1. Placebo / Standard of Care (PSoC)
Overview
Updated: 20 April 2017

2. Welcome to TransCelerate BioPharma Inc.
About TransCelerate
TransCelerate BioPharma Inc. was launched in 2012 as a non-profit organization with a mission to collaborate across the biopharmaceutical research and development community to identify, design and facilitate the implementation of solutions to drive efficient, effective and high-quality delivery of new medicines, improving the health of people around the world.
Our Mission Statement
TransCelerate BioPharma’s mission is to collaborate across the global biopharmaceutical research and development community to identify, prioritize, design and facilitate implementation of solutions designed to drive the efficient, effective and high quality delivery of new medicines.
Our Members
TransCelerate BioPharma is comprised of the world’s leading biopharmaceutical companies, with the vision of accelerating and enhancing the research and development of innovative new therapies. The spirit of innovation and collaboration occurs across TransCelerate Member Companies on Initiative goals.
Our Innovative Leaders
TransCelerate BioPharma is proud to have leadership from major biopharmaceutical companies – who have dedicated their careers to medicine, science and ensuring access to life-saving medicines through efficient and safe research and development.
We are fortunate to have the greatest minds in pharmaceutical R&D dedicated towards this common goal of identifying common issues and model solutions that will drive efficiencies into the R&D process.

3. TABLE OF CONTENTS Initiative Overview Value Proposition
Stakeholder Benefit / Impact
Accomplishments to Date
Upcoming Milestones
Who’s Involved
Additional Resources

4. Placebo / Standard of Care (PSoC)
Unmet Need:
No vehicle exists to maximize value of clinical data collected historically to improve study design, interpret safety signals contextually and improve subject recruitment.
Objective:
To establish a solution to share Placebo and Standard of Care (SoC) data.
Benefits:
Improved clinical trial design, faster clinical trial execution, enable a better understanding of disease, and improve the patient experience in a clinical trial.

5. Placebo / Standard of Care (PSoC)
Vision
Delivery of pooled data. Non-integrated placebo and SOC patient data* to be used to make targeted optimizations on study design.
Delivery of integrated database. Integrated data* to be used to make cross-study safety and study design assessments.
Enablement of modeling capabilities. Non-integrated placebo and SOC patient data* to be used for modeling purposes and replacement/reduction of control Phase III patients.
Approach
Baselined current thinking to identify areas of clinical development that could be improved with this solution.
Established 7 Use Cases to illustrate the value realized in improved study design and execution.
Established Use Case Playbooks to aid usage and development of White Paper.
Procured third party conversion partner to convert data, maintain and launch data repository.
Finalized a White Paper to socialize the solution with global Health Authorities.
Assess metrics to determine impact of, and update and improve the solution.
* NOTE: All data will be from prioritized disease areas aligned to the CDISC/FDA Standards Roadmap. Initial disease areas include: 1) Ulcerative colitis, 2) Diabetes, 3) Schizophrenia, 4) Alzheimer's Disease, 5) Rheumatoid Arthritis, 6) Asthma, 7) Cardiovascular Disease, 8) Chronic Obstructive Pulmonary Disease and 9) Medically Stable Subjects to Assess for Safety Signals.

6. Value Proposition / Benefits When Delivered
Through development of Placebo / Standard of Care…
Unmet Need
Value Proposition
Lack of data reuse: A lot of time and money is spent to recruit a large number of placebo patients and collect data that is used once and not reused for clinical research.
Limited historical data. Sample size calculations and study design optimization rely on limited data which is prone to inaccuracy.
Lack of readily available context. Needed to optimally interpret unexpected safety or laboratory observations.
Use of historical controls generated by the platform could save up to hundreds of patients, years of cumulative trial execution, and millions of dollars for each company.
More accurate study design and power calculations through enhanced model based approaches.
Allow rapid understanding of safety signals (e.g. adverse events, lab data) to better manage patient safety during trial execution.

7. Stakeholder Benefit/Impact
Member Company
Patients
Decreased subject recruitment costs.
Improved protocol development (including inclusion/exclusion criteria).
Reduction in protocol amendments.
Better understanding of disease models to identify the right targets.
Fewer patients required to execute clinical trials.
Better understanding of safety signals.
Improved product speed to market.
More patients can receive the study drug as opposed to the placebo
Regulatory Authorities
Sites
Work closely with Health Authorities to ensure appropriate application of this concept to clinical development.
Increased research into rare diseases.
Reduction in recruitment burden.
Less drug accountability (for trials using standard of care).

8. Accomplishments to Date
Since endorsement as a workstream by the TransCelerate Board of Directors:
Published White Paper: Development and Implementation of a Pharma-Collaborative Large Historical Control Database
Developed PSoC Video to increase awareness ature=youtu.be
Initiated trial submissions and conversions
94 trials have been submitted across 14 member companies
75 trials have been converted across 12 disease areas
62 trials harmonized trials have been delivered across 12 Therapeutic Areas

9. Upcoming Milestones 2017 Objectives
Submit at least 80 trials for conversion by Q3 (for a total of 150 submitted)
Deliver 120 converted trials into the data sharing environment by Q4
Conduct 2 meetings with Health Authorities
Demonstrate meaningful use of the data by at least 3 member companies by Q4
Submit for publication in a peer review journal a position paper by Q3
Academic (Harvard) Review of PSoC Data Initiated

10. Ulo Palm (Allergan) – Operations Committee Sponsor
Who’s Involved
Executive Leadership
Ulo Palm (Allergan) – Operations Committee Sponsor
Workstream Lead
Ed Bowen (GSK)
Core Team
* NOTE: Participating on the Working Groups does not require implement of the Platform solution

11. THANK YOU