Orphan Drugs and Rare Diseases

19th Orphan Drugs and Rare Diseases Global Congress 2023 Europe

Collaborations to Accelerate Rare Disease Drug Development and Access

Hilton London Kensington, London, UK

Monday 3rd - Tuesday 4th April 2023

KEY INDUSTRY EXPERT SPEAKERS

David Pearce, President of Innovation, Research & World Clinic, Sanford Health
Kristin Smedley, CEO, Curing Retinal Blindness Foundation
Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck
Graham Sidorowicz, EVP Commercial, Bionical Emas
Naomi Litchfield, Director, Patient Advocacy, Bionical Emas
Robert Palmer, Head of Value & Access Oncology, APAC, MEA, Novartis
Manolo Bellotto, CSO & General Manager, Gain Therapeutics
Sheela Upadhyaya, Chair Elect, Rare Disease Special Interest Group, ISPOR
Lara Bloom, President and CEO, The Ehlers-Danlos Society
Josie Godfrey, Director, JG Zebra Consulting, Joint CEO, Realise Advocacy
Robert Mitchell-Thain, CEO, PBC Foundation
Rüdiger Schulze, VP & GM, Germany & Central/Eastern Europe, Ultragenyx
Florence Barkats, Chief Commercial Officer, CISIV Ltd
Nicola Gillott, Head of Expanded Access, Amryt Pharma
Hannah Stark, Operations Lead, NIHR BioResource
Hannah Harvey, Senior Projects Officer, Beacon for Rare Diseases
Christian Girard, Co-Founder, OrphaPedia
Einar Andreassen, Director of HTA and Reimbursement, Norwegian Medicines Agency
Nick Sireau, Chair & CEO, AKU Society
Lisa Bashorum, Head, Int'l. Patient & Professional Advocacy, Amicus Therapeutics
Dr Peter Lanyon, Rare Diseases Clinical Lead, National Disease Registration Service, NHS England
Pr Daniel Scherman, Head of Medicine, Life Sciences Division, European Academy of Sciences EURASC
Bob Stevens, Group CEO, MPS Society
Anthony Hall, Chief Medical Officer, Healx

David Pearce

President of Innovation, Research, & World Clinic

Sanford Health

David Pearce

President of Innovation, Research, & World Clinic

David Pearce is President of Innovation, Research, & World Clinic for Sanford Health. He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986. He gained his PhD in 1990 at the University of Bath, UK, and did postdoctoral training at the University of Rochester, U.S., and Oxford University, UK. Dr. Pearce heads the leading lab in Juvenile Batten disease research. He has been researching Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease) since 1997. His research has led to the first clinical trial for Juvenile Batten disease. He has published over 100 research papers on Batten disease. He also oversees a national registry for rare diseases known as the Coordination of Rare Diseases at Sanford (CoRDS). He has served on numerous NIH review committees, has organized rare disease workshops for the National Institute for Neurological Disorders and Stroke (NINDS) arm of the National Institutes of Health (NIH) and is currently the chair of the consortium assembly for the International Rare Diseases Research Consortium (IRDiRC). As President of Innovation, Research, & World Clinic at Sanford, he oversees the development of research programs, including more than 450 researchers, eight research centers and more than 300 ongoing clinical trials.

Eric Crombez, M.D.

Chief Medical Officer, Gene Therapy and Inborn Errors of Metabolism

Ultragenyx

Eric Crombez, M.D.

Chief Medical Officer, Gene Therapy and Inborn Errors of Metabolism

Eric Crombez has extensive experience and expertise in the development and execution of clinical development programs for rare genetic disorders. Dr. Crombez currently serves as the Chief Medical Officer for Ultragenyx Gene Therapy and Inborn Errors of Metabolism and has been in this role since the acquisition of Dimension Therapeutics in November 2017. Dr. Crombez joined Dimension Therapeutics, a liver directed AAV gene therapy company, as Chief Medical Officer in November 2014. During this time, Dr. Crombez led the clinical development efforts for 4 clinical stage gene therapy programs including for hemophilia B, hemophilia A (in partnership with Bayer), ornithine transcarbamylase (OTC) deficiency and glycogen storage disorder type Ia (GSDIa). Dr. Crombez is also serving as an Industry Representative on the FDA Cellular, Tissue, and Gene Therapies Advisory Committee. Prior to this work in gene therapy, Dr. Crombez worked at Shire in their Human Genetics Therapy business unit for nearly 8 years. Before joining industry, Dr. Crombez was Assistant Professor, Department of Pediatrics, Division of Medical Genetics at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA). Dr. Crombez is a board-certified clinical geneticist and completed residencies in pediatrics and medical genetics and a fellowship in clinical biochemical genetics at the UCLA School of Medicine. Dr. Crombez has authored more than 40 peer-reviewed articles, book chapter and presentation abstracts. Dr. Crombez obtained his B.S. degree in biology from the University of Michigan, Ann Arbor, and his M.D. degree from Wayne State University School of Medicine, Detroit.

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP)

Merck

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP)

Pedro Franco holds a degree in Pharmaceutical Science (PharmD) from the University of Lisbon, a Master in Organic Chemistry (M.Sc) from the New University of Lisbon with collaboration with Imperial College in London and a PhD in Orphan drugs and Rare diseases from the University of Sheffield. Pedro Franco completed also his music studies (Cello) in Lisbon. Currently, Pedro is working as a Director for Global Regulatory & Scientific Policy (GRASP) at Merck and he is mainly focus on regulatory intelligence, strategy and policy. In addition, he is the liaison for the China and Latam region concerning regulatory and policy topics. Previously, He worked at the European Medicines Agency for 12 years in different roles and departments mainly focusing on regulatory affairs, international collaboration, assessment, authorisation of medicines, orphan drugs, paediatrics, peer review, quality and strategy. Beforehand, he was working at INFARMED (the Portuguese national competent authority) as a pharmaceutical assessor. Before joining regulatory competent authorities, he worked as a post graduate research fellow in the area of toxicology, carcinogenic chemistry and drug development.

Dr. Manolo Bellotto

CSO & General Manager

Gain Therapeutics

Dr. Manolo Bellotto

CSO & General Manager

Dr. Manolo Bellotto has served as Chief Strategy Officer and General Manager of the company and its subsidiary GT Gain Therapeutics SA since 2018. Dr. Bellotto has over 20 years of experience international pharmaceutical product, medical and advocacy development, commercialization, and international research collaborations in major and rare/orphan disease indications across various continents. Since 2013, Dr. Bellotto has served as director of strategic projects at Mederis, a service provider for pharmaceutical, medical device and medical food companies providing assessment, prioritization, planning and management of product development, medical and market-shaping activities. He developed his career as the director of business development at Aptitude Health, a global healthcare company focusing on oncology insights for the life sciences industry. Dr. Bellotto has also previously served as global product leader at Vifor Pharma, a global specialty pharmaceutical company. Dr. Bellotto has held various positions, such as head of international product management and head of marketing, at Helsinn Healthcare, DKSH Healthcare, ESMO – European Society for Medical Oncology, and Chemo SA. Dr. Bellotto received a Ph.D. in Molecular Developmental Genetics from the University of Zurich after having studied molecular biology at the Biocentre of the University of Basel and at the Fredrich Miescher Institute for Biomedical Research in Basel.

Kristin Smedley

CEO

Curing Retinal Blindness Foundation

Kristin Smedley

CEO

In 2011, Kristin launched a nonprofit to fund research and resources for children living with the blindness her two sons are affected by, CRB1 LCA/RP. The Curing Retinal Blindness Foundation has raised over 1.4 million dollars and achieved a National Rare Eye Disease Awareness Day. That legislation, H.R. #625, was the first in US history to be submitted in Braille and it advocates for better resources for blind and visually impaired Americans. Kristin partnered with Spark Therapeutics to help achieve the first ever FDA approved gene therapy to treat an inherited retinal disease in the United States. She has done a TEDx Talk in New York City to change perceptions of blindness and she partnered with Comcast media to spread awareness of the inclusive X1 product. In 2019, Kristin published her first book called Thriving Blind: Stories of Real People Succeeding Without Sight. It achieved #1 New Release on Amazon for paperback and kindle. Kristin partnered with TMobile and the National Braille Press to make the book available in electronic Braille. Kristin received one of the highest honors in the rare disease community, the Champion of Hope Award, from Global Genes and was appointed the Pennsylvania Ambassador for the National Organization of Rare Disorders (NORD). During the COVID19 pandemic Kristin co-founded an online show/podcast called Brilliantly Resilient which helps people Reset, Rise and Reveal their Brilliance during life’s challenges. Her book by the same title debuted on the Amazon Top 100 Best Seller List, right next to Deepak Chopra. In 2021 Kristin co-founded ThrivingblindAcademy.org to solve the employment, literacy, and financial crisis in the blind community. She is Co-Executive Producer of the short film, The Great Equalizer, (Feb. 2023) a partnership with Blind Institute of Technology, Insight Global and Salesforce to address the unemployment crisis of the blind.

FLORENCE BARKATS

Chief Commercial Officer

CISIV Ltd

FLORENCE BARKATS

Chief Commercial Officer

Florence Barkats is Chief Commercial Officer at CISIV Ltd and has 20 years of commercial and operational leadership experience. She has developed expertise in data strategies to bridge the gap between late-phase research and early-stage commercialisation. From her work at J&J and GE Healthcare, she has experience in growing digital health ecosystems for market access and increasing patient-led engagement. Florence is a Patient Advocate on an NHS Improvement taskforce aimed at increasing equality of access to care. She holds a degree in Molecular biology from Johns Hopkins University and an MBA from London Business School.

Lara Bloom

President and CEO

The Ehlers-Danlos Society

Lara Bloom

President and CEO

Lara Bloom is the President and CEO of The Ehlers-Danlos Society and responsible for globally raising awareness of rare, chronic, and invisible diseases, specializing in the Ehlers-Danlos syndromes, hypermobility spectrum disorders (HSD), and related conditions. Lara manages coordinated medical collaboration, raising funds for research, and focusing on the global progression of EDS and HSD. She speaks at conferences all over the world, lecturing to medical students and professionals, and supports specialists in the field by offering her experience as a leading Patient Expert. Commemorating ten years in the field of patient advocacy, Lara was officially appointed a Professor of Practice in Patient Engagement and Global Collaboration at Penn State College of Medicine, USA, on March 11, 2020.

HANNAH STARK

Operations Lead

NIHR BioResource

HANNAH STARK

Operations Lead

Hannah Stark leads the Operations team at the NIHR BioResource, which is responsible for the coordination of volunteer recruitment across all NIHR BioResource programmes, including rare diseases. The team support recruitment sites and BioResource Centres recruiting patients and those without health conditions into the BioResource and answers questions from members of the public and volunteers. The team also supports academic and industry researchers in inviting BioResource volunteers to their studies and ensuring they run to time and target.

RÜDIGER SCHULZE

VP & GM, Germany & Central/Eastern Europe

Ultragenyx

RÜDIGER SCHULZE

VP & GM, Germany & Central/Eastern Europe

Coming Soon.

HANNAH HARVEY

Senior Projects Officer

Beacon for Rare Diseases

HANNAH HARVEY

Senior Projects Officer

Hannah is a Senior Projects Officer at Beacon for Rare Diseases. She plays a leading role in coordinating and delivering parts of Beacon’s patient group training programme. This includes running the Patient Group Mentoring Programme, organising and hosting in-person workshops and delivering webinars and masterclasses. She also supports The Head of Operations in delivering community building events including showcases and conferences. Hannah has worked directly with over 40 patient groups and strives to keep patients at the centre of her work. She wants to see a world where all rare diseases have treatments developed for patients with patients.

ROBERT MITCHELL-THAIN

CEO

PBC Foundation

ROBERT MITCHELL-THAIN

CEO

Robert Mitchell-Thain is a patient advocate of 18 years, with experience in a number of roles, including currently Patient Insights Director at the Global Liver Institute, Vice-Chair of Liver Patients International, the Patient Voice in NHS England’s Hepatobiliary and Pancreas CCG, and was recently leading patient-centred solutions as Head of Education and Development at PBC Foundation. Robert has experience in taking the patient experience and incorporating it into clinical guidelines, abstracts, and journal papers, as well as into every day practice. He has presented at EASL, AASLD and APASL liver-specific conferences. He has also presented to and on behalf of patients in USA, Canada, Israel, China, Japan, Israel, and all over Europe. Robert has led a PBC-specific interactive App which can record GDPR-compliant anonymised data, allowing the patient experience to directly influence industry partners and help them to address unmet need. Using the app data, he presented in poster and orally at a number of national and international scientific conferences. He is currently working with GLI to design and release an interactive patient-driven App that will cover multiple liver diseases, giving patients equitable access to information and the opportunity to be part of the solution in their particular liver disease. Rüdiger Schulze, VP & GM, Germany & Central/Eastern Europe, Ultragenyx

Christian Girard

Co-Founder

OrphaPedia

Christian Girard

Co-Founder

Christian Girard, MiM, is a strategy consultant with a focus on value creation in the orphan drug area. A graduate of ESCP-Europe, a leading European business school, his entry in the life science industry dates back to 1994 when he joined the newly established French subsidiary of a US Medtech manufacturer, which was acquired one year later by Genzyme. He worked with Genzyme until 2002 in various sales and marketing roles, including Head of Carticel French operations (a first-generation autologous cartilage defects cell therapy). In 2002, he launched a consulting practice, which specialized in the orphan diseases arena. Leveraging his experience with Genzyme, he works with numerous European and US companies which develop and market orphan drugs. He has advised research institutions, tech transfer offices, patients organizations, start-up companies as well as mature biotechs and industry leaders, and Venture Capital funds. As a « hands-on » consultant, he is involved in different projects such as starting companies and subsidiaries, dilutive and non-dilutive funding campaigns, monetizing assets, spin-offs, In/Out-licensing R&D programs, portfolio prioritization, investment opportunities assessments, regulatory strategies, orphan drugs designations, and Early Access Programs.

Sheela Upadhyaya

Chair Elect, Rare Disease Special Interest Group

ISPOR

Sheela Upadhyaya

Chair Elect, Rare Disease Special Interest Group

Sheela Upadhyaya is a consultant to the life sciences industry and in her career has played several roles in the rare disease space. She has been in healthcare for over 25 years with the last 15 in the rare disease space having led the NICE 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 along with being their Rare Disease and COVID 19 strategic adviser. She now consults with the life sciences industry and is currently chair for Together for Rare Diseases, an initiative to support collaboration with European Reference Networks and Industry to improve the landscape for research in rare diseases. Sheela has extensive experience in understanding the issues that face the healthcare ecosystem when trying to secure access for medicines for 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 the value of orphan medicines and presented at many conference issue panels on the subject. Sheela also provides advice to the European Haemophilia Consortium Think Tank, is Chair for the ISPOR Rare Disease special interest group and Trustee of the My Name’5 Doddie Foundation. Sheela has a passion for partnership working and believes that collaboration across the sector is the key to delivering high quality outcomes for all.

Nick Sireau PhD

Chair and CEO

AKU Society

Nick Sireau PhD

Chair and CEO

Nick Sireau, PhD, is the CEO and Chair of Trustees at the AKU Society, a patient group that helps people with AKU, a rare genetic disease affecting both his children. The AKU Society and Nick are the winners of the 2021 Members Award by EURORDIS (the European Organisation of Rare Diseases) because of their work on successfully developing a new treatment for AKU. Nick is co-founder and Chair of Beacon, an organisation that helps all rare disease patient groups. He is the editor of ‘Rare Diseases: Challenges and Opportunities for Social Entrepreneurs’ (Greenleaf 2013) and of the 'Patient Group Handbook: A Practical Guide for Research and Drug Development' (Beacon 2016). Nick is co-founder and Chair of Orchard OCD, a medical charity that funds research into obsessive-compulsive disorder (OCD), a common yet debilitating mental health condition. He is also co-founder of Sirgartan Therapeutics, a biotech that focuses on new treatments for OCD. Nick has a BA from Oxford University, an MSc in management studies from the Lyon Graduate School of Business and a PhD in social psychology from City University. He is a fellow of the Ashoka Fellowship of Social Entrepreneurs.

Josie Godfrey

Co-Founder and CEO

Realise Advocacy, JG Zebra Consulting

Josie Godfrey

Co-Founder and CEO

Josie is the Director of JG Zebra Consulting which provides independent consultancy services with a focus on rare diseases and innovative technologies. She is Co-founder and CEO of Realise Advocacy which supports patient advocacy groups to maximise their impact in HTA and access. Her current projects include providing strategic market access, policy and stakeholder engagement advice, and support to pharmaceutical companies, trade bodies, and patient advocacy groups. She is the Strategic Director for Duchenne UK’s Project HERCULES, an award-winning international multi-stakeholder collaboration developing evidence and tools to support Health Technology Assessments (HTAs) for new treatments for Duchenne Muscular Dystrophy. Josie has extensive experience in rare diseases and led the establishment of the Highly Specialised Technologies programme at NICE, the National Institute for Health and Care Excellence in the UK which carries out Health Technology Assessments of treatments for very rare diseases.

Pr Daniel Scherman

Head of Medicine, Life Sciences Division

European Academy of Sciences EURASC

Pr Daniel Scherman

Head of Medicine, Life Sciences Division

- Director of the Foundation for Rare Diseases (FFRD) - Head of Medicine and Life Sciences - - Division of the of European Academy of Sciences EURASC - Corresponding Member of the French Académie Nationale de Pharmacie - Exceptional Class Director CNRS National Scientific Research Center - France. Competence fields : Rare diseases, Drug delivery and targeting, Gene therapy, Non-viral Gene delivery, In vivo imaging, Molecular pharmacology, Neurology, Cell biology. Scientific Publications: Scientific production: 547 publications; 16 900 quotations without self-citations; 5 reviews or books chapters; 40 independent patent applications. Main present and recent functions: - 2002 - 2018: creator and director of the Chemical and Genetic Pharmacology and Bioimaging Unit Partners : INSERM, CNRS, Pharmacy University, Ecole Nationale Supérieure de Chimie de Paris (staff 50). - 2009 - ongoing: President of the Committee of “Non Viral Gene Therapy” of the European Society of Cell and Gene Therapy (ESGCT). Member of the Non Viral Gene Therapy Committee of the American Society of Gene Therapy (ASGT) (two 3 years terms). - 2014-2018: President of the Pharmacology and Bioimaging Section of the French National Center for Scientific Research (CNRS). - 2017 – ongoing: Director of the French Foundation for Rare Diseases.

Christine Von Raesfeld

Founder & CEO

People with Empathy

Christine Von Raesfeld

Founder & CEO

Christine Von Raesfeld is a leader in bringing a critically needed patient perspective to cutting edge medical innovations. Committed to providing patients with chronic and rare diseases with the support they need, Christine works with patient advocacy organizations, industry representatives, and individual patients and their loved ones. Living with many rare and chronic diseases, she believes that in order to foster understanding and empowerment, patients must be treated as people first. As a patient involved in Stanford's Humanwide program, she speaks on the benefits and advantages of precision medicine, with a special interest in pharmacogenomics. Christine’s drive to make lives better for patients has been nationally recognized. In 2019, she spoke on stage at the Startup Health Festival as an invited guest of Sanguine Biosciences. In 2018, Christine served on the Team of Patient Advisors for PatientsLikeMe. She was also recognized as a Wego Health 2018 Top 10 Healthcare Collaborators Patient Leader as well as named one of Silicon Valley Business Journal's 100 Women of Influence for 2019. Christine’s conference speaking engagements, consulting efforts, and ability to share her personal experiences have allowed her to bring a much-needed change to the healthcare industry while bridging the gap between biopharmaceutical and patient stakeholders. https://www.peoplewithempathy.org/

GRAHAM SIDOROWICZ

EVP Commercial

Bionical Emas

GRAHAM SIDOROWICZ

EVP Commercial

Graham is an experienced senior leader with extensive experience in commercial, market access and medical functions working in small and large pharmaceutical organizations. He has worked implementing EAPs, both for pharmaceutical companies directly and through a provider organization. During his time working in Medical Affairs, Graham led multiple Real-World Data collection projects and has published data on prospective observational studies and a global registry. Graham currently leads commercial activities across the business and is responsible for external partnerships.

Robert Palmer

Head of Value & Access Oncology, APAC, MEA

Novartis

Robert Palmer

Head of Value & Access Oncology, APAC, MEA

Rob is a pricing and market access professional with >20 years experience in the pharmaceutical industry, including 5 years in operational management of market access teams. He’s led the access function in diverse regions including China, Asia pacific, Central Eastern Europe. He’s worked across numerous therapy areas including solid tumours, haematology, rare diseases, cardiovascular and immunology, including on blockbuster products such as Cosentyx, Entresto, Kisqali, Crestor and Lynparza.

NAOMI LITCHFIELD

Director, Patient Advocacy

Bionical Emas

NAOMI LITCHFIELD

Director, Patient Advocacy

Naomi Litchfield has extensive experience working in clinical research and with patient communities. As Director, Patient Advocacy at Bionical Emas, Naomi’s role is fundamental to the company mission of bringinglife changing medicines to patients around the world. Naomi and her team focus on ensuring the patient voice is always represented, creating a positive patient journey, and reducing burden where possible when designing Bionical Emas’ Clinical Trials (CTs) or Early Access Programs (EAPs). Naomi is accountable for all internal and external advocacy activities and fosters the Company’s thought leadership in patient centricity partnering with global patient organisations.

ANTHONY HALL

Chief Medical Officer

Healx

ANTHONY HALL

Chief Medical Officer

Dr Anthony Hall (Tony) graduated from King’s College London with first class honours in physiology and pharmacology before going on to qualify as a doctor in 1991 from the Royal Free Hospital London. He joined the pharmaceutical industry in 1994 and has spent many years working on the development of drugs for rare diseases. In recent years Tony has worked at Prosensa/Biomarin on the development of antisense oligonucleotides for the treatment of Duchenne muscular dystrophy and at Mereo BioPharma where, as Therapeutic Area Head Orphan Diseases, he worked on the development of a monoclonal antibody for the treatment of osteogenesis imperfecta. In January 2020 Tony joined Healx as Chief Medical Officer, with overall responsibility for the company’s clinical programmes. Tony was one of the key people who conceived the development programme and clinical trials for the repurposing of nitisinone for the ultra-rare disease alkaptonuria (AKU). The DevelopAKUre Consortium, in which his company participated, was awarded EUR 6 million under the EU’s 7 th Framework Programme and for which a Marketing Authorisation was granted in September 2020. Tony is Co-founder and Trustee of the rare diseases charity Beacon (formerly Findacure) and is a member of the Scientific Advisory Boards of two patient groups. Together with Beacon’s Co-founder, he published a book entitled “The Patient Group Handbook: A Practical Guide for Research and Drug Development”.

BOB STEVENS

Group CEO

MPS Society

BOB STEVENS

Group CEO

Having two sons diagnosed with a rare disease (MPSII) changes your perspective and priorities in life. Having spent over 20 years in construction, many of which were at Board level, Bob decided to pursue a career in the Charity sector. At first becoming a Trustee, then progressed to Managing Director and Group CEO of various patient organisations, supporting those living with rare diseases and complex needs. Bob wanted to bring together his family experiences of a; Rare life lived and commercial attributes in order to improve the lives of those most marginalised in society whilst, at the same time, helping to create a more sustainable financial future for patient organisations both in the UK and globally. As well as being Group CEO of the MPS Society/Rare Disease Research Partners he is proud to serve as Vice Chair of the LSD Collaborative, Board Member of the worldwide International MPS Network, Acting Chair of the New Born Screening Collaborative and work as a patient representative for MPS in various global charitable access programs that provide a possible lifeline to treatments in selected areas of the world. These are not job roles, but a way of life!

DR PETER LANYON

Rare Diseases Clinical Lead, National Disease Registration Service

NHS England

DR PETER LANYON

Rare Diseases Clinical Lead, National Disease Registration Service

Peter is a Consultant Rheumatologist, Nottingham University Hospitals NHS Trust and Honorary Professor, University of Nottingham. He is the Rare Diseases Clinical Lead in the National Disease Registration Service (NDRS, NHS England). NDRS collects patient data on cancer, congenital anomalies and rare diseases, and provides expert, timely analysis to support clinical teams, academics, charities and policy makers to help plan and improve treatments and healthcare in England. In previous roles in NHS England, he has led the clinical design and implementation of NHS commissioning policies for high-cost drugs in rare diseases.

NICOLA GILLOTT

Head of Expanded Access

Amryt Pharma

NICOLA GILLOTT

Head of Expanded Access

Nicola has spent 10 years designing and managing global Expanded Access Programs in all disease areas including rare/ultra-rare and orphan diseases. She has experience of managing Expanded Access Programs through specialist vendors and in-house for pharmaceutical companies. Most recently Nicola has joined Amryt Pharma, a biopharmaceutical company focussing on rare and orphan diseases as the Head of Expanded Access.

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