SMi’s 9th Annual Orphan Drugs and Rare Diseases Conference

Start Date : October 15, 2019
End Date : October 16, 2019
Time : 8:30 am to4:10 pm

Phone : 02078276000
Email :

Location :
Holiday Inn London - Kensington Forum, 97 Cromwell Road, London, SW7 4DN, United Kingdom


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SMi Group are proud to announce the 9th Annual Orphan Drugs and Rare Diseases Conference, taking place on the 15th and 16th October 2019 in London, UK. 

The orphan drug market is continuously growing and is expected 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). By 2020, orphan drugs are set to account for 19% of global prescription sales. 

This is due to the arising interest from pharmaceutical companies for underlying benefits, such as the potential discovery of mechanisms for more common diseases, and also the involvement of many smaller biotechnology start-up companies who are primarily focused on rare diseases.


The 2019 agenda features various key presentations by leading solution providers, biotechnology companies, clinical researchers, regulatory professionals and charity leaders discussing possible ways to accelerate 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.



•HEAR about the success of integrated approaches to develop a robust early access and evidence generation plan with Bionical Emas

•DISCUSS the intervention of pharmaceutical companies and patient advocates in developing lifelong rare disease treatments with Takeda

•EXPLORE the current development of strategies aimed to improve patient access to orphan drugs with Genetic Alliance UK

•GAIN INSIGHT into the various challenges facing orphan drug development with Minoryx Therapeutics



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


-Regulatory Affairs

-Rare Disease Charity Officers






Speakers: CHAIR: Mike Page, Executive Director Global Regulatory Affairs Portfolio Products, Alexion Pharmaceuticals, Tom Watson, Executive VP Early Access Programs, Bionical Emas, Sheela Upadhyaya, HST Associate Director, NICE (National Institute for Healthcare and Excellence), Rick Thompson, CEO, Findacure, Katrin Radl, Senior Manager Evidence Generation and Patient Access, Takeda, Kelly Du Plessis, CEO and Lead Patient Advocate, Rare Diseases South Africa, Maria Pascual, Chief Regulatory Officer, Minoryx, Martina Garau, Associate Director, Office of Health Economics, Mike Page, Executive Director Global Regulatory Affairs Portfolio Products, Alexion Pharmaceuticals, Jayne Spink, CEO, Genetic Alliance UK, Dan Donovan, CEO and Co-founder, Rare Life Solutions, Donatello Crocetta, Vice President Global Head Rare Immunology Franchise, Takeda, David Rose, Head of Business Development, Rare Revolution Magazine, Sara Carlot, Medical Advisor Rare Disease Unit Head, Chiesi Farmaceutici, Steve Smith, Head of Rare Disease and Cell and Gene Therapy, Next Phase Recruitment, Krysztof Potempa, CEO, BRAINCURES, Eddie Pease, Chief Technology Officer, PharmaForesight

Registration Info






Conference and Workshop: GBP 2098.0

Conference Only: GBP 1499.0

Workshop Only: GBP 599.0

Promotional Literature Distribution: GBP 999.0

Contact the Organizer

Organized by

Organized by LoveEvvnt1

SMi Group

Tel: 02078276000

Event Categories: Internal Medicine.

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