Rare diseases from clinical trials through approval to reimbursement - 2022

Challenges for orphan drugs in rare disease – from clinical trials to marketing authorisation and reimbursement

 

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The panel will discuss the broad scope of challenges for orphan drugs in rare disease starting from clinical trials (evidence generation) to marketing authorisation (by European Medicines Agency) to reimbursement from public funds. Different stakeholders, including patients, decision-makers, pharmaceutical industry and clinical research, will be represented in the discussion panel and they will present their perspectives on this important topic. International versus Polish experience in both availability and affordability issues for orphan drugs will be considered and discussed in details.   

Moderator - Ph.D. Iga Lipska
Speeches (11:00 - 13:00) Speakers
Representative of the Ministry of Health To be confirmed
prof. Jacques Demotes European Clinical Research Infrastructure Network (ECRIN)
Dariusz Żebrowski Patient representative
prof. Joanna Kwiatkowska Medical University of Gdańsk
Michał Byliniak Infarma
Elżbieta Bylina ABM

Moderator

dr n.med. Iga Lipska

Former Research Consortium & Project Manager, Strategic research grant “The implementation of Hospital-based HTA (HB-HTA) in Poland” & Head of Health Care Services Monitoring Department, National Health Fund HQ (public payer), Warsaw, Poland. Co-author of Polish National Medicines Policy for 2018-2022 – governmental strategic document. Former Director of Drug Policy and Pharmacy Department at Ministry of Health, former Economic Committee Member at Ministry of Health. Former Director of HTA Department at Polish HTA Agency

She’s a medical doctor by education with master degree in public health. She graduated from the University of Utrecht in the Netherlands, where she defended her PhD thesis on health technology assessment of new medicines. Iga Lipska has broad international and Polish experience in the healthcare sector, with a main focus in recent years on HTA, drug pricing, health insurance, public health and evidence-based decision making process in health care. Dr Lipska is an Advisory Board member of HTx project at Utrecht University and Core Member of Get Real Initiative Think Tank. She is also a member of International Scientific Committee for Health Technology Assessment international Annual Meeting in Utrecht in  2022. She has been co-chairing International Society for Pharmacoeconomics and Outcomes research (ISPOR) HTA Roundtable Europe for 3 years’ term (from 2019 to 2022). Starting June 2022 she has been co-chairing HB-HTA Interest Group at Health Technology Assessment international (influential global organization) for 3 years’ term.

 

Panelists

jacques demotes
MD PhD Jacques Demotes, MBA

Jacques Demotes is the Director General of the European Clinical Research Infrastructure Network (ECRIN), which he founded in 2004. In this role, he is responsible for the strategy and overall management of the infrastructure with the support of the ECRIN Management Office (located in Paris, France), the ECRIN European Correspondents (located in each Member / Observer country), and ECRIN’s Scientific Partners (i.e., national networks of clinical trial units).

A neurologist and professor of cell biology, Demotes is an advisor to the biology and health research department at the French Ministry of Higher Education and Research. While at ECRIN, he has contributed to numerous initiatives and collaborative projects related to multinational clinical trials. In particular, he chaired the working group that drafted the Organization for Economic Co-operation and Development (OECD) Council Recommendation on the Governance of Clinical Trials.  Prior to ECRIN, he worked as a clinical neurologist and basic neuroscientist, then as director of the clinical investigation centre in Bordeaux.  Jacques Demotes received his MD (with a specialization in neurology), a PhD in neuroscience, a Master of Science (MS) in neuroscience, and a Bachelor of Arts (BA) in mathematics and computer science from the University of Bordeaux. He also received an MBA from IAE Paris, and completed a training course in science policy with the Institute of Advanced Studies in Science and Technology in Paris.

 

Dariusz Żebrowski

Ojciec chorego na Dystrofię Mięśniową Duchenne’a Wojtka.  Od dziewięciu lat uczestniczy z synem w badaniach klinicznych w Paryżu, gdzie ostatnie cztery lata syn testuje badany produkt leczniczy o nazwie Givinostat.  Pasjonat rynku badań klinicznych, propaguje wśród rodziców dzieci chorych na DMD wiedzę o badaniach klinicznych, nowych lekach i samej chorobie. 

Od roku 2020 jest członkiem Stowarzyszenia na Rzecz Dobrej Praktyki Badań Klinicznych w Polsce (GCPpl), gdzie jest Ambasadorem Badań Klinicznych, członkiem Grupy ds. Chorób Rzadkich, a od czerwca 2022 członkiem zarządu. W raz z przyjaciółmi tworzy fundację Stop Duchenne Poland działającej na rzecz chorych na DMD i inne choroby rzadkie.

 

Michał Byliniak

Z  wykształcenia jest farmaceutą, absolwentem wydziału farmaceutycznego Warszawskiego Uniwersytetu Medycznego. Z rynkiem farmaceutycznym związany jest od ponad 15 lat, zdobywając bogate doświadczenie menedżerskie w zakresie dystrybucji, refundacji, badań klinicznych i rejestracji produktów leczniczych. Posiada doświadczenie w pracy w polskim systemie ochrony zdrowia w różnego rodzaju organizacjach, takich jak apteki, hurtownie krajowe, firmy farmaceutyczne oraz firmy konsultingowe. Jest autorem licznych opracowań związanych z sektorem farmaceutycznym. Był wieloletnim prezesem Okręgowej Izby Aptekarskiej w Warszawie, a  także  prezydentem Grupy Farmaceutycznej Unii Europejskiej.