Crowdsourcing Clinical Drug Development in a transparent model Tomasz Sablinski M.D., Ph.D. Founder, Transparency Life Sciences Head of Development, Celtic Therapeutics Open Science Summit Mountain View, CA Oct 23, 2011

Perspectives - clinical investigator - basic researcher - biotech consultant - CRO - big pharma - investor - entrepreneur

the drug development industry is facing extinction psychotherapy is not an answer

intensive care is the only hope

“open innovation” the abuse of terminology

Obvious fields: - pre-competitive space - sharing data on: placebo response failed clinical studies

Obstacles to crowdsourcing and transparency in pharma - legal - IP - arrogance of experts - fear for loosing jobs - secretive culture - antiquated computer sciences - development / marketing confluence

Drug Development - Sources of Waste partial list……… 8 Pharma Company Infrastructure + Multiple Patient Visits to Expensive Sites High-Cost Patient Recruitment Insular Approach to Protocol Design + 20 th Century Data Capture & Analyses + = + Attacking the sources of waste

New model: a platform for collecting key information, analyzing it, and building knowledge in a transparent, complete, easy to understand format to enable multiple parties to move a project ahead in a virtual global environment

secrecy = legacy transparency = currency

11 Transparency Life Sciences is a Drug Development Company We acquire IP and pharmaceutical assets from academia, biotech, and pharma, and add significant value by conducting clinical studies via open-source methods, and by executing with unmatched productivity.

Founders Tomasz Sablinski, MD, Ph.D. – Harvard, Novartis, Celtic Rx Marc Foster – FoldRx, Schrodinger, multiple exits in high tech Gareth Hicks, Ph.D. – Cambridge, GSK, Novartis, Tioga Lawrence Steinman, MD – Professor - Stanford, multiple biotechs Advisors Karim Lakhani – Harvard Business School Bernard Munos – Former strategist, Eli Lilly John L. Brown – Former President, Health Properties, Time-Warner Scott Becker – Co-Founder, Invite Media (acquired by Google) Passionate About Changing the World of Drug Development 12

Three major enablers: technology social changes / culture crisis

TLS Approach 14 1.Crowd-Sourcing – an approach to protocol design and data analysis, allowing for input from all available stakeholders “10,000 minds are better than 10” 2.Data & Technology Platform – web-based platform integrating multiple real-time data inputs via telemedicine Enhance trial cost-efficiency, safety, data 3.Transparency – Plans, data, analyses are shared openly, in real time Builds trust in TLS, provides best analyses

How Will it Work in Practice? High-Level View 15 Protocol Design Patient Recruit- ment Study Execution Data Collection Analysis Crowd- Sourcing Online Groups Tele- medicine Secure, Open Vendors More Crowd Input Trial Step Key Leverage Point

Why do people “go to” work? because of the context ($$ is the major incentive)

Why do people contribute? because of their passion for the content ($$ is NOT the major incentive!)

For TLS partnerships are essential Patients web groups Disease foundations Telemedicine companies Genetic testing enterprises Bioinformatics companies Focus: Clinical studies

Welcome to our project collaborative space !