Is Europe Inching Toward a Common Approach to Personalized Medicine?

Another attempt to focus European attention on the potential of personalized medicine is slowly making its way toward the operational stage. The European Partnership PerMed, which aims to support personalized medicine through transnational coordination, expects to kick off by the end of 2023, after a lengthy gestation. However, the innate diversity of Europe's constituent countries and regions means that circumstances and conditions vary widely, compromising the chances of benefiting from such innovations in care.

The plan, accordingly, is to stimulate countries to move more quickly—and on the basis of some common understanding—in exploiting personalized, or “precision” medicine, as it’s also sometimes referred as. This partnership of joint funding efforts and strategic activities over the next 10 years is intended to optimize the integration of personalized medicine (PM) into sustainable healthcare. Right now, the partnership is finalizing a “strategic research and innovation agenda,” with a view to that it plans to publish at the end of April.

At the heart of this agenda is the development of interdisciplinary research efforts in the context of what it anticipates as "a versatile PM-focused innovation system and successful implementation settings of PM approaches into health systems." The project claims that over the last decade, PM has come of age, moving beyond its earlier footholds of cancer and rare diseases. So much so, that nowadays not only research funders and researchers recognize the wider potential of PM, but also the public, healthcare providers and payers, industry, and policy circles.

The project has a long history. The European Commission and the EU member states, as well as regional entities, joined forces years ago in pursuit of this aspiration. Support came from the International Consortium for Personalized Medicine (ICPerMed) and related EC-funded coordination, as well as, over the last six years, the ERA PerMed fund for personalized medicine. A concept paper developed in May 2021 by some 50 national and regional organizations in the PM field underlined the need for establishing a European Partnership for Personalized Medicine. While recognizing that over recent years a number of international, pan-European, national, and regional activities have achieved progress, "to further develop and foster promising personalized medicine approaches, there is a need of an even closer collaboration and effort which could be achieved by such a European Partnership for Personalised Medicine," it says.

A wide consultation exercise has now been completed in preparation for the Strategic Research and Innovation Agenda (SRIA), revealing strong support within the PM community internationally for a list of key actions to be jointly pursued. Ranked by priority for funding by researchers participating in the consultation, top of the list is the generation of a collaborative approach between preclinical and clinical research, followed by identifying new targets for personalized therapies by making use of improved understanding of disease mechanisms. Also ranked highly were the pursuit of more biomarker evidence for PM, the use of single-cell technologies for PM in combination with artificial intelligence and machine learning, the creation of a network of national and regional innovation hubs, and the establishment of medical cohorts for collecting high-quality health and molecular data. Priority was also allocated to the development of new treatment modalities for PM, early cooperation between public research and the private sector, connected large-scale health databases, and broader biomarker approaches to enable more informed health decision-making.

Respondents to the consultation also listed a series of new initiatives they recommended including in the SRIA. High among these priorities was the creation of technology platforms for accelerating and de-risking of advanced therapy medical product (ATMP) innovations, to overcome the persistent challenges of time to market, risk of failure, and affordability and accessibility of personalized advanced therapies. The idea is that research should develop technology modules to rapidly generate and implement ATMPs that meet medical and market needs. Regulatory approved modules should be bundled into technology platforms, such as for manufacturing (gene transfer, gene editing, cell origin and enrichment, expansion); product analysis (potency testing, in-depth specification); preclinical testing in human in vitro test systems; therapy monitoring through biomarkers (safety, efficacy, mode of action, pharmacokinetics/dynamics); product delivery to the patient; and innovative reimbursement models.

In the research field, priorities identified included predictive patient-derived disease models for PM, early detection of non-communicable diseases, and high-throughput high-content profiling workflows for early-stage evaluation of drug candidates. Implementation issues such as improvement of patients’ adherence, the need to promote equity in PM clinical studies, and improving equity in health and wellbeing across individuals and groups also ranked highly.

According to Ejner Moltzen of Innovation Fund Denmark, who is the chair of ICPerMed, one of the predecessor organizations from which this initiative has sprung, "EP PerMed will help us to improve our understanding of diseases and to develop innovative therapies, diagnostics, and preventive strategies." Its ambition is to become "the most significant platform and initiative to connect key stakeholders in the personalized medicine field to share evidence, demonstrate solutions, and to drive supportive activities in policy, regulatory science, and health economics."