluation] process, at the level of the Food and Drug Administration [and] the European Medicines Agency,” are “not fit for purpose for rare conditions, for many reasons.”

He also argued that it was “not logical” for Luxturna to be available in some EU countries but not others, based on national HTA and reimbursement decisions.

“I think across the EU, we need to do a lot better and streamline the procedures a lot better,” Leroy said.

Luxturna was approved in the US in 2017, where it is marketed by Spark Therapeutics.

Creative Payment Models

While companies need to “have some protection” in terms of recouping the investment made in developing a therapy, HTA agencies “typically take a more static viewpoint” when it comes to reimbursement, said Lotte Steuten, deputy CEO of the UK’s Office of Health Economics, during the same discussion.

She explained that HTA organizations do not necessarily consider the cost-effectiveness of a product over an entire lifecycle, but acknowledged there are “good reasons” why they take this approach.

Steuten said that for companies, “creative payment models” can offer an incentive to develop innovative products, but these should be “coupled with evidence generation so that we indeed really know what the actual value is.”

“Another thing I see happening now, which is not necessarily a long-term sustainable solution, is asking [for] quite hefty price discounts from manufacturers because of all the uncertainty,” she continued.

This is because payers want to “offset some of that uncertainty by getting a lower price,” something that Steuten warned comes with the risk that future treatments for a given disease will be “compared against this very heavily discounted price.”

“It does not necessarily reflect the actual value,” she explained.

Steuten said that a solution could be the development of more flexible payment models and being “comprehensive and honest about the full societal value of these products.”

She added that it was positive to see some “different rules and approaches” emerging from HTA agencies such as England’s NICE.

However, Steuten pointed out that with more gene therapies coming down the pipeline, which is in itself a “good thing,” this means that they cannot continue to be treated “as an exception” to the pricing rules that other products are subjected to.

“Bringing this back to the population level is really important to make fair decisions and also decisions that really maximize the benefits that we get from our health care money.”

CSL Behring recently shared information with the Pink Sheet about how it successfully navigated the HTA processes of several European countries for its one-time hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec).

The company explained that one of its market access deals included a performance-based reimbursement arrangement in Denmark, whereby costs are only incurred to the payer if the gene therapy proves effective in the long-term.

CSL also stressed the need for creativity and flexibility when it comes to market access and reimbursement of gene therapy products.