EU GPL: A Shift Towards More Selective Incentives for Orphan Drug Manufacturers
In March 2026, the EU published the agreed framework for the reform of the General Pharmaceutical Legislation (GPL), following more than two years of negotiations since the European Commission’s (EC) initial proposal in April 2023.
The reform introduces a more conditional and harmonised approach to orphan medicines. Compared with the original Commission proposal, default exclusivity periods are reduced, although products demonstrating genuine innovation and meaningful patient benefit may still qualify for extended protection. (European Medicines Agency, 2026)
A key structural change is the integration of orphan medicines into a single “master” Regulation, replacing the current standalone framework. Under the revised system, the uniform 10-year Orphan Market Exclusivity (OME) period will be replaced by a modulated approach, designed to incentivise first-in-class, high-impact therapies. (European Council, 2026)
At the same time, lifecycle risk is increasing. Under the prior framework, the EMA cannot complete its assessment of generic and biosimilar applications until after OME expires, which delays competitor entry. (European Council, 2026) The new rules allow the EMA to assess these applications before expiry, meaning generics and biosimilars can launch as soon as exclusivity ends.
Access obligations are also being strengthened. Failure to launch in a Member State within three years of a request may result in the loss of Orphan Market Exclusivity (OME) extensions or market protection in that market. (European Medicines Agency, 2026)
Overall, the package represents a political compromise. In several areas, the final text appears more workable than the EC’s original proposal, but it remains less favourable for orphan developers than the current framework. Portfolio, filing and evidence strategies should therefore be reviewed now, rather than closer to 2028. The balance of risk has shifted from legislative uncertainty to implementation uncertainty, making active policy monitoring, early scenario planning and robust access strategies increasingly important.
What are the key implications of GPL for orphan drug manufacturers?
There are three key areas of the updated legislation that manufacturers should be paying attention to:
1. Orphan Medicine Exclusivity: A New Three-Tier Structure
Core to the new legislation is an update to the orphan medicine exclusivity modulation structure that determines the periods of regulatory data protection (RDP) and regulatory market protection (RMP) for orphan medicines. (European Council, 2026)
The new modulation structure has three categories (European Council , 2026):
- 9 years (baseline): Applies to all orphan medicines by default, unless they qualify for one of the two categories below.
- 11 years (breakthrough orphan medicines): Granted where two conditions are met: (i) no medicinal product is currently authorised in the EU for the relevant condition, and (ii) use of the orphan medicine results in a clinically relevant reduction in disease morbidity and mortality for the target population.
- 4 years (well-established use): Applies where the active substance has been in well-established medicinal use in the EU for at least 10 years.
This new modulated approach to exclusivity is designed to incentivise innovation and first-in-class therapies, as compared with the prior, uniform 10-year OME period.
2. New Global Orphan Marketing Authorisation structure (GOMA) linking orphan medicine exclusivity to the active substance, not the indication
In addition to the exclusivity modulation structure, orphan medicines will now be subject to the Global Orphan Marketing Authorisation (GOMA) framework. Under GOMA, market exclusivity is linked more closely to the active substance rather than individual indications, limiting the ability to secure full baseline protection for follow-on indications. Instead, incremental extensions of one year per additional orphan condition may be granted, up to a maximum of two years (European Council , 2026)
This shift means manufacturers will need to think more carefully about how they expand into additional indications, balancing launch timing, overall product value, and the need to show clear clinical benefit.
3. New iteration of the Access Framework
Under the previous iteration of the General Pharmaceutical Legislation (GPL), access provisions were a key point of concern for manufacturers.
In the current framework, a Member State may, within one year of marketing authorisation, request that a manufacturer:
- submit a full pricing and reimbursement (P&R) application,
- fulfil specific procurement requirements, or
- establish a launch rollout plan.
If the manufacturer subsequently fails to launch the product in that Member State within three years of such a request, they may lose their Regulatory Market Protection (RMP) in that market, as well as any Global Orphan Marketing Authorisation (GOMA) extensions. The baseline 8-year Regulatory Data Protection (RDP) period, however, remains unaffected.
Importantly, this represents a meaningful shift from earlier proposals. Under the previous iteration, failure to launch in a single requesting Member State could have resulted in the loss of market protection across the entire EU. In contrast, the revised framework limits this risk to the individual Member State concerned. This change has been viewed by EUCOPE and industry stakeholders as a significant improvement. (European Council , 2026)
This change reduces the risk of EU-wide loss of protection, but requires a more proactive and coordinated approach to launch planning at the Member State level.
What’s next for manufacturers?
Whilst all aspects of the reform (Orphan market exclusivity modulation, access framework and GOMA) have significant implications for manufacturers, GOMA is likely to be the most immediate priority with the implementation of this regulation coming into force later this year (European Medicines Agency, 2026):
- Q4 2026: Formal adoption by Ministers of Health (EPSCO) and European Parliament
- Q4 2026: Entry into application of GOMA
- Q4 2028: Entry into application of all other provisions (market exclusivity modulation structure and access framework)
For orphan developers, the implications of GOMA will need to be considered immediately, with approval planning potentially accelerated ahead of the Q4 2026 deadline. More broadly, manufacturers should prepare for earlier generic and biosimilar competition, particularly for follow-on indications.
Overall, manufacturers can now plan with greater confidence, as this iteration of the GPL is likely to remain in place for the foreseeable future, providing a more stable basis for long-term orphan launch planning.
Throughout this month, we have been publishing a series of articles on evidence planning. If you have any questions about these changes, the wider article series, or how GOMA may impact your pipeline and launch planning, please do not hesitate to get in touch.
References
- European Council. (2026). Document Register: Regulation laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rulesgoverning the European Medicines Agency . Retrieved from https://www.consilium.europa.eu/en/documents/public-register/document-details/?ImmcIdentifier=ST-6366-2026-INIT&DocumentLanguage=EN
- European Medicines Agency. (2026). Reform of the EU pharmaceutical legislation. Retrieved from https://www.ema.europa.eu/en/about-us/what-we-do/reform-eu-pharmaceutical-legislation