SBIR-STTR Award

Our new president vows to address the pervasive issue of cancer. Over 1.4 million Americans will be diagnosed with cancer this year, joining over 10 million living Americans who have experienced cancer. The medical costs of cancer care were $78 billion in 2008, and the overall economic impact was estimated to be over $200 billion. The current administration plans to double federal funding for cancer research within five years, primarily at NIH and NCI, with a focus on research for rare cancers. Our expert collaborator on this project, Dr. Daniel Von Hoff, describes two classes of rare cancers. There are the truly rare diseases (e.g., hairy cell leukemia), but also common histological cancer types like breast cancer, with rare molecular subtypes such as triple negative breast cancer. Cancer profiling into these specific ""rare cancer"" sub-classes and customized treatment for individual patients is now clinically realistic. Challenges to the development of tomorrow's therapies include identifying and connecting the right patients with the right treatments. Today, less than 5 percent of adult cancer patients participate in clinical trials. There are multiple reasons for this, including reimbursement problems in research funding, a daunting administrative burden, and a growing shortage of clinical researchers. The Obama administration has pledged to increase cancer trial participation to 10 percent of adult patients. In addition, the NCI Director has been charged with identifying the regulatory barriers that prevent the timely completion of successful clinical research. Pharmatech Oncology has conceptualized a unique solution that identifies patients first (Just-in-Time"", [JIT]), then rapidly connects patients and physicians to appropriate clinical trial opportunities. This differs from the traditional model, which focuses initially on administrative process and secondarily on patient enrollment. The traditional model can be effective for clinical trials in indications with millions of patients, but for rare cancers, it is not as effective. The goal of this Phase I SBIR project is to assess the feasibility of using the JIT approach to efficiently enroll and conduct analysis of two or more ongoing or completed clinical trials for rare cancers, compared with the traditional model. We expect to demonstrate that JIT can increase enrollment rates while maintaining patient safety and data quality, which will lead to a Phase II project designed to fully demonstrate/validate JIT across a larger number and variety of rare-cancer trials and to show how JIT streamlines the administrative workflow. We intend to demonstrate how the JIT system can be applied to community-based and institutional sites-becoming a platform for matching individual patients anywhere in the country to appropriate clinical trials without the typical and potentially harmful administrative delays. The nationwide JIT initiative will enable patient access to NCI- and privately funded clinical trials, will improve overall and representative participation, and will boost access to cancer cures and to personalized cancer treatments. , ,

Public Health Relevance:
Modern strategies for developing new, targeted cancer therapies and testing them in clinical trials increasingly focus on highly patient-specific molecular abnormalities-turning many cancer trials into what amount to ""rare cancer"" trials. Currently, these developments bypass the majority (85 percent or more) of candidate cancer patients because those patients go largely undetected, embedded in a nationwide, community practice treatment system that has limited access to clinical trials of targeted therapies. The overall goal of this multi-phase SBIR project is to develop, validate and commercialize Pharmatech Oncology's novel Just-In-Time method for facilitating rapid enrollment of rare-cancer patients, reducing administrative and technical barriers, and improving patient access to state-of-the-art cancer treatment through advanced, personalized-medicine clinical trials.

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The American Cancer Society estimates that more than 1.5 million new cancer victims will be diagnosed this year in the U.S., and that ~572,000 Americans will die of cancer in 2011.1 More than 13 million Americans are living today with a history of cancer, and this number is heading toward 18 million by 2020.1 Beyond the human costs in lives, productivity, and quality of life, the NIH estimated cancer's direct medical costs t $103 billion in 2010, with total costs being $263 billion.1-2 Clearly, adequate drug therapy is not yet available, and we need better ways to rapidly develop and test new drugs. At the heart of the problem is a ""one size fits all"" approach to research that is not aligned to a cancer population comprised of hundreds of rare treatment indications spread among thousands of separate practices? Innovations are needed to deliver greater access to patients and to connect the right patients to the right clinical trials at the right time. We need to get next-generation cancer drugs from benchtop to the clinic-faster and at a lower cost-and the Obama administration's ambitious plans to double federal funding for cancer research within five years and to also double enrollment in U.S. cancer clinical trials is certainly in jeopardy given the current budget situation.3 Pharmatech Oncology is addressing this critical need via research system innovations that increase access to patients for participation in clinical trials. Our Just-n-Time (JIT) research system being developed in this multi- phase SBIR project is designed to closely align and coordinate U.S. clinical research with cancer patient care. Our strong Phase I results demonstrate that the feasibility of using JIT is a superior methodology for conducting clinical trials in indications where patients are potentially rare and highly embedded in a large care system. Those results set the stage for this Phase II project, which is focused upon completing the system development, optimization, standardization, and customer interactive infrastructure needed to offer the JIT research service through a website portal product, the ""Pharmatech Nexus."" The Pharmatech R&D team will pursue the following Phase II Aims: 1) Develop and demonstrate optimized JIT workflow and economics at research site level;2) Develop and implement tools to standardize the JIT methodology;3) Develop a business platform to drive scalability and expanded connectivity;and 4) Prove the integration of JIT methodology with molecular screening. Building on the concepts proven in Phases I and II, a new means of conducting clinical trials will be made broadly available. When fully developed by the expert Pharmatech team, the Pharmatech Nexus platforms will 1) make a large number of clinical trials, spanning multiple indications and treatment stages, available to oncologists for their patients;2) provide a conduit for molecular testing to identify which targeted investigationl therapies may be appropriate;and 3) enable individual patients to enroll in the right trials very quickly and to receive research-based care on a schedule that accommodates their need to be treated.

Public Health Relevance:
As more-effective precision therapeutics become available to directly target the molecular causes of cancers, the costs and challenges of bringing a new generation of highly effective drugs from benchtop to clinic are escalating. The end goal of this multi-phase SBIR project is to establish a new, patient-centered clinical research system that is aligned with cancer patient care in the U.S. and to vastly expand patient/physician access to clinical trials as treatment options for individual patients. The Phase II work proposed here will position Pharmatech Oncology to lead a unique, collaborative marketplace initiative designed to drive cancer patient participation in clinical trials from 3% to 10% by 2020, enabling approval of next-generation cancer drugs more rapidly and at a lower cost.