LEAPS Design Lab

July 2018

The first LEAPS Design Lab was held on July 17-18, 2018 at the MIT Samberg Conference Center, 50 Memorial Drive, 7th Floor, Cambridge, MA 02142

The Design Lab’s objectives:

• Advance the evolving vision and strategic plan for the NEWDIGS LEAPS Project, as well as opportunities for deeper engagement following this Design Lab
• Shape the selection criteria for the target disease for the LEAPS Massachusetts (MA) Pilot through a structured analysis of four candidate diseases
• Discuss and advance elements of the Evaluation Framework for the MA Pilot
• Analyze two interactive case studies to inform the prospective design of evidence generation platforms in the MA Pilot

Download the Full Agenda

Attendee Bios

View the slides from day 1 here: Introduction; Evaluation Framework

View the slides from day 2 here: Platforms; GBM AGILE Case Study (pre-market); VA Case Study (post-market)

Target disease selection

On July 25, 2018, we announced our Target Disease Selection. After great discussions during the event, and careful deliberation with the MIT CBI Leadership Team and LEAPS Steering Committee, we are pleased to announce that we will advance rheumatoid arthritis (RA) as the primary target for the MA Pilot project. In addition, building on input from the Design Lab, we think it is important not to put all of our eggs in one basket, so we will advance—albeit at a less aggressive pace—asthma as a secondary disease target, in order to ensure that we have a back-up option if needed. The good news is there was a general consensus in the room that all four diseases considered would generate valuable learnings, and that there was no obvious right or wrong choice. We are thinking of RA as the first pilot, but hope to catalyze other pilots through dissemination of our generalizable principles for application in other disease ecosystems.

References

• Woodcock, J, LaVange, L. Master Protocols to Study Multiple Therapies, Multiple Diseases, or Both,New England Journal of Medicine. 2017;377:62-70.​
• Hirsch G, Trusheim M, Cobbs E, Bala M, et al. Adaptive Biomedical Innovation: Evolving Our Global System to Sustainably and Safely Bring New Medicines to Patients in Need, Clinical Pharmacology & Therapeutics, special issue on Adaptive Biomedical Innovation, published 14 November 2016 DOI:10.1002
• Trusheim M, Shrier A, Antonijevic Z, Beckman R, et al. PIPELINEs: Creating Comparable Clinical Knowledge Efficiently by Linking Trial Platforms, Clinical Pharmacology & Therapeutics, special issue on Adaptive Biomedical Innovation, published 14 November 2016 DOI:10.1002
• Gawer, A, and MA Cusumano. Industry Platforms and Ecosystem Innovation. J Prod Innov Manag 31, no. 3 (September 4, 2013): 417–43. doi: 10.1111/jpim.12105
• Choudary, SP. Platform Scale: How an emerging business model helps startups build large empires with minimum investment. United States: Platform Thinking Labs; 2015
• Parker, GG, Alstyne, MW, and SP Choudary. Platform Revolution: How Networked Markets Are Transforming the Economy - and How to Make Them Work for You. United States: Platform Thinking Labs; 2016

GBM AGILE Case Study (pre-market)

• Vanderbeek AM, Rahman R, Fell G, Ventz S, et al. The clinical trials landscape for glioblastoma: is it adequate to develop new treatments?, Neuro-Oncology, Volume 20, Issue 8, 5 July 2018, Pages 1034-1043, doi: 10.1093/neuonc/noy027
• Stern, AD, and S Mehta. Adaptive Platform Trials: The Clinical Trial of the Future?. Harvard Business School Case 618-025, September 2017. (Revised January 2018.)

VA Case Study (post-market)

• Fiore, LD, and PW Lavori. Integrating Randomized Comparative Effectiveness Research with Patient Care. N Engl J Med. 2016 Jun 2;374(22):2152-8. doi: 10.1056/NEJMra1510057.
• Fiore LD, Brophy MT, Ferguson RE, D'Avolio L, et al. A point-of-care clinical trial comparing insulin administered using a sliding scale versus a weight-based regimen. Clin Trials. 2011 Apr; 8(2):183-95. doi: 10.1177/1740774510395635.
• Lederle FA, Cushman WC, Ferguson RE, Brophy MT, Fiore LD. Chlorthalidone Versus Hydrochlorothiazide: A New Kind of Veterans Affairs Cooperative Study. Ann Intern Med. 2016; 165:663–664. doi: 10.7326/M16-1208
• Fiore, LD, and LW D’Avolio. Detours on the road to personalized medicine: barriers to biomarker validation and implementation. JAMA. 2011 Nov 2;306(17):1914-5. doi: 10.1001/jama.2011.1605.

Related

The Next Wave Forum where LEAPS was first announced
LEAPS Launch on MIT News
Post-Design Lab Press Release