Drug Repurposing for Rare

Diseases Conference


We pride ourselves on producing a diverse programme that delivers something for everyone, and ensures our audience has every opportunity to consider drug repurposing from different perspectives. With talks from industry, academia, patient groups, and of course, rare disease patients themselves, Drug Repurposing for Rare Diseases welcomes everyone with a place in the rare disease community.

The 2020 programme is no exception. This year, we are thrilled to highlight a new drug repurposing trial for stone-man syndrome (FOP) run out of Oxford University, delve into a multi-disease clinical trial, and hear the thoughts of NICE on the challenges and opportunities of reimbursement for repurposed drugs.

We also have the return of our annual lightning talk session, giving delegates the chance to take the stage and share their work with our community. 

We hope you will join the conversation in 2020!

Full programme

09:30   Registration

10:00   Welcome

10:10   Drug repurposing; driving research for rare diseases
            – Dr Rick Thompson, CEO, Findacure

10:30   Working together to rapidly advance a clinical trial; a collaboration between two rare disease charities and a biopharma company
            – Dr William Evans, Chair, Niemann Pick UK
            – Daniel Lewi, Co-founder, CATS Foundation
            – Taylor Fields, Senior Vice-President, Intrabio

11:15   Tea break

11:45   Repurposing saracatinib as ALK2 inhibitor to treat Fibrodysplasia Ossificans Progressiva (FOP)
            – Dr Alex Bullock, Principal Investigator, Structural Genomics Consortium, Nuffield Department of Clinical Medicine, Oxford University

12:15   Reimbursement in repurposing; a NICE perspective
              – Sheela Upadhyaya, HST and Topic Selection Specialist, NICE

12:45   Prizes for ‘The Student Voice Prize’ 2019

12:50   Lunch

14:00   Dual Deprivation: understanding the psychological burden that coexists with the physical struggles in individuals with rare diseases
            – Sandy Ayoub, Student Voice 2019 runner up, St George’s University London

14:20   Accelerating treatments through repurposing technology
            – Dr Anthony Hall, Chief Medical Officer, Healx

14:40   Building value in clinical repurposing
            – Barbara Goodman, President & COO, Cures Within Reach 

15:00   Tea break

15:30   Lightning talk session

A patient-centric approach to precision medicine
Fatima Sulaiman, Head of Research & Services, SRUK        

How CROs can add value to the development of drugs in rare diseases
Lisa Dilworth, Vice President Rare, Orphan & Pediatrics, Synteract

The challenge of Behcet’s
Tony Thornburn, Chair, Behcet’s UK

How digital health will accelerate drug discovery
Paul Wicks, Independent Consultant, Wicks Digital Health

The real cost of trimethylaminuria
Karen James, Public Relations Director, MEBO Research

15:50   From despair to hope: a repurposed drug transforms the outlook of women with LAM
              – Kelly Vance, Secretary & Patient Representative, LAM Action

16:20   Panel Session

17:00   Drinks reception in celebration of Rare Disease Day 2020

18:00   End of conference

Our speakers

Dr Alex Bullock

Principal Investigator, SGC


Alex Bullock is an Associate Professor at the Nuffield Department of Medicine, University of Oxford and Principal Investigator at the Structural Genomics Consortium (SGC), an international public-private partnership that aims to catalyse the discovery of new medicines through open access collaborative science.

In his current post, he has gained more than 15 years of experience working on proteins mediating phosphorylation and ubiquitylation. His work has revealed a Jekyll and Hyde behaviour of BMP signalling that drives different cancers and developmental diseases.

He is now working with patient groups to conduct a phase II drug repurposing study in FOP in early 2020.

Alex trained in biochemistry at the University of Cambridge where he completed his PhD with Sir Alan Fersht. He subsequently held a Wellcome Fellowship for positions with David Baker at the University of Washington, Seattle and later with Sir Peter Ratcliffe at the University of Oxford.

Dr Will Evans

Chair, Niemann Pick UK



Will’s eldest son Sam is 10 and has Niemann-Pick type C, an ultra-rare lysosomal storage disorder. He is the chairman of the board of trustees of NP-UK the charity that supports those affected by Niemann-Pick diseases. He has been heavily involved in bringing stakeholders together to advance therapy development for these diseases.

Will is a GP in Leeds and works part time in clinical practice. He is also an academic GP at the University of Nottingham.  His research looks at how we can use large primary data sets to refine how we identify and manage patients with inherited diseases. He is also involved with a health tech company Mendelian, who are using data driven approaches to shorten the diagnostic odyssey for rare disease patients.

Taylor Fields

Senior Vice President, Intrabio


Taylor Fields is the Senior Vice-President of IntraBio Inc. At IntraBio, she leads all efforts related to the development and commercialization of IntraBio’s novel treatment and therapies, and is principally responsible for the company’s clinical and regualtory development programs. Ms. Fields obtained a B.A. in International Studies and English from the University of Michigan, and received a Masters in systems design from the University of Oxford. 

Barbara Goodman

President & COO, Cures Within Reach


Barbara Goodman leads Cures Within Reach (CWR) and its mission of driving
more treatments to more patients more quickly through medical
repurposing research. She joined in 2017 to help expand the reach and
influence of CWR in both the philanthropic and commercial spaces, as well as to foster organizational growth and financial sustainability.

She joined CWR after leading iBIO Institute’s PROPEL Center for 10 years,where she led programs to increase the number and success rate of early-stage life sciences companies in Illinois, and supported iBIO’s international activities. Barbara previously worked in corporate strategy and business development at the Rehabilitation Institute of Chicago, Cardinal Health Medical Products & Services Division, and
Chesapeake Corporation.

Dr Anthony Hall

Chief Medical Officer, Healx


Dr Anthony Hall graduated from King’s College London with first class honours in physiology and pharmacology before going on to study medicine at the Royal Free Hospital. He joined the pharmaceutical industry in 1994 and has significant experience in the clinical and regulatory development of drugs for rare diseases. He speaks regularly at rare diseases conferences, is author of a number of articles on orphan drugs and is a member of the IRDiRC Working Group on Orphan drug-development and regulatory processes.

Daniel Lewi

Founder, CATS Foundation


Dan’s eldest daughter Amelie was diagnosed with Tay-Sachs in 2011 at 15 months of age and upon finding that there was no dedicated charity providing support for families affected by this disease he set set-up the Cure & Action for Tay-Sachs (CATS) Foundation with his wife Patricia.

Since the charity was launched, they have been able to grow The CATS Foundation so that it now offers a variety of services to its family members and has been actively involved in the research investigating therapeutic treatments for Tay-Sachs and Sandhoff disease. Dan is also the Business Development Manager at Aparito who are a technology company transforming healthcare by unlocking real world patient data through mobile applications and wearable devices.

Sheela Upadhyaya

HST and Topic Selection Specialist, NICE 


Sheela Upadhyaya is currently HST and Topic Selection Specialist at NICE. She was previously Associate Director Highly Specialised Technology program, responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS. She has extensive experience in understanding the issues that face companies and other stakeholders when trying to secure access for medicines for the orphan and ultra-orphan conditions. These include developing innovative access arrangements in liaison with industry, clinicians, patients and the NHS.

Sheela has co-authored several papers that discuss HTA methods for assessing value of orphan medicines and presented at many conference issue panels on the subject.   

Prior to joining NICE, she commissioned rare and ultra-orphan disease services in the NHS delivering improvements through collaborating with industry, clinicians and patient groups. During that time, she successfully decommissioned failing services and seamlessly transferred the care of patients to better quality services.

Sheela has a passion for partnership working and believes that collaboration across the sector is the key to delivering high quality outcomes for all. 

Kelly Vance

Trustee & Patient Rep. LAM Action 


Kelly Vance is a lawyer with the international law firm Covington & Burling LLP.  She relocated from New York to England in 2000 to head up the firm’s London U.S. corporate practice and in recent years has focused on business development for the London corporate team. 

Kelly was diagnosed with the rare lung disease Lymphangioleiomyomatosis (LAM) almost 25 years ago.  She has served as a trustee for LAM Action, the UK charity that supports women with LAM and funds research into the disease, since 2007 and served as its Chair from 2012 to 2017.  She also has participated in a LAM research study conducted by the U.S. National Institutes of Health (NIH) since 1998.  In her spare time, Kelly enjoys walking her two rescue dogs with her partner, Kevin, pursuing a nascent career in art and enjoying food and wine with friends. 

Our lightning talk speakers

Lisa Dilworth

Vice President Rare, Synteract


Recently designated the honour of being named to the list of the 100 Most Inspiring People by PharmaVoice, her breadth of experience encompasses more than 20 years of clinical research experience in all phases of drug development. Lisa is a passionate collaborator committed to working directly with patients and their families, physicians, sponsors, and other key stakeholders in the rare and orphan disease space. Lisa has extensive experience in study design, eligibility criteria, feasibility, endpoint selection, operations/project management, and overall project/program strategy. She combines her prior work as a study coordinator and neurophysiology technician with a patient-focused mindset and strong relationships with advocacy groups to ensure that the appropriate strategies are employed to successfully execute trials. Lisa also serves proudly as a committee member at the Myasthenia Gravis Foundation of America. 

Karen James

Public Relations Director, MEBO Research


I have lived with the metabolic malodour disorder known as Trimethylaminuria (TMAU) since 1997, but I was not officially diagnosed until 2010. Disappointed by the length of time it took me to find the reason for my symptoms and devastated by the highly negative impact of the disorder on all aspects of my life, I decided to raise awareness of the condition and to support the work of Maria De La Torre’s MEBO Research patient advocacy organisation. I appeared on ITV’s ‘This Morning’ programme to discuss the impact of living with TMAU, was interviewed for a magazine article and  wrote a patient’s perspective article for the Royal College of General Practitioners’ Innovait journal, which is aimed at introducing medical students to various disorders. I continue to work alongside many other members of the metabolic body and breath odour community to raise awareness and seek research opportunities.

Tony Thornburn

Chair, Behçet’s UK



Tony has been a member of Behçet’s UK (formerly Behçet’s Syndrome Society) since 2006 when his daughter, by chance, was eventually diagnosed with Behçet’s.  Immensely privileged to have been appointed Chair to such a supportive organisation, he set the aim to assist in building on all the hard work that has gone before to promote routine awareness of Behçet’s and achieve better coherence between patients and research, and all that should be in place to support them: as he puts it “a true systems challenge”. Tony served in the Armed Forces for 35 years, benefiting from a wide variety of appointments, before becoming a Lecturer in Systems Engineering at Cranfield University, where he remains a Visiting Fellow.  Presently supporting the STEM initiative in schools, the British Standards Institute, a Systems Thinking APM Special Interest Group and a Human Factors Integration Working Group.  He is a Fellow of both the Chartered Management Institute and the Institute of Leadership and Management.

Fatima Sulaiman

Head of Research and Services, SRUK


Biography coming soon! 

Paul Wicks

Independent Consultant, Wicks Digital Health


Paul Wicks, Ph.D., is a neuropsychologist and independent consultant in digital health, clinical trials, rare disease, and patient centricity. For 13 years he led the R&D team at PatientsLikeMe, an online community for people over 700,000 people living with medical conditions.

Specialising in clinical research using the Internet, Paul shaped the scientific validity of the platform in generating insights from patient-generated health data, leading to over 110 studies including a patient-driven observational trial of lithium in ALS/MND, numerous patient-reported outcome measures, a “dose-response” curve for the benefits of friendship between patients, and methods for patient-centered and virtual clinical trial designs.

He sits on the editorial boards of the BMJ, BMC Medicine, JMIR, Digital Biomarkers and The Patient. Prior to joining PatientsLikeMe, Paul worked at the Institute of Psychiatry (King’s College London) studying cognition and neuroimaging in rare forms of inherited ALS/MND.

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