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

Rosângela Caetano,Paulo Henrique Almeida Rodrigues,Marilena Cordeiro Dias Villela Correa,Claudia Garcia Serpa Osorio-De-Castro,Pedro Villardi,J E Trinidad Segovia

doi:10.1371/journal.pone.0247853

Rosângela Caetano, Paulo Henrique Almeida Rodrigues + Show 4 more

Open Access

https://doi.org/10.1371/journal.pone.0247853

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Abstract

BackgroundThis 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.MethodsA 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.ResultsOur 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.DiscussionOur 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.

Highlights

This case study focuses on eculizumab and the regulatory and financial aspects of the drug’s development trajectory
All clinical indications approved far refer to rare diseases
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

Summary

Introduction

This case study focuses on eculizumab and the regulatory and financial aspects of the drug’s development trajectory. Eculizumab is a drug approved to treat rare diseases, and many aspects related to its patenting, marketing authorization, therapeutic indications, and pricing render the study of this medicine relevant as an exemplary case concerning orphan drugs. The clinical indications for eculizumab have progressively expanded over time and share a common characteristic–rare diseases. The development of the molecule and drug marketing strategy were guided by a financialized business model [2,3,4] adopted by the patent owner, Alexion Pharmaceuticals, Inc, in 1992. 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.

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