In the largest and perhaps the most ambitious collaborative

FEATURES

THE BIRTH OF TRANSCELERATE BIOPHARMA, INC. Revolution in Clinical Research Partnerships by Dalvir Gill and Garry Neil

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n the largest – and perhaps the most ambitious – collaborative effort ever initiated to improve the quality of clinical studies and accelerate the development of new medicines, ten of the world’s largest healthcare and pharmaceutical companies agreed this past September to join forces and create a new ‘stand-alone,’ US-based, non-profit entity, TransCelerate BioPharma Inc., to better identify and address common pre-competitive drug development challenges facing the industry. The ten founding members of the new organization included five American (Abbott, Bristol-Myers Squibb, Eli Lilly and Company, Johnson & Johnson and Pfizer) and five European (Astra-Zeneca, Boehringer Ingelheim, GlaxoSmithKline, Roche and Sanofi) companies. Through their collaboration in TransCelerate, the founding members seek to advance innovation in research and development (R&D), to identify and solve common R&D challenges, and to further improve patient safety, with the goal of delivering more high quality medicines to patients. Acknowledging the widespread alignment among the heads of R&D at most pharma companies that there is a critical need to substantially increase the number and quality of innovative new medicines while eliminating inefficiencies that drive up R&D costs, the founding members also have agreed upon the urgent need to take concerted action to remedy the many ails of the current system. “Our mission is to work together across the global research and development community and share research and solutions that will simplify and accelerate the delivery of exciting new medicine for patients,” stated Dr. Garry Neil, partner at Apple Tree Partners, head of R&D at its company Braeburn Pharmaceuticals and founding chief executive of TransCelerate BioPharma at the time of the organization’s launch. “My colleagues from the member companies of TransCelerate and I have an unwavering commitment to assessing, refining and creating processes to bring innovative new medicines to the public faster,” adds Dalvir Gill, PhD, the current Chief Executive Officer appointed to lead TransCelerate effective January 1, 2013, and who now works closely with Dr. Neil, who

has remained with the organization as Chairman of the Board of Directors. Under TransCelerate, each member company has agreed to specific outcome-oriented deliverables and accepted clearly defined responsibilities. Each company also has agreed to commit significant financial resources and personnel, and to establish guidelines for sharing meaningful data and information among members in a way that will advance the collaborative efforts without disclosing any competitively sensitive information. To that end, TransCelerate’s members have identified clinical study execution as the new company’s initial area of focus, as this area is one of several key areas of R&D where it is believed there is significant potential to shorten timelines and reduce costs by working together to eliminate redundant efforts and remove process bottlenecks. As the current R&D system has evolved, the cost and time – and associated risk – to bring a new therapeutic medicine to market continues to increase for a variety of reasons including regulatory pressures, the increasing complexity of data, and the cost and complexity of healthcare systems. TransCelerate is a collaboration that in effect, rejects the concomitants of that evolution, and that will work aggressively to solve these challenges and speed the development of innovative therapies, while eliminating R&D inefficiencies and redundancies. As a combined entity, it will draw upon several key strengths: broad industry participation, standardized approach, lean and efficient operations, results-oriented focus and sufficient funding. According to sobering statistics from the 2012 annual SPRING 2013 - ISSUE 4

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report for the industry group, Pharmaceutical Research and Manufacturers of America (PhRMA), it now takes 10 to 15 years for a drug developer to negotiate the process from finding a compound to getting it approved by regulators,1 and the average cost of bringing a new drug to market has escalated from about $140 million in the 1970s1 to more than $1.2 billion,1 including the cost of failures.1 More troubling, for every 5,000 to 10,000 compounds that enter the discovery pipeline, only five make it to clinical trials and only one receives approval from the US Food and Drug Administration (FDA),2 and postapproval, only two of 10 marketed drugs return revenues that match or exceed R&D costs.1 In a recent annual analysis from PhRMA, President and CEO John J. Castellani summed up the current R&D dilemma in his annual message to the industry, “The biopharmaceutical industry faces many hurdles. The cost of developing new medicines has escalated, in part due

to the focus on more complex conditions and increasing regulatory requirements. Market conditions have also become more challenging, and generics now account for 80% of prescriptions filled.3” Looking forward, however, Castellani is far more optimistic, echoing many of the sentiments expressed by the TransCelerate members, “The industry is well focused on both the scientific potential and the business challenges. Companies are working to adapt to the changing conditions through reorganized R&D structures; more efficient drug discovery methods.3” Motivated by this demonstrated need for greater innovation, quality, and efficiency in clinical development, TransCelerate and its members identified five ‘priority’ projects for funding and development, to help rebuild the existing clinical trial process, and create a robust infrastructure for future trials

The Pharmaceutical Research & Development Process Developing a new medicine takes an average of 10-15 years

Pharmaceutical Research and Manufacturers of America

The Five Initiatives, all of which are now underway, are: 1. Establishment of a standardized approach for highquality risk-based site monitoring. Currently, risk to patients is measured in clinical studies without benefit of uniform guidelines for reporting low, medium and high levels of risk. By developing an industry-wide standard and approach for risk-based monitoring of clinical trials, TransCelerate’s objective is to both enhance patient safety and ensure the quality of clinical data. To facilitate this initiative, TransCelerate will consult with the FDA and European Medicines Agency (EMA) in key areas. [For example, TransCelerate would request feedback to ensure data standard scoping matches with regulatory guidance, and receive their input via the FDA’s Coalition for Accelerating Standards and Therapies (CFAST) Steering Committee, who will be partnering with TransCelerate to

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develop data standards. TransCelerate also will solicit feedback for its Risk-Based Monitoring Approach. 2. Creation of a shared investigator portal. At present, communications between investigators and among clinical trial sites are often cumbersome and difficult. With this initiative, TransCelerate will create and build a shared, cross-industry, web-based, investigator portal specifically designed to improve communications among users. Through the development of this common interface, TransCelerate’s goal is to streamline and standardize investigator and clinical trial site access, while enhancing communications through the harmonized delivery of content and services. Additionally, in terms of economic benefit, a shared investigator portal is expected to result in substantial cost savings to individual companies by

reducing the need for companies to run their own portals (which currently have different looks and feel), and require site-specific training for users. 3. Development of shared study site qualification and training. The common practice at the outset of every clinical trial is for pharmaceutical company sponsors to individually initiate clinical trial site selection, prequalification and Good Clinical Practice (GCP) training on a trial-by trail basis, a process that can be both paperwork-heavy and time-consuming for trial sponsors and investigators alike. With this initiative, TransCelerate’s objective is to centralize site prequalification and training by establishing agreements for mutual recognition of GCP training and site qualification credentials. Through this collaboration and by standardizing the process of training doctors and qualifying clinical trial sites, this initiative is expected to realize efficiencies and cost savings by reducing duplicative efforts. It also is expected to accelerate study start-up timelines, making it easier to identify appropriate trial sites and investigators for future clinical trials, while eliminating the need for investigators to go through separate but similar training programs in order to work in future clinical trials run by two or more companies. 4. Development of clinical data standards. Clinical data are now reported in individual trials in various ways, without benefit of an industry-wide set of standards. TransCelerate’s objective for this project is to work in partnership with the Clinical Data Interchange Standards Consortium (CDISC) to develop industry-wide data standards in priority therapeutic areas in order to support the exchange and submission of clinical research and meta-data, while improving patient safety and outcomes.

5. Establishment of a comparator drug supply model. As the ability to provide reliable, rapid sourcing of quality comparator products is critical to the success or failure of today’s clinical trials, so is the need to establish a comparator supply model for clinical trials in the future in order to better enable accelerated trial timelines and enhanced patient safety. TransCelerate’s aim for this initiative is to both speed up and standardize the process by which sponsoring companies strike deals to do clinical trials that compare an experimental drug to a standard regimen made by another company. Included within the new supply model would be a mechanism to enable participating companies to ship product directly to one another, as well as a distribution system, including terms upon which members might purchase product directly from each other and the development of Master Service Agreements (MSAs) with participating companies.

“Our mission is to work together across the global research and development community and share research and solutions that will simplify and accelerate the delivery of exciting new medicine for patients.”

© 2013 Pharmacogenomic Informatics

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Expected outcomes were also identified for each of the five clinical trial execution initiatives, as follows:

“Target Outcomes by Initiative” Source: TransCelerate BioPharma Inc.

To date, the launch of TransCelerate BioPharma has been enthusiastically welcomed by the wider drug development community. While not the first time that members of the biopharmaceutical industry have collaborated to solve common challenges, the mission of TransCelerate may well be the most comprehensive and promising, and it has garnered significant support from the biopharmaceutical industry at large, as well as regulators, other clinical trial collaborations and the general clinical trial ecosystem. Janet Woodcock, MD, director of FDA’s Center for Drug Evaluation and Research, and early supporter of TransCelerate’s praised the new company’s efforts, saying, “We applaud the companies in TransCelerate BioPharma for joining

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forces to address a series of longstanding challenges in new drug development. This collaborative approach in the pre-competitive arena, utilizing the collective experience and resources of ten leading drug companies and others to follow, has the promise to lead to new paradigms and cost savings in drug development, all of which would strengthen the industry and its ability to develop innovative and much-needed therapies for patients.” “These leading pharmaceutical companies are in a position to significantly influence changes in the way that clinical trials are done, so that better answers about the benefits and risks of drugs and other therapies are provided in a more efficient manner,” added Robert Califf, MD, Co-Chair of the Clinical Trials Transformation Initiative (CTTI) and Director of the Duke Translational Medicine Institute.  “This Initiative is complementary to efforts of CTTI, and we look forward to working with TransCelerate BioPharma to improve the conduct of clinical trials,” he said. And so far, the company’s progress to date has been encouraging. As Dr. Gill recently announced, “In just six months, we have made significant strides toward our initial aim of making selected components of clinical trials more efficient. The five ongoing initiatives are making strong progress toward their goals, and in the future, we will launch other projects also aimed at the pre-competitive drug development environment.”

“Initiative Milestones” Source: TransCelerate BioPharma Inc.

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As shared solutions in clinical research and other areas are developed, it is expected that more companies, regardless of size, will join, contribute to and benefit from the shared solutions of this combined effort. An additional six companies have joined since early second quarter of 2013 – Astellas Pharma Inc., Biogen Idec, Braeburn Pharmaceuticals, EMD Serono, Inc., Forest Laboratories and Onyx Pharmaceuticals. TransCelerate also is working collaboratively with industry alliances including Clinical Data Interchange Standards Consortium (CDISC), Critical-Path Institute (C-Path), Clinical Trials Transformation Initiative (CTTI), Innovative Medicines Initiative (IMI), regulatory bodies including the US Food and Drug Administration (FDA) and European Medicines Agency (EMA), and Contract Research Organizations (CROs). TransCelerate BioPharma Inc. was formed in 2012 and is a non-profit organization focused on advancing innovation in research and development (R&D), identifying and solving common R&D challenges and further improving patient safety, with the goal of delivering more high quality medicines to patients. TransCelerate  evolved from discussions at various forums for executive R&D leadership to discuss relevant issues facing the industry and solutions for addressing common challenges. Founding members include Abbott/AbbVie, AstraZeneca, Boehringer Ingelheim, Bristol-Myers Squibb, Eli Lilly and Company, GlaxoSmithKline, Johnson & Johnson, Pfizer, Genentech a member of the Roche Group, and Sanofi, and all have representation on the Board of Directors. Dalvir Gill, PhD, was named CEO in December of 2012. Membership in TransCelerate is open to all pharmaceutical and biotechnology companies who can contribute to and benefit from these shared solutions. Executive offices are located in Philadelphia, PA. For more information, please visit http://www.transceleratebiopharmainc.com.

Dalvir Gill, Ph.D. C.E.O. of TransCelerate

Dr. Dalvir Gill became C.E.O. of TransCelerate effective January 2013. Formerly President of Phase II-IV Drug Development at PharmaNet-i3, Dr. Gill has more than twenty-five years of drug development experience. He holds a B.S. from the University of Hertfordshire, a Ph.D. from the Royal Free Hospital School of Medicine in London and an executive program diploma in the health economics of pharmaceuticals from the Stockholm School of Economics. He is a fellow of the Royal Society of Medicine.

Garry Neil, M.D.

Founding C.E.O. of TransCelerate

*References 1. Pharmaceutical Research and Manufacturers of America, “Key Industry and PhRMA Facts,” http://www.phrma.org/newsmedia/related-resources/key-industry-factsabout-phrma. 2. Pharmaceutical Research and Manufacturers of America, “The R&D Process: The Road to New Medicines,” PhRMA 2012 Profile: 30. 3. Pharmaceutical Research and Manufacturers of America, “Letter from PhRMA’s President and CEO,” PhRMA 2012 Profile.

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Dr. Garry Neil, the founding C.E.O. of TransCelerate, is now Chairman of the Board of the organization. He is a Partner at Apple Tree Partners and head of R&D for Braeburn Pharmaceuticals, Apple Tree Partners company. Dr. Neil has more than thirty years of clinical development experience in the science, medicine and pharmaceutical industries, with approximately twenty years of pharmaceutical, science and technology research and development at Johnson & Johnson, AstraZeneca, Merck KGaA and Astra Merck.