Do protocols really help patients?

Referring physicians frequently ask whether protocols help or “harm” patients. In fact, the question is whether there is a self-serving interest on the part of principal investigators to enroll patients into their trials.  The answer is simple – protocols not only provide state-of-care therapeutics, but also ensure that patients adhere to, and physicians follow, close surveillance guidelines, which over the length of planned treatment results in closer observations than usual and better overall care. There are numerous publications attesting to this fact.

Government lawmakers realize this and are instituting policies for financial coverage of peer-reviewed clinical trials. Many states (e.g., Michigan) have passed legislation to finance clinical trials for patients and other states will follow. These legislative policies are driven by the enormous safety records of clinical trials performed in oncology of the years, which represents an excellent testament of good clinical practices. Trials at Montefiore Medical Center go through peer review at many levels. A large part of our trials are performed through the National Cancer Institute.

Patients are comforted by honest representation of peer-reviewed research and the ‘close observation and follow-up’ demanded by most if not all trials. Phase I studies are particularly strong in adhering to strict follow-up procedures.  In fact, the phase I research team visits patients in the field who are either non-compliant or have other reasons for poor follow-up. Data accumulated from the trials is discussed with patients at every stage of treatment. 

Post-protocol follow up is a requirement for most studies. Post-protocol follow up can extend over one year off-study. This ensures safety and brings the patient back to his or her physician as a “patient” and not a number or case study.  In contrast, non-protocol therapy - especially for most advanced malignancies where there is no established standard of therapy - has no such rule. Ad hoc follow-up and re-treatment can often lead to problems, especially in a non-compliant patient. Overall, it is clear to most oncologists that protocol therapy for cancer may still be the rule in most situations, especially for any patient with chemotherapy –refractory cancer considered a candidate for phase I-II therapy.