Ensuring Safety in PHN Studies

A clinical trial is planned to study a medicine or treatment to improve human health and medical practice. Studies can help healthcare workers know whether to use (or not to use) the treatment being studied. The purpose of the Pediatric Heart Network (PHN) is to develop studies to improve health in people with congenital heart defects or acquired heart disease . Studies also help doctors and nurses make better decisions in using certain medicines and treatments. It is very important to the PHN nurses and doctors to keep patients safe in clinical studies.

Study Design

Pediatric heart researchers suggest studies that they feel will answer important questions about how best to treat patients. Researchers will write a protocol which is like the instructions in a cookbook. A protocol describes:

• The problem to be studied;
• What will be learned from the study;
• Who may enter and what will happen during the study;
• How safety will be maintained;
• How success or failure will be measured

Studies are reviewed for safety by many groups

There are several steps a study has to go through before it can be started and patients can be enrolled:

1. The protocol is reviewed and discussed in detail by members of the PHN. The members have a wide variety of training and experience in nursing, medicine, statistics, special technologies and managing clinical trials. Changes are made to create the best and safest study possible.
2. The study protocol is then sent to a specially qualified, independent group appointed by the National Heart, Lung, and Blood Institute (NHLBI) . This group is called a Protocol Review Committee or PRC. It is their job to review the study protocol and make suggestions about how to carry out the study and to recommend changes to the protocol.
3. Once additional changes have been made, the protocol is sent to a second independent review committee called the Data and Safety Monitoring Board (DSMB) . The DSMB may also suggest possible changes to the protocol or consent form focusing mostly on the safety of the study subjects.
4. The protocol and consent form that has been reviewed by the PHN members, the PRC and the DSMB, will then go to a review board at each hospital that will enroll patients in the study. These boards are called different names such as Institutional Review Boards (IRB) or Research Ethics Boards (REB) . These boards are chosen by a hospital to review and approve the start of a study and to review the study at regular times. The primary purpose is to assure that the patients who participate in studies are protected. The IRB may also ask that changes be made before allowing the research team to begin the study at their hospital or clinic.

Special training on patient safety is required

Each member of the research team must complete special training on how to protect subjects in clinical trials. Additional specific training is given to the research team on how to safely conduct each PHN study. No one may contact a patient or family or participate in these studies unless there is proof that this training has been taken.

Dealing with adverse events

Each research team must collect and report all adverse events to their IRB or REB, the Pediatric Heart Network and the NHLBI in a short timeframe. Not all adverse events will occur because of the study but may occur as a result of the condition of the study participant. Adverse events are followed closely by the doctors and nurses doing the study and they will decide what to do as soon as the adverse event is known. Serious adverse events are reviewed by the DSMB chairperson, a medical monitor, and the NHLBI staff within a few days of the report.

Ongoing review for safety

It is the job of the DSMB and IRB or REB to review the study on a regular basis to make sure that participants are safe and that the study data is reliable. The DSMB will meet as often as necessary to:

• Assess how well each hospital is performing
• Review adverse events
• Suggest ending the study early if results show that the treatment is effective or not or because of safety concerns

All of the steps described above are taken to protect people who are enrolled in PHN studies.