Accelerating development, registration and access to medicines for rare diseases

in the European Union through customised approaches: features and perspectives

David Uguen, Thomas Lönngren, Yann Le Cam†, Sarah Garner‡, Emmanuelle Voisin, Carlo Incerti±, Marc Dunoyer° and Moncef Slaou˧

Published in Orphanet, May 2015

independent strategic advisor to NDA Advisory Services Ltd through his company Pharma Executive Consulting; †Chief Executive Officer and co-founder of EURODIS; ‡Associate Director for Research and Development at the National Institute for Health and Care Excellence (NICE) and project Director for Adaptive Licensing at the UK Centre for the Advancement of Sustainable Medical Innovation (CASMI); ±Genzyme Global Medical Research and Development leader; °Global Head of GSK’s Rare Diseases Unit; ˧Chairman of Global Research and Development (R&D) and Vaccines, R&D Management, at GSK.

CONTENTS

01 Introduction 3

02 Features, rationale and potential impact of novel licensing mechanisms 4

03 Voisin Consulting Life Sciences 5

04 Additional information 6

Rare Diseases: a collective effort and a customized approach

Timely patient access to novel treatments for rare diseases is based on collective engagement from:- Regulatory authorities, - Patient associations, - Pharmaceutical companies, - Legislators,- Payers

Approach• Adapting clinical data collection and development to the product/condition • Post-authorisation data collection • Ensuring access to market, not only marketing authorisation• Access to treatments should be cost-effective

How to integrate knowledge, evidence and experience in order to meet rare disease needs

Features, rationale and potential impact of adaptive licensing initiatives

EMA Road map to 2015: - how processes under existing regulatory legislation can be adapted to support innovation for rare diseases- Rationale for novel approaches to registration and launch:

- to allow certain new therapies to be marketed on basis of early positive risk:benefit ratio in a small group of patients

- to later confirm and broaden this appraisal with additional clinical data obtained post approval

Patients’perspective

- Support moves to allow faster access to safe and efficacious medicines

- Highlight the need for a pan-European approach, before and after marketing authorization, to ensure access is not fragmented at national level

Regulator’s perspective

- Role of regulators changes from ‘gate-keeper’ to enabler.

- Open dialogue with industry, experts, payers, patients and more

- Existing regulatory mechanisms currently evolving to respond to new needs

Policy-makers’perspective

- Therapeutic innovation not always successfully translated into patient benefits

- Flexible and innovative data collection- Timeframes for post-approval obligations to

be collaboratively explored upfront

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 development will be assessed by both regulators & payers

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

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Dr. Emmanuelle M. Voisin, CEO, created Voisin Consulting Life Sciences to support biomedical companies with fully integrated drug development programs

Learn more about her

VoisinConsulting.com

For more information