In 2023, the European Commission proposed changes to pharmaceutical legislation. One of the main objectives of these proposals is to ensure a high level of public health by guaranteeing the quality, safety and efficacy of medicines provided to patients across the EU. In particular, the changes aim to ensure timely and equitable access to safe, effective and affordable medicines, while creating an attractive, innovation- and competitiveness-friendly environment for research, development and production of medicines in Europe.
We take a look at the changes the pharmaceutical industry should prepare for.
Revolutions and evolutions in pharmaceutical regulation
A word of caution at the outset: the proposed changes, while comprehensive, focus only on the specific objectives mentioned above. They therefore do not include provisions on advertising, falsified medicinal products or homeopathic and traditional herbal medicinal products.
Understandably, the proposals that are causing the most controversy or doubt have been most widely discussed in the public sphere.
These include:
- Shorter periods of protection (data exclusivity and market exclusivity) for innovative medicines
- The possibility for innovative pharmaceutical companies to benefit from longer regulatory protection, but on the condition that they carry out their research on new medicines in Europe
However, it is worth looking at other, perhaps less controversial, proposals that are equally interesting and in some ways quite innovative.
In fact, the explanatory memoranda to the legislative acts of the pharmaceutical law reform contain one of the key concepts of this reform in the context of insufficient patient access to innovative and promising therapies, which “do not always reach the patient, and patients in the EU have differing levels of access” and the fact that “scientific and technological developments and digitalisation are not fully exploited”.
This is the concept of unmet medical needs.
Unmet medical needs of EU patients underpin new legislation
One of the proposed changes, designed to better exploit scientific potential and improve patient access to innovative and promising therapies to better meet their health needs, is the possibility of submitting significant non-clinical or clinical evidence for a new therapeutic indication that addresses an unmet medical need of patients.
Such evidence may be submitted, either to the European Medicines Agency or to the competent authority of a Member State, by an entity not engaged in an economic activity (a not-for-profit entity), i.e. not related to the marketing authorisation holder.
As a next step, at the request of a Member State, the European Commission or on its own initiative, the EMA will scientifically assess, on the basis of all available evidence, the benefit-risk balance of the product in the new therapeutic indication.
If the opinion is positive, the marketing authorisation holders of all medicinal products containing the active substance concerned will be obliged to request that the information on their products (such as the summary of product characteristics and package leaflet) be supplemented to include the new therapeutic indication.
The key points of this solution are:
- Entities other than the marketing authorisation holders will be allowed to conduct studies independently
- No regulatory data protection period for new therapeutic indications (data exclusivity)
- Applicable only to existing marketing authorisations and not to registration procedures for obtaining a marketing authorisation for the first time
The impact of the changes on patients
An ‘unmet medical need’ is defined in Article 83(1) of the revised Directive 2001/83.
In accordance with this provision, a medicinal product is considered as addressing an unmet medical need if at least one of its therapeutic indications relates to a life threatening or severely debilitating disease and the following conditions are also met:
- There is no medicinal product authorised in the Union for such disease, or, where despite medicinal products being authorised for such disease in the Union, the disease is associated with a remaining high morbidity or mortality, and
- The use of the medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population
The proposed solution appears to be highly beneficial from the patients’ perspective.
However, the requirement to meet the following two conditions will be important:
- Fulfilling the definition of an entity not engaged in an economic activity (not-for-profit entity) presenting research results for a new indication
- Documenting that this indication addresses an unmet medical need
It remains to be seen in practice whether this provision will provide a real improvement in access to new therapies or whether it will remain only a theoretical possibility.
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