Breakthrough?: Understanding the Drug Development and Testing Process
by Rich OBoyle, Publisher
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Everyone is gratified by news of a major drug breakthrough, especially if it promises help for people who are terminally ill or severely disabled. And if you or a loved one has been praying for such a drug, the news may seem like a miracle. Unfortunately all too often, reports in the press are exaggerated or seriously inaccurate interpretations of scientific findings. Really significant advances happen less often that the popular press (newspapers, the Internet, radio, television, and magazines) would have us believe.
A healthy skepticism coupled with a working knowledge of the scientific process for drug discovery can help you assess news reports and professional scientific journals. When hearing of advances in treatment for a particular condition or disease, it is helpful to ask the following questions and use the following guidelines:
The Drug Development and Approval Process
Controlled clinical trials, in which results observed in patients getting the drug are compared to the results observed in patients receiving a different treatment, are the best way science has come up with to determine what a new drug really does. Controlled clinical trials are a legal requirement that the U.S. Food and Drug Administration (FDA) makes drug makers prove that the new treatments are both safe and effective.
The process starts with the drug developer (usually a pharmaceutical research and development company), who is seeking to develop a useful and profitable drug. The researchers analyze the drugs chemical properties and test it in animals to determine any toxic reactions. The way that drugs react in animals is not always the same as they will behave in humans. Further tests are necessary before accurate conclusions can be drawn about the effect of drugs in humans.
They developer then asks the FDA for permission to test the drug in people. A panel of scientists, ethicists and nonscientists review the proposal from the drug developer and works to finalize a strict scientific protocol for the clinical trials.
Not all drugs that begin the three-phase process will make it to the pharmacy and medicine cabinet. Some are shown to be unsafe, ineffective, unprofitable, or all of the above. Of 100 drugs for which investigational new drug applications are submitted to the FDA, about 70% will complete Phase 1 and go on to Phase 2. About 33% of the original 100 will go from Phase 2 to Phase 3. And 25-30% of the original 100 will clear Phase 3. Of the original 100 experimental drugs, about 20% will be approved for sale.
Understanding the Design of Clinical Studies
Measuring the effectiveness of experimental drugs can be complicated, even though the process of investigation and analysis is grounded in science. Sometimes the disease simply goes away by itself (like a cold or allergy), varies in intensity and duration (like arthritis or asthma), or can only be measured subjectively by the doctor or patient (like depression or well-being). Scientists use "control subjects" to tease out these aberrations and subjective experiences.
In a controlled clinical trial, patients in one group receive the experimental drug, while those in a comparable group (the "controls") get either no treatment at all, a placebo (a "sugar pill") that looks like the drug, a drug known to be effective, or a different dose of the drug under study. Usually these test and control subjects are studied at the same time. Some studies continue even after the trial is stopped to allow comparisons before and after treatment.
It is important in organizing a group of people for study that they all be of similar age, weight, and general health status, or other characteristics (such as other treatments being received at the same time). Scientists use a process of "randomization" to eliminate problems where the statistical results of the study are distorted. Some studies have been disrupted when too many healthy people throw off the results, or when the drug reacts differently in women than men.
Further refinements to the study are made to remove any bias on the part of the researchers and patients. "Blinding" a study helps ensure that those participating in the study dont know what treatment they are receiving/giving so that they dont overrate the effectiveness of a drug or psychologically influence the results. A "single-blind" study consists of keeping patients from knowing whether they are receiving the experimental drug or a placebo. A "double-blind" study consists of neither the patient nor the data analysts/doctors knowing which patients got the drug. Only after the study is "unblended" do the participants know which drug is which.
Testing experimental drugs in people inevitably presents ethical questions. Is it ethical to give patients a placebo when effective treatment is available? Not all authorities agree on the answer. But the generally accepted practice in the USA is that fully informed patients can consent to take part in a controlled-randomized-blinded clinical trial, even when effective therapy exists, so long as they are not denied therapy that could alter survival or prevent irreversible injury. They can voluntarily agree to accept temporary discomfort and other potential risks in order to help evaluate a new treatment.
Resources for Participating in Clinical Trials
- Participating in
Clinical Trials: What You Need to Know by Drs. Mary Sano and Christine Weber
Resources for Assessing Health Information on the Internet
Source: U.S. Food and Drug Administration
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