Obtaining orphan status – A key milestone within a development strategy

Pauline Messina and Marie Deneux

Published in the Regulatory Rapporteur, July/August 2016

CONTENTS

01 Introduction

02 Eligibility criteria for EU and US orphan designation

03 Voisin Consulting Life Sciences

04 Additional information

Introduction

Although their prevalence is low, rare diseases affect six to eight per cent of the population worldwide and therefore represent a global public health concern. Orphan drug regulatory status has been established in various countries since the early 1980s to address the high unmet medical needs associated with orphan diseases. The attractiveness of the incentives associated with orphan designation is well recognized, encouraging drug development for rare diseases; hence, many drug developers, from start-ups to established pharmaceutical companies, have embarked on promising orphan drug development programmes.

Obtaining orphan status is therefore a key milestone within a development strategy, and its impact should be carefully considered upfront. Early and frequent interactions with regulators help in streamlining the development of OMPs and increasing the chance of accessing the market and ultimately, the patients, provided the data generated meet expectations.

This article describes the regulatory frameworks and incentives associated with orphan drug designation (ODD) in the EU and the US, as well as capturing key strategic considerations needed to achieve successful orphan drug product development and registration in both regions.

Eligibility criteria for EU and US orphan designation.

Voisin Consulting Life Sciences

Cambridge, MA, San Francisco, CA & Somerville, NJ, USA London, UK Paris & Rennes, France Lausanne, Switzerland Bangalore, India

Founded in 1997 by Dr. Emmanuelle M. Voisin, VCLS is a team of over 100 life science professionals located in the US, Europe and Asia.

VCLS supports Biotech, Pharma and Medtech manufacturers to:• Design global product development

strategies• Engage with local regulators & payers

We believe that product development must be driven by a solid understanding of the environment within which the product will be launched, and the criteria by which elements of the development will be assessed by both regulators and payers.

VCLS partners with life science companies across European, North American and international markets.

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Marie Deneux, PhD is senior director regulatory science drugs and biologics at Voisin Consulting Life Sciences. She has been assisting international health companies in the design and implementation of

global product development plans and associated regulatory strategy for more than 10 years. She also has extensive experience in interactions with European health authorities. Learn more about her

Pauline Messina, M.Sc. is regulatory scientist, drugs and biologics at Voisin Consulting Life Sciences. She is involved in different regulatory projects, conducting research and scientific writing in the framework of the

design and implementation of Regulatory Strategies for the development of drugs and biologics.

VoisinConsulting.com

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