UPDATE SEPTEMBER 8, 2016: The prepublication of
Neuroscience Trials of the Future: Proceedings of a Workshop
is now available online at the National Academies Press.
UPDATE APRIL 5, 2016: Presentations are now available for review at Neuroscience Trials of the Future: A Workshop (Click on Presentations at top right)
Institute of Medicine (IOM), Washington, DC, 3-4th March, 2016
Drs. Richard Keefe and Atul Pande co-chaired the 2-day public workshop that brought together stakeholders from technology companies, academia, NIMH, large and small pharma, CROs, and perhaps most importantly, representatives from both US and European regulatory authorities.
Steve Romano, SVP/CSO of Mallinckrodt Pharmaceuticals and Shitij Kapur, Executive Dean at the Institute of Psychiatry, London, set the scene by detailing the challenges that the industry faces and why the status quo cannot continue. A host of prestigious academic and industry leaders described the search for biomarkers, the RDoC approach as an alternative to DSM for recruitment criteria, novel trial designs and innovative statistical approaches.
The afternoon session on Day 1, moderated by Amir Kalali, Head of the Neuroscience Center of Excellence at Quintiles, brought together key players at the forefront of technological innovation to discuss how the current boom in the consumer market for wearables and mHealth products will be a driving force for changing how clinical trials are conducted in the future: obtaining real-world data in large sample sizes outside of scheduled trial visits; continuous and passive data collection; and definition of novel endpoints that better reflect diseases progression and treatment effects. To capitalize upon these opportunities, the participants also addressed the accompanying challenges of privacy and data security, ensuring the validity and veracity of the data, and reducing the cost of conducting clinical trials in the future.
Day 2 began with an excellent regulatory session moderated by Thomas Laughren, former Director for the FDA Division of Psychiatry Products, with newly-appointed Commissioner of the FDA, Robert Califf, and Director General of the Italian Medicines Agency (AIFA), Luca Pani. Speakers emphasized the need to design clinical trial programs with the product label and clinical practice guidelines in mind, because regulators want to see how an approvable drug fits in the marketplace, both in terms of clinical benefits and comparative effectiveness. Dr. Pani also provided a national payer’s perspective, with a fascinating insight into the AIFA process for reimbursement negotiations, that was supplemented in the afternoon by discussions regarding real world outcomes as the basis for drug pricing and reimbursement. .
In closing a very engaging two days, Dr. Keefe summarized the key take-home messages: we must find a new way of conducting neuroscience trials because the current model is not sustainable. The new and disruptive technologies offer up methods of data collection that have not been available to us before. These methods hold promise not just for clinical trials, but as ongoing methods of patient engagement and obtaining real-world treatment data that will help to address increasing need for comparative effectiveness for regulators and payers. The challenge however, is assuring that we apply the appropriate level of rigor to these new opportunities, so we can be confident that the decisions we make are based not on hope and marketing, but on good science. . And that was the strong message from the regulators: they are open to new methods, new technologies, and new outcomes, but to consider them, we must ‘show them the data’.