This guide, which will be published as a series of articles over the next couple of weeks, is intended to help clarify the clinical trial process and to answer common questions so that you can decide whether a clinical trial is the right option for you. The guide will explain the different types of clinical trials, address risks and benefits associated with participating in clinical trials, explain the details of participating in clinical trials, and provide resources for finding a clinical trial.

If you are considering participating in a clinical trial, it is important to be entirely comfortable with your decision. Here are some benefits and risks associated with clinical trial participation that should help you during the decision process. The most important thing, however, is whether the treatment regimen being studied is the best option for you. Your physician can help you determine this.

Why are clinical trials important?

Not only do clinical trials offer new treatment options for you, but they are also (in the long run) helping further research for the benefit of many, many patients in the near and far future. It takes a significant number of people who are willing to participate in order for disease research and treatment development to move forward, but every person counts and adds to the culmination of knowledge.

It takes years of preparation and research for one treatment to reach the market. Without the help of clinical trial participants, advances in treatment could not be made.

By taking part in a clinical trial, you can try a new treatment that may or may not be better than those that already exist. But in addition to receiving the treatment that you have decided is best for you, you can also help doctors and researchers better understand how the treatment works in people of different races, genders, ages, and stages of disease.

Potential benefits of participating in a clinical trial

• Health care provided by leading physicians in the field of cancer research
• Access to new drugs and interventions before they are widely available
• Close monitoring of your health care and any side effects
• A more active role in your own health care
• You may be among the first to benefit from a new treatment
• An opportunity to make a valuable contribution to cancer research.

Clinical trials are known for their meticulous detail and observation. So you will get the same, if not better, care and attention that you would get during regular treatment. There are frequent visits with your physician to provide thorough examinations. There are also various ways to record your everyday symptoms and to track your progress, such as diaries and hotlines. This allows the research coordinator and physician to physically see the progression of your symptoms and whether the treatment is having any effect.

Common reasons to participate in a MDS trial

If a patient is not responding to standard treatment and their quality of life is poor, there may not be many options left to exhaust. For those who have tried various treatments without results, clinical trials and their treatments offer new hope.

Potential risks of participating in a clinical trial

• New drugs and procedures may have side effects or risks unknown to the doctors
• New drugs and procedures may be ineffective, or less effective, than current approaches
• Even if a new approach has benefits, it may not work for you.

Common reasons patients choose not to participate in a MDS trial

Some patients may choose to continue trying established treatments that have been proven to work on other patients because those treatments have already been assessed for efficacy and safety factors. Unknown short term and long term side effects are one of the many reasons that patients choose not to participate in trials.

There is also a lifestyle factor. Participating in a clinical trial may not suit some patients’ lifestyles (i.e. too much traveling, too many doctor’s visits, unexpected financial troubles, etc.).

Another reason is a comfort level that patients feel with their doctors in a more private environment and a nervousness associated with switching to a different doctor and to a new environment. Additionally, local physicians may not be inclined to refer their patients to clinical trials at research institutions.

“Because of the infrastructure of clinical medicine in the United States, many patients are treated by local hematologists in their private practice. The incentives for these physicians to keep their patients in their clinics (rather than referring them to trials elsewhere) are quite strong,” said Dr. Ola Landgren, a clinical trial investigator at the National Cancer Institute.

For more information about clinical trials for MDS patients, please see Part 1: Learning About Clinical Trials and Part 3: Participating In Clinical Trials.