6th Expanded Access Programmes World Congress 2024 Europe.

6th Expanded Access Progammes World Congress 2024 Europe

Holiday Inn London - Bloomsbury, an IHG Hotel, London, United Kingdom

Thursday 11th - Friday 12th April 2024

KEY INDUSTRY EXPERT SPEAKERS

ANNIE O’KEEFE MARTIN

Global Medical Governance Lead for Managed Access Programs and Rare Humanitarian Programs

Sanofi

ANNIE O’KEEFE MARTIN

Global Medical Governance Lead for Managed Access Programs and Rare Humanitarian Programs

Annie is the Global Medical Governance Lead for Managed Access Programs and Rare Humanitarian Programs (Specialty Care). She represents Sanofi in the Managed Access Industry Forum and Managed Access Initiative Network. Prior to her current role Annie acquired 15 years’ experience in Regulatory Affairs through positions at Genzyme and AstraZeneca.

NORA PÖNTYNEN

Global Medical Affairs

Boehringer Ingelheim International GmbH

NORA PÖNTYNEN

Global Medical Affairs

Nora is an immunologist with a PhD in rare disease, she has worked in Medical Affairs for over a decade. Her specialty is stakeholder engagement strategies, focused on generating and collecting real world data: going beyond clinical trials. Her prior hands-on experience with the unique Nordic registries and advanced digital healthcare records studies and from planning and leading global managed access programs (MAPs) have led her to develop company-wide MAP strategy and operations. She is currently the Global Capability Owner, Early Access Treatments at Boehringer Ingelheim.

GORDON LUNDIE

Executive Director, Market Access and Pricing

Gilead Sciences

GORDON LUNDIE

Executive Director, Market Access and Pricing

Gordon Lundie joined Gilead Sciences in October 2017, and is their Executive Director of Market Access and Reimbursement for the UK and Ireland. His role is to lead the market access and pricing function for Gilead in the UK and Ireland, ensuring early access for the new treatments developed by the company, to create and implement their pricing strategy, and work with the NHS to manage their effective introduction into clinical practice. Prior to joining Gilead Sciences, Gordon held a range of Director level positions at GSK, Intercept Pharma and UCB in the UK, where most recently he led their market access and corporate affairs teams, supporting their significant UK investment in life science research at their research hub in Slough. Outside of work Gordon is involved in politics, was a local government Councillor for 16 years, and was Leader of West Berkshire Council, a Conservative controlled upper tier authority, from 2012 to 2015. Gordon was educated in Scotland, attending The University of Glasgow and holds a BSc (hons) in molecular biology. He lives in rural Oxfordshire with his wife and two sons.

Pedro Franco

Sr Director, Head, Europe Global Regulatory & Scientific Policy

Merck Healthcare

Pedro Franco

Sr Director, Head, Europe Global Regulatory & Scientific Policy

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.

Sara Radenovic

Director, Managed Access

GSK

Sara Radenovic

Director, Managed Access

Sara is the Director of Oncology Managed Access Programs at GSK. Her career started in clinical development at an academic CRO, after which she spent two years working in a biotech in Berlin. In 2018, Sara joined GSK, and prior to her role in the managed access field, she worked as a Global Clinical Development Manager in late-phase trials. She is a pharmacist by training, and before moving to the industry, Sara had a chance to work in academia and at a clinical trial site level. She obtained her doctorate at the Charité University Hospital in Berlin. Outside work, Sara volunteers in the Healthcare Businesswomen’s Association (HBA) as President for Education and Events for the Northern European region.

Katja Berg

Innovative Value and Access Strategy

AstraZeneca

Katja Berg

Innovative Value and Access Strategy

Katja Berg is a strategic leader in the biotech and pharmaceutical industry with 20+ years of science, management consulting, pricing and market access experience. In her global role of Innovative Value & Access Strategy within the Biopharmaceutical Business Unit at AstraZeneca, Katja is responsible for bringing novel approaches and future-looking access models for AstraZeneca’s portfolio to advance patient access and value-based healthcare. Katja spent her early years in the laboratory and Academia, mostly in Sweden, France and the US, authoring publications while nurturing her scientific curiosity and languages. She then became a senior consulting leader and Global Client Partner for Top 15 pharmaceutical companies for over 10 years at IMS Consulting [today IQVIA], leading among other things thought leadership for the practice. Transitioning from consulting to the biopharmaceutical industry, Katja led a multi-functional global team delivering above brand strategic, operational and policy-related pricing and access activities for Sanofi. During this time, she lead and coordinated Sanofi’s position on external access policy and working groups such as IFPMA and Access Accelerated, including work designing and incubating end-to-end patient access solutions in e.g. Asia and Africa. During her 3 years at AstraZeneca, Katja has delivered global pricing and access launch strategies for assets within the Cardiovascular, Renal & Metabolism (CVRM), Respiratory and Immunology (R&I) and Vaccines & Immune Therapies (V&I) Business Units, as well as acted as Head of Access Policy for the Australia & NZ team in Sydney.

JENNIFER GRIGONIS

Sr. Director Business Development, EAP

Bionical Emas

JENNIFER GRIGONIS

Sr. Director Business Development, EAP

Jennifer Grigonis has a strong background in business development and operations. Jennifer is currently working at Bionical Emas, where they hold the position of Director of Business Development, EAP. Prior to this role, they were an Associate Director of Business Development, EAP at the same company. Jennifer also served as the Business Development Operations Lead and US Proposal and Project Manager - EAP at Bionical Emas. Before joining Bionical Emas, Jennifer worked as a Business Development Operations Consultant at Social and Scientific Systems from 2010 to 2011. Jennifer also held various positions at Medifacts International, including Associate Director of Global Business Development Operations, Senior Manager of Business Operations & Strategic Partnerships, and Senior Proposal Manager. Jennifer started their career at PAREXEL International, where they worked in client relations, specifically in the field of Clinical Pharmacology. Jennifer Grigonis earned a Bachelor of Arts degree in Sociology from Temple University. No specific dates were provided for their education at this institution.

Dr David Gillen

Chief Medical Officer

Norgine

Dr David Gillen

Chief Medical Officer

David is the Chief Medical Officer at Norgine and has been in post since May 2022. David qualified in Medicine, Physiology and Clinical Pharmacology from St. Mary’s Hospital (Imperial College of Science, Technology and Medicine) in 1992. He retained this interest in Pharmaceutical Medicine during his postgraduate training in Cardiology and General Medicine and joined Pfizer Global Research and Development in Sandwich in 1999. After leaving Pfizer David held the role of Head of International Medical Affairs for Gilead Sciences. In 2011 he then moved to work at Celgene where he was Head of Drug Safety and Risk Management for EMEA / APAC for Celgene Ltd. In addition David acted as the QPPV for Celgene’s Pharmacovigilance System in Europe. In 2016 David moved to become Vice President of International Medical Affairs at Vertex. In 2019, he was given additional responsibility supervising the whole International Global Development and Medical Affairs function for Vertex. In 2022 David became CMO of Norgine based in London. David has served on the Innovation sub- committee of the ABPI (Initiating the ABPI Real – World data project), represented Industry on the NICE Appeal Panel and was a member of the important RCP working party looking at the relationship between Industry, Academic Medicine and the NHS. David is a visiting Lecturer at Queens University Belfast and Kings College London.

Dr. Alberto Calabrò

Patient Access Program and Supply Leader

F. Hoffmann-La Roche Ltd

Dr. Alberto Calabrò

Patient Access Program and Supply Leader

Dr. Calabro is a truly European Citizen who dedicated his career to the advancement of patient treatment opportunities in basic research first and then during his 15 years tenure in clinical research. He is fully dedicated to Early Access Programs since he really saw what benefits those mechanisms could bring to patients. In the last 8 years he has been involved in structuring the processes around EAPs at Roche and lately championed their FAIR data use.

Ana Tediosi

Head of Expanded Access Strategy

WEP Clinical

Ana Tediosi

Head of Expanded Access Strategy

With over 10 years of experience in the pharmaceutical industry, Ana is a recognized expert in the field of Expanded Access Programs (EAP). Her expertise lies in oncology, hematology, Rare Diseases, and she has held various roles in the big Pharma (Roche, Novartis, Menarini-Stemline), including leading asset transitions, commercial project lead, PV& compliance. Throughout her career, Ana has consistently demonstrated strong communication skills and a proven track record of successfully driving projects in challenging and international environments. Her commitment to delivering the best healthcare solutions to patients is unwavering, and she is equally passionate about promoting gender equality in both business and society. Ana is currently based in Switzerland working as a Business Development Director and Head of Expanded Access Strategy at WEP Clinical.

CAMERON MILLER

Director of Policy & Strategy

The Brain Tumour Charity

CAMERON MILLER

Director of Policy & Strategy

Cameron has worked for The Brain Tumour Charity for more than six years, having started by leading our support services before moving on to the policy and public affairs department. He recently re-joined as Director of Policy & Strategy following a four year hiatus where he worked in the pharmaceutical industry, helping to launch several medicines both in-house and as a consultant.

SHEELA UPADHYAYA

Chair Elect, Rare Disease Special Interest Group

ISPOR

SHEELA UPADHYAYA

Chair Elect, Rare Disease Special Interest Group

Sheela Upadhyaya is a seasoned life sciences consultant specializing in rare diseases, with over 25 years of experience in the healthcare industry. Her extensive international background covers the UK, including Scotland, the EU 5, and the USA. With a keen focus on healthcare research, service infrastructure, and commercial market access strategy, she excels in delivering innovative solutions while upholding the objectives of publicly funded services. Sheela commissioned rare disease services for NHS England and headed the NICE Highly Specialised Technology program, evaluating medicines and technologies for ultra-rare conditions. Her recent responsibilities at NICE included coordinating the response to the COVID pandemic successfully introducing 12 new therapeutics into the NHS during the COVID pandemic and strategically addressing the Rare Disease Framework. Sheela has co-authored papers on rare disease challenges and HTA methods, presenting at numerous conferences on the subject.

JULIE POWELL

Marketing & Communications Director

Pleco Therapeutics

JULIE POWELL

Marketing & Communications Director

Julie is an experienced commercial professional in the pharmaceutical industry with a proven track record of success in implementing innovative commercial marketing and distribution strategies to drive market expansion globally. Through her management and launch of pharmaceutical products in various therapeutic areas, Julie has developed a deep understanding of the pharmaceutical market and the key factors that contribute to successful product launches. She is also an expert in developing and executing marketing and distribution strategies that are tailored to specific markets and customer segments. She has gained her skills through the management of pharmaceutical products in various therapeutic areas. Julie holds a Master of Business and Marketing Product Management and is ABPI qualified.

DR JAMES DUBOFF

Strategic Partnerships Director

Genomics England

DR JAMES DUBOFF

Strategic Partnerships Director

Dr James Duboff works at Genomics England as Strategic Partnerships Director, driving the translational impact of National genomic healthcare data by partnering with global Pharmaceutical and Biotechnology companies working in Rare Disease and Cancer. He has previously worked in biopharmaceuticals at GSK and R&D management at P&G, with a Masters in Molecular and Cellular Biochemistry from the University of Oxford and a PhD in Genetics from the University of Cambridge.

ROBERT DONNELL

Executive Vice President – Medicines Access

Smartway Pharma

ROBERT DONNELL

Executive Vice President – Medicines Access

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 is a retained consultant to 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.

PAUL BLAKELEY

Sr Policy Advisor, Health and Life Sciences

Tony Blair Institute for Global Change

PAUL BLAKELEY

Sr Policy Advisor, Health and Life Sciences

Paul is an experienced life science and health policy professional, with over 10 years’ experience across the UK government, the NHS and the global life sciences sector. He has worked extensively on expanding clinical research delivery and increasing the adoption and spread of proven and cost-effective innovation within both the UK and global markets. Most recently, Paul has served as Health and Life Sciences lead for the Tony Blair Institute for Global Change, where he has co-authored several reports on the future of UK health and care, developed partnerships with multiple global organisations and supported governments around the world to grow their life sciences sector. Paul is deeply passionate about working with innovators and industry to ensure patients in the UK and beyond get faster access to cutting-edge treatments and technologies.

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!

Lindsay Randall

Founder, CEO, Mother

Arthur’s Quest

Lindsay Randall

Founder, CEO, Mother

Lindsay Randall graduated as a Children's Nurse in 2013, after 10 years in the property market. She worked at the Evelina London tertiary children's hospital across orthopaedics, neurology, cardiology and surgery, before taking on the role of Clinical Educator for Paediatric Surgery. In 2018, Lindsay's first child, age 2, was diagnosed with ultra rare condition SLC6A1 Developmental and Epileptic Encephalopathy. Lindsay founded Arthur's Quest in 2019, a UK registered non-profit, raising awareness and funds to support advancing research and developments for this disease. Lindsay relocated to her local DGH to be closer to home, working in the role of Paediatric Practice Development Nurse at MTW, Kent. She is currently completing a PG Cert in Leading Practice Education. In 2019, having tested negative for the SLC6A1 mutation, Lindsay and her husband welcomed their second child into the world, but SLC6A1 was not finished with them, and against all odds, their daughter was diagnosed in 2020 following increasing seizure activity. Arthur's Quest (www.arthursquest.org) is part of a global network of parent run organisations that work as a consortium of highly motivated CEO's and Trustee's, sharing ideas, progress, research initiatives and resources. They connect patients and families, clinicians, researchers, scientists, drug makers, funders and umbrella organisations with the primary focus of advancing translational science to treat this disease. Last year the organisation officially dual registered to include the name SLC6A1 Connect UK, in line with global partners.

Carole Scrafton

Director & Co-Founder

Flutters and Strutters

Carole Scrafton

Director & Co-Founder

Carole Sian Scrafton, Ba(Hons) MBS(dist), Director / Co-Founder – Flutters and Strutters Patient Advocacy Organisation. Carole has established Flutters and Strutters Patient Advocacy Organisation for chronic / rare illnesses, a patient-driven and focused patient advocacy organisation that is for everyone #notjustpatients. She is a patient with multiple chronic illnesses and a genetic connective tissue disorder, two rare gene mutations, and rare bone anomalies. Connecting different stakeholders from across the medical, clinical, healthcare industries, including digital health, pharma, research and development. She is helping to develop, nurture and advocate for multidisciplinary / multi-stakeholder approaches to all aspects of medical healthcare so that chronic illness & rare disease patients can receive the type of care and treatments that they need. Supporting disease education and awareness through better medical and health communications. Also, actively working on patient engagement projects collaboratively with multi-stakeholder teams with Patient Focused Medicine Development. Carole is a patient speaker, author, and reviewer. She campaigns and advocates at many levels including patient forums, as well as taking part in many global conferences, in person or via the internet. She encourages more efficient patient engagement practises with a view to the better development of therapies and treatments within the pharma, medical, and clinical environments.

Menno A. Kiel

former Global Medical Leader, Research Physician & Health Economist,

Roche

Menno A. Kiel

former Global Medical Leader, Research Physician & Health Economist,

Physician, Health economist & Global medical leader, originally from The Netherlands, currently living in the Zürich area of Switzerland. Lead Roche’s two “CD20xCD3-bispecifics” compassionate use programs (CUPs) in indolent and aggressive B-cell Non-Hodgkin Lymphoma from 2020 to 2023. Formerly of AstraZeneca, Janssen & Actelion, familiar with rare (hemato-)oncology in Medical Affairs. Sympathetic humanist, strong believer in medical ethics and patient’s rights, keen to expand access to innovative medicines to the far corners of the earth.

LORNA PENDER

Patient Group Lead

Clinigen

LORNA PENDER

Patient Group Lead

Dr Lorna Pender is the Global Patient Engagement Lead with Clinigen, a global specialist pharmaceutical services organisation. Dr Pender is an independent dermatology patient expert, a retired acute medicine doctor, an NIHR Research Champion, a medical writer, and a member of the PFMD patient engagement sustainability ESG task force.

HANNAH PLOWMAN

Real World Data Specialist

Clinigen

HANNAH PLOWMAN

Real World Data Specialist

Hannah Plowman is a Real World Data Specialist at Clinigen, leading the collection of patient data as part of managed access programs. Hannah is keen to support patient advocacy in the pharmaceutical space and is part of a patient involvement group herself for a rare disease.

JULIET HULSE

Head of Clinical Operations

Pulse infoframe Inc.

JULIET HULSE

Head of Clinical Operations

Juliet is Head of Clinical Operations at Pulse Infoframe. With over 20 years in clinical research, Juliet has established herself as a leading expert in developing patient centred clinical research strategies for global clients. Juliet's journey began with a strong foundation in cardiology research, eventually specializing in neurology, particularly multiple sclerosis. Her previous role as Executive Director for a mobile research nurse company, saw her spearheading innovative decentralized research nurse strategies and patient advocacy on a global scale, ensuring a focus on enhancing patient recruitment and retention. Her belief in ensuring the patient is central within the trial design process has been pivotal in executing successful research studies including diverse patient populations.

DAN LEWI

Head of Business Development and Patient Advocacy

Pulse infoframe Inc., Co- founder, CATS Foundation

DAN LEWI

Head of Business Development and Patient Advocacy

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