Featuring Expert Speakers

Orphan Drugs & Rare Diseases Global Congress 2022 Europe

  • Doug Danison, Head of Europe, bluebird bio
  • Pedro Franco, Director for Global Regulatory & Scientific Policy (GRASP), Merck
  • Helena Baker RGN, CEO, Rare Disease Nurse Network
  • Emily Crossley, Co-founder, Duchenne UK
  • Nick Sireau, PhD, Chair and CEO, AKU Society
  • Owen Marks, Country Head for Rare Diseases Unit, Pfizer UK*
  • Femida Gwadry-Sridhar, CEO and Founder, Pulse Infoframe Inc.
  • Senior Representative, Rare Diseases Unit, Pfizer, UK
  • Lesha D. Shah, MD, Medical Director, Icahn School of Medicine, Mount Sinai
  • Philippa Norman, Projects Coordinator, Findacure
  • Elisabetta Zanon, Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine
  • Josie Godfrey, Director, JG Zebra Consulting
  • Lindsay Weaver, Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases
  • Christian Girard, Strategic Consultant, Orphan Drugs Industry Advisory & Intelligence
  • Senior representative, Illingworth Research Group, a Syneos Health Company
  • Sophie Laribiere, Sr Director, Global Regulatory Affairs, Alexion Pharmaceuticals
  • Rhian Kiely, RWE Commercial Director, Europe, Open Health Evidence & Access
  • Martina Garau , Director, Office of Health Economics
  • Andrew Kim, VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines
  • Ryotaro Ishikawa, Global Market Access Director, Novartis
  • Simu Thomas PhD, Vice President, Global Head HEOR, Alexion Pharmaceuticals
  • Keith White, Head, Global Market Access, Amylyx Pharmaceuticals
  • Arvin M. Gouw, Ph.D., VP of Research & Development, Rare Genomics Institute
  • Angela Columbano, Head Business Development & Partnership, Genethon
  • Paul Beresford, International Biotech Executive
  • Saira A. Jan, VP & Chief Pharmacy Officer, Horizon Blue Cross Blue Shield of New Jersey
  • Paolo Morgese, EU Director Market Access, Alliance for Regenerative Medicine
  • Manolo Bellotto, PhD, President, Gain Therapeutics SA
  • Alexander Natz, LL.M., Secretary-General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
  • Karen Matthews, Business Development Manager, National Institute for Health Research*
  • Kerry Leeson-Beevers, National Development Manager & Project Lead, Alström Syndrome*
  • Senior Representative, National Institue for Health and Care Excellence (NICE)*
  • Paul Greenland, President EMEA, Amryt Pharma
  • Simon Jones, MD, Medical Director, NIHR Clinical Research Facility, NIHR
  • Sheela Upadhyaya, Associate Director - Highly Specialised Technologies, NICE
  • Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech Evaluation, Independence Blue Cross
  • David Rose, BD, RARE Revolution Magazine, Ultra-Rare Disease Patient Speaker
  • Karen Matthews, Business Development Manager, CRNCC, NIHR Clinical Research Network
  • Eric Alton, Professor, Imperial College London, Head, UK CF Gene Therapy Consortium
  • Joe Brice, Head, Franchise Public Affairs, EUCAN, Takeda*
  • Farhat Khanim MD, Director of Research, Institute of Clinical Sciences, University of Birmingham
  • Zizi Imatorbhebhe, MBA MS PMP® Senior Vice President, Global Strategy & Development, Rare Disease, Ergomed
  • Daniel Lewi, Head of Business Development and Patient Advocacy, Pulse Infoframe Inc.
  • David Ross, Patient Advocate, Rare Disease Mental Health
  • Peter MacLennan, CEO, Tailored Clinical Research Solutions Ltd
  • Juliet Hulse, Sr. Director, Global Research Nurse Strategy, Illingworth Research Group
  • Carole Scrafton, CEO & Co-Founder, FibroFlutters
  • And many more...

Alexander Natz, LL.M.

Secretary-General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)

Keith White

Head, Global Market Access, Amylyx Pharmaceuticals

Elisabetta Zanon

Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine

Andrew Kim

VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines

Robert Donnell

Executive Vice President – Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

Angela Columbano

Head Business Development and Partnership, Genethon

Helena Baker RGN

CEO, Rare Disease Nurse Network

Simu Thomas PhD

Vice President, Global Head HEOR, Alexion Pharmaceuticals

Emily Crossley

Co-founder and joint CEO, Duchenne UK

Lindsay Weaver

Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases

Philippa Norman

Projects Coordinator, Findacure

Eric Alton FRCP, FMedSci,

Professor of Gene Therapy and Respiratory Medicine, Imperial College, London

Paul Beresford

International Biotech Executive

Josie Godfrey

Director, JG Zebra Consulting, Project Director, Duchenne UK's Project Hercules

Doug Danison

Head of Europe, bluebird bio

Christian Girard

EU Business Operations Consultant, Acer Therapeutics

Ramona Reichenbach

Head Managed Access Center of Excellence (MACoE), Novartis

Dr Femida Gwadry-Sridhar

Chief Executive Officer and Founder, Pulse Infoframe

Ryotaro Ishikawa

Global Market Access Director, Novartis

Martina Garau

Director, Office of Health Economics

Saira A. Jan

VP & Chief Pharmacy Officer, Horizon Blue Cross Blue Shield of New Jersey

Manolo Bellotto, PhD

President & General Manager, Gain Therapeutics

Owen Marks

Country Head for Rare Diseases Unit, Pfizer UK

Lesha D. Shah, MD

Assistant Professor of Psychiatry, Medical Director, Child, Adolescent and Family Services, Icahn School of Medicine, Mount Sinai

Paul Greenland

President EMEA, Amryt Pharma

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP), Merck

Farhat Khanim MD

Director of Research, Institute of Clinical Sciences, University of Birmingham

Karen Matthews PhD

Business Development Manager, NIHR

David Edward Rose

Ultra-Rare Disease Speaker (1 in 300 million), Business Development & Sales, Rare Revolution Magazine

Eugean Jiwanmall,

Sr Research Analyst, Medical Policy & Technology Evaluation, Independence Blue Cross

Paolo Morgese,

EU Director Market Access, Alliance for Regenerative Medicine

Sheela Upadhyaya

Associate Director - Highly Specialised Technologies, NICE

Rhian Kiely

RWE Commercial Director, Europe, Open Health Evidence & Access

Senior Representative

Illingworth Research Group

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP), Merck

Dr. Nicolas Sireau

CEO and Chair of Trustees, AKU Society

Robert Donnell

Executive Vice President – Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

Zizi Imatorbhebhe, MBA MS PMP®

Senior Vice President, Global Strategy & Development, Rare Disease, Ergomed

Andrew Kim

VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines

Elisabetta Zanon

Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine

Arvin M. Gouw, Ph.D.

VP of Research & Development, Rare Genomics Institute

Peter MacLennan

CEO, Tailored Clinical Research Solutions Ltd

Juliet Hulse

Sr. Director, Global Research Nurse Strategy, Illingworth Research Group

Daniel Lewi

Head of Business Development and Patient Advocacy, Pulse Infoframe Inc.

David Ross

Patient Advocate, Rare Disease Mental Health

Carole Scrafton

CEO & Co-Founder, FibroFlutters

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Alexander Natz, LL.M.

Secretary-General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)

Alexander Natz is the Secretary-General of the European Confederation of Pharmaceutical Entrepreneurs (www.eucope.org) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective.

From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he has been a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals. Dr Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry. As a research assistant at Duke University (USA), he has dealt with international pharmaceutical law. His doctorate was supervised by the former judge at the European Court of Justice, Prof. Dr. Dr. Ulrich Everling.

Keith White

Head Global Market Access Amylyx Pharmaceuticals

Expertise lies in the development of pre-clinical gene therapies for neuromuscular diseases, in particular muscular dystrophies based on gene addition, endonuclease-mediated gene editing, antisense oligonucleotide (AO)-induced exon skipping and gene silencing. Having performed well in clinical trial, FDA approval is currently being sought for a patented AO developed in the Popplewell lab for a particular subset of Duchenne muscular dystrophy (DMD) patients. Dr Popplewell is PI of an upcoming clinical trial of an optimised microdystrophin AAV, which would have universal DMD patient applicability. She is an elected board member of the BSGCT, sits on the Management Committee of an eCOST Action and is an invited member of the MDEX Consortium.

Elisabetta Zanon

Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine

Elisabetta Zanon is a senior professional with over 20 years of experience in European affairs. This has included working for EU associations and representing different public sector organisations to the EU institutions. She has a successful track record in influencing EU policy and legislation and in designing and running EU communication campaigns. Elisabetta established the National Health Service (NHS) European Office in Brussels at the end of 2007 and was Director of that Office for ten years. In February 2018 Elisabetta joined the European Society of Cardiology as Advocacy Director taking responsibility for the Society’s activities in Advocacy, Regulatory Affairs, patient engagement and EU-Funded projects. Since November 2021 Elisabetta has been Director of EU Public Affairs & Advocacy at the Alliance for Regenerative Medicine with responsibility for Government relations. 

Andrew Kim

VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines

Andrew brings over 20 years of industry and clinical experience with a focus on precision oncology and fertility. Previously, he served as Blueprint Medicines’ International Head of Precision Medicine (2018- 2020). During this appointment he built and led a team of five Precision Medicine Liaisons (PMLs) developing strategic relationships with EU KOLs and investigators driving registrational trial enrolment in solid and haematological tumour programmes. He has also personally led KOL/investigator relationships in APAC ex-China.
Since 2020, Andrew has been leading the cross-functional preparation for full-scale AYVAKYT EU launch in EMA-filed orphan HaemOnc (and future rare disease) indications as the company’s International Launch Lead & General Manager for the Nordics.
Prior to joining Blueprint Medicines, Andrew served as Senior Director, European Operations at Igynta (2016 to 2018) where he led European operations including Medical Affairs and pre-commercial launch preparation. He informed global strategic decisions synthesising field perspectives and competitive intelligence including driving European patient enrolment for a/the Phase 2 basket study in solid tumours driven by rare NTRK and ROS1 gene fusions. He furthermore developed a European KOL network across Lung, Sarcoma, Head & Neck, GI and other tumour histologies.
Prior to that, he held roles of increasing responsibility in management consultancy (McKinsey & Company) and healthcare investment banking (Barclays Capital, Ares Life Sciences).
Andrew holds a Bachelor of Science in Chemical Engineering and a Master of Science in Chemical Engineering Practice from Massachusetts Institute of Technology (MIT) and an MBA from the London Business School. 

Robert Donnell

Executive Vice President – Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

Robert has designed and led some of the most innovative and largest EAPs in recent years including in rare diseases and gene therapy. He has experience across Europe, USA, and globally and has advised major blue-chip Pharma as well as smaller biotech companies. He started his career at P&G and AZ before moving to a number of international service providers working with pharma and biotech companies on supply chain solutions. He currently leads all global development and implementation of EAPs at Smartway where he specializes in working with manufacturers to develop the best Medicines Access plan for them.

Angela Columbano

Head Business Development and Partnership, Genethon

Angela Columbano has over 20-year experience in the pharmaceutical and Biomedical industry.

Prior to Genethon, Angela was Principal at Sanofi Sunrise an early investment and partnering vehicle. Angela has also held various positions with Sanofi in Marketing and Business Development and Licensing for a number of therapeutic areas such as neuro-degeneration, age-related muscular diseases, cardiovascular disease and diabetes.

In the early years of her career, Angela was a Research Scientist with positions at Pfizer in Oncology and SmithKline Beecham. She obtained her PhD in Pharmaceutical Sciences at the School of Pharmacy of London and an MBA from Kingston University, UK.

Helena Baker RGN

CEO, Rare Disease Nurse Network

Helena trained as a nurse and specialised in theatres and anaesthetics. She became the first-ever anaesthetic nurse at the Royal London Hospital, supporting some of the most seriously injured patients in the south of England, as part of the Helicopter Emergency Medical Service.

Helena then moved into the pharmaceutical industry in sales and product management, working across Europe in pharmaceuticals and medical devices. Clinically, Helena worked in urology and oncology and then moved to the Medical Research Network.

As one of the founders, Helena fulfilled several roles, including Director of Nursing and Global VP for Operations & Nursing.

Helena was born with a rare disease called Fibular Hemimelia and spent most of her early years in hospital. 4 years ago she had her right leg amputated, but has rehabilitated ridiculously well and has since done 2 parachute jumps, walked miles, and has recently taken up canoeing.

Helena joined the Rare Disease Nurse Network at its formation in 2018 and is now interim CEO of the charitable organisation. Passionate about improving access to specialist care in rare diseases, she aims to enable healthcare professionals to reach the expert advice and knowledge to help them improve care. 

Simu Thomas PhD

Vice President, Global Head HEOR, Alexion Pharmaceuticals

Dr Simu Thomas is the Vice President and Global Head of Health Economics & Outcomes Research at Alexion Pharmaceuticals. Simu brings 20 years of leadership experience in the area of Value, HEOR, RWE and Access, building and leading teams across the world.

Prior to Alexion, Simu was the Global Head of Value & Access for Rare Diseases at Novartis and previously, Global Head for the Cell & Gene Therapy Unit, where he developed the value access strategy for the first CAR-T therapy approved in the world. He was also the founding architect of the IMI HARMONY data initiative with public and EFPIA private partners in Europe, as the leading industry partner in the consortium. Simu ran the Economic Modeling organization at Novartis, was the Global Head of HEOR for Neuroscience and Ophthalmics franchises and US Oncology.

He has authored more than 35 manuscripts and 75 congress presentations and co-authored book chapters in the field of Health Economics. Simu holds a PhD in Pharmaceutical Economics from the University of Maryland and an MS in Pharmacy Administration from the University of Toledo and Pharmacy degrees. Simu also serves as Adjunct Assistant Professor at the University of Maryland and Rutgers University of New Jersey.

Emily Crossley

Co-founder and joint CEO, Duchenne UK

Emily set up the Duchenne Children’s Trust in 2012 after her son was diagnosed with Duchenne. Before that she was a reporter and anchor for Channel 4 News and CNN International. The Duchenne Children’s Trust raised £3.5million in its first three years, to spend on clinical trials, research, and clinical trial infrastructure. The charity set up the annual Duchenne Dash, which brings in money for research and raises awareness around Duchenne muscular dystrophy. Emily has advocated on behalf of patients around the world. She’s spoken many times in the Houses of Parliament. She is a member of the MHRA patient group consultative forum.

Lindsay Weaver

Co-Founder & CEO, Realise Advocacy, Trustee, Medics 4 Rare Diseases

Lindsay is the former CEO of MSUK where she served for 6 years and is a trustee at Medics 4 Rare Diseases. Lindsay also served as NHS England Metabolic Clinical Reference Group Patient and Public Voice member for over 4 years. During her time working in rare diseases, she has developed knowledge of the challenges that all stakeholders face in participating in NICE and NHS England access processes for new treatments.

Lindsay has become a strong advocate for supporting patient groups in access and has seen first-hand that well-supported patient advocates can have an impact on decision-making.

Philippa Norman

Projects Coordinator, Findacure

Philippa is the Projects Coordinator at Findacure. She is responsible for the coordination and delivery of major elements of Findacure’s Patient Group training projects. This includes leading the delivery of Findacure’s webinar programme, responsibility for a cohort of patient group mentees, supporting the Head of Operations in delivering the flagship workshop programme, and the launch of the new online Resource Hub. Philippa has worked directly with over 60 patient group leaders and advocates since joining the organisation and has gained insight into the needs of patients and the demands of patient group leaders. She is determined to see a world in which everyone cares about the stories and needs of the rare community and patient groups are a central cog and valued voice in the development of treatments.

Eric Alton FRCP, FMedSci,

Professor of Gene Therapy and Respiratory Medicine, Imperial College, London

Eric is Professor of Gene Therapy and Respiratory Medicine at Imperial College, London, an Honorary Consultant Physician at the Royal Brompton Hospital and NIHR Senior Investigator. Over the last 20 years he has coordinated the UK Cystic Fibrosis Gene Therapy Consortium, which brings together all the key centres in the UK (Edinburgh and Oxford Universities and Imperial College, London) in a programme to deliver gene therapy for these patients. Based on the cystic fibrosis programme, the Consortium is now also applying the underpinning gene delivery technology to a number of other diseases. He is the Chair of the NIHR ATMP Coordinating Group which has pulled together a dozen of the key stakeholders in the field to catalyse coordination and avoid duplication. He is the Lead for Cluster B of the NIHR CRN, coordinating portfolio studies in respiratory, ENT, gastroenterology, hepatology and infection.

Paul Beresford

International Biotech Executive

Paul has extensive biotech and pharmaceutical industry experience in ex-US markets. especially, Europe, encompassing general management, commercial leadership, and alliance management in specialty, rare and orphan diseases (including oncology)

He led Blueprint Medicines in International markets, establishing European offices and building the cross-functional team to drive the development and commercialization of the portfolio in major markets.

Paul has built a strong strategic and operational reputation, having built new organizations, developed product portfolio strategies, and driven great launches, as well as creating significant turnaround strategies to rejuvenate portfolios and accelerate growth.

Josie Godfrey

Director, JG Zebra Consulting, Project Director, Duchenne UK's Project Hercules

Josie Godfrey is the Director of JG Zebra Consulting. Josie is Strategic Director for Duchenne UK’s ground-breaking multi stakeholder collaboration, Project HERCULES. She is a member of the International Rare Disease Research Consortium (IRDiRC) Working Group 3 looking at methodologies for assessing treatments for rare diseases and has acted as co-Chair of the EUCOPE Gene and Cell Therapy Working Group. Josie is a trustee of Metabolic Support UK. Josie has 10 years of experience working in rare diseases with a particular focus on HTA, market access and policy and public affairs. Josie set up and ran the NICE Highly Specialised Technologies programme as well as the predecessor programme, the Advisory Board for National Specialised Services (AGNSS).

Doug Danison

Head of Europe, bluebird bio

Doug Danison is the Head of Europe at bluebird bio since January 2021. He joined bluebird bio in July 2015 to establish the global pricing, market access, and health economics team known as Access Value & Evidence Strategy (AVES).

Doug has more than 20 years of Pricing & Market Access experience, including roles with Eli Lilly & Company, Amgen, Millennium/Takeda Oncology. In that time, he has led or overseen the preparation of market access strategies for products in early development, pivotal study planning, launch, and post-launch lifecycle management. While addressing market access challenges from a variety of vantage points including Global, European Regional, and U.S. roles he’s built teams, processes, and a knowledge base that enables the execution of market access strategies that anticipate and meet the needs of access decision-makers in an increasingly challenging market access environment.

Christian Girard

Strategic Consultant, Orphan Drugs Industry Advisory & Intelligence

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. 

Femida Gwadry-Sridhar

CEO & Founder Pulse Infoframe Inc.

Femida is a pharmacist, clinical epidemiologist, and methodologist with over 25 years of experience in clinical trials, patient registries, knowledge translation, health analytics, and clinical disease outcomes. Femida has received more than $10 million in research funding as a principal investigator or co-investigator. She was awarded a career scientist award in 2006 from the Canadian Institute of Health Research and through her work founded the first knowledge translation health informatics lab in North America, creating a collaborative ecosystem for interdisciplinary research. Recently recognized as a lifetime fellow of CIHR for her contributions to global research with impact. Femida has published over 200 manuscripts and abstracts in top tier journals and actively continues her professional interests in enabling technologies for better provider workflow and patient engagement. Femida is a founding member of the International Society for Pharmacoeconomics and Outcomes Research medication adherence group, which has provided an early glimpse into how to conduct research in adherence. She is an expert reviewer in North America and Europe on large scale research network grants and a frequent international speaker on patient engagement, patient registries, innovation, health informatics and data visualization. The recipient of numerous nominations, awards and positive press, Femida has joined Springboard Enterprises as a female entrepreneur in digital health. She also holds her PhD in research methodology and health economics from McMaster University.

Ryotaro Ishikawa

Global Market Access Director, Novartis

Coming Soon! 

Martina Garau

Director, Office of Health Economics

Martina has over 15 years of experience leading projects for a variety of organisations, including global life sciences companies, non-profit research organisations, charities and trade associations. Her projects aim at expanding the evidence used in healthcare decisions and shaping the debate around critical health policy issues. Her areas of expertise include value-based assessments in high-and middle-income countries; economic issues posed by the development and provision of orphan medicines; methods and applications of structured decision-making approaches such as Multi-Criteria Decision Analysis (MCDA).

Saira A. Jan

VP & Chief Pharmacy Officer, Horizon Blue Cross Blue Shield of New Jersey

Dr Saira Jan is the Vice President and Chief Pharmacy Officer at Horizon Blue Cross Blue Shield of New Jersey and Professor Emerita at the Rutgers State University of New Jersey. With a passion for creating positive patient outcomes, driving innovation, and improving healthcare delivery, Dr Jan oversees Horizon’s pharmacy and medical integration initiatives for Commercial, Medicaid, and Medicare lines of business. She has over 25 years of experience in health care management, research, and academics, and during that time, she has worked closely with business units, clinical quality groups, and medical management teams to deliver integrated and comprehensive services for over 3.5 million lives.

 

Saira leads and established one of the first managed care curriculums and Managed Care Residency at Rutgers, one of the first programs in the US. She has extensive experience in outcome-based research projects with Harvard University and numerous other nationally acclaimed institutions. Dr Jan has also lead the opioid peer to peer collaboration model in New Jersey and has overseen the development of The Rutgers Opioid Toolkit, childhood obesity, adherence, diabetes and various other and community outreach program, involving Rutgers pharmacy students and Horizon clinical staff. Saira also works extensively with the National Blue Cross Blue Shield Association, CMS, FDA, as well as international healthcare groups on various programs and initiatives.

Dr. Jan has a Masters in Pharmacology from St John’s University and a doctorate in Pharmacy from Rutgers University.

Manolo Bellotto, PhD,

President & General Manager, Gain Therapeutics

Dr Manolo Bellotto has served as president and general manager of the company and its subsidiary GT Gain Therapeutics SA since 2018. Dr Bellotto has over 20 years of experience in 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 a global product leader at Vifor Pharma, a global speciality 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 PhD 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.

Owen Marks

Country Head for Rare Diseases Unit, Pfizer UK

Owen is Head of Rare Diseases for Pfizer in the UK covering a portfolio including Gene Therapies and Treatments for rare disorders. He is also Co-chair of the Pfizer UK Inclusive Diversity Group. A Physiology and Pharmacology Graduate from the University of Edinburgh, Owen joined the industry in 1997 and has held many commercial and marketing roles. Experienced in major organisational planning and change leadership, Owen has also held European Regional Marketing Leadership roles responsible for Western, Central, and Eastern Europe. 

Lesha D. Shah, MD

Assistant Professor of Psychiatry, Medical Director, Child, Adolescent and Family Services, Icahn School of Medicine, Mount Sinai

Lesha D. Shah, M.D. is Assistant Professor of Psychiatry and the Medical Director of Child, Adolescent and Family Services at the Icahn School of Medicine at Mount Sinai. Her primary academic focus is pediatric research ethics and physician perspectives around medical decision-making for children. She studies issues of consent and capacity as they interface with family complexity and innovative medicine with a focus on gene therapy clinical trials. She co-chairs the Pediatric Gene Therapy and Medical Ethics Working Group (PGTME), whose mission is to advance research, education, and policy in addressing emergent ethical issues around investigational gene therapy in pediatric populations. Dr. Shah serves on the Icahn School of Medicine at Mount Sinai Institutional Review Board and has published on issues related to medical ethics in Pediatrics, the Journal of the American Academy of Child and Adolescent Psychiatry, and the American Journal of Bioethics. She is dually board certified by the American Board of Psychiatry and Neurology.

Paul Greenland,

President EMEA, Amryt Pharma

Paul joined Amryt Pharma as President EMEA Region in September 2019 and brings over 30 years of experience across a broad range of International commercial roles in the pharmaceutical industry. Mr Greenland joined Amryt through the acquisition of Aegerion Pharmaceuticals where he held the position of President EMEA Region and had directed the launch and subsequent growth of Myalepta (metreleptin), a treatment for patients with Lipodystrophy. Prior to this Mr Greenland was Vice President of Biologics at Hospira where he directed the establish their fledgling commercial biosimilar business in Europe.

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP), Merck

Pedro Franco holds a degree in Pharmacy (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 Ph.D. 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 focused 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.

Farhat Khanim MD,

Director of Research, Institute of Clinical Sciences, University of Birmingham

Farhat Khanim is a translational cancer biologist using drug repurposing to identify novel, low toxicity and affordable therapies for rare diseases and diseases of unmet need. Her major research areas are haematological cancers of the elderly, urological cancers and paediatric cancer in low-middle income countries (LMICs) in Africa. Farhat’s research encompasses the full spectrum of translational research from in vitro drug screening, preclinical mechanism of action studies using a range of molecular, cellular and biochemical approaches, through to clinical trials in the UK and Africa. Farhat is also Director for Research for the School of Biomedical Sciences. As a full-time academic, Farhat teaches and supervises undergraduate and postgraduate students including clinical and non-clinical Masters and PhD students.

Karen Matthews PhD

Business Development Manager, NIHR

The National Institute for Health Research (NIHR) Clinical Research Network works in partnership with the National Health Service (NHS) to deliver high-quality research. We support all phases of clinical research studies and trials across all disease areas.

Dr Karen Matthews facilitates discussions between non-commercial funders of research, such as charities, and the Clinical Research Network. She provides advice to both charities and companies on how they are able to collaborate with the Clinical Research Network. Karen also provides guidance on how to access and optimise our Study Support Service to ensure that clinical studies are set up efficiently and deliver to their recruitment targets.

Karen has been with the Clinical Research Network since December 2007. She was previously the operational lead for the Coordinated System for gaining NHS Permission (CSP). The system, which has now been superseded, was instrumental in demonstrating that the UK could set up clinical trials rapidly in a coordinated manner.

Karen has previously worked as a research scientist within the oncology team in Glaxo Wellcomne (1995-6). She gained a PhD in developmental genetics (2000) and conducted post-doctoral research within the Institute of Child Health, London. She subsequently went into research administration and has held posts with Cancer Research UK and Barts and The London NHS Trust.

David Edward Rose

Ultra-Rare Disease Speaker (1 in 300 million), Business Development & Sales, Rare Revolution Magazine

David is an accomplished public speaker, delivering talks on his ultra-rare disease, Occipital Horn Syndrome. David talks about all areas of what it’s like living with an ultra-rare disease, both medically and in general life. Occipital Horn Syndrome is ultra-rare, David often talks about the isolation and what it’s like for those living with an ultra-rare disease with no community set up.

Alongside his speaking engagements, he works in business development for Rare Revolution Magazine – a rare disease digital magazine based in the UK. He is also a volunteer ambassador for the famous children’s hospital – Great Ormond Street Hospital – where he spent a lot of time as a child and teenager.

Eugean Jiwanmall

Sr Research Analyst, Medical Policy & Technology Evaluation, Independence Blue Cross

Eugean Jiwanmall’s past professional experiences include basic science and clinical research. In his current role as a Senior Research Analyst for Medical Policy & Technology Evaluation at Independence Blue Cross’ Medical & Claim Payment Policy Department within Facilitated Health Networks and Medical Affairs, he has served as the subject-matter-expert, writer, reviewer, communicator, presenter, consultant, adviser/facilitator, and decision-maker for hundreds of medical policy topics and technology assessments within dozens of clinical disciplines and multiple business areas. Eugean has participated in numerous roles & capacities in a number of conferences, summits, and other national & international events, etc. upon invitations. He has been invited by renowned organizations, evaluator bodies, & thinktanks, etc. to be a key informant on a variety of complex and challenging topics, and recognized in communications & publications for these works.

Eugean has taken and completed graduate coursework in bioethics at the University of Pennsylvania School of Medicine. He holds a bachelor’s degree in biological sciences with honours from Drexel University (minor was in business administration), a master’s degree in public health in evaluative clinical sciences from Dartmouth Medical School, and an MBA in healthcare management and economics with honours from Drexel University.

Paolo Morgese

EU Director Market Access, Alliance for Regenerative Medicine

Paolo is a healthcare investment and market access professional with more than 15 years of experience in both assessing and supporting access to innovative healthcare technologies. Paolo is currently VP Market Access at Alira Health a healthcare advisory company and EU Director Market Access and Member Relations at the Alliance for Regenerative Medicine (ARM). Between 2012 and 2017 he worked at Deerfield Management, a healthcare investment company, where he was Director of EU Research and Market Access. Prior to that, he worked at Merck Serono for five years, Kyphon, which became Medtronic, and also spent several years with the Italian HTA Agency Agenas. Paolo’s interests and expertise are centered around healthcare technology value assessment and maximization, with a particular interest in the early stages of product development.

Sheela Upadhyaya

Associate Director - Highly Specialised Technologies, NICE

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is 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 to 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 them 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 collaboration 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.

Rhian Kiely

RWE Commercial Director, Europe Open Health Evidence & Access

Dr Rhian Kiely has experience in the planning and execution of real-world evidence projects in order to generate impactful evidence to drive change. Rhian brings over 10 years’ experience in clinical research in order to develop the best solutions for our clients.

Rhian has experience in developing strategic evidence generation plans both at the local and global level to support clients to navigate the launch of their product and beyond. Rhian also supports the OPEN Health operational teams in generation of evidence from both primary and secondary data sources and leverages expertise from across the OPEN Health group in market access, communications and modelling to ensure that the value of evidence generated is maximised for clients.

Rhian has a degree in Applied Biology from Cardiff University and a DPhil in Clinical Medicine from Oxford University. Rhian has published her work in several peer-reviewed journals and has presented her work at national and international conferences.

Rhian is a member of the ISPOR Precision Medicine and Advanced Therapies Specialist Interest Group and is part of the RWE & Digital Health Working Group within the European CRO Federation.

Helen Springford

Chief Operating Officer, Illingworth Research Group

Helen Springford has over 28 years’ experience in the Clinical Trials arena. Having worked as a nurse in cardiac transplantation in the UK and Australia, Helen moved into clinical research in 1992, initially as a research nurse. Since then, she has managed an SMO and held various positions in both CROs and big pharma within several functional areas including Clinical Operations, Project Management, Business Development and Proposals. Helen was appointed Vice President, Strategic Development at Illingworth Research in August 2016 and was promoted to Chief Operating Officer in April 2019. Helen has always promoted the patient as an active participant in their care and has brought this to the current role where patient focused solutions such as mobile research nursing allows people, irrespective of their geography or state of health to participate in clinical trials. Patient empowerment, whether adult or child is an important part of Helen’s work and has been something of personal interest throughout her career.

Pedro Franco

Director for Global Regulatory & Scientific Policy (GRASP), Merck

Pedro Franco holds a degree in Pharmacy (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 Ph.D. 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 focused 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. Nicolas Sireau

CEO and Chair of Trustees, AKU Society

Dr Nicolas Sireau is the CEO and Chair of Trustees at the AKU Society, a patient group that helps people with AKU (short for alkaptonuria), a rare genetic disease affecting both his children. He is also co-founder and Chair of Findacure, an organisation that helps rare disease patient groups. Previously, Nick was the CEO of SolarAid, an NGO working in Africa. He is a fellow of the Ashoka Fellowship of Social Entrepreneurs and has a PhD in the social psychology of social movements. 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’ (Findacure 2016).

Zizi Imatorbhebhe, MBA MS PMP®

Senior Vice President, Global Strategy & Development, Rare Disease, Ergomed PLC

  • Seasoned executive with over 30+ years of global commercial, consulting and clinical development experience in the Biopharmaceutical and CRO industry
  • Instrumental in working with many small to large biopharmaceutical companies to identify Drug Development challenges  in the Rare Disease space and provide solutions to address them
  • Leverage innovations and data insights to build differentiated solutions including Decentralized Trials, Patient Success grow business.
  • Developed and commercialized over 25 pharmaceutical and medical device products
  • Active with Patient Advocacy Group sand Diversity Community Outreach efforts 

Educational background includes :

  • MS in Biotechnology from University College London, University of London
  • MBA from Chapman University, California
  • Management Certificate in Disruptive Strategies and Innovation, Harvard Business School, Boston, Massachusetts
  • PMP® certified and Lean six sigma trained
  • All providing a strong commercial and business perspective as well as clinical development expertise in numerous therapeutic areas e.g., CNS, Rare Disease

Andrew Kim

VP International Strategic Operations & General Manager, Global Distributor Markets, Blueprint Medicines

Andrew brings over 20 years of industry and clinical experience with a focus on precision oncology and fertility. Previously, he served as Blueprint Medicines’ International Head of Precision Medicine (2018- 2020). During this appointment he built and led a team of five Precision Medicine Liaisons (PMLs) developing strategic relationships with EU KOLs and investigators driving registrational trial enrolment in solid and haematological tumour programmes. He has also personally led KOL/investigator relationships in APAC ex-China.
Since 2020, Andrew has been leading the cross-functional preparation for full-scale AYVAKYT EU launch in EMA-filed orphan HaemOnc (and future rare disease) indications as the company’s International Launch Lead & General Manager for the Nordics.
Prior to joining Blueprint Medicines, Andrew served as Senior Director, European Operations at Igynta (2016 to 2018) where he led European operations including Medical Affairs and pre-commercial launch preparation. He informed global strategic decisions synthesising field perspectives and competitive intelligence including driving European patient enrolment for a/the Phase 2 basket study in solid tumours driven by rare NTRK and ROS1 gene fusions. He furthermore developed a European KOL network across Lung, Sarcoma, Head & Neck, GI and other tumour histologies.
Prior to that, he held roles of increasing responsibility in management consultancy (McKinsey & Company) and healthcare investment banking (Barclays Capital, Ares Life Sciences).
Andrew holds a Bachelor of Science in Chemical Engineering and a Master of Science in Chemical Engineering Practice from Massachusetts Institute of Technology (MIT) and an MBA from the London Business School.

Elisabetta Zanon

Director, EU Public Affairs & Advocacy, European Alliance for Regenerative Medicine

Elisabetta Zanon is a senior professional with over 20 years of experience in European affairs. This has included working for EU associations and representing different public sector organisations to the EU institutions. She has a successful track record in influencing EU policy and legislation and in designing and running EU communication campaigns. Elisabetta established the National Health Service (NHS) European Office in Brussels at the end of 2007 and was Director of that Office for ten years. In February 2018 Elisabetta joined the European Society of Cardiology as Advocacy Director taking responsibility for the Society’s activities in Advocacy, Regulatory Affairs, patient engagement and EU-Funded projects. Since November 2021 Elisabetta has been Director of EU Public Affairs & Advocacy at the Alliance for Regenerative Medicine with responsibility for Government relations. 

Arvin M. Gouw, Ph.D.,

VP of Research & Development, Rare Genomics Institute

Arvin Gouw, PhD is the vice president for research and development overseeing the BeHEARD (Help Empower & Accelerate Research Discoveries) program of Rare Genomics Institute, where he leads crowdfunding efforts for rare disease personalized medicine research, predominantly for children, and develops novel platforms to connect patients to medical experts. He was an instructor in oncology where he also did his postdoctoral work in cancer metabolism at Stanford University School Medicine. 

Peter MacLennan

CEO, Tailored Clinical Research Solutions Ltd

Peter MacLennan, TCRS CEO, is an experienced professional with a track record of success in academia, pharma, biotech and CROs.
He has in-depth knowledge of clinical development with an emphasis on risk management, project governance, metrics, and budgeting.
With his TCRS colleagues, his task is to ensure that our clients’ objectives are delivered on time, on budget and to the highest standards of quality.

Juliet Hulse

Sr. Director, Global Research Nurse Strategy, Illingworth Research Group

Juliet joined the company in 2011 as a Senior Research Nurse and after several promotions was appointed to her current the position in 2020. Juliet has over 20 years’ experience within clinical research. Commencing her career in cardiology research covering multiple Phase I- IV trials. More recently she has specialised in neurology research, in particular, the field of multiple sclerosis. Juliet has wide experience in the management of trial projects from initiation to close out as well as working with site investigators to improve patient recruitment and patient retention to the study protocols. She believes developing a strategy that places the patient in the centre of the trial is vital for a successful study.

Daniel Lewi

Head of Business Development and Patient Advocacy, Pulse Infoframe Inc.

Dan Lewi is the Head of Business Development and Patient Advocacy at Pulse Infoframe. He develops relationships with the many clients who can benefit from building registries for the purpose of accelerating research and drug development. Dan’s varied background in rare disease advocacy began when he and his wife founded the Cure & Action for Tay-Sachs (CATS) Foundation in the UK, after their daughter Amélie was diagnosed with Tay-Sachs. With no national charities to provide support, the CATS Foundation has nonetheless proved instrumental in helping pharma develop treatments for Tay-Sachs and Sandhoff diseases, ranging from gene therapy programs to drug repurposing studies. Dan has over ten years of experience in medtech and advocacy, and he has also served as Chairman of the European Tay-Sachs and Sandhoff Charity Consortium since 2012. Dan is fully aware of the patient voice, and he consistently puts patients and their needs at the forefront of clinical discussion.

David Ross,

Patient Advocate, Rare Disease Mental Health

David Ross is a patient advocate and rare disease male mental mental health collaborator. David’s activism began in 2017 when he was diagnosed with a rare disease called Cowden Syndrome due to being given a letter from his mother about getting tested before she passed away due to having the same condition.

After a period of coming to terms with this he became committed to raising awareness and supporting others impacted by this condition by helping set up a support group and also applying to join the Pten Foundation International Family Council which looks to helping my rare disease community by raising awareness about Cowden Syndrome.

In 2020 he attended 3 virtual Eurordis rare disease schools and is currently on the Findacure mentoring program. His latest project has been to set up rare disease male mental health International zoom calls for patients and caregivers and will be looking to set up an online support group for males in 2021

Carole Scrafton

CEO & Co-Founder, FibroFlutters

Carole is a patient with chronic and genetic rare disease, Patient Partner, Expert Patient, Patient Speaker, Author and Researcher.

She is the CEO & Co-founder of FibroFlutters Patient Advocacy Organisation for chronic / rare illnesses. Helping to develop, nurture and advocate for a multidisciplinary approach to all aspects of medical healthcare so that chronic illness & rare disease patients can receive the type of care and treatments that they need.
Carole utilises varying social media platforms to support people with Chronic illness & Rare Disease, #notjustpatients, and with a multi-stakeholder following. A network used for raising disease awareness and sharing as much up to date information.
Carole also advocates for patient centricity, especially patient engagement best practices and the need for patients to be included within research at early development / preclinical phases. She recognises the need to help educate patients about clinical trials and the reason why they should get involved.
Recently as a Patient, and with FibroFlutters, was a team member of an initiative to create: ‘How-To guide’. How to implement Patient Engagement into Early Development and Preclinical phases with Patients Focused Medicines Development.
 

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