Long-View Innovation Hopes for Pharma Policy in the EU

From the outside, it may look as if Europe is falling apart in health policy, with the painfully slow vaccination campaign leaving it still vulnerable to the COVID-19 pandemic, and with squabbles over the safety of vaccines and access to supplies. But there is some steadier planning for the longer term going on behind the scenes, suggestive of a more confident and coherent approach to the future—and some of it could spell good news for pharmaceutical executives.

Unobtrusively, an outline agreement was reached in March over Horizon Europe, the European Union’s framework program for research and innovation for the next seven years, which will dedicate a sizeable chunk of its $75 billion budget to drug research. Even less obtrusively, a draft agenda is now circulating among insiders that indicates just how far the EU has got in identifying its priority challenges for the future. But it is eloquent of a determination to promote R&D that should provide better care for Europe’s citizens than the current difficult circumstances allow.

Rare diseases

“New effective therapies for rare diseases” is one of the targeted areas, with financial support to be offered to researchers and developers to increase the success rate of these therapies by employing “robust preclinical models, methods, technologies, validated biomarkers, reliable patient reported outcomes, and/or innovative clinical trials designs,” and by moving faster toward market approval through increased testing in late-stage clinical trials.

Despite the increase in orphan medicines approved in recent years, still fewer than 6% of rare diseases have an approved treatment option, the agenda points out. EU incentive packages “have not yet adequately managed to direct research and innovation in areas of greatest unmet medical need,” it laments. So projects will be financed that deploy translational, preclinical, and clinical research and validation in the clinical and/or real-world setting, as well as the use of biomarkers defining robust surrogate and clinical endpoints. There is an emphasis on making use of artificial intelligence tools, medical devices, biosensors, companion or complementary diagnostics for defining reliable patient reported outcomes, and modeling and simulation and in silico trials methodologies.

Advanced therapies

At the other end of the spectrum of unmet medical needs is the agenda’s offer of funding for the development of next-generation advanced therapies to treat highly prevalent and high-burden diseases. To respond to the lack of robust research on parameters such as safety, upscaling, immunity, potency assays, and cost-effectiveness, this topic aims to ensure that the next wave of advanced therapies, based on either pluripotent stem cells, gene editing, or RNA, are established in a timely fashion and in accordance with the appropriate regulatory standards for further testing. The funding will be given to preclinical research platforms that tackle the bottlenecks currently encountered, with a view to bringing promising advanced therapies to the market within the next decade.

Biomarkers

Another topic to be funded is the use of biomarkers for optimizing the effectiveness of existing prescription drugs for major diseases, with support available for moves by diagnostics companies toward market approval for companion diagnostics. Applicants should perform the clinical validation of qualified biomarkers (not limited to molecular biomarkers) that will enable the identification of appropriate patients to ensure an effective and efficient use of existing drugs. The relevant biomarkers should allow providing the right medicinal product, at the right dose and the right time.

'An innovative industry'

But of most particular interest to drug industry executives is doubtless the entire section of the agenda devoted to “Maintaining an innovative, sustainable, and globally competitive health industry.” This is based on a very positive assessment by the EU of the industry’s merits as “a key driver for growth” with “the capacity to provide health technologies to the benefit of patients and providers of healthcare services.” One of the specific focuses of this section is the development of better methodologies and interdisciplinary approaches to equip public authorities to assess and value new health technologies and interventions. Still more welcome will be the prospect of specific support for the development of new pricing and payment models for health innovations.

Reflector is Pharm Exec’s correspondent in Brussels