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An Initiative to Support Non–Industry-Sponsored Clinical Research Is Needed

Author's Affiliation: The Cancer Institute of New Jersey, New Brunswick, New Jersey

Requests for reprints: Roger Strair, Cancer Institute of New Jersey, Robert Wood Johnson Medical School, 195 Little Albany Street, New Brunswick, NJ 08901. Phone: 732-235-6044; Fax: 732-235-8098; E-mail: StrairRK{at}umdnj.edu.

Healthcare costs in the United States exceed $1.7 trillion dollars per year, approximately 15% of the country's gross domestic product. On a national level, drug expenditures are the most rapidly increasing component of healthcare costs. Drugs have the potential to cure some diseases, delay or prevent other diseases, improve the quality of life, and complement standard medical therapies. Increasing drug expenditures are explained by the availability of new and effective drugs that are marketed at very high prices, broader use of drug therapies that delay or prevent common diseases, insurance coverage for medication costs, and potent marketing programs targeting healthcare professionals and patients. Both high drug prices and the increased prevalence of prescription drug use contribute to increasing costs. In fact, some healthcare analyses indicate that prescription drug expenditures are not as high as they should be because many patients with cardiovascular conditions, pulmonary diseases, or diabetes are either undertreated or not treated at all.

Antineoplastic drugs and drugs prescribed to treat the symptoms of cancer (or treatment-related toxicities) provide the foundation of therapy for patients with cancer. Generally, the efficacy and applicability of a new therapy is shown by formal clinical trials before regulatory approval is granted. Over the past several years, many exciting new cancer therapies, such as rituximab, imatinib mesylate, herceptin, bevacizumab, alemtuzumab, bortezomib, erlotinib, sutinib, sorafinib, additional kinase inhibitors, and other novel drugs have been developed. Regulatory approval has resulted in the availability of these new, effective, and costly therapies to increasing populations of patients with cancer.

It is very hard to combat the ever-increasing cost of cancer drugs. No one would want to slow the pace of drug development, as these treatments increase disease response, reduce symptoms, and/or prolong survival. Pricing of some new anticancer therapies, however, may approach $100,000 per year per patient in some cases, and the clinical trials supporting the use of these new drugs or drug combinations may show only marginal benefit. In addition, there does not seem to be an effective mechanism to regulate drug pricing, at least early after drug approval.

In this context, it is important to have mechanisms available that support the development and testing of novel drug therapies, procedures, or devices that may be effective, but that do not meet the profit-driven agenda of pharmaceutical companies. Such therapies might use off-patent medications, new cellular therapies that will not be awarded a patent, or other approaches that do not translate into large profits. Without the potential of profit, however, there is no financial support for the development of these therapies.

One problem is that the processes required to show the efficacy of a drug or drug combination are very costly. Even the final stage of development, the demonstration of efficacy in a clinical trial, is extremely expensive. So expensive, that drugs or drug combinations that are not sponsored by pharmaceutical companies often cannot be tested, even at major academic medical centers. Drugs, drug combinations, devices or procedures that cannot be patented, are currently off-patent, or from which there is no way to envision a profit, even if clinically effective and inexpensive, may not be developed because there is no sponsor to incur development costs, including clinical trial costs. In oncology, approaches such as low-dose metronomic therapies, novel cellular therapies, new combination chemotherapy regimens with old drugs, novel surgical therapies, and modified hematopoietic stem cell transplants often cannot be formally tested because of the costs involved. For example, there are no effective standard therapies for most elderly patients with acute myelogenous leukemia. Several drugs under recent development by pharmaceutical companies are anticipated to have low response rates. Such therapies, if approved by the Food and Drug Administration after clinical trials, will likely be very expensive, despite the low response rates. In contrast to these newly developed therapies, oral melphalan is an off-patent medication that has been used in very low doses for patients with acute myelogenous leukemia. There are no large clinical trials of melphalan or other low-dose metronomic approaches in this setting, however, and clinical trial costs preclude a formal trial in almost every medical center. Although testing the use of melphalan for patients with acute myelogenous leukemia is not exciting and likely would not receive funding from the NIH or alternate sources, it is a treatment option that has some clinical data support and should be formally tested because it might fill an open therapeutic niche as a low-cost, low side effect therapy for elderly patients with acute myelogenous leukemia.

Although there may be NIH or other national funding for more novel clinical research not sponsored by the drug industry, such support often is limited and difficult to obtain for early stage research, particularly when there is not a wealth of preliminary correlative data. Therefore, it is often very hard to get off the ground without a push motivated by the potential of a large profit.

There must be some initiative that allows clinical research developed independent of a pharmaceutical company to get to the table, particularly if the trial tests a novel hypothesis or is accompanied by a health-economic evaluation that indicates the potential for cost-efficient care. Otherwise, we will be paying large bills for expensive therapies developed by pharmaceutical companies, while allowing cheaper therapies developed without pharmaceutical company support to languish without testing.

Who will pay the clinical trial costs for such studies? It depends on who is likely to receive economic gain. Trials with a health-economic evaluation indicating the potential to save money should be supported by insurers and/or Medicare. If even 0.01% of the Medicare budget were devoted to supporting clinical trials that have cost-effectiveness as an outcome, there could be great financial benefit. Alternatively, the costs of medications developed by pharmaceutical companies are so expensive, even when marginal clinical benefit is seen, that a tax to support the development of nonprofitable therapies might also be appropriate.

Our system is costly and we are often restricted to only testing therapies that increase our healthcare expenditures. There needs to be some sponsored seats at the table and some motivation to develop less costly therapies. We need a clinical trial initiative that will allow a full spectrum of non–industry-sponsored therapies to be tested.

Received 12/13/06; revised 12/30/06; accepted 2/ 8/07.

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