Day 1: When Overall Survival Cannot be the Primary Endpoint
Session Chairs: Denis Lacombe (EORTC, BE) and Ralf Herold (EMA, NL)
Axel Glasmacher (CDDF, DE)
Prof Axel Glasmacher has been an adjunct professor of medicine since 2009 at the University of Bonn, Germany. He worked in Haematology and Oncology from 1989 to 2006, focusing on haematological malignancies (notably, acute leukaemias and multiple myeloma) and infections in neutropenic patients. Prof Glasmacher coordinated the clinical study group “Refractory Multiple Myeloma”, served as Vice-chair and Chair (2002-2006) of Antifungal Therapy at the Paul-Ehrlich-Society for Chemotherapy, and was Editor for the Cochrane Haematological Malignancies Group (2003-2006). He has coordinated and co-authored medical practice guidelines for several scientific societies, including the German Society of Haematology and Oncology and the British Society of Haematology. Prof Glasmacher worked from 2006-2018 as Medical Director, Germany, Vice-President Medical Affairs (Europe, Middle East, Africa), and Head of Global Clinical Research and Development Haematology-Oncology. In these roles, he developed risk management plans for thalidomide and lenalidomide, and clinical development and regulatory submissions for drugs such as lenalidomide, azacitidine, enasidenib and luspatercept. Prof Glasmacher established an independent consultancy in March 2018, focusing on drug development, medical communication, and leadership development for medical and scientific talents in the pharmaceutical industry. These experiences have facilitated Prof Glasmacher’s engagement in the drug development process from different viewpoints. His understanding of the scientific, economic and people-oriented perspectives in this arena allow him to make a valuable contribution to current research interest in drug development at the interface between industry, healthcare structures and society.
Chitkala Kalidas (Bayer, USA)
Chitkala Kalidas, Ph.D., is Vice President and Head of Global Regulatory Affairs for Oncology and in vitro diagnostics at Bayer. Dr. Kalidas leads a team of Global Regulatory Strategists who are responsible for developing appropriate global strategies for programs in all stages of drug development and for the registration of in vitro diagnostics as well. Programs in her group include VITRAKVI® (larotrectinib), STIVARGA® (regorafenib), XOFIGO® (radium-223), ALIQOPA® (copanlisib), NEXAVAR® (sorafenib) and NUBEQA® (darolutamide), among others. Precision medicine is an area of interest for her group as well as novel development strategies including tissue agnostic development, novel clinical trial designs, pediatric development in Oncology, and drug development in special populations. Dr. Kalidas has over 18 years of experience in drug development. Prior to joining Bayer, Dr. Kalidas was at Merck & Co. Inc. where she worked across multiple therapeutic areas and geographic regions. Prior to joining the pharmaceutical industry, she was a Management Consultant at the Boston Consulting Group. Dr. Kalidas has a Ph.D. in Microbiology from Cornell University.
Elmar Schmitt (Merck Healthcare KGaA, DE)
Elmar gained a diploma in chemistry with a doctorate in experimental toxicology. He finished his post-graduate Master in Drug Regulatory Affairs at the University of Bonn (DGRA/MDRA). He is a professional in Global Drug Regulatory Affairs, Merck in Darmstadt since March 2002. Elmar’s current position is an Executive Director at Global Regulatory Oncology since 2019. He is a Global Regulatory project leader in the Therapeutic Area Oncology for early and late-stage development projects & group leader of Global Regulatory Strategist in the Therapeutic Area of Oncology. Elmar contributed to several publications over the last years such as Strategic considerations for implementing the revised EMA guideline on FIH clinical trials (2019), Clinical Development and Initial Approval of Novel Immune Checkpoint Inhibitors in Oncology: Insights From a Global Regulatory Perspective (2018), and A sponsor’s experience with the Voluntary Harmonization Procedure for clinical trial applications in the European Union (2011).
Filip Josephson (EMA, SWE)
Filip Josephson M.D., Ph.D. has been employed at the Swedish Medical Products Agency (MPA) since 2008. His main area of prior scientific endeavor and expertise is the pharmacology of antiviral agents. Since 2014 he has served as a representative of the MPA at the Committee for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA). As such, he is extensively involved in the evaluation of medicinal products, as well as regulatory policymaking, in the therapeutic areas of oncology and infectious diseases.
Day 2: Endpoints in Expedited Approval Pathways
Session Chairs: Axel Glasmacher (CDDF, DE) and Serban Ghiorghiu (AstraZeneca, UK)
Hans Scheurer (MPE, NL)
Hans Scheurer is president of the pan-European organization Myeloma Patients Europe (MPE) since 2016. He is a myeloma patient since 2005, and active as a patient advocate for the myeloma community since 2008. Being an educationalist, he started writing for patients about medical developments and all innovation in the myeloma field, with a passion for understandable language for patients. In his current role, he focuses on good governance of the patient community of MPE and mentoring/coaching new patient groups across the EU, with the goal of shaping meaningful patient involvement and, and in general the professional development of patient advocacy. He is active in many consortia representing the patient perspective, like EMA procedures and committees, the WECAN consortium of Cancer patient umbrella organizations, the HOVON Myeloma workgroup in the Netherlands, SISAQOL-IMI.
Vishal Bhatnagar (FDA, USA)
Vishal Bhatnagar is a medical oncologist and Associate Director of Oncology Patient Outcomes at the Oncology Center of Excellence (OCE) at the U.S. Food and Drug Administration. His clinical focus is in multiple myeloma. Prior to his time in OCE, he was a primary reviewer and team leader in the Division of Hematology Products (DHP), where he reviewed myeloma product applications and advised sponsors on efficient drug development. His current regulatory work focuses on management of the OCE’s patient-focused drug development program. His interests include patient-reported outcomes, patient preference, and incorporation of patient experience in oncology trials. Dr. Bhatnagar received his B.A. in Political Science and his M.D. at the George Washington University. He completed his internal medicine residency and hematology/oncology fellowship at the University of Maryland.
Carole Longson (NICE, UK)
Carole is a respected leader in health technology assessment with considerable achievements in research, public and private sectors. She has unparalleled insight into the fast-evolving health technology innovation pipeline having pioneered NICE’s health technology evaluation programs. Carole was an Executive Director at NICE from 2000-2018 and Chief Scientific Officer at the Association of the British Pharmaceutical Industry from 2018-2020. She was formerly President of Health Technology Assessment International and now has advisory roles including Life Science Advisor at NICE. Carole is currently vice-chair of the Medicines Discovery Catapult in the UK, was previously on Scientific Advisory Committee for Innovative Medicines Initiative (IMI), and holds non-Executive Director and advisory roles in scientific endeavors in the UK and abroad. Throughout her career, she has championed the public and private sector collaborating to ensure medical innovation benefits patients in a way that is sustainable for healthcare systems.
Day 3: PRO Endpoints – Review of Strategies
Session Chairs: Anne-Sophie Darlington (EORTC, UK), Michael Zaiac (Novartis, CH)
Jayne Galinsky (MPE, UK)
Jayne is Head of Patient Evidence at the patient organization Myeloma Patients Europe and has a Ph.D. in Health Sciences from the University of Stirling. She is interested in the impact of rare and life-threatening conditions and generating evidence to support access to health technologies that improve the quality of life for individuals and families. Jayne previously worked as the Health Services Research Manager at Myeloma UK, as a lecturer in Psychology at the University of Stirling, and on clinical trials at the University of Glasgow (Neurology) and University College London (Institute of Child Health). She is an honorary research fellow at the University of Stirling. She has published work in the fields of patient preferences, the economic and societal impacts of disease, perceptions of patient-reported outcomes (PROs), and patient and carer perceptions of clinical neurology trials in stroke. She lives and works in Edinburgh, Scotland.
Vishal Bhatnagar (FDA, USA)
Vishal Bhatnagar is a medical oncologist and Associate Director of Oncology Patient Outcomes at the Oncology Center of Excellence (OCE) at the U.S. Food and Drug Administration. His clinical focus is on multiple myeloma. Prior to his time in OCE, he was a primary reviewer and team leader in the Division of Hematology Products (DHP), where he reviewed myeloma product applications and advised sponsors on efficient drug development. His current regulatory work focuses on the management of the OCE’s patient-focused drug development program. His interests include patient-reported outcomes, patient preference, and incorporation of patient experience in oncology trials. Dr. Bhatnagar received his B.A. in Political Science and his M.D. at the George Washington University. He completed his internal medicine residency and hematology/oncology fellowship at the University of Maryland.
Paul Kamudoni (Merck Healthcare KGaA, UK)
Paul is a Scientific Director for Patient-Centered Research at Merck Healthcare KGaA, Darmstadt, Germany, working in the R&D function, where he supports the design and implementation of strategies to integrate patient input and patient-centered measurements in clinical programs as well as post-launch studies. Paul currently co-chairs the Critical Path Institute PRO Consortium MS Working Group, which is working on the qualification of patient-reported measures in MS as drug development tools (DDTs) at the FDA. Before joining Merck Healthcare KgaA, Paul worked as a research consultant for several years. Paul earned his Ph.D. in health outcomes measurement at Cardiff University in the UK in 2014 and is an author of recently published a book on PROs – Living with Chronic Disease: Measuring Important Patient-Reported Outcomes – a practical guide in PRO measure development and patient engagement (2018). Main research interests: methodological research on how to capture, structure, and apply patient input and patient experience in drug development, Use of modern test theory in PRO measure development and application, Integration of qualitative research in clinical trials.
Leeza Osipenko (London School of Economics, UK)
Leeza Osipenko, Ph.D. is a CEO of Consilium Scientific and a Senior Lecturer in Practice at the LSE Department of Health Policy. After completing her Ph.D. in Systems Engineering in the US, Leeza moved to the UK and worked as Senior Research Fellow at the University of Warwick Medical School. This experience was followed by two years at a public sector consultancy, Optimity Matrix where she was a Principal Economist. Leeza led NICE Scientific Advice between 2014 and 2018. She chaired over 150 advisory meetings at NICE and at the EMA and provided methodological, operational, and strategic leadership for the team which became well-known internationally. During those years Leeza represented NICE as a chief analyst at the Department of Health Appraisal Alignment Working Group. She is an expert in Health Technology Assessment. Currently, her research work focuses on examining the status quo of clinical research, through quality improvement, transparency, and methodological rigor. In particular, Leeza is interested in clinical data integrity, health policy, applied clinical research, and clinical trial design.
Corneel Coens (EORTC, BE)
Corneel Coens is currently working as a lead statistician at the EORTC Headquarters (Brussels, Belgium). As part of his tasks for the EORTC Quality of Life Department, he is involved in various aspects of Quality of Life in many oncological clinical trials ranging from instrument validation to design, analysis, and reporting of QoL endpoints. He is one of the lead investigators in the international SISAQOL initiative. He is also the designated study statistician for the EORTC Gynaecological Cancer Group since 2001. He is a regular contributor for various EORTC educational courses such as “Clinical Trial Statistics for Non-Statisticians” and has been course director for the Joint FECS/AACR/ASCO Workshop “Methods in Clinical Cancer Research”.