The clinical trials journey

IT takes at least 10 to 13 years for a new medicine to complete the research and development process — approximately the same time needed for a child to finish his K to 12 basic education.

Biopharmaceutical research and development is set in motion by the discovery process, which takes three to six years to complete. Another six to seven years is required for the next stage called the development process.

Apart from the time necessary to discover and develop potential new medicines, biopharmaceutical companies also invest about $2.6 billion in the complex search for life-saving drugs. It includes the cost of failures where thousands, or millions, of compounds are screened but only few eventually receive regulatory approval.

Determining the quality, safety, and efficacy of medicines is primary to the drug development process. Clinical trials are at the center of the development process, where all potential medicines undergo extensive studies to demonstrate that they are safe and effective for human use.

Phase I of the clinical trials is the first time that the candidate medicine is tested on people. It involves initial safety testing with a small group of healthy volunteers, usually 100 people or less. Scientists examine the pharmacokinetics of the drug, such as how it is absorbed, metabolized, and eliminated from the body. They also look into its pharmacodynamics, zeroing in on any potential side effects. Results of Phase I clinical trials will help identify the safe dosing range for the candidate medicine.

The next stage of development is the Phase II clinical trial where researchers assess the safety and efficacy of the candidate drug in about 100 to 500 patient volunteers. Phase II trials compare patients receiving the drug with patients receiving either an inactive substance (placebo) or a different medicine that is the standard of care for the disease. Scientists continue to identify optimal dose strength and schedules for using the candidate medicine, as well as any possible adverse events.

If the results are positive during this stage, researchers advance to the much larger Phase III trials. This third and final stage in clinical trials is also considered the costliest and longest phase in the development process.

There is a huge expectation for the candidate drug at this stage. A candidate medicine must demonstrate safety and efficacy in a large group of patients, enrolling 1,000 to 5,000 people or more in different countries. This often requires the need to coordinate study results in various sites at hospitals and centers across the world. Biopharmaceutical companies work closely with each of the trial sites as well as the Institutional Review Board (IRB) or Ethics Committee and the regulatory agency. Moreover, the expertise of a clinical research organization is often needed to aid in the day-to-day operations of the trial.

The main objective of Phase III trials is to generate statistically significant data about the safety, efficacy, and the overall benefit-risk relationship of the potential medicine. It also ensures the proper use of the drug such as generating data for potential interactions with other medicines and specific dosing instructions, among others.

If the drug continues to show relevant outcomes, biopharmaceutical companies prepare for high quality production for use in the trials while planning for the full-scale production of the medicine after approval. (For more information about the clinical trials process, read the “Biopharmaceutical Research and Development: The Process Behind New Medicines” on phrma.org.)

(To be continued)

Medicine Cabinet is a column of the Pharmaceutical and Healthcare Association of the Philippines (PHAP), representing the research-based medicines and vaccines sector in the country. The author is the executive director of PHAP. E-mail the author at medicinecabinet@phap.org.ph.