Advocacy >  Budget Cuts: A Crisis in Clinical Research

Last update: 04/25/2014

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The cycle of basic and clinical science (101 - a draft)

It's a circle.  Basic science guides clinical research, which in turn guides basic research.  One type of research supports,  validates, and informs the other - and generates new questions.   An insight about a disease process leads to the design of a new drug. The new drug leads to a concept about how to meet the needs of patients afflicted with a disease.  Clinical trials test the concept. The outcomes of the clinical trial informs clinical practice but also the larger body of science - particularly when patient biospecimens are captured and evaluated before and after the intervention that is tested.  The validated or exploratory findings are published and shared with the scientific community - generating new ideas and leading to new discoveries. 

Obviously, the scientific research benefits patients (all of us eventually) -- relieving us from pain and suffering.  It also contributes to the body of scientific knowledge -- which fosters cross fertilization of scientific discovery (HIV research advancing cancer research, and vice versa)  ... leading to innumerable new products, companies, jobs ...  the exuberance of our economy.    

Scientific research that is grounded in solving the problem of human suffering from cancer - striking 3 in 5 Americans, deserves our full support.     

The critical role of cooperative group (tax-payer funded) clinical science

Clinical research can be sponsored by the industry or by taxpayers (through the National Institute of Health (NHI) grants).  There is much to learn about how to use approved drugs that's of critical importance to patients -- that would not be done by industry sponsors.   Here we provide an overview about the critical role of publicly funded research.

The importance of the commercial sponsors to the development of new treatment products cannot be understated. Clearly it plays a critical role in the development and manufacture of important new treatments and tests.   However, it's for-profit mission limits its focus to product registration (marketing approval), expanding its market share, and to increasing shareholder value. 

We can think of cooperative group research as science that's motivated to advance the common good, funded by the common man (taxpayers).  It is a cooperative group endeavor carried out by dedicated scientists, clinicians, patient advocates, and academic institutions.  It is research focused on solving problems and clinical questions of vital interest to patients - such as how to optimize and tailor treatment for the benefit of patients.  Its charter is to improve the public health.  Its purpose is also to create and share new scientific knowledge, which supports also commercial innovation and the growth of our broader economy.

Publicly Funded Clinical Trials and the Future of Cancer Care

What follows are our notes from a lecture by Richard L. Schilsky, M.D. on the critical role of publicly funded clinical trials and the future of cancer care. Doctor Schilsky is a distinguished Professor and Chief Section of Hematology-Oncology, University of Chicago, Deputy Director, Comprehensive Cancer Center

Fundamental goals of publicly-funded versus commercial funded research

	Optimize Treatment versus drug registration for market approval (determine and advance best practice)
	Create New Knowledge versus  expand market share (publish negative results)
	Improve Public Health versus  create shareholder value (optimize and tailor therapy to patient subgroups)

Examples of Publicly-funded goals

	Compare the effectiveness of various treatment options (identify best practice)
	Combine/compare drugs developed by different sponsors
	Develop therapies for rare diseases
	Address optimal dosing
	Test multi-modality therapies such as radiation therapy in combination with drugs
	Identify patient and tumor subsets most likely to benefit from interventions
	Study screening and prevention strategies
	Focus on survivorship and quality of life
	Publish negative results
	Assess cost and cost-effectiveness of equivalent treatments
	Provide “gold standard” databases for registry studies
	Comparing Efficacy
	Compare Treatments from Different Sponsors
	Exploratory (Correlative) Biomarker Studies Biomarker Validation Studies (to predict risk of disease or response to treatment) Cooperative groups have the capacity to conduct many types of biomarker studies, including formal validation trials
	Identify Patient Subsets (clinical or molecular biomarkers)
	Test treatments on Patient Subsets (clinical or molecular biomarkers)
	Tackle practical problems: Most people who are diagnosed with cancer are elderly Most people who are on clinical trials of anti-cancer therapy are not elderly The risks and benefits of anti-cancer therapies in the elderly is uncertain
	Rare Disease Treatments
	Optimize Dosing
	Combine Treatment Modalities
	Who Benefits from Adjuvant treatments
	Assess Cost Effectiveness (for equivalent treatments)
	Provide Gold Standard Databases SEER-Medicare Analysis of RT Use in Older Women Following Publication of C9343
	Translational Science Infrastructure and correlative science infrastructure

Summary - publicly funded clinical trials are essential to:

	Directly compare drug treatments;
	Develop combined modality treatments;
	Study chemoprevention and rare diseases;
	Identify patient subsets;
	Study health outcomes, cost and cost-effectiveness
	Creation of the National Clinical Trials Network National Clinical Trials Network (NCTN) provides essential infrastructure for publically funded trials in treatment, control, screening, diagnosis, and prevention NCTN provides a unified clinical and translational infrastructure for the extramural cancer community: investigators, patients, advocates, and industry NCTN efficiently functions to answer critical questions not well supported in a commercial environment NCTN will eventually replace the program we have known as the cooperative groups

Risks of Replacing the Cooperative Groups

	Loss of institutional allegiance and cost sharing
	Fewer publically-funded trials
	Loss of young investigator mentoring
	Loss of competition to drive innovation
	The NCTN becomes a research infrastructure but not a research engine The future of cancer care depends on a robust, publically funded clinical trials system!

In the News

Group Chairs: Research in "Crisis" - The Cancer Letter Publications http://bit.ly/1hOHx31 The chairs of the adult clinical trials groups that make up the NCI National Clinical Trials Network said in a letter that recent budget cuts have triggered a “crisis” in clinical research. Concern about the three-month funding gap, coupled with implications of patient harm—ASCO alludes to a “dangerous disruption of cancer care”—comes from people who usually keep their rhetoric in check. These protestations amount to a resounding invitation for congressional oversight (of Congress?). Ending ongoing trials presents ethical problems, the group chairs wrote. “It is a serious ethical dilemma to consider stopping any of these critical trials in progress, to which our researchers have committed and our patients have consented,” they wrote.

* Statement on Forthcoming Changes in NCI's Clinical Trials Programs - National Cancer Institute http://1.usa.gov/1e9Bwia  assures that trials will continue. What is the truth?

Budget resources

	Fiscal Year 2015 Budget Request - The NIH Director - National Institutes of Health (NIH) http://1.usa.gov/1hxnrpg
	NIH budget background http://nih.gov/about/budget.htm
	Fiscal Year 2014 Budget Request - The NIH Director - National Institutes of Health (NIH) http://1.usa.gov/1jlcSdj

Related topics

PAL topic on Costs