Rare Diseases and Orphan Medicinal Products
Favourable Environment for Orphan Drugs in Europe since 2000
Orphan Medicinal Products are designed to diagnose or treat rare diseases that are serious, life-threatening or chronically debilitating and that affect 5 or fewer people in every 10 000 in the EU. These small patient populations mean that companies have little opportunity to develop, register, and place an orphan product on the market at normal prices while obtaining an adequate return on their investment. However, a favourable environment and incentives for orphan drug development have been facilitated in Europe by the Orphan Medicinal Products (OMP) Regulation, in place since 2000.
Stakeholder Collaboration is Essential at EU and Member State Levels
Collaboration between industry and academia in rare disease research is essential to convert an initial scientific idea into a safe and effective medicine for patients. This collaboration also needs to include other key stakeholders, such as patient organisations and regulators.
EBE has therefore welcomed the greater prominence given to rare disease research in the 7th EC Framework Research Programme (FP7). Since the introduction of the OMP Regulation, 58 treatments have been authorised to date, and over 630 medicinal products in development have received the “orphan” designation. This has been achieved thanks to cooperation between all stakeholders. But it is vital that this cooperation continues at all stages, including at Member State level, ensuring that European patients get equal and real access to authorised treatments in all Member States.
EBE Contributes to the European Commission Strategy on Rare Diseases
Member companies of EBE and EuropaBio have joined forces to coordinate on policy and strategic issues and speak with a single voice on issues related to orphan medicinal products in Europe. This has been established through a joint OMP Task Force. In 2008 and 2009, we submitted detailed recommendations to the European Institutions as part of the public consultation in the field of rare diseases.
In summary, we stressed that improving the treatment of patients with rare diseases requires:
1. Greater acknowledgement that an orphan medicinal product treats life-threatening or serious debilitating conditions for which no, or no satisfactory, alternative exists in the EU; or is a product that offers clinical superiority for patients.
2. Creative solutions in clinical development, regulatory requirements and reimbursement to address the challenges of rarity. These challenges increase in direct correlation to increasing rarity.
3. Greater urgency in determining reimbursement at Member State level. We support an improved system for ensuring early patient access.
- EBE-EuropaBio Poster 2010.pdf
(861.249 KBkb) Download
- Industry Recommendations on Clinical Added Value of Orphan Drugs, Nov 2009.pdf
(130.739 KBkb) Download
- EBE-EuropaBio Comments on COMP Recommendation on Significant Benefit for Orphan Designation, July 2009.pdf
(192.881 KBkb) Download
- Council Recommendation in the Field of Rare Diseases, June 2009.pdf
(742.061 KBkb) Download
- EBE-EuropaBio Press Release on Council Recommendation in the Field of Rare Diseases, June 2009.pdf
(122.081 KBkb) Download
- EBE-EuropaBio Press Release on Commission Communication on Rare Diseases, Nov 2008.pdf
(126.389 KBkb) Download
- EBE-EuropaBio Response to Commission Consultation on Rare Diseases, Feb 2008.pdf
(207.079 KBkb) Download
- EBE-EuropaBio welcome draft Commission guideline providing legal certainty on market exclusivity for Orphan Medicines, 9 Mar 2007
(110.05 KBkb) Download
- Draft Guideline on aspects of the application of Article 8(2) of Regulation (EC) No 141/2000, 7 Mar 2007
(44.264 KBkb) Download
- EBE-EuropaBio Press Release Orphan Medicines Regulation for rare diseases on the road to success, 27 Jun 2006
(137.201 KBkb) Download
- Commission Staff Working Document on the experience acquired on Regulation (EC) No 141/2000
on orphan medicinal products, 20 Jun 2006
(568.2 KBkb) Download
- EBE Comments to Guideline on Clinical Trials for small populations, Sept 2005
(158.096 KBkb) Download
- Commission Regulation No 847/2000 on the criteria for designation of Orphan Medicinal Products and definitions of concepts 'similar medicinal product' and 'clinical superiority', 27 Apr 2000
(112.663 KBkb) Download
- Regulation 141/2000 on Orphan Medicinal Products, 16 Dec 1999
(119.111 KBkb) Download