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Preparing for a Clinical Trial

As the third blog in a series introducing drug development, I am thrilled to be discussing participation in a clinical trial. Clinical trials are the only way to get new treatments to market and choosing whether or not to participate in a trial is a significant decision. As you can imagine, it’s important to evaluate the trial to determine if it’s a good fit for your family. Here are the key things to focus on:

Doing diligence

Do your own research so that discussions with your healthcare providers are as comprehensive as possible. What is known about the treatment being studied? What is the expected benefit of the treatment and will that be meaningful to your family? Has it been tested in other patients and what are the side effects? If there are publications about the treatment from other clinical trials, ask for them, not only to get up to speed on your own, but to help you speak to experts that can help you decide.

Get opinions from educated sources about the science supporting the treatment and any actual data. Consistent evidence from animal studies or human studies supporting the treatment’s use in patients should be available. Though the study physician will be knowledgeable about the study, their hat is already in the ring, and it’s important to talk to experts in the field that are not directly involved in the research as well.

Studies that have sound science to support therapeutic effects will be important if the treatment has been in limited or no human trials before, but clinical trial data will be key for knowing what to expect if the treatment already has a history in humans. The answers to all of your questions may not be known even if there is a lot of experience with the treatment. Keep in mind that not all studies will be a good fit based on the data available, and that’s ok. Just because a study is available does not obligate you to participate.

Does the study work for me?

Once you’ve done your diligence and feel the study is worth doing, here are ten things to keep in mind when you’re ready to commit:

Determine if your child is eligible

Trials have entrance criteria to identify and maintain patients that are appropriate to receive the treatment. Some criteria are included to detect changes with the treatment, like a disorder severity score, to ensure the right group to see change is included. Other criteria are required for safety reasons, like excluding certain medications or medical conditions thought to be or known to be harmful in combination with the treatment. Therefore, don’t fudge the numbers here. If there’s any question about whether eligibility is met, err on the side of caution and wait for the next study. Usually entrance criteria are narrow and very restrictive early in development. But they can be relaxed later as data accumulate to show what is necessary for safety and efficacy and what might not be.

You may not benefit

Not everyone who receives a treatment will benefit, and if a placebo is involved, not everyone may receive treatment. Whether or not you benefit directly in a clinical trial, you are contributing in a meaningful way by helping to define what works and what doesn’t, and how much of an effect is detectable over another treatment or no treatment. These data are critical for making educated decisions for the next stage of development.

Know the risks

All treatments have risk. Those not yet approved by the FDA need to be thoroughly evaluated to know how safe (or dangerous) they really are. Even if a side effect is very rare, it could still occur. Be sure you understand and accept all of the risks of the treatment before you consent to the study, and as new information becomes available.

Be informed when you provide your consent

As a participant or a caregiver of a participant you are protected to ensure that you are aware of all of the responsibilities of being in a study, the risks involved, and the option to withdraw your consent. Understand the time commitment it will take to attend all of the study visits. Understand exactly what will be required of you when you’re not in the clinic. What do you have to monitor? Can you commit to daily or weekly or monthly evaluations? Can you commit for the duration of the study? Follow and take seriously all instructions you receive from the study team.

Randomization means your treatment is assigned randomly

Requests can’t be made – you get what you get. If the study is also blinded (meaning treatment assignments are unknown) no one can guarantee you are receiving any one of the particular treatment options, and a specific dose level can’t be assured.

Stay diligent

Be diligent about completing diaries and other at-home activities, at home. Don’t wait until you’re in the parking lot before a visit to fill in data that should have been completed in real time. Data obtained under those conditions can derail a drug development program, for better or for worse.

Don’t throw anything away

Keep all packaging from any treatment administered outside of the study center and bring it with you to every visit. Studies must account for every dose that was taken.

Honor your commitment

Even if you think the treatment is not working, stick it out! Patients that discontinue the study leave holes in the dataset. If too many people leave the study because they are disappointed with their results, there won’t be enough data to draw conclusions about whether the treatment is effective. Data that show placebo doesn’t work or a dose level is not effective is just as valuable and important as data that show effective dose levels.

Treatment may be limited

You may not be able to take the treatment outside of the trial, including if you leave early or after your participation is complete. If the treatment is investigational it can only be taken under the conditions in the protocol at those centers approved to participate in the study. You can’t leave the study and then change your mind.

You can be in a study and still see your regular physician

Your regular physician is still your physician and will be your physician when the study is over. Study physicians are qualified and selected by pharmaceutical or biotech companies to treat patients for that specific study. Study physicians may also have their own practice, just like your regular physician. You don’t have to give up your regular physician while participating in a study. However, they should be made aware that you are participating in a study so that medication changes or other medical care can be discussed with the study physician to ensure any changes won’t disqualify you from participating.

Deciding to participate in a clinical trial is a personal decision, but not one that should be made in isolation. Be sure you agree with the study concept and don’t stop asking questions until you are satisfied. And then make sure to be realistic about what it will really take for you to participate, if you choose to participate. There is no better setting for “knowledge is power” than when considering a clinical trial.  Make sure you know everything you can to make the best decision.