Rare essentials drugs for rare diseases as essential medicines

Rare essentials: drugs for rare diseases as essential medicines.

Since 1977, the WHO Model List of Essential Medicines (EML), published by WHO, has provided advice for Member States that struggle to decide which pharmaceutical technologies should be provided to patients within their public health systems. Originating from outside WHO, an incentive system has been put in place by various governments for the development of medicines for rare diseases ("orphan ...

Drugs for Rare Diseases: Paying for Innovation

Rare diseases and expensive drugs

Immunologists/allergists are a physician group that is familiar with caring for patients with rare diseases. Conditions that come under the umbrella of our specialty include various primary immunodeficiency diseases, mast cell disorders, various autoinflammatory diseases and diseases of the complement cascade including Hereditary Angioedema (HAE). A rare or orphan disease is defined by the Euro...

Rare diseases and orphan drugs.

According to the Regulation (EC) N. 141/2000 of the European Parliament and of the Council, rare diseases are life-threatening or chronically debilitating conditions, affecting no more than 5 in 10 000 persons in the European Community. It is estimated that between 6000 to 8000 distinct rare diseases affect up to 6% of the total EU population. Therefore, these conditions can be considered rare ...

[The pharmacist, rare diseases and orphan medicines].

Treatment of rare diseases is often complicated due to the difficulties posed by the research and development of medicines by the pharmacological industry. One of the greatest concerns of this group of patients, which is both scarce and diverse, is access to available medicines. The European Medicine Agency establishes the term orphan medicines and the incentives and bases for their commerciali...