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Impact of Data Sharing on the Rare Disease Ecosystem

Updated: Oct 2, 2022

Decentralized Blockchain Data Platforms Offer Major Advantages Sean Gordon


Current Data Collection and Management Impede Patient Participation

One can argue that putting the control of data in the hands of the individual addresses the many issues around trust. Historically, lack of trust has limited participation in research, to the detriment of the broader communities in need of improved therapies and treatments.[1]

Healthcare data is critical for people’s health in general and rare patients in particular. Data drives diagnosis, drug discovery, development, and regulatory approval. Moreover, it is the centerpiece of today’s Precision Medicine movement. Therefore, an ethical, participatory, trust centered system collecting, storing, sharing, and managing healthcare data, across a number of silos has a massive impact on health.


Fundamentally, rare disease patients are not in control over their own health data. Independent of the moral and ethical concerns, this current state-of-affairs creates numerous negative healthcare effects.


The authors, Effy Vayena and Alessandro Blasimme argue that greater control and management mechanisms have a positive impact on patient participation. [2]Moreover, EURORDIS, the European umbrella organization for Rare Disease discovered that patients are interested in sharing their data, “Rare disease patients want to have control over the data they are sharing. 80% said they wanted to have full (47%) or near to full (33%). However, they have the following concerns, limiting their participation.

· 50% - Fear their information being shared with 3rd parties without their consent

· 47% Fear their information being used in a different context from the ones where they disclosed it

· 35% Fear their information being used without their knowledge

· 34% Fear becoming the victim of discrimination[3]

The importance of sharing data was summarized by Health tech chief medical officer Dr Shaun O’Hanlon of the UKs National Health Service “Quite simply, sharing data is essential if we are to provide the very best care we can to patients.”[4]

Several organizations, public and private have created Digital Health Platforms (DHPs). They act as interfaces among the patient’s data and the ecosystem partners which include:

1. Patients

2. Caregivers

3. Healthcare Providers

4. Patient Advocacy Groups

5. Centers of Excellence / Key Opinion Leaders

6. Payers

7. Regulators

8. Scientific Societies / Academia

In addition to providing a “safe space” for patients to contribute their data, the DHPs provide the opportunity for cross-disease analysis, offering greater synergies in research and drug development.

Currently, rare disease DHPs, such as Rare-X, C-PATH, Pulse Infofram, Healx, and All-Stripes go a long way to empower patients with their data. However, the patients are still not masters of their own data and we believe that will still hinder maximal patient participation and possible returns.

Non-Patient Centered Healthcare Data Silos Impede Progress

et al.et al in a seminal article on the negative impact of data silos on rare disease research point out, “However, we fear the tendency of various stakeholders to Balkanize databases in proprietary formats, driven by current economic and academic incentives, will inevitably fragment the expanding knowledge base and undermine current and future research efforts to develop much-needed treatments.”[5]

Healthcare Silos presupposes patient data is under the custodianship of a top-down third-party. These third parties limit access to Ecosystem partners.

Siloed data, however, also creates a range of problems for researchers looking to make that data useful to the general population. Silos, for example, block researchers from accessing the most up-to-date information or the most diverse, comprehensive datasets. They can slow the development of new treatments and, therefore, curtail key findings that can lead to much needed treatments or cures [Emphasis Mine].[6]

Slow drug development and R&D

“There is growing agreement that patient involvement (PI) in medicines research and development (R&D) provides value for all stakeholders, including patients, researchers, industry, regulatory bodies, payors, and policy makers.”[7] The depth of participation and impact is visualized in the graphic below.








[1] Dr. Scott Kahn, https://www.weforum.org/agenda/2020/07/medical-data-has-a-silo-problem-these-models-could-help-fix-it/, July 2020 [2] Vayena, E., Blasimme, A. Biomedical Big Data: New Models of Control Over Access, Use and Governance. Bioethical Inquiry 14, 501–513 (2017). https://doi.org/10.1007/s11673-017-9809-6 [3] Eurordis, Share and protect our health data!, Rare Disease Barometer Initiative [4] https://www.nhsconfed.org/articles/why-data-sharing-matters-excellent-care [5] Denton, N., Molloy, M., Charleston, S. et al. Data silos are undermining drug development and failing rare disease patients. Orphanet J Rare Dis 16, 161 (2021). https://doi.org/10.1186/s13023-021-01806-4 [6] https://www.weforum.org/agenda/2020/07/medical-data-has-a-silo-problem-these-models-could-help-fix-it/ [7] Improving Patient Involvement in Medicines Research and Development: A Practical Roadmap,Therapeutic Innovation& Regulatory Scienc, 1-8, The Author(s) 2017, Jan Geissler, Dipl.-Kfm, Bettina Ryll, PhD, MD, Susanna Leto di Priolo, PharmD, and Mary Uhlenhopp, RN, MS, MPH5,

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