Convergent Drivers of European Medicines Policy

It isn't just the COVID crisis that is changing the landscape of pharmaceutical policy in Europe—although it’s the COVID-induced changes that are getting most of the headline coverage, in multiple "lessons learned" reviews, and in the continued entrenchment of joint procurement for vaccines, which is giving the EU a new foothold in the hitherto sacred territory of national sovereignty over drug pricing. Not only is the total of vaccines ordered through its advanced purchase agreements now in excess of 4 billion, but its concerted choices of vaccine technology—shifting markedly towards mRNA and other novel approaches—is a striking intervention in previously independent national decisions over acquisitions.

But in what is currently the background of policy, other moves are occurring that will impact on the world of medicines—and particularly the development of innovative medicines, often driven by a broader search by the EU to boost its ability to compete internationally but holding out the prospect of some potentially valuable improvements in the operating context. Early hints emerged at the Global Health Summit (which ACT commented on in May), with its 'Rome Declaration' call for an efficient and effective end-to-end R&D ecosystem to accelerate research.

That call has since been endorsed and amplified by the G20 and then the G7, giving rise to a lengthy G7 communiqué that recommended greater international collaboration and coordination of clinical trials "to facilitate rapid availability of safe, effective and high-quality medical countermeasures while protecting trial participants’ safety and rights and based on good clinical practice." It expressed support for a 'G7 Therapeutics and Vaccines Clinical Trials Charter' of shared principles on accelerating the generation of "robust evidence", and for large-scale international trials to avoid unnecessary duplication and "produce clinical evidence that is generalizable to a larger number of populations and places." Crucially, the G7 also threw its weight behind greater harmonization and standardization in the digital transformation of health, with open health data standards, open-source software tools and related infrastructure to maximize the return on investments in a defined secured framework, such as the promised European Health Data Space, and with the development and international recognition of a framework for the clinical evaluation of artificial intelligence in health.

The EU-U.S. summit in Brussels in mid-June—again ostensibly about post-COVID recovery—offered further encouraging noises about innovation, with a commitment to continue to engage in health research initiatives, "including to develop medical countermeasures in a much shorter period of time." The two sides also intend to explore a new research initiative on biotechnology and genomics with a view to setting common standards. And they backed the drive to digital transformation, and to cooperate on the development and deployment of new technologies.

Just days later, a meeting of Europe's economic and finance ministers added to the pro-innovation music with a statement that pushed back firmly against the selective patent waivers on which the World Trade Organization is now embarking in the context of the pandemic. The ministers' conclusions restated the EU position that intellectual property is a major driver for innovation, competitiveness, economic growth and sustainable development, and a key enabler for knowledge and tech-transfer: "Well-calibrated policies must be put in place for purposes of helping companies to capitalize on their inventions and creations, ensuring that these serve the economy and society as a whole," the ministers insisted.

Progress at last seems to be on track for the introduction of the long-awaited EU regulation on clinical trials, with a satisfactory independent audit of progress in the development of the Clinical Trial Information System that the new system will depend on, operated by the European Medicines Agency. Publication of the final piece of the regulatory framework is now expected at the end of July, which will enable everything to go live on January 31, 2022. The onus is now on stakeholder organizations, clinical trial sponsors and authorities to prepare their staff and processes to work with the new system.

And the EMA itself has just been reinforced (once again with COVID as the impetus) to give permanence to some of the emergency measures introduced over the last eighteen months—including a new scientific task force to swiftly advise clinical trial sponsors on innovative products, and a legal basis to access and analyze real-world healthcare data.

Meanwhile, plans have emerged for a new EU-backed public-private partnership with healthcare industries—the Innovative Health Initiative—which aims to foster a convergence between medical devices, radiological electromedical and IT companies along with biotech, vaccines and pharmaceuticals. With a budget of nearly $3 billion, it will follow on from the long-running Innovative Medicines Initiative with a new strategic research agenda geared to personalized treatments and improved individual health outcomes. This affirms the partners' determination to break down "still existing silos" across discovery science and translational research, as well as between different academic research disciplines and industry sectors. The ambitions relate to the discovery of new molecules, mechanisms of action, processes, technologies, as well as their development and testing, along with methodologies for assessing safety, health outcomes and health-economic evaluation. It has set objectives for 2030 of launching 30 large-scale, cross-sectoral projects focusing on health innovations, with an accent on integrating cost-effective health care products or services with demonstrated suitability for uptake by health care systems.

Almost simultaneously, the EU has started to put into motion its new $100 billion dollar research support program, Horizon Europe, with clear aims that include "generating new knowledge and developing innovative solutions" to prevent, diagnose, monitor, treat and cure diseases. Between now and the end of the decade it will be funding activities as diverse as molecular and neurobiological understanding of mental health and mental illness, understanding the individual host response to viruses, pre-clinical development of the next generation of immunotherapies, biomarkers in treatment optimization, the use of real-world data, patient stratification, smart medical devices, advanced therapies for unmet needs, development of new antimicrobials, and public procurement of innovative solutions. Just as random examples, calls are opening shortly to obtain funding for boosting understanding of the chronic inflammation factors triggering the health-to-disease transition, or development of therapies for rare diseases, or building a European platform for repurposing medicines, or scaling up data anonymization techniques and synthetic data generation or improving the access for clinicians and researchers to electronic health data and metadata.

Big changes are underway to health technology assessment, too. After three years of often vigorous discussion, the EU proposal for closer coordination of national and regional HTA work across Europe finally won acceptance in June. It is a compromise that satisfies both the big countries like France and Germany that didn't want other countries to interfere with their well-established processes, and the many smaller countries that can benefit from shared work. Even the European Commission, which initially wanted a system that eliminated any duplication of HTA work in the EU, has pronounced itself satisfied with the deal. Over the next few years its provisions for joint scientific consultations will provide medicine developers with some greater degree of certainty in planning their evidence development both for marketing authorization and for reimbursement negotiations. The European drug industry wanted something more binding on member states, to require the use of joint assessments, but companies will nevertheless learn to derive the maximum benefit from the less-than-perfect outcome.

Not only is the EU trying to ease the HTA experience. An analogous process is underway in the semi-detached-no longer in the EU, but still an important (if reluctant at senior government levels) part of Europe, where NICE is updating its procedures for the review of commercial and managed access requests. It says it "wishes to ensure that those who are bringing technologies forward for possible use in the National Health Service can make the best plausible case for the use of their product." Initial discussions with NICE will focus on submission of a simple patient access scheme proposal—the default commercial proposal, which NICE says can cover most situations when a company needs to improve the cost effectiveness of a technology. However, if NICE identifies that a technology is not likely to be cost effective with a simple patient access scheme, it will liaise with the company to assess the potential for submitting a managed entry request.

And as it that wasn't enough, the EU's determination to press ahead with the creation of its European Health Union is behind spasmodic progress on a raft of other medicine-related initiatives, from the Beating Cancer Plan to the European Health Data Space, and from the revision of the decades-old pharmaceutical legislation to urgent reviews of the incentives to researchers in the orphan drug and pediatric medicines schemes. As an industry executive closely involved in EU policy on medicines innovation remarked to ACT recently, "There's plenty of change coming that's going to see me through to my pension." And that was a young man speaking.