Patient disease registries: potential use for regulatory purposes

Patient disease registries are becoming increasingly widespread and more frequently used by professionals, such as patient associations, doctors and public research institutions. Moreover, pharmaceutical companies are often involved in supporting or sponsoring these registries. The creation and development of these registries is made easier with the Internet and with digital communications media, such as apps and related devices, that facilitate the collection and transmission of information between patients, doctors and research centers.

The multiplication of these registries leads to the collection of enormous amounts of information on the progress of diseases and the patients’ responses to specific treatments, which represent information of significant value both for scientific research and for monitoring the safety and effectiveness of medicines taken by patients.

EMA projects

Since 2015, the EMA has been working on a project for the implementation and improvement of the use of patient registries, enhancing the role they can play for regulatory purposes and, specifically, in the monitoring of drugs. Additionally, the EMA has set up a special committee composed of representatives of the EMA scientific committees and experts from different Member States in order to create a European framework for patient registries. On November 8, 2018, the Committee published a discussion paper containing methodological and operational suggestions on the use of patient disease registries for regulatory purposes, which will remain open to comments from stakeholders until June 20, 2019.

In addition, the EMA has established an inventory of patient registries within the database of the European network of centers for pharmacoepidemiology and pharmacovigilance (ENCePP), in order to facilitate the retrieval of useful information by stakeholders from patient registries.

EMA discussion paper on the use of registries for regulatory purposes

The document prepared by the EMA distinguishes and defines various types of registries. The main distinction is between disease registries, in which data on patients with the same conditions are collected, and product registries, which instead include information on subjects taking the same medicinal products. Disease records are usually collected by organizations like academic groups, medical associations or research bodies, in accordance with national data security requirements.

As highlighted in the discussion paper, the data contained in the disease registries is particularly useful for monitoring the effectiveness and safety of treatments and for supporting the assessment of the benefits and risks of medicines. Therefore, the EMA’s paper also promotes access to the data collected in patient disease registries for pharmaceutical companies that apply for or already hold a marketing authorization for a specific medicinal product.

Governance elements

The suggested governance principles are aimed at the correct coordination and functioning of the procedures for accessing and sharing data. For instance, the EMA document recommends that registry coordinators publish policies governing collaboration with interested parties (such as pharmaceutical companies) by establishing the purpose of the collaboration, the activities that can be carried out on the registries, the scope of monitoring and the type of data that can be collected.

Security monitoring and updating

The Committee also specifies how access to the data contained in patient disease registries can be of particular benefit to pharmaceutical companies for the post-authorization monitoring of their medicines. In fact, pharmaceutical companies could use the data collected in the registries to complete the Periodic safety update report (PSUR), taking advantage of the registry’s collection and cataloging of any adverse reactions to the drug.

Furthermore, the registries facilitate the monitoring of the medicinal product, which is extremely useful for the safety and efficacy assessments required for a thorough examination of the risk-benefit balance of the medicine. The registries can also make an important contribution to Post-authorization safety studies (PASS) or Post-authorization efficacy studies (PAES), where required from the pharmaceutical company by the regulatory authorities.

Importance of protecting patients’ personal data

The collection, accessibility and sharing of patient data in disease registries are critical from the point of view of the confidentiality of the information collected, since sensitive data regarding the health of patients are involved. The EMA discussion paper clarifies that patients are free to consent to the use of their data, as well as having the option to revoke their consent at a later date and that the data shall be collected and shared by the responsible coordinators of the registries in accordance with the European regulation on the protection of personal data (GDPR) and the informed consent given by patients.

Moreover, the document recommends the creation of an electronic system of informed consent to facilitate the alignment of forms for obtaining consent, as well as the proper storage of consents, limitations and consent withdrawals. In cases where patients’ data is shared with pharmaceutical companies under a contractual agreement, the contract must clearly describe the level of access to the data and the intellectual property rights arising from the use of the data and the possible dissemination of the results.

Lastly, greater interaction between pharmaceutical companies, regulators and registry coordinators is encouraged. To this end, it is recommended that stakeholder dialogue be initiated on the appropriateness and feasibility of using the data in the registries at an early stage of the authorization process.