Development process of Orphan Medicinal Products

EuropaBio-EFPIA Joint Task Force on Rare Diseases and Orphan Medicinal Products

Report from the EuropaBio-EFPIA Joint Task Force on Rare Diseases and Orphan Medicinal Products looks at key facts about oprhan medicines, their life cycle and specific features. It also gives a glossary of regulatory terms relevant for orphan medicines.

Did you know that...

  1. A rare disease is officially defined as a lifethreatening or chronically debilitating condition that affects no more than 5 in 10,000
  2. There is very little knowledge about the underlying disease mechanism for a large majority of these conditions.
  3. Only 1 out of the 5,000 to 10,000 substances initially tested gets through to the marketing authorisation phase.
  4. The EU has a dedicated piece of legislation for orphan medicinal products (OMPs) – Regulation No 141/2000 of 16 December 1999 on Orphan Medicinal Products.
  5. It is estimated that between 6,000 and 8,000 distinct rare diseases exist today.
  6. It takes on average 10 to 12 years and 1,1172 million EUR to develop and bring a new treatment to the market.
  7. Overall, employment in all departments of companies working in the area of orphan drugs more than doubled between 2000 and 2008 increasing from about 2,000 in 2000 to more than 5,000 in 2008.
  8. 80% of rare diseases have a genetic cause. Other rare diseases are due to degenerative and proliferative causes.
  9. Prior to the creation of the EU’s Orphan Medicinal Products Regulation, just 8 products had been authorised for treating such diseases. The European Commission subsequently formally identified these as “Orphan-like” products. Almost 15 years after its entry into force, more than 90 orphan medicinal products were approved to treat rare diseases.

About the Joint EFPIA-EuropaBio Task Force on Rare Diseases and Orphan Medicines

The Joint Task Force between EFPIA (the European Federation of Pharmaceutical Industries and Associations) and EuropaBio (the European Association for Bioindustries) on Orphan Medicinal Products and Rare Diseases is a European alliance of over 45 member companies committed to the development of orphan medicinal products (OMPs) and improving the lives of patients with rare diseases.

Read the report on your screen or download a copy below.