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Outrageous prices of orphan drugs: a call for collaboration

2018; Elsevier BV; Volume: 392; Issue: 10149 Linguagem: Inglês

10.1016/s0140-6736(18)31069-9

1474-547X

Lucio Luzzatto, Hanna I. Hyry, Arrigo Schieppati, Enrico Costa, Steven Simoens, Franz Schaefer, Jonathan C. P. Roos, Giampaolo Merlini, Helena Kääriäinen, Silvio Garattini, Carla E. M. Hollak, Giuseppe Remuzzi, Tiziano Barbui, Ariela Benigni, Enrico Costa, Erica Daina, Silvio Garattini, Donatella Gramaglia, Carla E. M. Hollak, Hanna I. Hyry, Helena Kaarinen, Christoph Licht, Lucio Luzzatto, Giampaolo Merlini, Rosario Notaro, Giuseppe Remuzzi, Franz Schaefer, Arrigo Schieppati, Steven Simoens,

Pharmaceutical Economics and Policy

Few 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 US Food and Drug AdministrationOrphan Drug Act, Pub L. No. 97-414, 96 Stat. 2049. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm364750.htmDate accessed: March 1, 2018 Google Scholar 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 Administration 2 Office of Orphan Products DevelopmentList of orphan designations and approvals. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htmDate accessed: March 1, 2018 Google Scholar (FDA) approving 575 drugs and biological products for rare diseases between 1983 and 2017—a real success. In 2000, the European Commission passed similar legislation for orphan medicinal products (OMPs). As a matter of fact, the diseases, not the drugs, are the orphans because all drugs are very expensive, 3 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (consolidated version .7/8/2009). http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2000:018:0001:0005:en:PDFDate accessed: March 1, 2018 Google Scholar having marrying this success story (table). TableThe most expensive drugs Disorder Affected population Estimated price (US$) Manufacturer Eculizumab (Soliris) Paroxysmal nocturnal haemoglobinuria; atypical haemolytic-uremic syndrome 2000 $409 500 Alexion Pharmaceuticals Idursulfase (Elaprase) Mucopolysaccharidosis II 2000 $375 000 Shire Galsulfase (Naglazyme) Mucopolysaccharidosis VI 1100 $365 000 BioMarin Pharmaceuticals Alglucosidase alpha (Myozyme) Pompe disease 900 $300 000 Genzyme, BioMarin Rilonacept (Arkalyst) Muckle-Wells disease 2000 $250 000 Regeneron Algasidase beta (Fabrazyme) Fabry disease 2200 $200 000 Genzyme Imiglucerase (Cerezyme) Imiglucerase (Cerezyme) 5200 $200 000 Genzyme Laronidase (Aldurazyme) Mucopolysaccharidosis I 600 $200 000 Genzyme Drug names are followed by brand names in parenthesis. Affected population sizes are estimates. Source: M Harper (2010). 4 Harper M The world most expensive drugs. https://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html#2fb30c285e10Date: Feb 22, 2010 Google Scholar Open table in a new tab Drug names are followed by brand names in parenthesis. Affected population sizes are estimates. Source: M Harper (2010). 4 Harper M The world most expensive drugs. https://www.forbes.com/2010/02/19/expensive-drugs-cost-business-healthcare-rare-diseases.html#2fb30c285e10Date: Feb 22, 2010 Google Scholar 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 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 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 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 Dealing with drug pricing: not just one solutionOn Dec 11, US Food and Drug Administration Commissioner Scott Gottlieb announced a set of rules that would change the way insulin production is regulated in the USA, potentially leading to increased accessibility and lower prices for the drug. Those changes will not take effect until 2020. The soaring cost and limited supply of insulin (which has been available for nearly a century) is just one example of an ongoing crisis of global drug prices, from treatments for hepatitis C that cost US$100 000 for a single course to cancer drugs that cost $400 000 per year per patient. Full-Text PDF