BioCentury On The Road. Amsterdam, 2023

by Erik Doevendans, Technical Head NL & Principal Consultant at Scendea

12th June 2023 - Amsterdam, NL

Introduction

This June, Scendea’s Technical Head NL & Principal Consultant, Erik Doevendans, had the pleasure of attending On the Road Amsterdam, an event organised by BioCentury, as part of their educational series: “On the Road in 2023”.

For three decades BioCentury has helped biotech executives and investors make business-critical decisions and build larger networks with peers across the innovation ecosystem. To kick off a European tour celebrating the 30th anniversary of the organisation, an in-depth interview with Emer Cooke, executive director of the European Medicines Agency was held in Amsterdam. Emer was interviewed by Steve Usdin who is the Washington Editor & Head of Policy and Regulation at Biocentury Publications. Main topics addressed during the interview included the reform of the EU pharmaceutical legislation, lessons learned from the SARS-CoV-2 pandemic and leveraging of real-world clinical data.

Reform of EU Pharmaceutical Legislation

The EU legislation has not been changed since 2004, in April of this year, the Commission adopted and disclosed a proposal for a new Directive and a new Regulation, which revise and replace the existing general pharmaceutical legislation, including the legislation on rare diseases and children. Key messages:

• Streamlining of regulatory processes, such as a reduction of review time to 180 days for assessment of Marketing Authorisation application, and 46 days for authorisation by the Commission. Promoting the ‘perfect dossier’ also through early agency interactions.

• Initiative for regulatory pilots in a ‘sandbox’ environment provided by the EMA and the Commission to test the adaptability of the pharmaceutical frameworks for new cutting-edge product developments.

• Regulatory data protection duration will be incentive driven. Importantly, a reduction of the baseline data protection period is proposed, while extending the number of possibilities of prolongation of this period. Amongst others, the proposal encompasses major changes in data protection and market exclusivity through:

o   Rewarding innovation through standard regulatory data and market protection.

o   Listening to patient organizations, ‘patients voice’.

o   Streamline the committees, going from 7 to 3; CHMP, CVPM and PRAC.

o   Additional regulatory data protection for medicine developers who provide access for all patients in the EU (+2 years).

o   Additional regulatory data protection for developers of medicines for unmet medical needs of patients (life-threatening or seriously debilitating diseases) (+6 months).

o   Additional data protection if comparative trials are conducted (+6 months)

o   Developers of medicines for rare disease will be rewarded with market exclusivity (the standard duration of market exclusivity will be 9 years, + up to 2 years when a new therapeutic indication is developed for an already authorised orphan medicine, + 1 year if UMN, + 1 year if available in all MS – max Total 13 years).

o   Developers of new medicines continue to benefit from patents and patent extension rights, known as supplementary protection Certificates (SPCs).

o   Extra 4 years of data protection for repurposed off-patent medicines with added value for patients.

SARS-CoV-2 Pandemic, Lessons Learned

Even when the mortal danger of a pandemic has passed, will there be a process of collective remembering? Instinctively, one assumes the answer must be yes, as a disturbing global event touched us all. History, though, suggests we may not remember. The ‘Spanish’ flu that struck in 1918 infected one in three people on the planet, leaving 50 million dead, still no monuments are built remembering the plague. The COVID pandemic kicked off November 28, 2020. The European Medicines Agency together with stakeholders had to adapt swiftly, first learning to understand the symptomology of COVID disease, supporting the development of COVID vaccines and later accommodating fast Market Access of these vaccines without compromising on safety and efficacy.

In that process, EMA learned that communication and collaboration with stakeholders is of the utmost importance in addressing a public health threat such as COVID. It also realized that experts in infectious disease are scarce when a global pandemic hits in. EMA made use of ad-hoc advisory groups with an understanding of the regulatory principles as well, the existing network of European Member States proved valuable in that respect. Rolling reviews were implemented making available vaccines on relatively short notice whilst ensuring safety and efficacy.

In addressing the risk of ‘pandemic amnesia’, the Agency has accumulated the lessons learned into an EMA plan for emerging health threats; the plan sets out the roles and responsibilities of EMA’s scientific committees and staff during a public health threat aiming at involvement of all interested parties at EMA and in the European medicines regulatory framework, including expert groups, national competent authorities and the European Commission, as well as the European Centre for Disease Control and Official Medicines Control Laboratories,  where relevant. Furthermore, in case of an emerging health threat, an Emergency Task Force will be set up to manage and coordinate discussions on the development, authorisation and surveillance of medicines addressing the health threat. EMA will provide support stakeholders involved in the development of medicines and to public health authorities outside Europe, including the World Health Organization.

Real World Data

In EMA’s vision for 2025, the enabling of the use of real-world evidence (RWE) and establishing its value for regulatory decision-making on the development, authorisation and supervision of medicines is laid down. In summary, it will be important to advance the debate on the value of RWE compared to randomised clinical trials (RCTs), the gold standard to demonstrate efficacy of a medicine. The vision is that RWE and RCTs should be seen as complementary, each having strengths and weaknesses, with their relative importance depending on the regulatory question. A rigorous and systematic approach to learning from doing will help to identify and establish the use-cases in regulatory decision-making for which RWE will add most value.

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