In view of the growing burden of metastatic breast cancer (MBC), data on recurrence of the disease is urgently needed to support further research into the specific needs of patients.
That has proved a stiff challenge to health authorities and policymakers, as health authorities do not always monitor and collect data from diseases such as MBC separately from breast cancer as a whole. Traditionally, they tend to rely on the clinical trial process.
But trials are just the tip of the iceberg. Real World Data (RWD) encompasses data gathered from electronic health records (EHRs), product and disease registries, patient-related activities in out-patient or in-home use settings and health-monitoring devices.
While there are many sources of pan-European RWD, a recent report suggests that health authorities still tend to maintain a degree of scepticism about its collection, while clinicians remain unconvinced of the utility of capturing RWD as part of their day-to-day practices.
The US has a head start on Europe on the matter. In 2016, the 21st Century Cures Act included provisions to increase the use of RWD to support regulatory decision making.
The American Society of Clinical Oncology’s CancerLinQ began a partnership with the Food and Drug Administration in June 2017, aimed at studying the real-world use of newly approved cancer treatments. CancerLinq, a national coalition of more than 85 health organisations, contends that data from clinical trials tap into only 3% of the cancer patient population.
“There is major potential to increase the use of RWE to support lifecycle product development and monitoring and to improve decision-making for regulation and HTA,” the European Medicines Agency has said.
“RWE has significant potential to support drug regulatory and HTA decision-making by providing information on a medicine’s use,” says the EMA, though it adds that “for the vast majority of products RWE will not replace the gold standard evidence for pre-authorisation development derived from randomised controlled trials (RCTs).”
The European Commission, for its part, is anxious to increase the role of real-world data across the 28-country bloc.
“Big data, in particular, real-world data, holds enormous and obvious potential in the field of medical research”, EU Health Commissioner Vytenis Andriukaitis told an event on 26 January.
“Regarding the real world data, we will need to further explore the opportunities and limitations of its use for decision making on medicinal products and in the development of new products,” he added.
RWD is also part of the health technology assessment (HTA) process on which the EU executive tabled legislation in January.
“We support it,” a Commission official told EURACTIV, pointing to the recent creation of the European Reference Networks (ERNs) – virtual networks bringing together healthcare providers across Europe to tackle complex or rare medical conditions.
Since March 2017, 24 thematic ERNs are now up and running with more than 900 highly specialised healthcare units from 25 EU countries and Norway.
The EU executive hopes that these Networks will help patients and, as they reach a critical mass of patients being analysed, diagnosed and treated, will generate more research on rare diseases and their treatments.
The Commission also intends to table a communication on digital health and care in June, EURACTIV has learned. This paper is expected to urge EU countries to support the development of a secure data infrastructure to maximise the use of big data to advance research, disease prevention and personalised health and care.
It is also expected to propose pilot actions to test the practical application of EU data sharing in the area of rare and infectious diseases and the use of real-world data.
One such programme is the DO-IT Big Data for Better Outcomes programme, a two-year project funded by the Commission’s Innovative Medicines Initiative (IMI) 2 programme aimed at facilitating the transition towards outcomes-focused healthcare systems across Europe.
“The Big Data for Better Outcomes Programme will provide a structure for empowering patients as data owners and partners in research,” Lydia Makaroff, director of the European Cancer Patient Coalition, which is a partner in the programme, told EURACTIV.
“It will ensure that informed consent and data privacy standards strike a balance between protecting individual privacy and promoting innovation. The use of Big Data will identify patient-relevant outcomes that will determine which interventions improve outcomes and increase patients’ quality of life,” she added.