Successful pharmaceutical development requires the balancing of three elements, each of which imposes constraints on the process.
For potential therapies emerging from basic academic research, finding a means of interfering with cancer biology is usually the only concern. This is a critical part of the process – there would be no new drugs without it. It is, however, only a first of many steps to a licensed drug.
Regulatory agencies, such as Health Canada or the FDA, are responsible for ensuring that therapies are safe and efficacious before approving the sale a drug. Their prime concern is minimizing risk to patients. These agencies mandate a structured, step-wise approach to collecting the evidence for safety and efficacy. Quality standards are in place governing how an investigational drug can be produced, and what studies are needed prior to testing in humans. Agency review of the evidence, and agency approval, are required before each study of the drug candidate in humans. Initial testing in humans is for safety only, in a Phase I clinical trial typically involving about 10 – 20 people. Subsequent larger Phase II and III clinical trials clearly define the drug’s safety and efficacy. If the risk/benefit ratio is acceptable to the relevant agency, a new drug is brought to market.
While estimates vary hugely, bringing a new therapy to market requires the investment of hundreds of millions of dollars. This partially reflects the cost of working to mandated quality standards, which increase in stringency through each Phase. Bringing a new therapy to market can take 10 years of development. While an academic investigator may find the several million dollars needed to get through a Phase I clinical trial, development beyond this will be done within the pharmaceutical industry.
While market launch of a product occurs years later, discovery researchers need to thoroughly examine a project’s commercial aspects before starting on the product development path. Investigators need to build (and maintain) a credible business case for their potential therapy. As a minimum, this would identify and quantify the unmet medical need, and establish intellectual property protection. To get your therapy to patients, you will eventually need to satisfy an investor that they can recoup the considerable investment of money needed to bring it to the market.