Orphan medicines launch excellence: sustaining launch success

Overview

Launching orphan medicines excellently will become even more important over the next five years as more patients with rare diseases have pharmacotherapies available.

There are also a growing number of disease-focussed registries, increasing public and policy maker awareness, and significant R&D investment in orphan drugs, as well as in digital technologies to support trials and treatment.

But a new frontier of challenge faces orphan medicines companies as the gap between orphan medicines and mainstream specialty products narrows.

To understand how to succeed in launching an orphan medicine in the coming years, companies must learn from past launches and apply an orphan medicines-focused launch excellence framework for success.

Focus

This internationally relevant webinar provided US and European examples to illustrate the ways in which the pharmaceutical market for orphan medicines is changing and how, consequently, launch strategies must evolve.

This pharmaphorum debate, held in partnership with IQVIA, took place on Thursday 13^th June from 15:00-16:00 BST and looked at how pharmaceutical companies can optimise all the activities needed to take orphan medicines from clinical development to commercialisation, including:

• Identifying the three fundamental challenges for orphan medicine launch excellence
• Discussing the ways in which companies launching orphan medicines can learn from excellence in mainstream launches
• Examining, in-depth, the specific areas of focus which are crucial for orphan medicines excellence.

To view the debate on demand, please click here or on the button above or below.

Our Panel

Angela McFarlane, Market Development Director, IQVIA

Angela is Market Development Director for IQVIA, a human data sciences company formed through the 2016 merger of IMS Health and Quintiles. She works with Government, the NHS, the Pharmaceutical Industry and Patient Organisations on pioneering collaborations that will improve NHS patient access to medicines. She has over ten years’ experience in the commercialisation of rare disease medicines. Angela’s passion is working on initiatives that ensure grows post-Brexit as a priority country for inward investment in clinical research, real world evidence studies and new medicines launches. Angela founded and sold her own Market Access company to IMS Health in 2013; prior to this she was marketing director of a managed care hospital in Manchester, preceded by working for BMS and MSD in marketing.  In 2018, she was nominated by Pharmaceutical Marketing Europe as one of the 30 most influential women in UK Healthcare.

Peter Meeus, Commercial Excellence Executive, European Orphan Medicines

Up until recently, Peter Meeus was the Head of Region Europe at Shire plc, the global leader in Rare Diseases, acquired by Takeda in January 2019.  Peter has over 25 years of pharma experience, principally at Novo Nordisk, Novartis and Shire.  This experience covers local, regional and global responsibilities in many disease areas ranging from primary and specialty care to rare diseases.

In his latest position as Head of Region Europe, based at the International Operational Headquarters in Zug, Switzerland, Peter had responsibility for all operations in Europe and was a member of the International Leadership Team.  He was also Shire’s representative on both the European Markets Committee and Patient Access Committee of the European Federation of Pharmaceutical Industries and Associations (EFPIA).

Peter is 49, a Belgian national and is married with two children. Originally a graduate in Applied Economic Sciences from The Limburg University Centre, Belgium, Peter went on to gain a Post Graduate distinction in International and Industrial Marketing Management. Additionally he undertook an Executive MBA programme at the Scandinavian International Management Institute in Copenhagen, Denmark between 2000 and 2002.

Sarah Rickwood, Vice President, European Thought Leadership, IQVIA

Sarah Rickwood has 26 years’ experience as a consultant to the pharmaceutical industry, having worked in Accenture’s pharmaceutical strategy practice prior to joining IQVIA. She has an extremely wide experience of international pharmaceutical industry issues, having worked in most of the world’s leading pharmaceutical companies on issues in the US, Europe, Japan, and leading emerging markets, and is now Vice President, European Thought Leadership in IQVIA, a team she has run for 8 years. Sarah presents to hundreds of pharmaceutical industry clients every year on a wide range of global pharmaceutical industry issues, and has published white papers on many topics, including:

• Uptake and impact of innovative medicines, and challenges for Launch Excellence
• The relative strength and prognosis for the developed and the emerging pharmaceutical markets
• Orphan drugs launch challenges
• Cell and Gene therapies commercialisation challenges and opportunities

Sarah holds a degree in biochemistry from Oxford University.

Jeffrey Keefer, Head, Pediatric and Rare Disease Center of Excellence, IQVIA

Jeffrey Keefer MD, PhD is a board-certified pediatric hematologist/oncologist with 12 years of academic faculty experience at the Johns Hopkins University School of Medicine in the Division of Pediatric Hematology where he was in clinical practice, led basic and clinical research programs and was the co-director of the joint NIH/Johns Hopkins Pediatric Hematology/Oncology Fellowship program. In addition to caring for patients with sickle cell disease and rare bleeding disorders, he practiced general pediatric hematology and attended on the general pediatric ward service.

For the past four years at IQVIA, Jeff has worked in the Therapeutic Science and Strategy Unit as a medical strategy lead. Two years ago, he also assumed the leadership of the Pediatric and Rare Disease Center of Excellence where he heads a team that supports clinical trials and other initiatives in pediatrics and rare disease throughout the organisation.

Jeff received his BA in Biology from the University of Virginia and his MD and PhD degrees from Vanderbilt University School of Medicine where his graduate work was in Pharmacology. He completed his residency in Pediatrics and served as Chief Resident at the Johns Hopkins Hospital. He continued there for Fellowship in Pediatric Hematology and Oncology before joining the faculty.

Maximilian Newton, Consultant, European Thought Leadership, IQVIA

Max is a consultant within IQVIA's European Thought Leadership team, advising on major upcoming trends within the pharmaceutical industry. He has previous experience at GSK Global Manufacturing & Supply and four years experience in pharmaceutical strategy. His areas of expertise are rare disease and orphan medicines, European HTA processes, speciality care and biosimilars, funding route viability and early access options. He project managed Roche Pharmaceuticals first UK evidence-driven rare disease whitepaper ins 2017-18 and published analyses on NICE HST.

Moderated by Paul Tunnah, CEO, pharmaphorum

Paul Tunnah is CEO and Founder of pharmaphorum media, which facilitates productive engagement for pharma, bringing healthcare together to drive medical innovation. It combines industry-leading content and social media engagement services with the globally recognised news, information and insight portal pharmaphorum.com, working with pharmaceutical companies, service providers and broader healthcare organisations to help communicate their thought leadership and connect them with relevant stakeholders.