Report from the 21st EURORDIS Round Table of Companies (ERTC) meting, Barcelona, Spain.
Initiated in 2004, the EURORDIS Round Table of Companies (ERTC), is a “club” of pharmaceutical companies with a common interest in rare diseases and orphan drug development. The 21st ERTC was an occasion to review and discuss issues around collecting, registering and sharing patient data in order to foster the development of treatments for rare diseases. There were 80 participants from 14 different countries attending this meeting, which looked at ongoing challenges in sharing rare disease patient data.
A need for cultural change
While progress has been made in the area of patient registries and data – notably through the adoption of the basic principles delineated in the EUCERD Recommendations on Rare Disease Patient Registration and Data Collection and the upcoming establishment of the European Platform of Rare Diseases Registration, which will also subscribe to these principles – there is still a need for cultural change. As Hanns Lochmüller, coordinator of RD-Connect pointed out, “While competition between different research groups and drug development companies is a driving force to advance science and innovation, harmonisation and sharing of data is ultimately required to compare, combine and make best use of the results. This is especially true in rare diseases, where individuals with the conditions may be scattered across the world”.
Other outstanding issues also remain: Technically, the collection and sharing of patient data represents a major challenge: how to create interoperability between geographically and structurally dispersed entities and ‘siloed’ sources of data? There are also legal challenges, particularly the anticipated impact of the proposed European legislation on data protection. Socially & ethically, it is still not completely understood how to achieve a comfortable balance for patients between sharing data and protecting privacy and confidentiality.
The European regulatory framework for data protection
In 2012, the European Commission proposed a major reform of the EU legal framework on the protection of personal data to incorporate considerations brought into play by the introduction of new technologies. The new legislation favours a high standard of protection of personal data that is widely considered detrimental to research. In particular, for health and medical research, the issue of “consent” remains critical. Many patients and researchers alike were hoping for a “consent –mechanism” that would address all concerns around processing and re-use of data. The proposal for a Regulation is still undergoing the legislative process.
What do patients want?
According to results of the EPIRARE patient survey and ongoing patient consultation on data sharing in the RD Connect project, a majority of patients want to share their data in order to move forward knowledge, treatments and care for their conditions. However, within this context of general willingness, patients desire an active role in the establishment, design, operating, and governance of data collection programmes. Patients need to trust the data-sharing network and the purposes of the research projects, want to see a firm process for consent, and want to be kept informed of progress gathered from their data sharing. This is particularly true for initiatives such as RD Connect, which heighten the sensitivity of patient information by integrating genetic material and data. Furthermore, patients are in favour of a common European registry infrastructure as well as a uniform European legislative framework for rare disease patient registries.
Toward a public-private model
The ERTC provided the occasion to examine the feasibility of establishing public-private partnerships in patient data collection which could include collaboration between members of industry and the European Platform of Rare Diseases Registration to be set up at the Joint Research Centre (JRC) in Ispra Italy, rare disease Centers of Expertise, European Reference Networks, and academics through EU-funded research projects such as RD-Connect.
As a community, we can congratulate ourselves on coming a long way in building consensus around guiding and aspiring principles for collecting and sharing patient data to maximise opportunities for improved diagnosis and treatment development.
“Many disease-specific communities – researchers, companies and patient groups - have demonstrated the translation of these principles into action. Yet for many others, this is not yet a reality,” observes Anna Kole, Registry and Biobanks Manager, EURORDIS.
We will continue this open dialogue on how to best build common incentive structures and effect a true cultural change in data sharing to ultimately deliver concrete benefits to patients.