The role of orphan medicinal products and the access to treatment

1. The role of orphan medicinal products and the access to treatment Maria Puiu, Dorica Dan ANBRaRo

2. About Orphan Drugs "Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases.

3. Rare diseases • In the EU, a rare disease is one which affects fewer than 5 people per 10 000. • The number of sufferers may still be high, however, as there are some 7 000 known rare diseases.

4. Rare diseases While signs may be detected at birth or in childhood, more than 50% of rare diseases appear during adulthood, and are often life-threatening or chronically debilitating. Most are caused by genetic defects, but environmental exposure during pregnancy or later in life, often in combination with genetic susceptibility, is another cause.

5. Rare diseases The EU is promoting optimal prevention, diagnosis and treatment of rare diseases by creating networks, sharing experience and training, and disseminating knowledge.

6. Rare diseases Rare diseases are a serious public health concern and a priority in the EU health and research programmes. EU law and funding for R&D projects aim to promote the development of orphan drugs for patients with rare diseases.

8. Usually there is no effective treatment, but screening for early diagnosis, followed by suitable care, can improve quality of life and life expectancy.

9. It became increasingly obvious that these patients need support from European community.

10. All the national single efforts in this field are not sufficient and those diseases are so rare, there we need centers and experts in each European State.

11. About Orphan Drugs They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.

12. To provide care for these “forgotten patients” and to encourage pharmaceutical and biotechnology companies to invest in treatment for rare diseases, governments have created various legal and financial incentives.

13. In 1983 the Orphan Drug Act was introduced in the US and in 1999 the “Regulation on orphan medicinal products” was approved by the European Parliament. • This regulation grants, among other things, a ten-year market exclusivity for any new orphan drug.

14. Orphan Europe has been developing and providing orphan drugs for the treatment of rare diseases since 1990, well before the orphan drug legislation was introduced in Europe. • We have the conviction that every patient has the right to the best possible treatment, especially patients suffering from a rare disease.

15. Scientific Committee on Medicinal Products and Medical Devices 1997-2004

16. The European Medicines Agency is a decentralised agency of the European Union, located in London. The Agency is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union.

17. http://www.ema.europa.eu/ • A designation from the European Medicines Agency's Committee on Orphan Medicinal Products (COMP) permits a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease such as a genetic disorder or a rare cancer. A large number of these diseases affect children and newborn babies.

21. In America: • The U.S. Food and Drug Administration (FDA) website offers a list of orphan drugs that have received marketing approval in America. www.fda.gov In Europe: • European Union registry of designated orphan drugs

22. In Romania, the growinginterest of the govermental health system is due to the Rare Disease Patients Associations effort in the last 5 years (RPWA then RoNaRD).

23. Law of 20 November 2003 establishing the criteria for designation of orphan medicinal products and how the marketing authorization of its. • Published in Monitorul Oficial 874/ 9 December 2003 (M. Of. 874/2003)

24. Health Programs • HP chronic disease in children • HP for hemophilia, thalassemia and other rare diseases- 2008 • HP for rare diseases and severe sepsis -2009 • HP for Rare diseases - 2010

25. National Medicines Agency • 47 orphan drugs - 2009 • http://www.anm.ro/_/Lista%20medicamentelor%20orfane%20valide%20in%20Romania.xls

35. Treatment of patients with rare diseases through the National Plan for Rare Diseases…not through Health Programs…

36. The future of orphan drugs… Together Thank you!