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Understanding Research

By Cherie C. Binns
laboratory-2815641.jpgDid you know it takes an average of fifteen years and a cost of one billion dollars to bring a single drug to market? For every one hundred drugs that start in the research pipeline only one makes it to FDA approval. Let’s take a few minutes to look at why this happens to understand the process that currently takes place in research.

All research begins with an idea. A researcher might think of a way to reduce inflammation in the central nervous system thereby preventing damaged nerves. This is where research animals come into play. Mice are given a virus that mimics MS in the damage that is caused in the CNS so that researchers can test drugs on them to determine if the drugs do what the researcher thinks they can do. If the drug does what is hoped, the next step is to try it on a small group of humans to see if results are similar. The process of developing the idea and successfully testing on mice is slow, often taking 8-10 years.

When desired results are seen in the lab, a Phase I clinical trial starts. These trials are small and generally do not have more than a hundred people in them across the country. Everyone in the trial gets the new drug in one of two or three doses calculated to have worked in the lab. This generally lasts about a year. Phase I clinical trials look at safety and effectiveness. Is this drug safe to give to humans? What dose works best? What side effects occur at different doses?

If Phase I shows that a drug has benefit at one or more doses, it then goes into a Phase II clinical trial where there may be 500 or more people. These trials have what are called “treatment arms.” A treatment arm may be a placebo (inactive substance that is administered exactly the same way the real drug is given), or actual drug in one or more arms with different dosing in each arm. This shows researchers if the drug has benefit over doing nothing (placebo) and if there is benefit, which dosage gives greater benefit without increasing potential for harm. This phase of a Clinical trial generally takes a couple of years to complete and there may be only a half dozen participants at each site and 90 to 100 trial sites around the country.

If the benefit of the new drug is greater than any risk it may pose to the individual, it moves into Phase III Clinical Trials where the study population increases to a couple thousand individuals. Phase III trials go for about three years. Generally, about half of the group being studied is on the new drug and half on placebo. Today, however, if the drug is a disease-modifying therapy, researchers are not using placebo because it is no longer thought to be ethical to allow an individual to go the length of time a Phase III clinical trial must run with nothing to protect their CNS from damage. The control group in these trials generally consists of individuals who have been randomized (chosen by a lottery of sorts) to receive a proven DMT. The remainder are getting the new drug. Often the individual does not know which group they are in and the researcher also does not know. This is what is known as a double-blind trial.

The data is then gathered from all of the trial sites around the country and tabulated. They look at percent improvement over that of the control group. Side effects that were reported by even one of the 2,000 participants are noted as well as the effect of those side effects. This generally takes about six months to complete after the trial is over. These reports then go to the FDA for approval of the drug, another six-month process.

There are ways that we all can shorten this process by adding our voices to patient directories that are then deidentified and shared with researchers so that they can better understand what patients want for symptom relief, disease modification, quality of life. One of these directories is www.iconquerms.org which is in a relationship with MS Focus: the Multiple Sclerosis Foundation to recruit members for whom research is important. To get an informational packet from MS Focus, call 888-673-6287 and ask to speak with Kathryn and she will send it to you. Or go directly to the website for I Conquer MS, create an account and start answering questions to add your voice to the research. In so doing, we have the potential to shorten this process by years as our input helps researchers focus on what those living with multiple sclerosis really want. There is also a place for you to add an “idea” for research!