A new European framework for assessing medicines has the potential to speed up access to groundbreaking treatments and reduce the administrative burden on pharmaceutical companies, according to its developers.
Health technology assessments, which examine the clinical effectiveness of a new medicine or medical device compared with existing technologies, are currently conducted by individual EU member states. But reformers believe a new, unified EU approach towards assessing the clinical evidence will reduce wasteful duplication of effort by regulators and manufacturers, by removing the need to produce separate scientific files for each nation to justify a drug’s cost.
Marcus Guardian — chief operating officer of the European Network for Health Technology Assessment, the body that has spent seven years developing the new framework — is now leading a team to fine-tune the details before its full implementation in 2025.
Under the existing system, each country “would assess each new medicine by themselves”, he says, even though they were evaluating exactly the same data. This, he suggests, represented “a waste of time and resources”.
The idea now is that, rather than all 27 EU member states being involved in every assessment, at least two would be involved in each, but the resulting evaluation would be accepted across the bloc.
While countries would continue to take individual decisions about whether they wished to pay for a medicine, Guardian considers that the new approach would particularly help small and medium-sized companies. For them, the ability to prepare a single dossier in support of a new medicine “compared to having to apply 27 times separately, in different languages” might be especially valuable.
Mihai Rotaru, a senior manager for market access at the European Federation of Pharmaceutical Industries and Associations (EFPIA), says when major pharma companies prepare their global clinical development plans, a single HTA should allow them to understand what clinical evidence “the totality of 27 member states added together will want in one single requirement”. This would allow them to prepare submissions to regulators and HTA bodies in a “quicker and more streamlined” way.
Rotaru adds that he did not think the new framework would make a difference to the ease, or otherwise, with which a pharma company would be able to conduct a price negotiation. But since the clinical evidence that would form the basis for negotiations would be the same across member states “there should be less time spent on fruitless discussions about what the drug will do from a clinical point of view”, he says. That question would in future be answered at EU level.
However, the EFPIA, which represents drugmakers, has expressed concern that member states might still decide on a case-by-case basis if and how they commit to use the new jointly conducted clinical assessments — threatening to create an even more complex system.
While emphasising EFPIA’s support for the concept of unified assessment, director-general Nathalie Moll says the “proof would be in the pudding”, as the implementation arrangements are worked on over the next three years.
“We really need to make sure that by having a European joint clinical assessment, we don’t duplicate: so we don’t have a national one and a European one”, she says.
Moll suggests it would be necessary to have “a certain level of flexibility” about the way in which assessments were conducted, recognising that “state of the art approaches” — such as incorporating “real world data”, gathered when a medicine is already in use — would be important if Europe’s pharma industry was to remain competitive globally.
The criteria for assessment also need to be “very clear and predictable” for manufacturers, she adds.
Campaigners are also seeking assurances that the voice not only of manufacturers, but of patients, will be heard in the new system.
Adela Maghear, senior EU affairs officer at European Cancer Patient Coalition, says the new framework holds out the hope of far faster access for patients to innovative medicines. But she also emphasises how important it will be to secure collaboration between all the member states if the new Europe-wide approach was not to add an additional layer of complexity to the system.
“The challenge is in the implementation”, she says. To achieve the objectives of the new regulatory framework, there is “a need for shared responsibility between the European Commission, the member states, HTA agencies but also between patient representatives, and industry.”
Only if all the relevant stakeholders work together over the next few years “can [we] build a system that is future proof and is able to deliver high quality outputs that can be relevant for decision making in member states,” she adds.
Maghear points out that there are already many barriers to patients’ access to medicines. “So we don’t want the new regulation to become an additional barrier.” The “unique input and expertise of patients” should also be reflected in the joint HTA work, she says.
Acknowledging that much work remained to be done, Guardian says: “It is a very intricate dance. It is a lot of work to [develop] mechanisms to ensure that the quality and the scientific integrity of these assessments are upheld.”
Health technology assessments, he acknowledges, “are one of these arcane fields that nobody knows about, but without which our healthcare systems would be significantly poorer”.
Nevertheless to have reached the goal of a shared framework after so many years of planning “shows that scientific collaboration across the continent is not only not dead, but really thrives, and it will allow us to get access to medicines to patients faster and, hopefully, with even higher quality, than in the past,” he says. “And I think that in itself is a huge achievement.”