Medicine Safety Journey

Explore this section to view how safety is integrated into every phase of testing and development, and how efforts to manage potential safety issues in approved medicines continue for many years.

Drug Development Overview

Developing a medicine that is effective in treating or preventing a specific disease with an acceptably low risk of side effects is a major endeavour that can take up to 15 years. Only about 250 of every 10,000 compounds that enter the research and development pipeline will make it into human testing—and only 1 of those original 10,000 will actually become an approved medicine.

During the research and development process-and after a medicine is approved—pharmaceutical companies have ethical and regulatory responsibilities to collect, analyze, and communicate information about the benefit-risk profiles of its products. In order to ensure that companies meet these obligations, regulatory agencies require them to submit many different types of reports and documents during the lifetime of a medicine—from pre-approval to post-approval.

PRE-APPROVAL

Safety in Drug Development View

During the research and development process, medicines are evaluated for safety continuously against the rigorous standards expected by the scientific and medical communities, government and other health agencies, and the pharmaceutical companies themselves. The more common side effects of a medicine are identified during the research and development process. However, rare side effects can sometimes only be identified when the medicine has been taken by many thousands of patients in the general community.

Safeguards for Study Participants

The first clinical studies in humans are conducted to test safety in small numbers of subjects who are usually (but not always) healthy volunteers—that is, individuals who do not have the disease for which the medication is being developed. In these early studies, information is also obtained to help design the safest and most appropriate dosing in later clinical trials. As the process continues, a larger number of subjects—patients with the condition or disease to be treated—are recruited. Throughout this rigorous testing process, multiple safeguards are in place to protect the rights and safety of study participants.

POST-APPROVAL

The Need for Post-Approval Safety Monitoring

Although regulatory drug approval represents a significant judgment about the safety and efficacy of a new medicine, it is not the final safety checkpoint. Rather, it is an important threshold based on sufficient clinical data to show that the benefits of a medicine for the intended population and use outweigh its known risks at the time of approval.

Safety surveillance continues for as long as the medicine is prescribed. This is because the rigorous clinical testing that medicines undergo before approval cannot detect every possible side effect. In fact, it is recognized by regulatory agencies, by the pharmaceutical company sponsoring the new medicine, and by the doctors who prescribe it that additional safety risks may become apparent as the newly approved medicine is used:

• More widely—in much larger numbers and broader groups of patients
• For longer periods of time—sometimes for months or years
• Under "real-world" circumstances rather than the more predictable and closely monitored conditions of clinical trials

Therefore, pharmaceutical companies and regulatory agencies take steps post-approval to monitor emerging safety issues and manage known risks. This set of strategies is collectively known as pharmacovigilance.