December 12, 2024 5pm London, UK time
This panel will critically examine the recent FDA approval processes for oncology and Alzheimer's treatments. Can a case be made for “regulatory capture?” This discussion will highlight specific cases where the FDA's decisions have sparked controversy, focusing on concerns about the accelerated approval pathways, the reliance on surrogate endpoints, and the pressure to approve drugs with limited efficacy and safety data. Experts will dissect the balance between the fear-driven alleged urgency to provide new treatments to patients and the necessity of maintaining rigorous standards to ensure these treatments' safety and effectiveness. By exploring and comparing these high-profile examples, the panel aims to shed light on systemic issues within the FDA's regulatory framework, debating the influence of pharmaceutical lobbying, the potential for conflicts of interest, and the broader implications for public health. This dialogue seeks to provide actionable insights into how the FDA can reform its approval process to better serve patients and the medical community.
Speakers:
Chair: Peter Whitehouse, MD, PhD
John H. Powers, MD (George Washington University School of Medicine)
Joel S Perlmutter, MD (Washington University St Louis)
Antonio Tito Fojo, MD (Columbia University)
October 17, 2024 5pm London time
Video
Chair: Dr. Leeza Osipenko, Consilium Scientific
Speakers:
Prof John Hickman (Consilium Scientific)
Prof George Perry (University of Texas at San Antonio)
Prof Rudy Tanzi (Harvard University)
Prof John Lazo (University of Virginia)
Cancers and Alzheimer’s disease are the major health challenges of our era, with cases predicted to rise dramatically over the next decade. Both age-associated diseases share the characteristic of having multiple genes implicated in the onset and evolution of their complex pathologies. In the case of cancer, sequencing the DNA from human malignancies has revealed aberrantly activated genes (mutations and/or translocations) that drive malignancy. These present disease biomarkers and drug targets. Similarly, multiple genetic alterations and biomarkers have emerged for Alzheimer’s disease.
Failure of early- and late-stage clinical drug candidates is a commonality between cancer and Alzheimer’s disease, reflecting the challenges of target validation and the inherent limitations of preclinical models. Should the past decades of biomarker identification, imaging, and drug discovery in oncology act as a robust model for securing advances in the diagnosis and therapy of Alzheimer’s disease? Does an understanding of cell death/apoptosis mechanisms provide opportunities in both diseases, where Alzheimer’s is characterized by post-mitotic cell death and cancer often by stubborn cell survival and replicative potential? Does the inflammation associated with both diseases point to the potential of novel immunological approaches? In this dialogue, experts in cancer pharmacology will be joined by experts in the molecular pathology and treatment of Alzheimer’s disease. They will discuss the question of whether progress in the diagnosis and treatment of cancer could be a template for making advances in Alzheimer’s disease.
November 14, 2024 5pm London UK time
Chair: David Crosby, PhD
Speakers:
Sir John Burn, PhD
Katie Spencer, PhD
Nick Wooley
Matt Fagg
An estimated 22 million people worldwide will have cancer in 2030. Policy goals of reducing cancer mortality often default to a discourse of advancing therapeutic strategies, strongly supported by the pharmaceutical industry. However, the impact of cancer therapeutics on survival remains limited. A cancer diagnosis and its treatment, successful or not, impose a heavy burden on patients and their loved ones. Preventing cancers from arising would reduce mortality and the negative social and psychological impact of this disease. Current estimates suggest that at least 30-50% of cancers are preventable with opportunities to go further by better understanding the underlying mechanisms of cancer genesis and risk. In this shared Cancer Research UK and Consilium Scientific webinar, experts in broad aspects of health policy and economics will discuss the historical funding limitations of cancer prevention research compared to therapeutics. What are the current regulatory and economic barriers to advancing cancer prevention? Do we need novel models of health economics to promote cancer prevention? What are the challenges of behavioural and interventional prevention strategies? How can they be overcome? Join us at this webinar focused on highlighting the growing global importance of cancer prevention and the vast opportunities ahead.
Video
September 19, 2024, 5:30pm London time
Video
Many expensive oncology medicines which have entered clinical practice have resulted in lucrative profits for pharma, an excessive financial burden for healthcare systems and questionable benefits for patients. Recent approvals of new Alzheimer’s drugs were about to set a similar trend in neurological diseases but were timely questioned.
The panel discussion will delve into the striking parallels currently observed in the efforts to capitalize on oncology and Alzheimer`s drugs, emphasizing the significant investments being poured into these areas and the rush to bring these medicines to market. The panel will explore trends such as accelerated approval pathways, breakthrough designations, and the intense competition to lead the market.
Additionally, the discussion will address controversies surrounding the balance between innovation and patient safety, the ethical implications of fast-tracking drugs with limited long-term data, and the potential for financial incentives to overshadow rigorous scientific validation. The role of regulatory bodies, especially the FDA, will be scrutinized as to who they actually serve. This conversation aims to provide a comprehensive overview of the current landscape, highlighting both the promise and the pitfalls of these high-stakes medical advancements. The panel will also focus on the need for a broader approach in preventing both conditions, advocating not only for lifestyle changes, but more importantly public health and environmental interventions over an exclusive focus on molecular solutions.
Speakers:
Chair: Dr. Leeza Osipenko, Consilium Scientific
Dr. Michael Kolodziej (ADVI Health)
Prof Aaron Kesselheim (Harvard University)
Prof Sharon A. Brangman (SUNY Upstate Medical University)
Prof Peter Whitehouse (Case Western University)
September 5, 2024, 5pm London time
Waste in cancer research has been disappointing to many whilst allowing significant capitalisation for pharmaceutical industry. Many improvements have been made and many more are yet to come. How should cancer research evolve? How can we decrease the waste and ensure better selection of pre-clinical models moving to clinic? Will we need phase 3 trials and what role RWE evidence will play? Will synthetic comparators give answers clinical practice needs? How can we overcome current incentives in the oncology market and stop over-production of expensive me-toos?
Speakers:
Chair: Dr. Leeza Osipenko
Dr. Marty Tenenbaum (Cancer Commons)
Dr. Julian Adams (Stand Up to Cancer)
Prof. Ian Tannock (University of Toronto)
Dr. Jonathan Kimmelman (McGill University)
Video
15 June 2023 5pm BST
Video | Slides | Transcript | Podcast
If you live long enough, either you will have Alzheimer’s yourself or it’ll be one of your relatives or one of your friends or… maybe more than one. This is a public health issue of the 21st century which is getting very personal. Impressive failures in all our attempts to figure out the mechanism of the disease and find any disease modifying solutions have been disappointing. Where are we today? What are the new -mabs which are making the headlines? Is this a hype promising billion-dollar earnings to Pharma or is this a hope for disease modification?
Discussants:
Professor Aaron Kesselheim (Harvard Medical School)
Professor Peter Whitehouse (Neurology, Case Western Reserve University)
Professor Frank Harrell (Biostatistics, Vanderbilt University)
Dr Richard Oakley (Alzheimer’s Society, UK)
Read:
Rare Alzheimer's Mutation Delays Onset of Disease Nature | Vol617 | 25May2023
9 November 2023 17:00 BST
Chair: Clare Turnbull, MD, PhD
Susan Domchek, MD
Peter Sasieni, PhD
Rebecca Beeken, PhD
Bethany Shinkins, PhD
David Crosby, PhD (CRUK)
Kate Hamilton-West, PhD
Cancers are a leading cause of mortality, accounting for nearly 10 million annual deaths worldwide, or 1 in 6 deaths. It has been estimated that at least 40% of cancers are preventable. Recent cancer policies from both the USA (The Cancer Moonshot), Europe (Conquering Cancer-Mission Possible) and Cancer Research UK (The Future of Cancer Prevention) have an increased focus on cancer prevention. For Consilium Scientific, a panel with medical, scientific, policy, economic and behavioural science expertise will discuss the opportunities for and challenges of promoting cancer prevention. What emerging scientific insights could guide novel strategies for prevention? How can a strong population-led demand for cancer prevention be generated? What economic and societal changes are required to support this in order to reduce cancer mortality? How will gains in reduced cancer burden be attributed to preventative interventions? After the panel discussion, time will be allotted for questions and further discussion.
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December 7, 2023 5pm BST
Chair : Ewan Birney FRS (Sanger Institute)
Clare Turnbull MD PhD (Institute of Cancer Research, London)
Paul Pharoah MD PhD (Cedars-Sinai, Los Angeles)
Anneke Lucassen MD DPhil (Nuffield Dept of Medicine, Oxford)
Raghib Ali, MD (Cambridge University)
Aroon Hingorani PhD (University College London)
Sir Peter Donnelly PhD (University of Oxford)
It has been proposed that polygenic scores may enable better targeting of interventions for disease prevention and early detection, reducing cost, improving efficiency and extending opportunities into new age groups and even new diseases. In the UK, the Our Future Health Programme is being rolled out this year in conjunction with NHS England. This programme will focus specifically on SNP-genotyping and generation of polygenic scores in up to 5 million adult NHS users: patients will be offered opportunity to receive their results. Are polygenic scores "ready for primetime"? In which diseases might they be useful? Are additional research studies required? What are the possible harms of PGS-stratified screening/interventions? These questions will be discussed by a distinguished panel of experts chaired by Ewan Birney.
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4 May 2023
Under United States and European Union laws, sponsors who do not make certain clinical trial results public risk fines and, in extreme cases, prison. The UK plans to introduce a law allowing the drug regulator to withhold approval for new trials if old trial results are not made public. Meanwhile, research funders across the world are
increasingly starting to monitor trial reporting and are considering introducing sanctions. But are such sanctions really a good idea? In this lively debate, four experts agree that transparency is important - but strongly disagree on whether sanctions are the best way forward.
Video | Transcript
27 April 2023 2:30pm BST
This event brings together a diverse mix of actors to jointly brainstorm new ways to speed up and improve clinical trial reporting.
What can we learn from the successes and failures of the past? What new tools and models are available? How can we mobilise other groups to drive political and policy change?
Video | Transcript
Moderated by:
Emma Thompson, Cochrane
Discussants:
Stephanie Mueller-Ohlraun, Germany
Nicholas DeVito, UK
Delwen Franzen, Germany
Claire Veryard, ISRCTN trial registry
Gustav Nilsonne, Sweden
Valerie Labonte, Germany
Dinesh Thakur, India
Florian Naudet, France
08 September 2022 at 18:00-19:00 CEST (Berlin time) / 17:00-18:00 London time
Video | Transcript
The global network of 17 clinical trial registries provides the world’s only comprehensive overview of who is researching which medical treatments. Whether it’s a funding agency trying to decide where to target its resources, or a patient trying to find trials to enrol in, registries are indispensable. But the entire system is riddled with inconsistent, incorrect and out-of-date data, slowing down trial recruitment, undermining health policy making, and fuelling costly research waste. This seminar brings together registry managers and data users to jointly brainstorm realistic and practical options for improving individual registries and the system as a whole.
Participants:
Till Bruckner, PhD
Senior Policy Researcher
Till Bruckner is the founder of TranspariMED, a campaign that works to end evidence distortion in medicine, and a Research Fellow at the QUEST Center for Responsible Research. His work focuses on regulatory transparency and clinical trial registration and reporting. He previously worked for the AllTrials campaign, the Transparify think tank initiative, the anti-corruption group Transparency International, a commercial microfinance consulting company, and various international development NGOs. Till holds a PhD in political science from the University of Bristol.
Nicholas DeVito, DPhil
Nicholas DeVito is a postdoctoral researcher in health policy at the Bennett Institute for Applied Data Science at the University of Oxford. Nick recently completed his DPhil at Oxford on the impact of clinical trial registries on transparency and accountability with a particular focus on compliance with legislative requirements to report in the US and EU.
Perrine Janiaud
Perrine Janiaud is a researcher at the Research Center for Clinical Neuroimmunology and Neuroscience Basel and at the Department of Clinical Research at University of Basel and University Hospital Basel, in Switzerland. She is a trained clinical epidemiologist and her research focuses on clinical trial methodology, more specifically pragmatic trials, real world evidence and more broadly meta-research. She was one of the lead investigators for COVID-evidence a living database of all planned, ongoing, completed, and published COVID-19 randomized controlled trials.
Gayatri Saberwal
Prof. Gayatri Saberwal is a faculty member and Dean at the Institute of Bioinformatics and Applied Biotechnology (IBAB) in Bangalore. She did her PhD in the life sciences at the Centre for Cellular and Molecular Biology, Hyderabad, and then did post-doctoral work at at the Weill Medical College of Cornell University, New York. She has worked on various aspects of public health policy, and is currently focussed on determining what clinical trial registries can, and cannot, tell us.
Thomas Wicks
Thomas Wicks is the Head of Data and Partnerships at Informa Pharma Intelligence, where he is responsible for the organization’s data governance and interoperability as well as tracking regulatory requirements and clinical data sharing trends that shape the company’s clinical transparency solutions and services. He has over 25 years of experience with compliance management solutions, specializing in applications for life sciences with a focus on clinical trial disclosure and transparency since 2007.
C Marc Taylor
Marc Taylor chairs the not-for-profit company which owns the ISRCTN registry. Based in the United Kingdom, ISRCTN is a primary clinical trials registry in the WHO system. BMC, part of Springer Nature, maintains and publishes it under contract. Marc has chaired ISRCTN since he retired in 2011. For ten years, Marc led system reforms in the regulation, governance, management and funding of health research in England. He was a member of the team at the Department of Health which established the National Institute for Health Research and the Health Research Authority. As an official he took part in setting up the UK Research Integrity Office and the ISRCTN registry. Until 2022 Marc was a member of the HRA’s audit and risk committee. His earlier career in the Department of Health gave him a strong background in health service finance, building on what he learnt in the 1980s as a budget holder for official British development assistance programmes. He headed the development section of the British High Commission in India in 1986-9. Marc is a British and French citizen.
Deborah Zarin
Deborah Zarin is currently the Director of the Program for Advancement of the Clinical Research Enterprise at the MRCT Center of Brigham and Women’s Hospital and Harvard. Prior to that, she was the Director of ClinicalTrials.gov. Dr. Zarin has focused her career on applying evidence to improve the quality of critical clinical, policy and research decisions. Specific positions have enabled her to focus on clinical decisions in psychiatry, payment and coverage decisions for the Medicare program, and overarching issues related to the reporting and conduct of clinical trials.
As the Director of ClinicalTrials.gov at the National Institutes of Health, she led the development of the world’s largest trial registry, contributed to a series of policy initiatives in the US and internationally designed to improve the reporting of clinical trials, developed the first ever structured database of summary trial results, and oversaw the implementation of Section 8 of the Food and Drug Administration Amendments Act (FDAAA) that mandates the reporting of most clinical trials conducted at US academic medical centers, as well as trials conducted by industry throughout the world.
In addition to serving as a key advisor for the various policies and overseeing the development and operations at ClinicalTrials.gov, she led a research program focused on improving trial reporting policies, and using data generated from ClinicalTrials.gov to analyze issues within the clinical research enterprise that interfere with the optimal generation of evidence to guide clinical and policy decisions.
Since joining the MRCT Center, Dr. Zarin has focused on improving the quality of clinical trials, as well as the quality of clinical trials reporting. She worked with colleagues to develop a framework for understanding the sources of uninformative (low value) clinical trials and is now engaged in several research projects to identify points of leverage for decreasing the initiation of low value trials.
Thursday 26 January 2023, 15:30-17:00 Berlin time (14:30-16:00 London time)
Video | Transcript ENG | Transcript GER
Slides: Introduction to the problem in Germany | A possible solution for Germany
The UK is the first country worldwide to set up a watertight national system that will ensure that all interventional clinical trials are pre-registered and make their results public. In future, the UK ethics regulator will directly register all trials immediately after they receive ethics approval, and will later check whether their results were made public, ending research waste once and for all. Quick overview of the UK model here.
In many other countries, there has been significant progress in curbing medical research waste in recent years, but that progress has largely been limited to the minority of clinical trials that are subject to legal reporting requirements. For most other trials, the problem continues unchecked. For example, a recent study of German university trials found that 29% had never made their results public, wasting public money, slowing down scientific progress, and undermining public health.
This live discussion organised by the TranspariMED campaign is bringing together some of the key players involved in developing the UK’s pioneering system to discuss with experts from other countries how this model could be applied to other jurisdictions, with a particular focus on Germany.
All attendees are strongly encouraged to contribute questions and comments during the debate.
Marc Taylor:
In 2011-2022, Marc Taylor chaired the not-for-profit company which owns the ISRCTN registry of clinical research. ISRCTN, based in the UK, is a primary registry in the World Health Organization's system of clinical trial registries.
Before leaving government service in 2011, Marc was one of a team of British officials who established the National Institute for Health Research and related systems, including the Health Research Authority, to enable high quality health research in England.
In earlier roles in government, he was responsible for finance policy development and budgets in health care and overseas development assistance.
Christoph Stein:
Christoph Stein studied Medicine at Ludwig-Maximilians-University (LMU) München, Germany, and underwent specialty training in Anesthesiology, Pain Medicine and Neuropharmacology at State University of New York, University of California Los Angeles, LMU and Max-Planck Society. From 1992-97 he served as Professor at Johns Hopkins University and the National Institute on Drug Abuse (Baltimore, USA). In 1997 he assumed the Chair of Anaesthesiology and Intensive Care Medicine at Freie Universität Berlin, Germany (since 2003: Charité Campus Benjamin Franklin). He is a board member of TI-D and represents its working groups on Science and Health Care.
Martin Smith:
Martin Smith worked as a Specialist for the UK House of Commons Science and Technology Committee from 2016 -19, leading on a range of inquiries for the Committee – including on research integrity and
clinical trials transparency. Martin has worked in policy roles at a range of UK learned societies, including the Royal Society and the London Mathematical Society. He is now Head of Policy Lab at the Wellcome Trust.
Valerie Labonte:
Valerie is originally a biologist and is now working as a scientific staff member at Cochrane Germany and the Institute for Evidence in Medicine at the university clinic Freiburg, Germany. Besides working on evidence synthesis, Valerie is part of Cochrane Germany’s advocacy team, currently working on trial transparency.
