Clinical trials are driven by hope

www.clinicaltrials.gov summarizes why someone might want to be in a clinical trial and covers the risks.

Benefits to a "well-designed and well-executed" study include the ability to play a more active role in your own health care, to access new research treatments before they are widely available, to obtain expert care — sometimes more extensive than you would normally receive — and to contribute to medical knowledge.

The flip side is potential "unpleasant, serious or even life-threatening side effects to treatment," treatment that may not help the participant, and protocols that may require more visits to the study site, more treatments, hospital stays, dosage requirements, need to log information, etc.

The basics

Clinical trials are divided into phases, each with a different goal. In Phase I, a new drug or treatment is tried in a very small group of people to find out if it's safe, what the safe dosage range would be and to identify side effects.

In Phase II, the participant pool grows to 100 to 300 people to assess if it works and to look more closely at safety.

Phase III tests it in thousands of people to confirm treatment works, to monitor side effects, to see how it stacks up against the standard care.

The medical motto "first do no harm" applies to trials. Participants receive solid standard treatment as well throughout the trial, Prescott said. No one's deprived of needed treatment already known to work.

The phases must be complete before the U.S. Food and Drug Administration will approve a new treatment, device or process. Even then, it's not over. Phase IV trials take place after that wonder drug is on the store shelves, providing additional information about risks, benefits, optimal use and more. A drug may even be pulled after it's been on the market.

Each clinical trial is designed to answer specific questions, so there are tight criteria of who can participate and who is excluded. Some clinical trials need healthy patients, others those with a specific medical condition. Some exclude a person based on previous treatment, others embrace someone because of it. The goal is simply to find out in a "careful, scientific way" whether a proposed treatment works, Prescott said.

All trials are approved by institutional review boards, which view a proposed study from the ethics and safety angles. Some research facilities, like the University of Utah, have an in-house IRB. Others, like Radiant Research, contract IRB work out to a company that specializes in that line of work.

Eureka! I have an idea!