2024 Predictions and Trends for the Life Science Industry

There have been several changes to the pharmaceutical and biotech regulatory landscape in 2023. The short and long-term impacts of these changes are relatively unknown; however, all could shape the regulatory landscape for life sciences in 2024 and beyond.

What are the regulatory changes?

1. Reform of the European Union’s Pharmaceutical Legislation.
2. Medicines & Healthcare products Regulatory Agency’s (MHRA’s) International Recognition Procedure (IRP).
3. Food and Drug Administration’s (FDA’s) Benefit-Risk guidance.
4. European Medicines Agency’s (EMA’s) revised CTIS transparency rules.

Reform of the European Union’s Pharmaceutical Legislation

The European Commission is proposing a plethora of changes to the European Pharmaceutical legislation (which incorporates a new directive¹ & regulation²), including:^1-2

• Adding variable exclusivity periods for orphan medicines,
• reducing regulatory data protection from 8 years to 6 years¹ (with variable extensions),
• increasing transparency of public funding contributions to research,
• reducing the European Medicines Agency’s (EMA’s) evaluation timeline for new medicines from 210 days to 180¹days for most medicines,
• reducing the European Commission’s decision timeline for new medicines from 67 days to 46 days,¹
• reorganising and reducing the EMA committees to two: The Committee for Medicinal Products for Human Use (CHMP) and the Pharmacovigilance Risk Assessment Committee (PRAC),
• changing paediatric development to evolutionary and simplified Paediatric Investigation Plans (PIPs),
• requiring developers to study a medicine’s use in children if it has a molecular mechanism of action that may be efficacious in children (even if that disease is different to the disease being investigated in adults), and
• introducing transferrable data exclusivity vouchers granting one extra year of regulatory data protection (to be used or sold on) to a developer of a priority antimicrobial medicine.

*This list only includes a smattering of the proposed changes to the EU pharmaceutical legislation.

Impact in 2024

Although the proposed updates aim to improve the affordability, accessibility and innovation of medicines, they have been met with mixed reviews and could have cascading implications for medicine developers seeking marketing authorisation in Europe

MHRA’s International Recognition Procedure (IRP)

As of the 1^st of January 2024,³ the IRP will replace the European Commission Decision Reliance Procedure (ECDRP).

The IRP is an initiative to accelerate approval of new medicines by recognising approvals given by seven international ‘Reference Regulators’ (RRs).³ With this route (IRP), it is possible to get a marketing authorisation (MA) in the United Kingdom (UK) in as little as 2-4 months.³

The MHRA’s IRP offers significant advantages for pharmaceutical and biotech companies to fast-track marketing authorization in the UK. As an initiative based on recognising an existing approval, the IRP aims to streamline the approval process, which should save time and resources.

However, there are caveats.

The 60-day evaluation procedure is subject to strict eligibility criteria, meaning medicine developers are more likely to find themselves on the 110-day evaluation timeline.³

Also, there are UK-specific requirements for some aspects of the CTD, and this requires an understanding of the UK and EU regulatory landscape, approved precedents and treatment paradigms.

You can read more about the IRP in this article.

Impact in 2024

The IRP means medicine developers won’t be required to create and submit an entirely new Marketing Authorization Application (MAA). The IRP could also act as a springboard to a wider marketing authorization with the EMA because the MHRA generally accepts European guidelines and templates, including a country-specific Module 1

Food and Drug Administration’s (FDA’s) Benefit-Risk guidance

The FDA published guidance clarifying how it assesses the benefits and risks of a New Drug Application (NDA) or biologics Licensing Application (BLA) using its Benefit-Risk Framework.

Essentially, the FDA assesses several factors including:⁴

• The therapeutic context,
• the evidence submitted,
• uncertainties, and
• regulatory options to reduce the amount of uncertainty.

The Benefit-Risk Framework⁴

• The framework considers the therapeutic landscape to include analyses of the condition itself and current treatment paradigms.
• Then, the medicinal product is evaluated to analyse the purported benefits and risks, as well as the strengths and limitations of the evidence regarding safety and risk management activities.
• Regulators then weigh the evidence supplied, against each of the dimensions, to determine the strengths and uncertainties.
• The conclusions incorporate all aspects within the table to evaluate benefit-risk based on the evidence, any uncertainties, the condition’s severity, and the unmet needs of the patient population targeted.

The guidance details benefit-risk considerations medicine developers should incorporate throughout drug development, including:⁴

• including benefit-risk planning during drug development,
• interactions with the FDA throughout drug development,
• including patient experience data,
• conducting additional analyses, and
• presenting a drug’s benefit-risk considerations in the NDA/BLA.

Impact in 2024

The FDA iterates and reiterates the importance of incorporating patient experience data in drug development programmes, with a focus on patient-reported outcomes in relation to endpoint development. Therefore, medicine developers should consider the impact of collecting this type of data and how it could affect how clinically relevant a primary endpoint for a registrational trial will be assessed in terms of a drug’s benefits and risks

European Medicines Agency’s (EMA’s) revised CTIS transparency rules

The Clinical Trials Regulation (CTR) (EU) 536/2004⁵ came into force on 31^st January 2022.⁶ Its associated database; the Clinical Trials Information System (CTIS), is the portal medicine developers now use to compile and submit clinical trial applications and associated amendments.

Using CTIS, a Sponsor can redact certain information about the clinical trial relating to personal data and commercially confidential data. The EMA also allowed Sponsors to request deferred publishing of relevant clinical trial information, which—depending on the type of clinical trial—could be up to 7 years from the end of a trial in the EU/EEA.⁶

However, a survey of CTIS users highlighted the need for CTIS transparency rules to be amended.

This led to the EMA amending the following CTIS transparency rules:⁶

• Publishing clinical trial information that is relevant to the public,
• revision to structured data fields (e.g., study title, endpoints, inclusion/exclusion criteria, study design, investigator sites and sponsor contact information, etc),
• removing the deferral system for all clinical trial categories (which correspond to trial phases), and
• reducing the documents published to only documents essential to patients and researchers.

Impact in 2024

The removal of the deferral option is the biggest change that could impact medicine developers' strategy for protecting commercially confidential data going forward.

References

1. Proposal for a DIRECTIVE OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC and Directive 2009/35/EC. (2023). COM/2023/192 final. Document 52023PC0192. Available at: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX%3A52023PC0192
2. Proposal for a REGULATION OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006. COM/2023/193 final. Document 52023PC0193. Available at: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX%3A52023PC0193
3. Medicines and Healthcare Products Regulatory Agency (2023). International Recognition Procedure. Available at: https://www.gov.uk/government/publications/international-recognition-procedure/international-recognition-procedure
4. The Food and Drug Administration (2023). Benefit-Risk Assessment for New Drug and Biological Products. Guidance for industry. Available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/benefit-risk-assessment-new-drug-and-biological-products
5. 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, and repealing Directive 2001/20/EC. (2014). Official Journal Available at: https://eur-lex.europa.eu/eli/reg/2014/536/2022-12-05
6. European Medicines Agency (2023). Revised CTIS Transparency Rules. EMA/263067/2023. P1-8. Available at: https://www.ema.europa.eu/en/documents/other/revised-ctis-transparency-rules_en.pdf