THE QUALITY, safety, and efficacy of medicines are primary to the drug discovery and development process. When a candidate medicine demonstrates promising results, its entire history from the early discovery to the development stage will be written in a 100,000-page report for regulatory review.
The lengthy report contains results and data analysis from the entire clinical development program, pre-clinical testing, and proposals for manufacturing and labeling. It also includes results of extensive studies that demonstrate the safety and efficacy of a potential medicine.
With understanding about diseases at the molecular level, potential new medicines go through the discovery process, which includes early phases of basic research, drug discovery, and pre-clinical stages.
The discovery process could take approximately three to six years of identifying an investigational drug and conducting initial laboratory tests. It involves identifying potential targets and eventually narrowing it to one lead compound, or a promising molecule that could influence the target and, potentially, become a medicine.
Another six to seven years are required for the next stage called the development process. Clinical trials are an integral part of the development process. These are conducted in three phases with specific purposes to help researchers answer particular questions on safety, efficacy, and the benefits and risks of the candidate medicine. It involves careful coordination in the various clinical trial sites and close monitoring of about 5,000 patients across the globe.
After hurdling the complex clinical trials stage, and when results indicate that a candidate medicine is both safe and effective, a biopharmaceutical company may now submit a new drug application (NDA) or biologics license application (BLA) to the regulatory agency requesting approval to market the drug.
Accelerating the availability of medicines to patients with serious diseases — or where there is an unmet medical need — is possible when the regulatory agency implements expedited approaches to accelerate the development and review of new medicines. The agency may grant a fast track, breakthrough therapy, accelerated approval, or priority review status to a medicine which meets its criteria.
A medicine under “fast track” status allows an expedited review of drugs that treat serious conditions and fulfill an unmet medical need. “Breakthrough therapy” status, on the other hand, provides for an expedited development and review of drugs that may demonstrate substantial improvement over available therapy.
“Accelerated approval” status gives accelerated approval for drugs that address a serious condition or fulfill an unmet medical need, based on a surrogate or an intermediate clinical endpoint.
“Priority review” status, meanwhile, accelerates regulatory agency evaluation of drugs that would have significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.
The regulatory agency, on the other hand, reviews the report containing all the results of the studies with their own scientists, physicians and statisticians. They will weigh the benefits and risks of the potential medicine and decide whether to grant approval. They may also ask for additional data before granting approval or convene an independent expert advisory panel to further review the data submissions.
(For more information about R&D, 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 email@example.com.