Masterstudiengang "Drug Regulatory Affairs"

Master-Thesis

Impact assessment of the proposed Pharma Legislation of the European Union on a pharmaceutical industry portfolio with focus on Regulatory Data Protection and Orphan Market Exclusivity and associated conditions ***

Dr. Isabel Ramtohul (Abschlußjahr: 2024)

Summary
Language: English
On 26 April 2023, the “Pharma Package” was published by the European Commission (EC) targeting the revision of the European Union’s (EU) pharmaceutical legislation. This package proposes a more streamlined legal framework and comprises only one new directive and one new regulation. These two documents will cover the General Pharmaceutical Legislation (GPL) as well as the specific legislation for rare diseases and for children. The new directive will replace Directive 2001/83/EC and will incorporate relevant parts of the Paediatric Regulation (Regulation (EC) No 1901/2006), whereas the new regulation will replace Regulation (EC) No 726/2004, and the Orphan Regulation (Regulation (EC) No 141/2000) and will incorporate relevant parts of the Paediatric Regulation (Regulation (EC) No 1901/2006).
The proposed legislative revision aims at increasing the effectiveness of incentives for innovations while promoting public health interests with a modular approach of regulatory data protection (RDP) for non-orphan pharmaceuticals as well as orphan market exclusivity (OME) for orphans. This includes a minimum period of baseline protection and the possibility of extensions if different conditions are met, thus providing a protective environment for highly needed medicines while ensuring timely and equal access to medicines across the EU.
However, this approach has been heavily criticized by the industry, pharmaceutical associations, and governments, mainly because of the potential shortening of the protection period, the greater uncertainty regarding the overall length of the protection period due to the modular approach, and the very high hurdles for meeting the different conditions, which are at least partly outside the control of the pharmaceutical companies.
Against this background, the overall objective of this thesis was to assess the impact of the proposed changes to the regulatory data protection and orphan market exclusivity mechanisms for the pharmaceutical industry.
For this analysis, a representative subset of 21 pharmaceuticals, authorised in the European Union via the Centralized Procedure, was chosen from the portfolio of a global pharmaceutical company with focus on innovative pharmaceuticals. This subset consists of 13 non-orphan medicinal products and 8 orphan medicinal products and covers the therapeutic areas “cardiovascular, renal and metabolic”, “immunology and neuroscience” as well as “oncology”.
Based on a detailed examination of the prevailing protection mechanisms for orphan and non-orphan medicines and their proposed changes in the ongoing revision, a retrospective analysis approach based on publicly available documents from EMA, mainly the EPAR, was developed, described in detail and carried out using the selected subset of pharmaceuticals.
Key results regarding the fulfilment of the newly introduced conditions for extending total RDP period and OME period were the following: The introduction of the condition of launch in all EU member states within two years after initial MA has the highest requirements. As a result, only one pharmaceutical product out of the 21 analysed pharmaceuticals managed to fulfil this condition. For the other conditions, like addressing unmet medical need, conducting comparative pivotal clinical trials and introducing a new indication with significant clinical benefit in comparison with existing therapies, about half of the 13 non-orphan medicinal products managed to fulfil the requirements. When looking at the conditions for extending the OME period, the degree of fulfilment was on average lower, which suggests higher requirements in the context of orphan medicinal products and conditions. In general, the performed analysis confirmed the considerably higher degree of complexity and uncertainty of the proposed modular approach compared to the prevailing legal framework.
When analysing the impact on the total protection period, especially the impact on the RDP period was tremendous. When comparing the total RDP period for all 13 non-orphan medicinal products of the current legal framework with the EC proposal, a total loss of 17.5 years of RDP period was calculated. Comparing however the total RDP period of the current legal framework with the EP Rapporteurs’ draft report, a total gain of 28 years of RDP period was calculated.
On 15 March 2024, compromise amendments have been adopted with a baseline RDP period of 7.5 years and a baseline OME period of nine years together with further adjustments like the deletion of the launch condition and a limited extension of the protection periods by one year when fulfilling different conditions. However, this legislative procedure is not yet complete, and the performed impact analysis should be repeated with the finally adopted legislation. In the meantime, pharmaceutical companies are advised to already integrate the potential new requirements for RDP and OME into their current development efforts.
Pages: 72
Annexes: 2, Pages: 28