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.

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.

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.

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.