The Long and Winding Road of Drug Trials

Patients often wonder why there aren’t more options for medications. Getting a new drug to market can take anywhere from 8 to 20 years from development to Food and Drug Administration (FDA) approval. It is costly and the route is often serpentine.

Only about 10 percent of the medicines that are tested in the lab ever make it to human trials. And then only one in five of these will see the light of a pharmacy shelf. Along the way a pharmaceutical company will spend an average of $360 million.

There are seven phases in the Food and Drug Administration’s approval process for new pharmaceutical agents. In the pre-clinical phase, researches look to determine the causes of a disease or, in the case of a disease such as diabetes, they may look at how the disease has distorted normal body processes.

This leads to an investigation of compounds that can treat these abnormalities. For example, diabetes affects not only the body’s ability to manufacture insulin but also dispose of glucose and metabolize fat appropriately. That is why there are drugs that treat insulin release such as Glipizide® and drugs that treat insulin resistance such as Actos®.

Drug companies will search through thousands of compounds to find a few of therapeutic value. Once a compound is selected, it’s purified and its affects are tested on animals. If the company is satisfied with the results they will apply to the Food and Drug Administration (FDA) for an Investigational New Drug Application (IND) and the clinical part of drug testing begins.

Phase I of clinical trials is conducted on a small group of healthy volunteers to evaluate how the drug works in the human body and its safety profile. This process usually takes about a year. Drugs that make it out of Phase I will advance to Phase II

This part of the process looks at safety, effectiveness and dose response. Instead of using healthy volunteers, researchers begin testing the drugs on the people who have the disease under study. If the drug does well in this phase, the FDA and the company representatives meet to discuss the protocols for phase III and any concerns brought up in phase II.

Phase III takes the longest costs the most money and involves the most people. In addition to further reviews of safety and efficacy, this part of the study compares the study drugs results to similar drugs on the market. And because the drug is tested in a very large number of people, issues of side effects and more subtle safety concerns usually become apparent in this phase. If the drug passes phase III, the pharmaceutical company can

By Nora Saul, M.S., R.D., C.D.E., Manager of Nutritional Services at Joslin

apply to the FDA for the authorization to market the drug.

And it’s not over yet. Companies often continue evaluating their drugs post market release. In fact, given the recent spate of drug recalls and black box warnings, some people think the FDA’s current process isn’t stringent enough.

As it happens diabetes is a hot disease and over the past twenty years a plethora of drugs has been released and more are on the way.   With the many companies researching treatment options, perhaps one will turn out to be a cure.

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2 Responses to The Long and Winding Road of Drug Trials

  1. Ryan Luce says:

    Interesting summary about the challenges of approving drugs. It should be emphasized, however, that there are lots of other types of research – observational, diet/exercise, even surgical – and all are potentially very helpful at driving research forward.

    If you’re interested in finding research for type 1 diabetes, JDRF has a great resource:
    https://trials.jdrf.org/patient/

    If you’re interested in finding research for type 2, there’s a tool to help you do at http://www.corengi.com. (I’m biased – this is a website I founded :)

  2. Tom Beatson says:

    Fortunately, when insulin was first discovered, there was not the long wait for testing and approval.

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