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03 January 2022

The EU Clinical Trial Regulation: big data and … human rights

The new EU Clinical Trial Regulation ("Regulation No 536/2014" or "CTR") was published in May 2014 as Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use. It repeals Directive 2001/20/EC and is applicable to a certain extent as of the end of January 2022.

The CTR will simplify and harmonise the procedures for authorising clinical trials in the EU. It is focused on more in-depth safety analyses and introduces damage compensation principles and the category of low-intervention clinical trials.

How will the Clinical Trial Regulation affect business?

The CTR will make it much easier for pharmaceutical companies to conduct multinational clinical trials, which is likely to increase the number of studies conducted in the EU.

As the CTR has been subject to public discussions for several years, most businesses involved are already in compliance with the requirements. The implementation of the CTR may have a significant impact on project budgets and will require additional time and resource management.

Since the CTR provides extensive guidance for safety reporting processes and strict timelines, businesses involved in clinical trials must rethink their timetables and leave more room for communication with the controlling authorities while preparing and submitting/updating safety reports.

As clinical trials must be summarised and published in a manner that is understandable to laypersons and within one year of their completion, coordinating and performing this task may also take additional time and have to be rationally budgeted. Businesses also must be aware that the controlling authorities may require updates of the published summaries in case of technical progress or international regulatory developments.

Businesses involved in clinical trials need to review their CTR compliance policies, align them with their data protection policies and ensure that both comply with GDPR requirements.


"Businesses involved in clinical trials need to review their CTR compliance policies, align them with their data protection policies and ensure that both comply with GDPR requirements."


What is CTIS and why is it so crucial?

According to the European Medical Agency ("EMA"), the Clinical Trials Information System ("CTIS" or the System) will be the single entry point for submitting information (the so-called EU portal) as stipulated in the CTR. The CTIS application form for a clinical trial and the supporting dossier have to comply with the regulatory and ethical evaluations by the Member States concerned. However, the centralised System aims to allow all clinical trial stakeholders, including sponsors, researchers and national competent authorities to cooperate in the hope that it will lead to better knowledge sharing.

The CTIS will store the data for the clinical trials conducted in the EU. The EMA will manage the data in cooperation with the Member States and the European Commission and will make the stored information publicly available to the extent specified in the CTR.

A smoothly operating CTIS is vital for the actual implementation of Regulation No 536/2014. The absence or non-viability of the CTIS makes the existence of the CTR pointless.

Healthcare big data for dummies

Speaking of CTIS, another noteworthy aspect is the potential and benefits of utilising the vast volume of data which will be generated thereby. In recent years, the EMA has acknowledged the potential opportunities of analysing large data sets or big data in healthcare.

But what is big data exactly? Although there is no commonly accepted definition, for the purposes of this article we will refer to the definition provided by the EMA: "extremely large datasets which may be complex, multi-dimensional, unstructured and heterogeneous, which are accumulating rapidly and which may be analysed computationally to reveal patterns, trends, and associations".

In other words, big data means large databases from different sources, which to be properly utilised must be processed with new technologies, including machine learning and artificial intelligence.

Big data could be used to complement the results from clinical trials and, therefore, to provide an invaluable source for pharmacovigilance or patient satisfaction analyses. This could help in finding a better (personalised) treatment (including predicting treatment responses and patient behaviour), developing medicines and identifying adverse effects, more accurately characterising diseases, etc.

With the introduction of the higher GDPR consent standard and additional legal bases for processing special categories of personal data, including health data, identifying an appropriate legal basis for health research purposes has become a complex task that must be handled on a case-by-case basis. The diverging Member State laws providing the necessary national exceptions for processing special categories of personal data (e.g. public interest or scientific research) have made the task even trickier in a cross-border context.

Fortunately, the European Data Protection Board ("EDPB") and the European Commission ("EC") have provided helpful guidance to tackle these challenges in the EC's Q&A and EDPB's opinion on the interplay between the CTR and the GDPR, as well as in the EDPB's clarifications on the consistent application of the GDPR, focusing on health research. However, many other issues for which further input will be useful remain, including the anonymisation of health data, the use of health data from social media, activity trackers or publicly available databases for research purposes, conditions for transfer to third countries of special categories of data in the context of scientific research, retention periods and others.

The Cambridge Five1 in your pocket

Data on our health, food, medicine, sport and hobby preferences has been collected for a long time. Each of us willingly or not uploads such data to the likes of Facebook, Amazon, Apple, Netflix or Google daily. These companies' silent data analysis structures the information that we provide in our searches. It determines our consumer profile and influences our decisions, including those that affect our health. That is why data analysis is often associated with limitation of human rights. But can it benefit them?

Human rights and orphan medicines

Thousands of Europeans are discriminated against daily in a way that endangers their lives. This fact cannot be changed either in view of Europe's usual east-west axis or in terms of origin, religion or gender, because we are referring to patients with rare diseases. The EU legislator has taken care to create conditions conducive for the development of rare diseases drugs ("orphan medicines"). However, these products often do not reach the people who need them. Why? Because orphan medicines are expensive (burdened by the cost of years of research and product development), oriented to small markets and usually covered by public health insurance funds. The latter "suffer" from orphan medicines' inability to achieve an effective ratio between the cost and the number of people who need them. Thus, solidarity steps back and gives way to a healthcare budget in favour of the majority. This puts people with rare diseases at a disadvantage.

If healthcare is fragmented on a sovereign principle, the economic evaluation of orphan medicines is becoming even more difficult due to limited clinical data. In the context of rare diseases, it may be a problem to gather enough patients and medical centres to conduct locally efficient clinical trials. The availability of adequate data to monitor the long-term efficacy and cost-effectiveness of orphan drugs influences whether Member States decide to reimburse them and hence their availability.

This is where the CTR and the CTIS can bridge the gap and provide useful big data analysis to facilitate access to adequate healthcare.

"Good intelligence work… was gradual and rested on a kind of gentleness"2

Intelligence as a term has always been explained as the ability to acquire useful knowledge or to collect valuable information. The CTR aims to guarantee the safe gathering and availability of such medical information summarised in an easily understandable manner. We may choose to think that the CTR via the CTIS will try to harness the artificial intelligence created by the human mind into the idea of guaranteeing human dignity, equality and solidarity.


1 A group of spies active in the UK that passed information to the Soviet Union during World War II in the early 1950s.
2 Tinker Tailor Soldier Spy by John le Carré, a novel based on the Cambridge Five case.

authors: Elena Todorova and Kristina Bozhinova

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