Exploiting Research Infrastructures for Personalized Medicine in Europe

There is some powerful support for personalized and precision medicine in the 'Porto declaration on cancer research' that was opened for signature on May 3^rd in the Portuguese city famed for its more traditional approaches to a sensation of well-being. The occasion was the EU Cancer Research Summit, where the accent was on developing a network of well-distributed and interconnected high-quality infrastructures for translational research, clinical and prevention trials, and outcomes research.

In particular, it recommends action across three specific areas of research: translational, clinical, and outcomes. European-wide deployment of high-quality infrastructure could achieve a 10-year cancer-specific survival for 75% of patients diagnosed in EU member states by 2030—a literally vital contribution in the face of an expected increase of cases in EU from the current 3.5 million to more than 4.3 million by 2035.

With successful proof of principle clinical/prevention trials, translation will continue by means of clinical research to achieve effective healthcare system implementation, at the same time reducing the timespan from scientific discovery to patient benefit, says the declaration. But complex and advanced infrastructures are required to make the bridge to healthcare from basic/preclinical research and clinical and prevention research, building on inventions and innovations from basic/preclinical research to directly impact therapeutic and prevention research. The use of molecular and digital pathology for stratifying patients for systemic treatment with anticancer agents is increasing and liquid biopsies are being implemented as a complementary diagnostic/monitoring tool, and infrastructure support is also increasing in complexity for radiation and surgical therapy, imaging, and immunotherapy. But consortia of advanced cancer care centres are required to make the necessary links.

Infrastructures for clinical and prevention trials can take 'proof-of-concept’ studies as a starting point for further clinical and prevention research, with a practice-changing aim, including the assessment of its utility in healthcare or prevention, patients and individuals at risk, cure and survival, and health-related quality of life. But it will require well-developed clinical trial structures, and advanced diagnostic methods such as state-of-the-art molecular pathology, omics technologies, and pharmacology, as well as innovative imaging, to stratify patients are crucial.

Prevention will depend on infrastructures that include strong epidemiology closely connected to basic research, data acquisition capacity, and advanced computational capabilities, Appropriate funding mechanisms are needed to stimulate these activities and guarantee sustainability, including for proof-of-concept trials initiated by academic investigators, says the declaration.

Infrastructures for outcomes research will make it possible to deliver on the assessment of clinical utility, cost-effectiveness, accessibility, sustainability, and prioritization. Outcomes research in therapeutics must address all aspects of the clinical pathway, including treatment optimization, side effects of treatments, long-term follow-up with assessment of health-related quality of life, rehabilitation, and survivorship, as well as attention to social and economic aspects, and should be a collaborative effort between clinicians, researchers, and epidemiologists.

"Precision oncology isn't only about what is new. It's also about making better use of what we already have," Denis Lacombe, director general of the European Organisation for Research into and Treatment of Cancer, told the Porto conference to launch the declaration. He cited EORTC's SPECTA infrastructure for access to personalized medicine clinical research programs as an example of a successful quality assured clinical trial’s structure, arguing in favour of harmonisation of technical requirements to support multinational collaborations in next-generation clinical trials. "Current regulatory and research approaches leave many areas of treatment knowledge under addressed," he said, pointing to issues of dosage, sequence, combination, or possibilities of de-escalation.

He insisted that society will have to develop the means to generate datasets aiming at documenting the optimal treatment for cancer patients though the integration of clinical research, free of commercial interest in the process of access to treatments, and to inform healthcare systems. This means that for optimized treatments in healthcare, society must re-engineer new models of partnership between commercial and non-commercial research in the continuum of clinical science, regulatory science, and into health technology assessment. Public health priorities will need to command upstream research, to focus innovation where needed, and avoid multiplication of redundant agents of the same class.

While the needs were great, not everything needed creating de novo, he argued, "Europe should build on existing solutions and infrastructures which deliver, optimizing competences and avoiding duplication."