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The Regulatory Road to Innovation
At EFPIA we are working to discover and develop innovative therapies, to deliver people the treatments they need, and eventually ensure a healthier future for European patients.  Developing new medicines is a long, complex and risky process with no guarantee of success. It begins with the spark of an idea.

The regulatory framework is the process that allows to take this idea for a new treatment through clinical trials and tests to ensure it’s safe and effective, all the way through the final authorisation, before it can be made available to patients. 

The European regulatory system has done great so far. For instance:
  • It served patients: Since its foundation in 1995, the European Medicines Agency has recommended the authorisation of over 1,400 new medicines[1].
  • It supported investments: It has helped to attract the €35 billion that the pharmaceutical industry invest in European research and development every year[2]. Investments are needed to ensure continuous innovation of new treatments.
But more can be done, to keep pace with new ideas in science and technology, as well as to face unprecedented challenges. This has never been more evident than in the fight against COVID-19.

To deliver safe and high quality diagnostics, vaccines and treatments available to patients, as fast as possible, we have identified four areas for action within the existing legislative framework:
  1. Encourage the use of new types of clinical trials;
  2. Allow greater use of data from real world use;
  3. Allow ongoing dialogue and discussion about a treatment throughout development;
  4. Simplify how medicines and other healthcare products are regulated.


Innovative Clinical trial approaches Innovative Clinical trial approaches

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INNOVATIVE CLINICAL TRIAL APPROACHES

ISSUE

Complex (and innovative) clinical trials (CCT) can enable several trials to be done in parallel to investigate the effect of a new medicine in different diseases or groups of patients. This gives more patients the opportunity to join a trial and can shorten the time needed to get the data to decide if a medicine can be licenced to treat a disease.

80% of EFPIA companies have used CCT designs to collect clinical data. Encouraging the use of complex trial designs and their acceptance by Health Authorities will help bring new treatments to patients earlier and more efficiently.

RECOMMENDATIONS

EMA and the national authorities to lead a strategic initiative to broaden the appropriate use and acceptability of CCT, including the design of an IT platform for CCT application.

The European Commission to ensure the compatibility of CCT with the EU Clinical Trial Regulation.

Real World Data And Real World Evidence Real World Data And Real World Evidence

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Real World Data And Real World Evidence

ISSUE

Industry and regulators recognise the value of using good quality RWD/RWE as additional sources of evidence for decision-making, since they allow to consider real-life impact of medicines, instead of standard effects as considered in traditional clinical trials. RWE has already shown its value in medicine safety surveillance and for the development of medicines for rare diseases, when traditional clinical trials are impossible to conduct due to the low number of patients.

Aware of the potential to use RWD and RWE even more effectively and broadly to support medicine development and assessment of benefit and risk, the FDA is already developing a RWE regulatory framework.

RECOMMENDATIONS

EMA to develop and adopt guidance on a RWD/RWE framework with clear principles for data quality and interoperability, access, analysis and regulatory acceptance.

Dynamic regulatory assessement Dynamic regulatory assessement

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DYNAMIC REGULATORY ASSESSMENT

ISSUE

Today an application for approval of a new medicine needs a large amount of data to be collected over many years and then submitted as a single dossier to be evaluated. This means that any gaps in the data package may not be identified until a late stage in the medicine’s assessment. An iterative scientific dialogue with Health Authorities during the collection of data will help reduce the uncertainties and optimise the application process. This would allow more rapid approval of medicines and faster access for patients to new treatments.

Even though PRIME already allows for an early and ongoing dialogue, resulting in an accelerated assessment, criteria for PRIME eligibility are narrow. The EMA accepted only 22% of the 215 requests for eligibility.

RECOMMENDATIONS

EMA to design guidelines for a flexible regulatory pathway which includes an iterative process for seeking early and continuous dialogue on data, as they are generated.

The European Commission to accelerate the review of EMA recommendations for approval.

Drug-device combinations & biomarker validation Drug-device combinations & biomarker validation

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DRUG-DEVICE COMBINATIONS & BIOMARKER VALIDATION

ISSUE

1 in 4 medicines approved at EU level includes a device component. In the EU, while medicines are assessed by the EMA, different authorities (notified bodies namely) are responsible for assessing the performance of medicines used in combination with medical devices and in vitro diagnostics. Experience gained by the FDA underlined the importance of an integrated approach.

Advances in personalised medicine require additional regulatory requirements, such as biomarker validation. Current biomarker qualification process lengthens already cumbersome procedures thus jeopardising access of patients to innovation.

RECOMMENDATIONS

EMA to adopt an integrated EU pathway for the assessment of drug-device combinations and in vitro diagnostics.

EMA to incorporate best practices in order to include additional regulatory requirements coming from advances in science, such as biomarker validation, without overburdening existing procedures.

Discover all our recommendations Discover all our recommendations

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[1] European Commission, Union Register of medicinal products for human use.
[2] EFPIA, The Pharmaceutical Industry in Figures, 2019