Are drugs for rare diseases essential?

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 Against Rare Diseases: Are The Regulatory Standards Higher?

The US Food and Drug Administration (FDA) recently issued a draft Guidance for Industry for Rare Diseases: Common Issues in Drug Development (referred to as "Rare Diseases Guidance"). In our opinion, the FDA should consider: (a) explicitly acknowledging the standards are higher for rare diseases for the reasons presented in this article; and (b) illustrating innovative development pathways that...

Drugs for Rare Diseases: Paying for Innovation

Negotiating prices of drugs for rare diseases

Negotiating prices of drugs for rare diseases Séverine Henrard & Francis Arickx a Institute of Health and Society (IRSS), Université catholique de Louvain, Clos Chapelle-aux-Champs, 30 B1.30.15, 1200 Brussels, Belgium. b National Institute for Health and Disability Insurance (RIZIV/INAMI), Brussels, Belgium. Correspondence to Séverine Henrard (email: [email protected]). (Submitted: ...

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...