With the arrival of precision medicine, the days of the old ‘one size fits all’ treatments seem long in the past. Precision oncology and new approaches to clinical research have meant a dramatic change in the field of cancer treatment. But precision medicine, promising as it is, brings its own challenge – how can we be sure that these targeted treatments are used optimally to the benefit of the individual patient?
New treatments now become available based on solid scientific rationale, thanks to our understanding of molecular biology and immunology. However, optimal use of new anti-cancer treatments remains poorly documented. Optimal patient population and cut-off values of biomarkers, treatment duration, sequence and combination are rarely informed, leaving patients, doctors and society facing many questions. In addition, all this is exacerbated by the often very high cost of new treatments. It is therefore essential to find an equilibrium between the interests and needs of all stakeholders, and define it around patient-centredness.
A newer version of the Clinical Trial Regulation will soon be implemented in Europe. However, it is not yet known whether it will address the issue of the qualification of studies designed to answer questions centred on patient care. “On the contrary, it raises the risks that all Member States could adopt a position not necessarily based on medically informed criteria, and risks making therapeutic strategy trials yet more challenging to perform in the EU,” says EORTC Director General, Dr Denis Lacombe.
A recent EORTC manifesto aims to try to find the balance between the interests and requirements of stakeholders “The current drug development paradigm has been criticised for being too drug-centred, and not focusing sufficiently on the patients who will eventually be consumers of the new therapies,” says Dr Lacombe. “To this end we carried out interviews with stakeholder groups across ten EU Member States to see what their views on this subject are.”
The EORTC consulted academic clinicians, representatives of patient organisations, regulatory and payer authorities, health technology assessments agencies, and industry. “We now have much greater clarity on what is needed to put patients at the centre of the drug development process,” says Dr Lacombe, “and we were pleased to find that all stakeholders considered that treatment optimisation tools to be valuable tools to address current gaps in evidence.”
Treatment optimisation is a process intended to enhance the long term efficacy, adherence, safety, convenience and affordability of a therapy. Its ultimate goal is to expand access to effective treatment to all of those it will benefit.
“Currently, European patients and the healthcare system are penalised rather than aided by the regulatory hurdle. We need greater independence in assessing the role of treatments. Urgent reform is needed to assess interventional clinical research based on purpose and treatment modalities, not necessarily through the rigid application of inappropriate frameworks” Dr Lacombe concludes.