Wednesday, February 5, 1997
"The Clinical Trial Dilemma: How to Choose and Prioritize": A Summary*

Introduction - Debu Tripathy, M.D.

Dr. Tripathy began the discussion by talking about his goals for improving clinical trials. He would like to identify ideas that could be implemented to make clinical trials better, more accessible and more useful. He discussed how it is difficult for investigators to know what to study without knowing what the patient's needs are. He would like to better understand the area of clinical trials, patients' interests and the obstacles that prevent patients from enrolling on clinical trials. In addition, Dr. Tripathy would like to ask women who have gone through clinical trials, or those who have considered trials, about their experiences.

Eligibility Criteria

• One woman stated that she had a very difficult time getting onto a HER2/neu trial because she did not fit the requirements of having failed 2 chemotherapies. She intervened with the system and eventually got onto the trial, but was frustrated that she had to go to so much effort to get enrolled.

• The reasons for eligibility criteria were discussed. The point of eligibility criteria is to be able to answer a research question as well as possible. Researchers would like to test a homogenous population (or as homogenous as possible) to be able to reduce the number of factors that could affect a treatment and skew the findings. The criteria that is picked is often arbitrary - there just needs to be a common ground. For HER2/neu, a patient must fail 2 chemotherapy treatments.

• The old dogma has been to test people who have no other options. Increasingly researchers are willing to test different therapies on people who are receiving treatment for the first time.

• The Principle Investigator of a study should justify why a trial is using it's particular exclusion criteria.

• The FDA also imposes some eligibility criteria and is very strict about who can take new drugs and treatments. Their argument is that women with breast cancer have very unpredictable outcomes. The FDA is less likely to permit the administration of new drugs or therapies to patients who do not fit their criteria.

• A solution would be having a trial that is as inclusive as possible. By testing a drug or treatment on a large population, it could be determined how it affects a cancer population as a whole. This is a quicker way of deriving answers than by conducting a longer study on a limited population of patients.

• It's important to note that the recent loosening of eligibility criteria and the move to larger studies would not have happened if people had not mobilized to change the former system.

The Dissemination of Information

• A question was posed about the politics of patients enrolling onto clinical trials: whether a woman in the Midwest can have access to a trial being sponsored at another cancer center or if her doctor will only recommend the trials offered at their facility. It was agreed that this scenario could occur and that hearing about trials can be the luck of where you live.

• On the other hand, it was pointed out that since trials exist to test new treatments, no one really knows which trials are good or bad and, therefore, it makes no sense to travel around the country in search of good trials when there might be good ones right in you area.

• One way for women to find out about their options is to become proactive and look for available trials themselves.

• The National Cancer Institute (NCI) has a web site called PDQ where women can learn about the clinical trials available. For women who are not computer literate, the NCI will fax trials to patients. Unfortunately, their list is not comprehensive. There is also no guidance about the possible side effects or how to differentiate between good and bad trials.

• There is also the huge problem of patients not being Internet-savvy or not being able to understand the trials once they are able to find them. PDQ was designed for physicians and the trials are described in complex medical terms.

• The ideal way of getting information about clinical trials to patients would be to organize the trials in a structured way and integrate them into standard medical care.

• Dr. Tripathy said clinical trials should be an integral part of our medical culture and should be conducted for every medical problem or procedure. This would allow scientists, researchers and doctors to learn the answers to many medical questions very quickly.

• Deborah Collyar, a patient advocate, plans on getting clinical trials on the agendas of primary care physicians. At the beginning of their diagnosis is an ideal point for patients to learn about clinical trials.

• There was also the suggestion of having clinical trial phone numbers listed in the phone book. There could be a special clinical trial office at each medical facility.

• Deborah Collyar founded the Clinical Trial Information Project which is working on centralizing, organizing and distributing information about clinical trials. The information collected will be distributed over the Internet, in a printed guide and available through a fax retrieval service. The Project has collected information on the 60+ clinical trials that exist in the Northern California area. The clinical trials will be "translated" into plain English. The one-page summaries will comprise one-third of the guide and the rest of the guide will contain advice on clinical trials, research and breast cancer issues.

• As great as it is to have huge web sites of clinical trials on the Internet, many people do not have access to the Internet or simply do not know how to use it. A proposed solution is to have computers in the waiting rooms of medical offices and someone available to teach patients how to use the Internet.

Alternative Therapies

• Many patients who can not find appropriate clinical trials or who feel that their cancer is not "serious" enough would like more alternative medicine options.

• The F.D.A. is considering licensing herbs. This regulation will help determine what quantities of herbs are safe.

• In the meantime, it is important to record all information about herbal remedies in an outcomes database. Questions that need to be asked are: What medications are people taking? What alternative drugs or treatments are they using? How often? What are the outcomes? What are the side effects? What are the benefits?

• Patients would like to find alternatives to the types of treatments that alter other important bodily systems (heart, nervous, immune systems).

• Many women who are diagnosed at a late stage would like to have aggressive treatments, but not necessarily stem cell treatments because they take too much time. These women are very receptive to trials, but they can't find good ones.

• Ideally, trials would have crossover arms that would allow patients to switch to a different arm of the study if their cancer was progressing, or if it was determined that one arm was better than the other.

• One problem with crossover arms is that many trials need to be completed before it can be determined whether the treatment or drug being tested works. This is especially true with new drugs and trials that measure the effect of different doses of drugs.

The Cost of Trials

• Usually insurance companies or the clinical trial will cover the costs of standard treatments.

• Some insurance companies won't pay for any experimental treatments.

• There are also many hidden costs involved in clinical trials. There are additional office visits, >which is an added cost in terms of the doctor's fees, but also costs the patient in terms of time away from work, transportation fees, child care fees, etc.

The idea of having treatment consultants or facilitators was proposed. These would be women who have already been through breast cancer treatments and would lead patients through the breast cancer treatment process. They could help patients find information about treatments, support groups, clinical trials, insurance, etc. Ideally, hospitals and breast care centers would hire, train and pay for the consultants.

Thought for the night: