Orphan Drugs and Rare Diseases 2019 Conference

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The market for rare disease products  is continuously  growing, expecting to reach $176 billion by 2020, with a CGR of 10.5% just for orphan drugs - this is twice the growth rate of the overall prescription drug market (5.3% CGR from 2014-2020). Explanations for this include the arising interest for pharmaceutical companies, primarily  for  underlying benefits, including the potential discovery of mechanisms for more common diseases, and the involvement of many smaller biotechnology start up companies who are more focused on rare diseases.

Following this, we are proud to announce our 9th Annual Orphan Drugs and Rare Diseases conference, aiming  to bring together solution providers, biotechnology companies, clinical researchers, regulatory professionals and charity leaders together to discuss possible ways to accelerative orphan drug development and access to rare disease patients, including the introduction of recent technologies and products to help aid the access of  orphan drugs.

We look forward to welcoming you to the conference this October.

Early registration is strongly advised to avoid disappointment.

Benefits of Attending

  • HEAR about the success of orphan drug launches following the heavy involvement of patient advocacy programs
  • DISCUSS the potential changes in assessing the development of medicines across Europe
  • EXPLORE the future landscape orphan drugs following the market access and reimbursement challenges providers are currently facing.

Who should attend

CEOs, CSOs, Vice Presidents, Directors, Heads, Principals working in:
  • Medical Affairs
  • Medical Advisory
  • Clinical Operations
  • Rare Disease Research
  • Patient Advocacy
  • Commercial Officer
  • Orphan Drug Therapeutics
  • Healthcare
  • Regulatory  Affairs
  • Rare Disease Charity Officers

Plus an Interactive Half Day Post-Conference Workshops | Thursday 17th October 2019

A deep dive into strategies for accelerating patient access of orphan drugs
Workshop Leaders: Solange Corriol-Rohou, Senior Director Regulatory Affairs & Policy Europe, AstraZeneca
Cecile Ollivier, Chief Operating Officer, Aparito
08.30 - 12.30


  • Annamaria Merico, Head of Technology Transfer, Fondazione Telethon
  • Dan Donovan, Co-Founder & CEO, Clear Pharma
  • David Rose, Business Development, Rare Revolution Magazine
  • Donatello Crocetta, VP, Global Head of Rare Immunology Franchise, Takeda
  • Eddie Pease, CTO, PharmaForesight.ai
  • Jayne Spink, Chief Executive Officer , Genetic Alliance UK
  • Katrin Jack, Sr. Manager Patient Access & Evidence Generation, Takeda Pharma AG
  • Krzysztof Potempa, Founder and Director, BRAINCURES
  • Maria Pascual, Regulatory Affairs, Minoryx Therapeutics S.L.
  • Martina Garau, Associate Director, Office Of Health Economics
  • Michael Page, Executive Director, Portfolio Products, Alexion Pharmaceuticals Inc
  • Rick Thompson, CEO, Findacure
  • Sara Carlot, Global Medical Advisor - Rare Disease, Chiesi Farmaceutici S. p. A.
  • Sheela Upadhyaya, Associate Director - Highly Specialised Technologies , NICE - National Institute for Health and Care Excellence
  • Tom Watson, Executive Vice President, Bionical Emas

Please fill in your name and email to receive the Conference Agenda of this event.


Holiday Inn Kensington Forum
97 Cromwell Rd, Kensington, SW7 4DN
London, UK