Drug Discovery & Development: From Lab to Market

Pharmaceutical Research and Development Journey

From Concept to Market: The Drug Development Timeline

From the initial idea that a chemical compound could become a drug until the molecule is ready for marketing, the process typically spans about seven to twelve years.

Sources of Medicinal Compounds

Chemical defenses from fungi and and plants against viruses and bacteria are significant sources of drugs. The contribution of the marine world has achieved a high profile, exemplified by AZT for AIDS, which was extracted from herring sperm. The plant kingdom is a paradise for pharmacology. Animal-derived medicines are also obtained; for instance, insulin was first extracted from the pancreas of a dog.

Sometimes, medicines designed for one objective prove useful for another. During the course of investigation, the need for animal products that could become drugs stems from the careful consideration not to expose humans to possible risks. For some time, animal testing has been a controversial issue. Animal testing, although not sufficient since the results cannot be completely comparable to humans, may still be necessary.

The Unexpected Discovery: A Case Study

In 1985, an investigation began to find a drug against hypertension. Several years later, in 1991, it was time for clinical trials in healthy volunteers to analyze their effects. Headaches, vision problems, and stomach issues were administered and recorded. However, to the surprise of all, the volunteers reported an increase in the frequency and intensity of erections. The lab soon converted their search for a coronary vasodilator into the first oral drug for the treatment of impotence.

In recent years, the use of this drug has been expanding among young people, often to compensate for the antagonistic effects of other drugs or alcohol. Doctors warn of the dangers of this cocktail, which can cause sudden death.

Drug Testing and Approval Phases

For a drug to be accepted, it must undergo rigorous testing:

• Bacterial Testing: In bacteria, tests show whether compounds induce mutations. If so, the drugs are not developed further.
• Animal Testing: Initial studies are performed in mice, and later must include at least one non-rodent animal. Initially, large doses of the compound are administered to determine the toxic dose and identify which organs have been damaged. Subsequent tests assess the effect on fertility, particularly the possibility of fetal abnormalities.

Human Clinical Trials

1. Phase 1: In the first stage, the drug is administered to healthy volunteers who often receive an economic incentive.
2. Phase 2: The second stage involves hundreds of volunteers, typically without economic incentive, who are suffering from the disease being treated. Over several months, some patients receive the investigational medicine, while others receive standard treatment. This is known as a double-blind study, where neither the doctors nor the patients know who is receiving which treatment.
3. Phase 3: The third stage aims to determine the optimal dose. Pilot programs may also be proposed for terminal cancer patients.

Regulatory Approval and Generic Drugs

Medicines are subject to very stringent checks before being allowed onto the market. The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) are responsible for monitoring this process.

If all goes well, the pharmaceutical company that funded the drug patents it. After approximately 20 years, when the patent expires, the drug enters the public market, allowing other companies to manufacture and market copies of the original, known as generics. A generic drug is a product identical in composition and dosage to one that already exists in the market. Among the advantages of generics is their lower price, resulting in significant savings for patients.