Orphan drugs

Abstract

The Viewpoint by 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 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,1Luzzatto 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 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.2Rawson NS Health technology assessment of new drugs for rare disorders in Canada: impact of disease prevalence and cost.Orphanet J Rare Dis. 2017; 12: 59Crossref PubMed Scopus (18) Google Scholar, 3Medic G Korchagina D Young KE et al.Do payers value rarity? An analysis of the relationship between disease rarity and orphan drug prices in Europe.J Mark Access Health Policy. 2017; 5: 1299665Crossref PubMed Google Scholar, 4Messori A Cicchetti A Patregani L Orphan drugs. Relating price determination to disease prevalence.BMJ. 2010; 341: c4615Crossref PubMed Scopus (16) Google Scholar Needless to say, patent expiration and price competition are also involved in the pricing process. In our view, the main point of controversy is the role that production costs should play in the price of the new agent.1Luzzatto 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 In decisions regarding the price of Sovaldi,5Pierson R US senators ask Gilead to explain cost of Sovaldi hepatitis drug.https://www.reuters.com/article/us-gilead-sciences-sovaldi-senators/u-s-senators-ask-gilead-to-explain-cost-of-sovaldi-hepatitis-drug-idUSKBN0FG1JV20140711Date: July 11, 2014Date accessed: July 23, 2018Google Scholar the role of production costs was emphasised in 2014 by two American senators of the opinion that knowing the production costs could be useful information for determining a fair price for the drug. We are of the opinion that production costs essentially have a political nature rather than a scientific one. Importantly, production costs have the disadvantage that information regarding the reasonable profit recognisable to the manufacturer (as well as on the ratio between research costs vs promotion costs and the balance between research on successful drugs vs research on unsuccessful ones) is lacking. Additionally, the patients' interests would regrettably conflict with those of the manufacturer's shareholders. In conclusion, accounting for production costs in drug pricing is a poorly standardisable process that remains outside the boundaries of evidence-based medicine and previous health technology assessments. Additionally, discretion would be unnecessarily increased by adopting an approach based on production costs. 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 drugsLucio Luzzatto and colleagues1 (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,2 are in need of orphan drugs and should join forces in this area. 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