How EMA’s Adaptive Pathways Fits Into The Complex Drug Pricing Puzzle

The European Medicines Agency’s adaptive pathways concept may have a role to play in reducing the prices of medicines. But early market access initiatives such as these are just one piece in the complex drug reimbursement puzzle.

There are suggestions that EU regulatory initiatives aimed at bringing innovative medicines to the market faster, especially where the medicines address areas of high unmet medical needs and rare diseases, may also play a role in lowering the prices of orphan drugs. There is, however, no evidence that this has happened as yet. Health technology assessment bodies and payors point out that such initiatives are only one part of the complex drug reimbursement puzzle, and that it might be difficult to assess the direct impact of such early access initiatives on drug pricing.

The European Medicines Agency’s adaptive pathways project – which can result in eligible drugs being authorized earlier on in the development process for a limited population – is an example of an initiative that some say could help bring down drug orphan drug prices. “It should. I am not saying it does, but in our view we think that it should,” said Yann Le Cam, chief executive officer of EURORDIS, the patient-driven alliance of rare disease organizations in Europe.

Le Cam’s reasoning is based on the fact that under such accelerated review initiatives, companies can get their products to market earlier, potentially with conditional approval. “This is usually at the end of Phase II [trials]… because after that you want to generate real world evidence and have more knowledge about the efficacy and effectiveness of the product,” Le Cam told reporters during a virtual press conference on Feb. 22 that was organized on the sidelines of EURORDIS’s multi-stakeholder symposium on Improving Patients' Access to Rare Disease Therapies.

This, in turn, means that the investment that is needed to support the approval process is lower for such products compared to drugs authorized under conventional conditions. That should help lower development costs and therefore the market price, says Le Cam, but a practical demonstration of this concept actually leading to cheaper medicines “as far as I know has not been done,” he added.

Payors point to the complexity of the issue. Ri de Ridder, the director general of Belgium’s National Institute for Health and Disability Insurance (RIZIV-INAMI), does not believe that “just one change in the decision chain will automatically lead to another outcome – that is lowering of drug prices”.

De Ridder, who also addressed the press conference, said that several questions needed to be addressed at the point of reimbursement decision, such as how to manage the many uncertainties that exist, how to be best informed about the value of the medicine based on available knowledge and how to translate that into something which would be an acceptable cost for society.

Over the past 10 years, said de Ridder, there has been a huge change in how reimbursement decisions are dealt with for new medicines, including orphan drugs. In Belgium, for example, he explained that the entry of new innovative medicines is now handled through managed entry agreements. Decisions are no longer based “on the classical pathway of having an HTA and an economic evaluation… It is much more fluid now”, he said.

The adaptive pathways initiative is being followed by “adaptive reimbursement negotiated contracts” and this approach will likely undergo more changes in the future, de Ridder said. As such, he believes that the EMA’s adaptive pathways initiative alone cannot lead to lowered prices. To achieve price reductions, he explained, there is a need to redesign the entire process so that it leads to “fair decisions [on] meeting patients’ expectations… and also give[s] the guarantee that what we are investing in terms of money is the best investment we can make for everyone”.

Sheela Upadhyaya from UK HTA body NICE, believes that it is very hard to demonstrate the improvements that initiatives such as the adaptive pathways project can deliver in terms of price reduction. Upadhyaya emphasized the need for a “more holistic, wider concept” that would need buy-in and engagement from the industry, payors, HTA bodies and patient groups.

“It’s not just one process that is going to allow us to make those changes and reduce the prices… The adaptive pathways element is one small piece of the puzzle and we need to think how we can enhance and build on that in order to enable [patient] access to be a more real scenario,” said Upadhyaya, who is associate director for highly specialized technologies at NICE’s Centre for Health Technology Evaluation.

From the editors of Scrip Regulatory Affairs.