Big changes ahead for medicinal products in the EU – EU regulatory framework and incentives set to change

On 26 April 2023, the European Commission finally published its proposed amendments to the regulatory framework governing medicinal products in the EU. This the first major review of the EU regulatory framework for medicinal products since 2004, and in the Commission's own words, it is aimed at "adapting the legislation to the needs of the 21 century".

The proposals involve some big changes, including:

• Reducing the regular period of regulatory data protection for medicinal products
  • The period of data protection for medicinal products will be reduced from 8 years to 6 years. However, extensions to this 6 year term would be available subject to various conditions:
    • +2 years: if MA holders launch the medicinal products in all Member States covered by the MA. Said prolongation will be granted if the medicinal product is supplied in accordance with the needs of the Member States concerned within two years from the marketing authorisation (or within three years in some cases, e.g. in the case of SMEs). Interestingly, Member States have the possibility to waive the condition of launch in their territory for the purpose of the prolongation, e.g. for situations where launch in a particular Member State is materially impossible or because there are special reasons why a Member State wishes that launch takes place later.
    • +6 months: for the Unmet Medical Needs (as explained below).
    • +6 months: if MA holders conduct comparative clinical trials.
    • +1 year: for an additional therapeutic indication.
  • The period of market protection (two years) remains unchanged.
  • In summary, the period of regulatory protection (data and market protection) can now add up in principle to 12 years, compared to the maximum today of 11 years, but only for innovative medicines (if a new therapeutic indication is added after the initial marketing authorisation).
  • For medicines for rare diseases, the standard duration of market exclusivity will be 9 years. Companies can benefit from additional periods of market exclusivity if they address a high unmet medical need (1 year), launch the medicine in all Member States (+ 1 year), or develop new therapeutic indications for an already authorised orphan medicine (up to 2 extra years). The regulatory production periods can therefore add up to maximum 13 years (while today the maximum was 10 years), but only if the aforementioned strict conditions are met.

• Introduction of new classification of medicinal products
  • The commission has introduced two new classifications of medicinal products:
    • UMN (Unmet Medical need) –  These are medicinal products where least one of its indications relates to a life-threatening or seriously debilitating condition and no medicinal product is authorized in the EU for that condition or where, despite medicinal products being authorized for such disease in the EU, the disease is associated with a remaining high morbidity or mortality and the use of a medicinal product results in meaningful reduction of morbidity or mortality for the relevant patient population. All orphan medicinal products are designated as UMN.
    • HUMN (High Unmet Medical Need) – These are medicinal products that prevent, diagnose or treat conditions where either no other method of prevention, diagnosis or treatment exists or, if such method already exists, they would bring exceptional therapeutic advancement.
  • In both cases, the criterion of meaningful reduction in disease morbidity or mortality for the relevant patient population should ensure that only most effective medicinal products are covered.

• Bouquet of new incentives
  • The new proposal aims to replace the existing incentives with a range of possible incentives that medicines may benefit from, on a case by case basis, which is designed to move away from the current "one-size-fits-all" system.
  • Some examples include:
    • Regulatory data protection for a repurposed medical product – the MA holder could obtain 4 extra years of protection for a new therapeutic indication that is of significant clinical benefit. This incentive would be available to medicines that have not previously enjoyed regulatory data protection and are authorised as a generic, or medicines that obtained the initial marketing authorisation more than 25 years ago.
    • Voucher system for "game changing" antimicrobials – the Commission is incentivising development of novel antimicrobials by introducing 'transferable data exclusivity vouchers', which will provide an additional year of data protection to the MA holder.  The MA holder can either use this additional period for any other medicinal product that they produce, or sell it to a third party. The system is aimed at incentivising research into new antimicrobials capable of fighting multi-drug resistant pathogens in the future, and also encouraging the responsible use of existing antimicrobials in the EU and at a national level.
    • Flexible Orphan regime – for "Orphan" medicinal products, the standard duration of market exclusivity will reduce to 9 years instead of 10. Orphan drugs that qualify for the HUMN status, as defined above, would be entitled to 10 years of market exclusivity (instead of the new standard of 9 years). Market exclusivity for orphan medicinal products based on well-established use shall be 5 years. Further extensions may also be available based on prompt and continuous supply of drugs, or new therapeutic indications.

• A streamlined and faster MA authorisation procedure and leaner EMA structure
  • The proposal introduces the reduction of overall period for MA authorisation procedure from 210 days to 180 days. The time between the opinion of the Committee for Medicinal Products for Human Use (CHMP) and the final decision on any Commission Decision concerning national marketing authorisations, in particular for referrals, should be reduced to, in principle, 46 days instead of 67 days.
  • To support the development of innovative medicines, the proposal also introduces measures such as early scientific advice by EMA (even years before the filing of the MA application) and "rolling reviews" of the data as they become available.
  • Interestingly, the proposal simplifies the EMA's structure to two main scientific committees for human medicines: the Committee for Human medicinal products, and the Safety Committee (PRAC). The orphan, paediatric and ATMP committees would be discontinued.
  • The new, leaner structure is complemented by a stronger support for the two remaining EMA committees, through working parties and expert pools. An inspectorate should be established within EMA to reinforce Member States' capacities, in particular for inspections in third countries to build efficiency in surveillance and support marketing authorisation procedures.

• A new duty of disclosure of the R&D costs
  • Pharmaceutical companies will be required to publish information on all direct financial support for the research and development of medicines received from public authorities or publicly funded bodies. This information shall be easily accessible to the public on a dedicated webpage of the company and through the database of medicinal products for human use authorised in the Union.
  • This is aimed at strengthen the negotiating position of authorities responsible for pricing and reimbursement of medicines when negotiating prices with the pharmaceutical companies.

The measures discussed above are designed to promote innovation and support the development of, and access to, innovative medicines. The new measures are not intended to have an impact on the intellectual property protections that currently cover medicinal products in the EU.