Written by Iro Malekou, Senior Analyst & Srishti Gupta, Senior Consultant
Emails: [email protected], [email protected]
Over a year ago, the European Commission (EC) set out to address the recognised shortcomings of the Orphan Medicinal Product (OMP) legislation.1 Partners4Access published an article in January 2023 outlining our predictions of the likely revisions, called ‘OMP legislation changes: can it improve access, innovation and affordability of new therapies?’.2 On the 26th of April 2023, the EC released its proposed revisions to the Orphan Regulation (regulation EC No 141/2000) and the Paediatric Regulation (regulation (EC) No 1901/2006).3 Table 1 provides a non-exhaustive overview of some of the key proposed legislation changes.
Table 1: Key proposed OMP legislation changes 3
| Updates | Details |
| Market exclusivity changes | – For rare disease therapeutics, market exclusivity will be reduced from 10 to 9 years – Market exclusivity can be increased if drugs address a high unmet medical need (+1 year), if they are launched in all member states of the EU within 2 years of marketing authorisation (+1 year), or if a new therapeutic indication for an already authorised orphan medicine is developed (+2 years) – These extensions could increase the market exclusivity period from the current maximum of 10 years to 13 years |
| Updated unmet need definition | – The definition of unmet need no longer refers to the diagnosis and characterisation of a disease and only focuses on the medicinal product – A medicine will address high unmet need “if there is no medicinal product authorised in the Union for such condition” or “if it demonstrates that, in addition to having a significant benefit, it will bring exceptional therapeutic advancement; where a significant benefit means a clinically relevant advantage or a major contribution to patient care of an OMP if such an advantage or contribution benefits a substantial part of the target population” – An additional requirement is that “the use of the OMP results in a meaningful reduction in disease morbidity or mortality for the relevant patient population” |
| Streamlining regulatory processes | – The Commission proposed a simplified regulatory framework for faster authorisations – The European Medicines Agency (EMA) assessment process will be reduced from 210 to 180 days, and the authorisation by the Commission from 67 to 46 days – For medicines of major public interest, the EMA assessment process will only take 150 days |
| Joint procurement | – The Commission will support Member States seeking joint procurement by coordinating access of medicines across member state patients and offering information exchange – These arrangements fall under Directive 2014/24/EU, which sets out purchasing procedures for public buyers, the Joint Procurement Agreement, and the proposed revised Financial Regulation |
Theoretically, the outlined proposed reforms hold great promise in improving the orphan medicinal landscape. By providing more tailored incentives and streamlining regulatory processes, pharmaceutical companies may be encouraged to invest more in Research & Development (R&D) and clinical trials of innovative OMPs. However, in practice, this may not be the case. The EU Federation’s response is an echo of multiple concerns that have been raised by the pharmaceutical industry and other key organisations in Europe. “The proposed changes will harm patient access to medicines and innovation in the EU”, as highlighted by the EU Federation.4
Many believe that the implementation of the proposed changes will lead to reduced innovation, unaddressed medical need, and decreased investment activities within the EU. The most discussed issues include a) the proposed penalty in market exclusivity if a medicine is not launched in all EU Member States within 2 years of marketing authorisation (MA) and b) the uncertainty associated with the definition of unmet need.
The market exclusivity period for a rare disease medicine could be extended if the drug addresses a high unmet need (+1 year) and if it is launched across Europe (+1 year). These proposed measures could help improve access and can be considered as strong incentives for manufacturers to invest in the development of easily accessible drugs for patients that need them the most. However, there are concerns regarding the feasibility of meeting the relevant criteria.
- Feasibility of launching across Europe within 2 years of MA
Launching across the EU may be extremely difficult to achieve within the 2-year time frame and could disadvantage small companies with limited resources. In support of these concerns, the European Federation of Pharmaceutical Industries and Associations (EFPIA) explored the root causes of delays in launching in the EU. They identified that “25% of the total time to availability was time between MA and pricing & reimbursement (P&R) filing and 75% was time between P&R filing and the reimbursement decision”, with the value assessment being a frequent cause for delays.5 This analysis proves that most delays are caused by factors beyond the companies’ control and highlights the need to extend market exclusivity periods rather than making them shorter.6
- Uncertainty associated with the definition of unmet need
There have also been calls for a more inclusive definition of (high) unmet need. Le Cam, the head of the rare-disease patients’ group in Europe (Eurordis) was concerned that the ‘high unmet need’ definition is unclear.7 Many available rare disease treatments are often in the early stages of their lifecycle and may therefore not meet the criteria for unmet need because of lack of sufficient evidence.8 Another study conducted by the EFPIA evaluated the likelihood of active substances authorised between 2019 – 2022 achieving the currently proposed criteria for unmet need. Only 29 of 58 of the included orphan drugs would satisfy the criteria for high unmet need.9 This may discourage manufacturers from investing in orphan drugs and will negatively impact patients affected by rare diseases, who may be denied access to life-changing treatments because of this.
Further turmoil was caused by the EU Parliament’s (EP) proposed revisions to the legislation changes, which were shared in October 2023. Revisions relevant to the rare disease space include reduced market exclusivity periods for orphan drugs and stricter orphan designation criteria than those proposed by the EC. Specifically, the EP is proposing that an orphan designation is only granted to drugs that are unlikely to “generate a sufficient return to justify the necessary investment” and where necessary, exclusivity should be reduced if a product is sufficiently profitable. On the contrary, the EC proposed the abolishment of this criterion. 10,11
The terms of the proposals of the EU Commission are being discussed by legislators in the European Parliament and the Council. However, the reforms are not expected to be completed prior to the June 2024 elections to the Parliament, especially considering the conflicting views of the EC and EP on the legislation.12 The outcome of these proposals and their impact remains to be seen. In the meantime, it is important for pharmaceutical manufacturers to stay up to date on these activities to understand the threat of OMP legislation changes to their assets.
Partners4Access can help you navigate the changing landscape in Europe and understand how these changes may affect you in the future.
References:
- https://www.europarl.europa.eu/legislative-train/theme-promoting-our-european-way-of-life/file-revision-of-the-legislation-on-medicines-for-rare-diseases-and-children
- https://partners4access.com/whitepapers/trends-to-look-out-for-in-2023/
- https://ec.europa.eu/commission/presscorner/detail/en/qanda_23_1844
- https://www.linkedin.com/posts/neil-grubert_the-current-proposals-for-revision-of-the-activity-7116653753780297728-ZBFJ/?utm_source=share&utm_medium=member_desktop
- https://www.efpia.eu/media/677291/european-access-hurdles-portal-efpia-cra-report-200423-final.pdf
- https://www.apmhealtheurope.com/story/20192/84080/efpia-bounces-straight-back-against-eu-talk-of–launch-everywhere–requirement
- https://www.apmhealtheurope.com/story/20192/84059/rare-disease-patients-see-much%2C-want-more%2C-in-eu-pharma-reform-proposals
- https://www.apmhealtheurope.com/story/20192/84259/smaller-drug-research-firms-see-commission-proposals-on-unmet-medical-need-as–troubling-
- https://www.efpia.eu/media/4aep340e/criteria-to-define-umn-and-humn-implications-of-a-proposed-incentive-framework.pdf
- https://www.europarl.europa.eu/doceo/document/ENVI-PR-753470_EN.pdf
- https://www.europarl.europa.eu/doceo/document/ENVI-PR-753550_EN.pdf
- https://www.linkedin.com/posts/neil-grubert_eu-pharmaceutical-legislation-activity-7112094146688868352-8sft/?utm_source=share&utm_medium=member_desktop
