scholarly journals Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab

PLoS ONE ◽  
2021 ◽  
Vol 16 (3) ◽  
pp. e0247853
Author(s):  
Rosângela Caetano ◽  
Marilena Cordeiro Dias Villela Correa ◽  
Pedro Villardi ◽  
Paulo Henrique Almeida Rodrigues ◽  
Claudia Garcia Serpa Osorio-de-Castro
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Public Procurement ◽  
Atypical Hemolytic Uremic Syndrome ◽  
Orphan Drug Designation ◽  
Marketing Authorization ◽  
Drug Status ◽  
Retrospective Case ◽  
Market Expansion ◽  
Clinical Indications

Background This study examines the dynamics of the eculizumab patenting, orphan designation, and marketing authorization process in different countries and regulatory systems and analyzes drug revenues since its first marketing authorization. Methods A retrospective case study was conducted. Multiple information sources were used to: determine the status of eculizumab patents; examine the designation of orphan drug status by US, European, Japanese, and Brazilian regulatory authorities to determine registration status and approved clinical indications; estimate the prevalence of associated clinical conditions; investigate the history of the drug manufacturer, Alexion Pharmaceuticals, Inc., and its financialized business model; and examine global eculizumab sales revenues since its first marketing authorization. Results Our search yielded 32 patent families divided into 98 applications. The first patent granted was filed in 1995 by Alexion Pharmaceuticals, Inc. in the US. Eculizumab has always been as an orphan drug, except in the Brazilian regulatory agency. All clinical indications approved thus far refer to rare diseases (e.g., paroxysmal nocturnal hemoglobinuria syndrome, atypical hemolytic-uremic syndrome, refractory and generalized myasthenia gravis, and neuromyelitis optica spectrum disorder). Alexion’s revenues amounted to more than US$25 billion between 2007 and 2019, showing a growing trend. Eculizumab led sales from the beginning, being the only product in the company’s portfolio until 2015. In 2019, the drug accounted for 79.1% of all revenues. Discussion Our findings show that a strategy focused on obtaining orphan drug designation, expanding therapeutic indications and the geographic range of marketing approvals, extending monopoly periods, and prioritizing public procurement niches has enhanced revenues and helped the company achieve leadership in a highly specific and profitable market.

scholarly journals European and Italian regulation on orphan medicinal products

2013 ◽  
Vol 14 (2) ◽  
pp. 89-98
Author(s):  
Roberta Joppi
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Potential Benefit ◽  
Orphan Drugs ◽  
Pharmaceutical Companies ◽  
Drug Status ◽  
Medicinal Products ◽  
Eu Legislation ◽  
Orphan Medicinal Products ◽  
The Eu

The paper presents an overview of the European and Italian Regulation on Orphan Medicinal Products (OMPs), along with some data on the OMPs licensed in the EU from 2000 to 2012. The EU legislation encourages pharmaceutical companies to develop drugs for rare diseases, so-called “orphan drugs”. The European Medicine Agency recognizes orphan drug status mainly on the basis of the prevalence of the disease (≤ 5/10,000), and potential benefit. Orphan status implies incentives for pharmaceutical companies. From 2000 up to 2012 890 candidate orphan drug designations received a positive opinion and the marketing authorization was granted to 72 OMPs corresponding to 80 different indications. Currently, 59 OMPs are available to Italian patients either because licensed to the market by the AIFA or included in the list of the L. 648/96. Despite of an encouraging regulation nearly all the currently estimated rare diseases still await treatments.

scholarly journals Analysis of patient access to orphan drugs in Turkey

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Güvenç Koçkaya ◽  
Sibel Atalay ◽  
Gülpembe Oğuzhan ◽  
Mustafa Kurnaz ◽  
Selin Ökçün ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Anatomical Therapeutic Chemical ◽  
Descriptive Analysis ◽  
Orphan Drugs ◽  
Orphan Drug Status ◽  
European Medicines Agency ◽  
Drug Status ◽  
The European Union ◽  
Early Access

Abstract Background Rare diseases are life-threatening, serious, and chronic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. Methods The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey. Results Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status except for drugs that have lost orphan drug status, decommissioned in the European Union and withdrawn from the European Community Register by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. 17 (74%) of licensed products and 17 (35%) of unlicensed products were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L—Antineoplastic and Immunomodulatory” agents. Conclusion An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. Considering the capacity and prices for orphan drugs in Turkey, it can be said that many patients with rare diseases have difficulty in their treatment. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

scholarly journals An International Comparative Analysis Highlighting that Canada is Not Keeping Pace With Patient Access to Drugs for Rare Diseases

2021 ◽  
Author(s):  
Leanne Marie Ward ◽  
Alexandra Chambers ◽  
Emine Mechichi ◽  
Durhane Wong-Rieger ◽  
Craig Campbell
Keyword(s):  
Comparative Analysis ◽  
Rare Disease ◽  
Orphan Drug ◽  
Median Duration ◽  
Rare Diseases ◽  
Orphan Drug Designation ◽  
Patient Access ◽  
Number Of Patients ◽  
Health Canada ◽  
The U.S

Abstract Background: The Canadian government has committed to developing a national strategy for high-cost drugs for rare diseases starting in 2022. Considering this announcement, we conducted a comparative analysis to examine patient access to therapies for rare disease in each of Canada’s 10 provinces relative to Europe and the U.S., to understand how Canada measures up relative to other countries. Methods: We analyzed all of the therapies with an orphan drug designation approved by the European Medicine Agency (EMA) from 1 January 2015 to 31 March 2020 as the reference for the comparative analysis. We then contrasted access to these drugs in Canada (Health Canada) and the U.S. (Food and Drug Administration, FDA). Our analysis focused on: 1) the number of therapies for rare diseases entering the Canadian market; 2) the percentage of these therapies that are publicly available to Canadians; and 3) the timeliness for patient access to these therapies in Canada. Results: Our analysis identified 63 approved therapies with an orphan drug designation from the EMA. The FDA has approved 53/63 (84%) of these drugs, while Health Canada has approved 41/63 (65%). In Europe, Germany, Denmark, and the U.K. had the highest percentage of publicly reimbursed orphan drugs (84%, 70%, 68%, respectively). In comparison, Ontario (20/63, 32%), Quebec (16/63, 25%), and Alberta (16/63, 25%) had the highest percentage of drugs reimbursed among the Canadian provinces. The shortest median duration (in months) from EMA approval to jurisdictional decision on reimbursement was in Austria (3.2), followed by Germany (4.1), and Finland (6.0). In Canada, the shortest median duration (in months) from regulatory approval to reimbursement was in British Columbia (17.3), Quebec (19.6) and Manitoba (19.6), while the longest duration was in P.E.I (38.5), followed by Nova Scotia (25.9), and Newfoundland (25.1).Conclusions: Our comparative analysis found that Canadians had less frequent and timely access to therapies for rare disease than their global counterparts, highlighting the need for a strategy for rare disease in Canada that optimizes the number of patients with access to rare disease therapies, as quickly as possible.

scholarly journals Analysis of Patient Access to Orphan Drugs in Turkey

2020 ◽  
Author(s):  
GÜVENÇ KOÇKAYA ◽  
Sibel Atalay ◽  
Gulpembe Oguzhan ◽  
Mustafa Kurnaz ◽  
Selin Okcun ◽  
...  
Keyword(s):  
Orphan Drug ◽  
Rare Diseases ◽  
Anatomical Therapeutic Chemical ◽  
Descriptive Analysis ◽  
Orphan Drugs ◽  
European Medicines Agency ◽  
Drug Status ◽  
Early Access ◽  
Life Threatening ◽  
Low Prevalence

Abstract Background: Rare diseases are life-threatening, serious, and chronic genetic conditions that require complex care and have a low prevalence. An estimated one in 15 people worldwide are affected by rare diseases. This study aims to analyze the accessibility, reimbursement status, licensed status, and Anatomical Therapeutic Chemical (ATC) codes of drugs that the European Medicines Agency (EMA) in Turkey considers to be “orphan” pharmaceuticals. Methods: The drugs included in this analysis were obtained from the list of orphan drugs published by the EMA. Orphan drugs’ accessibility and licensing status in Turkey were obtained from the Health Implementation Communiqué published by the Social Security Institution (SGK) and the List of Abroad Active Substance and List of Licensed Products published by the Turkey Pharmaceuticals and Medical Devices Agency (TİTCK). Descriptive analysis was applied to determine the accessibility status of orphan drugs identified by the EMA in Turkey.Results: Based on the EMA, 105 pharmaceuticals were approved with “orphan drug” status by January 2020. Of the 105 rare drugs on the EMA list, 34 were inaccessible in Turkey. Of the 71 available drugs, 23 (32%) were licensed and 48 (68%) were unlicensed in Turkey. Seventeen licensed products (74%) and 17 unlicensed products (35%) were covered by reimbursement. When orphan drugs’ ATC codes were examined, the most common ATC group was found to be “L –Antineoplastic and Immunomodulatory” agents.Conclusion: An orphan drug incentive policy is very important to ensure early access to the drugs used to treat rare diseases. It is obvious that such a policy must prepare for the regulation of orphan drugs in Turkey.

scholarly journals The role of globalization in drug development and access to orphan drugs: orphan drug legislation in the US/EU and in Latin America

F1000Research ◽  
2015 ◽  
Vol 4 ◽  
pp. 57 ◽  
Author(s):  
Renée J.G. Arnold ◽  
Lida Bighash ◽  
Alejandro Bryón Nieto ◽  
Gabriela Tannus Branco de Araújo ◽  
Juan Gabriel Gay-Molina ◽  
...  
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Latin American ◽  
Rare Diseases ◽  
Market Access ◽  
Orphan Drugs ◽  
The United States ◽  
Drug Status ◽  
Drug Legislation ◽  
The Us

Compared to a decade ago, nearly three times as many drugs for rare diseases are slated for development. This article addresses the market access issues associated with orphan drug status in Europe and the United States in contrast to the legislation in five Latin American (LA) countries that have made strides in this regard--Mexico, Brazil, Colombia, Chile and Argentina. Based on the success of orphan drug legislation in the EU and US, LA countries should strive to adopt similar strategies with regard to rare diseases and drug development. With the implementation of new targeted regulations, reimbursement strategies, and drug approvals, accessibility to treatment will be improved for people afflicted with rare diseases in these developing countries.

Neues aus der Pharmakotherapie

2010 ◽  
Vol 29 (04) ◽  
pp. 191-198
Author(s):  
G. Kluger ◽  
S. Arnold
Keyword(s):  
Orphan Drug ◽  
Orphan Drug Status ◽  
Drug Status

ZusammenfassungRund ein Drittel aller Patienten mit fokaler Epilepsie ist trotz medikamentöser Behandlung nicht anfallsfrei (1). Insbesondere für diese Patienten mit schwer behandelbaren Epilepsien bieten medikamentöse Neuentwicklungen neue Chancen. Seit 2008 steht in Deutschland für Patienten mit fokaler Epilepsie mit Lacosamid ein Wirkstoff mit einem neuen Wirkungsmechanismus zur Verfügung. 2009 wurde die Medikamentenpalette um Eslicarbazepinacetat erweitert. Beide Substanzen haben in großen randomisierten Doppelblindstudien eine signifikante Reduktion der Anfallshäufigkeit im Vergleich zu Placebo belegen können. Zur Behandlung seltener Erkrankungen können Substanzen mit der Option des “Orphan- Drug”-Status auch nach Untersuchung vergleichsweise geringer Patientenzahlen unter besonderen Auflagen zur Verfügung gestellt werden. Als “Orphan Drug” zur Zusatzbehandlung des Lennox-Gastaut-Syndroms wurde 2007 Rufinamid von der EMEA zugelassen. Bereits seit 2001 ist Stiripentol als “Orphan Drug” von der EMEA zur Zusatzbehandlung des Dravet-Syndromes ausgewiesen und seit 2007 als “Orphan Drug” mit Auflagen für Europa zugelassen. 2008 konnte Stiripentol auch in Deutschland eingeführt werden. In dieser Übersicht sollen die wesentlichen Merkmale der genannten Substanzen dargestellt werden.Da selten auftretende Nebenwirkungen nach der Markteinführung einer Substanz auftreten können, sind weitere Untersuchungen notwendig,um die langfristige Sicherheit der vorgestellten Substanzen zu überprüfen.

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