The rare disease biopharmaceutical landscape continues to evolve, but despite the advances made, only five percent of an estimated 7,000 rare diseases have an available treatment option. As life sciences companies begin to address the significant unmet need in the rare disease space, clinical-stage companies developing treatments for rare diseases face a number of challenges as they aim to bring products to market, particularly when on the global scale.
This webinar will explore PTC’s journey from an R&D-only focus to a fully integrated, global commercial company as they launched Translarna™ the first disease-modifying treatment for Duchenne Muscular Dystrophy in the European market. The speakers will discuss the challenges of entering a new disease area for the first time, the balance of achieving the right size global organization to meet the customer population and the considerations for proceeding independently with a global launch rather than partnering.
Mark Rothera, Chief Commercial Officer, PTC THERAPEUTICS
Mark Rothera is responsible for establishing the global strategy and operational plan to commercialize PTC’s novel therapeutics for the treatment of a range of devastating rare diseases. Over the last 4.5 years, Mark has transformed PTC from an R&D company, in New Jersey, to a global commercial stage company covering almost 50 countries on the back of launching the first therapy approved for Duchenne Muscular Dystrophy, Translarna.
Mark has spent 28 years in the biopharmaceutical sector, of which the last 19 have been spent pre-launching and launching orphan drugs internationally for rare diseases such as Duchenne Muscular Dystrophy, Cystic Fibrosis, Hunter Syndrome, Hereditary Angioedema and Gaucher Disease. Mark has built international operations from the very earliest stages at PathoGeneis, Chiron BioPharmacetucals, Shire Human Genetic Therapies and most recently PTC Therapeutics.
A patient-centered approach has motivated Mark and his teams to enable patients to gain access to life-saving therapy as early as possible through existing and innovative early access mechanisms. Mark has engaged with the investor community as part of PTC’s IPO team and through multiple financing events. He has been a board member of the UK Bio Industry Association, has a Masters in Natural Science from Cambridge University, an MBA from INSEAD and a Diploma of Company Direction from the Institute of Directors, UK.Message Presenter
Ned Kitfield, Vice President, Charles River Associates
Ned Kitfield has extensive experience providing commercial decision support for large and small biotech, pharmaceutical, and healthcare companies. While Ned has worked across a broad spectrum of therapeutic categories, he brings particular insight into rare and ultra-orphan diseases. His expertise includes everything from commercialization strategy to advanced analytics and primary market research designed for these often overlooked rare spaces.
Prior to joining CRA in 2017, Ned acted as one of three Managing Directors at C1 Consulting, a life sciences decision support firm, where he headed up the Rare Disease Practice for 3 years. Ned also held a variety of senior level sales and commercial roles at Shire, Sanofi, Bayer and Adolor where he was responsible for domestic and global commercial operation for a variety of specialty and orphan products. Ned received his MBA from the University of Massachusetts and his BA from McGill University.Message Presenter
Who Should Attend?
Senior professionals from pharmaceutical and biotechnology companies involved in the following areas within Rare Disease Therapies:
- Clinical Operations
- Market Access
- Regulatory Affairs
- Research & Development
Charles River Associates
Charles River Associates is a leading global consulting firm that offers economic, financial, and strategic expertise to major law firms, corporations, accounting firms, and governments around the world.
With proven skills in complex cases and exceptional strength in analytics, CRA consultants have provided astute guidance to clients in thousands of successful engagements. We offer litigation and regulatory support, business strategy and planning, market and demand forecasting, policy analysis, and risk management consulting.
Our success stems from the outstanding capabilities of our consultants, many of whom are recognized as experts in their respective fields; our close relationships with a select group of respected academic and industry experts; and from a corporate philosophy that stresses interdisciplinary collaboration and responsive service.
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