What should I ask when considering taking part in a clinical trial?

Clinical trials are research studies that explore whether a medical device, treatment or strategy is safe and effective for humans. Laws and regulations governing clinical trials differ from country to country, but reputable clinical trials share important criteria, including respect for participants’ rights, strong scientific evidence, oversight by independent committees and compliance with appropriate laws and regulations concerning research on human subjects.

You should ask many questions about the treatment being offered and also seek a second opinion from a trusted healthcare provider with related qualifications, competency, and expertise who can give accurate and credible answers. You should not be rushed to make a decision—make sure you understand the entire treatment plan and any associated risks.

The doctors involved should specialize in your disease, describe alternative treatment options, and provide an Informed Consent Form or Treatment Consent Form that should address many of your questions. Not all clinical trials are created equal. If you are considering a clinical trial, below are recommended questions to ask when considering taking part in a clinical trial.

Learn more about clinical trials here.

The Treatment

• Is the treatment routine for this specific disease or condition?

• Is the treatment part of a formal clinical trial?

• What are the alternative treatment options for my disease or condition?

• If I have this treatment, could it affect whether I get into another clinical trial in the future or exclude me from other potential treatments?

• What are the possible benefits I can expect? How will this be measured and how long will this take?

• Am I likely to experience personal benefit as a result of being in this study, or is my participation likely to benefit future patients rather than me?

• What other medications or special care might I need?

• How is this stem cell treatment or procedure done?

• What is the source of the stem cells?

• How are the stem cells identified, isolated, and grown?

• Are the cells differentiated into specialized cells before treatment?

• How are the cells delivered to the correct part of the body?

• If the cells are not my own, how will my immune system be prevented from reacting to the transplanted cells?

• Are there any advantages of being in a clinical trial that I should know about before I decide whether to participate?

• Are there any disadvantages connected to being in a clinical trial that I should know about before I decide whether to participate?

Scientific Evidence and Oversight

• What is the scientific evidence that this stem cell treatment or procedure could treat my disease or condition? Where is this published? Is the journal credible? Are the findings credible?

• Have there been previous clinical trials? What was learned from these trials?

• Is there any independent oversight of the treatment plan, for example, an Institutional Review Board? Can you provide several names of qualified scientists and clinicians who can give independent advice?

• Is there any independent oversight or accreditation of the clinic where the treatment will be done and the facility where the cells are processed? Is this accreditation or oversight meaningful? Does it tell me anything about the credibility of this institution or the treatment I am considering?

• Has this treatment for this specific disease or condition received approval from a national or regulatory agency, such as the EMA, FDA, PMDA, or TGA?

Safety and Emergencies

• What are the risks of the treatment itself and the possible side effects, both immediate and long-term?

• Are there any other risks to me by participating in the trial?

• What will be done if an adverse reaction (bad side effect) develops? Who is the person to contact in an emergency or trial-related injury? Who will provide emergency medical care? What do I do if I cannot get in touch with someone from the clinic in the event of an emergency?

• Is the clinic adequately prepared to handle emergencies such as a serious allergic reaction?

• What will happen if I am no longer at the clinic when I experience a serious side effect?

• Will the clinic maintain communication with me and provide follow-ups for an extended period of time, potentially spanning months or even years, once I enroll in this trial?

• What follow-up treatment will be received, and for how long? What will I need to do?

• Who is the doctor in charge of the treatment? What specialized training does this doctor have? How well trained are the other doctors and the technical support staff?

• Who is responsible for paying the cost of care if I experience a medical emergency and subsequently require additional medical care?

Patient Rights

• What are my rights as a participant? For example, in terms of confidentiality, my right to be informed of any new information that may arise, and the ability to withdraw from the treatment process?

• Who has access to my health data and how often can I access my own health data?

• What compensation am I entitled to if I am injured as a result of taking part in this trial?

• If I am injured, will researchers understand that it happened because I was in the trial or will the burden of proof be on me to make the case that the injury resulted from participating in the trial?

Cost

• Are there any costs that I will have to pay if I participate? If so, what does this include? What other costs will I incur? Note, it is not customary for someone to pay to be in a clinical trial (other than perhaps travel and other personal expenses), and in fact, charging research participants fees to participate in a clinical trial is often a “red flag” of a problematic study or one that could have ethical, legal, or scientific shortcomings.

• What would be the cost of emergency treatment if something goes wrong? Who would provide emergency care and who would pay for this? Before traveling or agreeing to treatment, find out what costs your travel insurance, health insurance provider, or national health program will cover, in what circumstances, and in what countries.

• What happens if there is a serious complication that results in long-term disability? Who is responsible for paying associated costs?

• If I have to go to court and seek remedies through litigation, who is responsible for paying legal fees?