Background

Clinical trials are essential to ensure the highest level of safety and effectiveness of medicinal products in Europe. Data generated during the trials aims to demonstrate the safety and efficacy of the tested medicine.

When applying for authorisation to market their medicinal products in the EU, companies need to submit large amounts of clinical trial data to the European Medicines Agency (EMA). EMA analyses this data and provides advice to the European Commission, which is responsible for granting marketing authorisations for medicinal products.

In 2014, EMA adopted a policy ensuring the proactive publication of this data on a dedicated portal.[1]

Ensuring broad public access to clinical trial data can foster innovation and avoid duplication of clinical trials. Importantly, by improving the accountability of the assessment process, it can strengthen public trust in decision making on medicinal products and, more generally, their safety. Although issues concerning the protection of confidential commercial information may arise,[2] the goal should be to ensure extensive transparency of clinical trial data.[3] Under its founding regulation, EMA is obliged to ensure an appropriate level of transparency and availability to the public of scientific information concerning the authorisation of medicinal products, among other things.[4]

However, EMA suspended the policy in 2018 due to the relocation of its premises from London to Amsterdam in the aftermath of Brexit and, subsequently, prolonged the suspension due to the need to reassign its resources to other urgent tasks linked to the COVID-19 pandemic. During that period, only data related to COVID-19 treatments and vaccines was proactively published.

In December 2022, EMA announced that it would relaunch its policy. In May 2023, it specified that the relaunch would be gradual, and that it would first apply to clinical trial data for medicinal products containing ‘new active substances’ that EMA envisaged for examination from September 2023 onward.

The strategic initiative

The Ombudsman opened a strategic initiative to seek more information about how EMA chose the priority area of ‘new active substances’ for the partial relaunch of the publication policy. She also asked how EMA will deal with the backlog of clinical trial data examined at the time when the publication policy was suspended and what its plans were regarding data underpinning new applications that do not immediately fall under the relaunched policy. Finally, the Ombudsman asked how EMA ensured transparency and public participation in the process for relaunching the policy. The Ombudsman sent a list of questions to EMA in June 2023.[5] EMA replied in September 2023.[6]

1. EMA’s reasons to prioritise the data concerning new active substances in relaunching its publication policy

The Ombudsman asked what motivated EMA to prioritise the publication of data concerning new active substances and whether other criteria had been considered. EMA explained why it initially focused on publishing clinical trial data concerning new active substances.

EMA chose this priority area to accelerate the relaunch. Relaunching the full original scope of the publication policy, including data unrelated to new active substances, requires more time. Due to the innovative nature of new active substances, EMA considered that the data concerning these substances was likely to be of greater public interest. It also took into account the importance of medicinal products containing such substances in the areas of oncology, cardiovascular diseases, diabetes, HIV and AIDS, immune-system diseases, neurodegenerative diseases and viral diseases, as well orphan and advanced therapy medicinal products. To inform its decision, EMA also analysed the activity and the number of downloads from its data portal, as well as requests for public access to clinical trial data, to determine which data category generates the greatest public interest. However, the analysis showed no clear trends.

The Ombudsman welcomes the fact that, in deciding which category of data to publish first, EMA sought to also take into account where there is greater public interest. For future decisions concerning publication policy, this approach, in conjunction with stakeholder consultation, could help ensure EMA best addresses the public interest.

2. EMA’s plans to extend the scope of the relaunch

The Ombudsman asked about EMA’s plans to deal with the data received during the suspension of the policy and data underpinning new applications that do not immediately fall under the relaunched policy.

EMA explained that there were over 1 600 procedures conducted between 2018 and 2023, for which the clinical data remains unpublished. EMA is still working on its strategy concerning the publication of this data. It finds this task challenging, in part, as it depends on the cooperation of the companies that applied for authorisation, which must invest resources to prepare the clinical data for publication. According to EMA, many companies stopped devoting resources to this activity while the policy was suspended, and have asked EMA for assistance with this task, putting additional pressure on its resources. EMA is currently working on improving its processes, technical tools and documentation, such as relevant templates, in preparation for the next phase of relaunching the policy. Moreover, it is working with an external service provider and Health Canada[7], which has implemented a similar policy.

The Ombudsman is aware of the challenges that EMA is facing in publishing other clinical trial data, given the limited resources available. That said, it is EMA’s duty to ensure transparency and the accessibility of scientific information concerning the authorisation of medicinal products. The Ombudsman trusts that EMA will share its preliminary plans for dealing with the backlog as soon as possible, allowing industry, civil society and experts to provide their views.

3. Transparency and consultation of stakeholders when choosing the priority area for relaunching the policy

The Ombudsman asked EMA whether it had consulted stakeholders when deciding on the phased approach to relaunching the proactive publication of clinical trial data.

In reply, EMA detailed the steps it had taken to inform the stakeholders about the details of the relaunch of the policy, including a conference, a “stakeholder platform meeting”, and a webinar. In addition, EMA updated a Q&A on its guidance relating to the publication of clinical trial data.[8] In its reply to the Ombudsman, EMA emphasised that the phased approach was not opposed by stakeholders informed following the announcement. Finally, EMA said that, if it intends to update the policy in the future, it will consider undertaking a written formal consultation with all relevant stakeholders.

The Ombudsman has consistently emphasised the importance of transparent and inclusive decision making.

To this end, the Ombudsman trusts that EMA will share with interested stakeholders - including the pharmaceutical industry, civil society, and academics - how it is planning to proceed with publishing other clinical data, and that it will undertake to involve stakeholders in this process.

Conclusion

This initiative has enabled the Ombudsman to gain insight into how EMA relaunched its proactive publication policy for clinical trial data.

The Ombudsman welcomes EMA’s efforts to fully relaunch its proactive publication policy, and encourages it to work through the backlog of unpublished data as quickly as possible. She trusts that EMA will share information about options for the next phases of the relaunch with the public as soon as possible.

On this basis, the Ombudsman closes the initiative. The Ombudsman thanks EMA for the explanations provided and invites it to take into account the observations made above. She looks forward to exchanging with EMA on these matters in the future.

 

Emily O'Reilly
European Ombudsman

Strasbourg, 16/11/2023

 

[1] https://clinicaldata.ema.europa.eu/web/cdp/home; more information: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/clinical-data-publication

[2] See decision on own-initiative inquiry OI/3/2014/FOR concerning the partial refusal of the European Medicines Agency to give public access to studies related to the approval of a medicinal product, https://www.ombudsman.europa.eu/en/decision/en/68107,

[3] The clinical trials regulation introduces increased transparency requirements. See, Regulation 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, OJ L 158/1, https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=celex%3A32014R0536

[4] Article 80 of Regulation 726/2014 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency, OJ L 136/1, https://eur-lex.europa.eu/legal-content/en/TXT/?uri=CELEX%3A32004R0726

[5] https://www.ombudsman.europa.eu/en/opening-summary/en/171508

[6] https://www.ombudsman.europa.eu/en/doc/correspondence/en/176095

[7] A department of the Government of Canada responsible for health policy.

[8] Available at https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/questions-answers-qas-external-guidance-policy-0070-clinical-data-publication-cdp_en.pdf