Policy on Compassionate Access to Investigational Therapies

Breast Cancer Action (BCA) is a nonprofit grassroots organization of people who are willing to take the actions necessary to end the breast cancer epidemic, including breast cancer survivors and their supporters. Our mission is to serve as a catalyst for the prevention and cure of breast cancer by working toward three major goals:

  1. To make breast cancer a national priority through education and advocacy.
  2. To promote and refocus research into the causes, treatment, cure, and prevention of breast cancer.
  3. To empower both women and men so they can fully participate in decisions relating to breast cancer.

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From its inception, Breast Cancer Action has worked with advocates across the country as part of the National Breast Cancer Coalition to increase federal funding for breast cancer research. The results have been dramatic, more than quintupling the level of funding from $90 million in 1990 to $465 million for 1995. However, private sector funding for pharmaceutical company research is much larger than that sum and that research, in all likelihood, will eventually yield the major treatment modalities for women with breast cancer.

A significant proportion of federal research funding supports research studies of promising new approaches to primary prevention, chemoprevention, and treatment of breast cancer. Research studies such as clinical trials expand the knowledge base of clinicians and researchers. In addition, research studies can become an important treatment option program for thousands of women with breast cancer and other life-threatening illnesses. In fact, BCA believes that all cancer patients should be offered information about all clinical trials, whether sponsored by government or private industry, in the form of a clinical trials directory. We call upon industry to fund such directories in cooperation with members of the affected community.

An experimental drug cannot be prescribed outside of a clinical trial setting, but there are exceptions to this rule. Experimental drugs (including biological products) can be released to individuals with cancer outside of clinical trials through various compassionate mechanisms that currently exist within the National Cancer Institute (NCI) and the Food & Drug Administration (FDA). These compassionate mechanisms, such as Treatment IND, Group C cancer drugs, and Special Exception of Cancer Treatment Evaluation Program (CTEP), are often confusing or unfamiliar to ordinary women with breast cancer and to many doctors, who often have no special knowledge of the procedures to obtain experimental drugs outside of a clinical trial. The procedures and policies of NCI and FDA need to be reviewed and revised so that these mechanisms are widely understood and accessible.

Although these mechanisms can dispense experimental drugs on a widespread basis, most of the existing mechanisms are for a case-by-case, individual exception for those facing immediate life-threatening illness. However, as NCI points out, most of these exceptions are given to people who are too ill to benefit from them, and in many cases, these therapies hasten their demise. Requiring that all conventional or standard therapies be exhausted is cruel and unusual punishment for cancer patients, as the more heavily pretreated they are, the less likely they are to respond to any treatment, whether experimental or standard. A new paradigm for design of compassionate use policies must include providing drugs at earlier stages of disease, when the drugs could play an important role in extending lives.

The FDA and NCI need to fundamentally change the definition and use of the existing compassionate drug access programs. There must be a shift from an individual, case-by-case approach to one that has as its goal large-scale compassionate programs devised through the collaboration of advocates representing the affected community and pharmaceutical companies, with approval by the FDA. The mechanisms and policies of the NCI and FDA need to be revised to reflect the urgent needs of women with metastatic cancer whose disease is fatal and expected lifespan is short.

None of the existing NCI and FDA procedures/mechanisms address the issue of private pharmaceutical companies’ refusal to provide experimental drugs on a compassionate basis. Pharmaceutical manufacturers design clinical trials so that their product can be licensed/indicated for a specific use or uses. Thus, manufacturers can narrowly structure the clinical trial so it will answer the particular question that is asked in that trial. Just because a clinical trial has certain exclusionary criteria, however, does not mean that people who do not fit that criteria would not benefit from that drug. In fact, after licensing, a drug is commonly used for a wide variety of uses (called “off-label” use). There is no economic incentive for a drug company to seek further labeling uses, given the widespread “off-label” use of drugs. Eventually, most insurers do pay for “off-label” use.

While drugs are in the clinical trial stage, women with metastatic cancer need to have large-scale programmatic access, with full information about any known side effects and risks, to drugs that treat life-threatening illness at a time when the use of the experimental drug can potentially have a significant impact on extension and quality of life. Current treatment for metastatic cancer is essentially palliative, as it cannot effect long-term survival but can relieve some symptoms.

Treatment is entering a new era, and much of current approach and thinking is reflective of the “chemotherapy era,” which was a broad span approach to therapy. The new biological products are often more specific to the individual woman’s tumor and disease state. This specificity means that some experimental products would not be appropriate at all for women who do not manifest such a specific trait. However, new biological products that are specific for an individual woman’s case could potentially have greater positive impact on life extension and quality of life. This is yet another reason why compassionate drug access is so critical and must be available before the patient has exhausted all other therapies.

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Summary

  • Existing procedures and policies of NCI and FDA regarding compassionate mechanisms need to be reviewed, revised, and eventually publicized in a way that is accessible to the ordinary women with breast cancer and her ordinary doctor.
  • The requirement that all conventional or standard therapies be exhausted is cruel and unusual punishment for cancer patients, as the more heavily pretreated women are, the less likely they are to respond to any treatment, whether experimental or standard.
  • The focus of the FDA and NCI on a case-by-case exception for compassionate drug access needs to be revised to reflect a treatment strategy for women with metastatic cancer — to use experimental therapies as a conscious treatment option for larger groups of women to extend life before the disease process and the effect of past therapies make it highly unlikely that any therapy would prolong life for any reasonable period of time.
  • Disease process and treatment toxicity make it imperative that compassionate access be available early rather than late as newer and experimental drugs need time to work and the body needs time to adjust to any new product.
  • Compassionate drug access must be part of the drug approval process, and the FDA needs to contact pharmaceutical companies who are developing a drug for treatment of a life-threatening disease when the initial safety evaluations are completed during Phase II and ask them to submit a compassionate program.
  • BCA urges that the Pharmaceutical Research and Manufacturers Association (PHRMA) develop voluntary programs of compassionate drug access and that developing these programs shall be a condition of retaining membership in the organization. PHRMA should assist smaller companies in this effort, and encourage all its members to utilize input from patients, physicians, and consumer advocacy groups.
  • PHRMA shall fund a mediation service between the affected community and the pharmaceutical company when the pharmaceutical company refuses/delays in developing a compassionate access program.
  • BCA recommends that the pharmaceutical industry fund publication of a clinical trials directory on a regional and national basis. In that way women and their doctors can know and evaluate what drugs and trials maybe appropriate for them.
  • BCA recommends that private pharmaceutical companies list their clinical trials on 1-800-4-CANCER. If there are any regulatory impediments to this, these regulations should be evaluated and revised.

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Adopted September 15, 1995, by the Breast Cancer Action Board of Directors.