Orphan drugs

Lucio Luzzatto and colleagues1Luzzatto L Hyry HI Schieppati A et al.Outrageous prices of orphan drugs: a call for collaboration.Lancet. 2018; 392: 791-794Summary Full Text Full Text PDF PubMed Scopus (99) Google Scholar (Sept 1, 2018, p 791) have called for collaboration from EU member states on negotiation of orphan drug prices to take advantage of the fact that with 500 million inhabitants, the EU is the largest customer for any new drug. Low-income and middle-income countries (LMICs), with more than 6 billion inhabitants and 360–480 million patients with rare diseases,2Auvin S Irwin J Abi-Aad P Battersby A The problem of rarity: estimation of prevalence in rare disease.Value Health. 2018; 21: 501-507Summary Full Text Full Text PDF PubMed Scopus (38) Google Scholar are in need of orphan drugs and should join forces in this area. Rare diseases cannot continue to be neglected in developing countries, and international collaboration among these countries is vital to change the situation. Screening programmes should be introduced for more diseases, and educational programmes for doctors (especially paediatricians and general practitioners) and nurses should be implemented; these would raise the number of diagnoses and reduce misdiagnoses. Most importantly, more patients with rare diseases for which drugs are available should be treated. Legislations, regulations, and policies for orphan drugs should be drafted and implemented (a review article3Gammie T Lu CY Babar ZU-D Access to orphan drugs: a comprehensive review of legislations, regulations and policies in 35 countries.PLoS One. 2015; 10: e0140002Crossref PubMed Scopus (145) Google Scholar failed to identify any pertinent legislation in Latin American and African countries). Patient organisations should be created in LMICs and should play a prominent role in increasing awareness and urging governments to provide treatment for rare diseases, particularly for children. Provision of antiretrovirals to HIV-infected patients in LMICs was considered impossible on economic grounds 20 years ago, and became a reality in 2010; a joint effort from patient organisations and governments could make the provision of orphan drugs to patients in need in the same countries a reality. We declare no competing interests. Outrageous prices of orphan drugs: a call for collaborationFew instances of a single act of legislation have shifted industrial policy in the pharmaceutical industry like the Orphan Drugs Act did when it was signed in the USA in 1983. The Act was written to facilitate the development of drugs for rare diseases and health conditions,1 and the incentives provided by the Act, such as 7 year exclusivity, tax credits of up to 50% of research and development costs, and access to research and development grants, resulted in the US Food and Drug Administration2 (FDA) approving 575 drugs and biological products for rare diseases between 1983 and 2017—a real success. Full-Text PDF Orphan drugsThe Viewpoint by Luzzatto and colleagues1 on drug pricing addresses a very crucial issue because an increasing number of orphan drugs have been marketed in the past decade, and genetic treatments costing more than €300 000 are being made available. Among the determinants of drug pricing reported in the Viewpoint,1 benefit to the patient is the main factor that is typically examined in cost-effectiveness analyses; disease-specific factors are already recognised to influence drug prices because an inverse association exists between treatment cost and disease prevalence. Full-Text PDF Orphan drugsLuzzatto and colleagues1 give three recommendations for pricing orphan drugs. We will focus on the first recommendation (European price negotiation) and on the first part of the second (cost-based pricing). Full-Text PDF Orphan drugs – Authors' replyWe agree with Francesca Cainelli and Sandro Vento that patients with orphan diseases, regardless of location, ought to receive the best treatment available. We share their appeal on behalf of low-income and middle-income countries for access to orphan drugs. Full-Text PDF