Evaluating CAM

Many patients with cancer perceive a benefit to the use of CAM therapies, yet most have not been adequately tested for safety, efficacy, and interactions with chemotherapy and radiation. Often, CAM therapies are available over the counter without the regulatory oversight or quality controls that govern conventional medications. In January 2005, the Institute of Medicine Report noted that the healthcare system should strive to be both comprehensive and evidence-based and should therefore hold conventional and complementary treatments to the same standards and use the same general research principles in evaluating both types of treatments.

Below are 3 steps in how to get started with clinical research of CAM therapies and children with cancer.

• Step 1: Develop plausible data-based hypotheses
  1. Assess the Risk-Benefit Ratio
     Ask: What is known about the effectiveness of the CAM therapy? Is there any reported toxicity of the CAM therapy? How effective is the conventional anti-cancer therapy? Are there any known interactions?
  2. Consider developing a Best Case Series to generate data that may be used for hypothesis generation.
  3. Develop a systematic review to summarize existing data, refine hypotheses, estimate sample sizes, and help define future research agendas
• Step 2: Decide what study design to use to evaluate the CAM therapy. Determine if there is enough known from observational and preclinical studies prior to implementing an intervention trial.
• Step 3: Practical Issues to consider
  1. Regulatory issues include submitting an IND application to the FDA if needed and obtaining human subject protection approval through an institutional review board and data safety monitoring board.
  2. Because clinical trials can be expensive, sources of funding should be sought out. Sources of funding include institutional pilot funds, philanthropic support, private foundations, the National Institute of Health, and industry support.

Further research of CAM therapies with the use of the same general research principles that are used in evaluating conventional treatments is needed. Strong research designs are necessary to evaluate CAM therapies and ultimately influence clinical practice and public awareness. To review a listing of clinical trials on CAM, visit http://clinicaltrials.gov.

(Modified from the lecture: “Research Methods: Clinical Trials and Lessons Learned,” Kara Kelly, MD)