Changing the face of medicine

Zoomer | April 1st, 2000

Research and Development of new drugs costly, time consumingTraditionally, medical progress was measured in terms of decades, even centuries. But thanks to the billions of dollars provided annually by the pharmaceutical industry, governments and private investors, today’s advances in medicine come at a pace that only a generation ago seemed impossible.

Research and Development (R&D) is a long and complex process, marked at each stage by risk of failure. If all goes well, the end result may be the introduction of a new prescription medicine. But even the most promising of ideas can amount to nothing. In fact, although thousands of drugs are created each year, only a handful will become candidates for further development and, of these, few are likely to endure the arduous tests and trials before being approved for use.

Although this painstaking, repetitive, process of synthesis and testing may continue for years, a few successful researchers will eventually see thei drug tested on humans. In Canada, before human testing can begin, researchers must submit a summary of their preliminary findings to the Therapeutic Products Program of Health Canada, includg chemistry and manufacturing data, pharmacologic test results, safety testing results and a plan for clinical testing on people. If the TPP feels potential benefits outweigh the risks, clinical trials involving humans will occur in the following phases:

  • Phase I: Is the drug safe? This initial phase emphasizes safety, not effectiveness – What are the side effects? How will it be absorbed into the bloodstream? What is a safe dosage? Low doses are given to a small group of healthy volunteers under the close supervision of doctors who study how the participants’ bodies react as dosages are increased.
  • Phase II: Does the drug do what it’s supposed to? This phase focuses on the drug’s effectiveness in treating the illness for which it was designed. Researchers also look for effective dosages and methods of delivery. Phase II usually involves 100 to 300 volunteer patients in need of treatment. Three out of four prospective drugs are usually eliminated during this critical phase due to ineffectiveness or severe side effects.
  • Phase III: Making sure. Involving up to 10,000 patients and lasting up to five years, this phase gives researchers the best possible picture of a drug’s effectiveness and limitations. One in five potential drugs still fail at this stage.
  • Phase IV: Post approval. Even after approval and being brought to market, research doesn’t stop. Other uses and indications are investigated, and different forms of the medication, such as intravenous and pediatric formulations, developed. Safety-monitoring studies are conducted on a continuous basis.

Despite the lengthy, complicated process of drug development, the R&D system has been extremely successful in providing doctors and their patients with safe, effective, cures and therapies. Indeed, the hard work and dedication of researchers has seen what were once the six deadliest diseases of the early 1900s – pneumonia, tuberculosis, rheumatic fever, peptic ulcer disease and painful arthritis – all but eliminated as life-threatening ailments. And in the past decade alone, critical pharmaceutical breakthroughs have been made in the treatment of immune system dysfunction, hypertension, mental disorders, and severe AIDS complications.