Clinical trials play a critical role in evaluating new medicines to ensure they are safe and effective. These studies provide important information to healthcare providers so that they can provide the best care possible for people living with the conditions being researched.

Research starts well before clinical trials begin. Preclinical (or non-human) testing helps ensure that clinical trial participants are not undertaking unreasonable risk and that the potential medicine may ultimately offer a benefit. Once preclinical testing is complete, an Investigational New Drug (IND) application is submitted to a regulatory agency, such as the U.S. Food and Drug Administration. Health agencies must approve the submission before a clinical trial can proceed.

Once a trial has concluded, an application is submitted to regulatory agencies. The application, which usually follows the completion of a Phase 3 or pivotal study, contains all data gathered about the safety and efficacy of a study medicine. The application also contains information about the preclinical studies, chemistry, toxicology, pharmacology and manufacturing processes of the product. The regulatory agency reviews the information and, if approved, the new medicine can be provided, usually through a prescription.

Doctors or patient advocacy organization can provide information about opportunities to participate in clinical trials. Potential participants and caregivers can also search on https://clinicaltrials.biomarin.com or www.clinicaltrials.gov.

Study teams discuss the requirements of a trial and review potential participants’ health status and medical history. Each study has eligibility criteria, such as age, birth sex and treatment history. Criteria vary from study to study, even for the same condition.

Interventional studies involve the use of a study medicine, medical device or procedure. Observational studies usually involve the collection of health information, but study participants do not receive any intervention. Collection of health data could include exams with a study doctor, completion of surveys and tracking of how you feel both in the clinic and at home.

Placebo-controlled clinical trials assess a study medicine compared with an inactive version of the intervention. Typically, one group of participants is randomly assigned to receive a placebo. Sometimes the study doctors and participants know who gets the placebo (open label) and other times they do not (blinded).