Clinical Trials: A Guide to the FDA Approval Process

Clinical Trials: A Guide to FDA Approval

All new drugs and therapies in the U.S. go through lots of testing and monitoring before they are approved by the FDA. During the approval process, though, there are clinical trials. These trials can be especially helpful and potentially lifesaving if you have a rare cancer or orphan disease with few treatment options available. Some of these clinical trials may go through a quicker approval process than others depending on a number of factors.

What are the FDA's different approval processes?

If you’re interested in being part of a clinical trial, you need to know some key terms. The FDA has a regular (standard) approval process, but many clinical trials, especially those for patients with rare cancers, go through accelerated or expedited processes.

According to the FDA, “Over the past three decades, Congress has established five programs aimed at expediting patient access to important drugs that treat serious or life-threatening conditions.”

These five programs are:

  • Fast Track Designation

  • Breakthrough Therapy designation

  • Regenerative Medicine Advanced Therapy designation (RMAT)

  • Priority Review designation

  • Accelerated Approval

If a drug or therapy has one of these designations, it could mean that the FDA likely sees that it has the potential to fill a need. It can be helpful to search these designations for new and upcoming treatments that could meet your specific needs.

  • Fast Track designation can usually be granted if there is an unmet medical need, and a drug’s data thus far shows promise.

  • Breakthrough Therapy designation can be given to drugs that show a significant improvement over therapies or drugs that are currently available.

  • Regenerative Medicine Advanced Therapy designation (RMAT) is for getting regenerative medicine therapies (i.e., stem cell transplants) with the potential of meeting an unmet need for a serious condition on the market quicker.

  • Priority Review designation is opposed to the FDA’s standard review. Many of the new treatments the FDA approves fall under standard review. This is a process that usually takes about 10 months for review. With Priority Review, the goal is six months. This is for treatments that have shown proven, significant improvements for a serious condition.

  • Accelerated Approval is a little different than the other four programs. It allows a drug to be approved based upon what’s called a “surrogate endpoint.” That’s medical jargon for “a blood test" or "a CT scan” that shows improvement that proves the drug is working.

    For example, if you had a tumor and were on a drug in the Accelerated Approval program, and the tumor was shrinking every few weeks, that might prove the drug was working without having to wait to see how you feel or how long the treatment extends your life since those things can take years to find out.


It’s also important to note that clinical trials go through three different phases.

You may find it difficult to keep up with new treatment options like clinical trials. You might not feel comfortable bringing them up to their doctor.

Dr. Berenson is a myeloma specialist and has been practicing medicine for nearly 40 years. He is also part of a non-profit that conducts clinical research. with many groups of oncologists throughout the country.

He says, “If your doctor doesn’t want to look into treatment options that make you more comfortable, find someone who is willing. You don’t have to stick with that doctor. You don’t have to go with their approach.”

If you find it difficult to keep up with your treatment options while in the thick of a diagnosis, here are two quick and simple resources for you to find new treatments:

  • - Here you can search your condition and see the status of clinical trials for it. Look for ones that say “recruiting.” Even if you don’t understand all the jargon, you can always present it to your doctor and see if they think it’s a viable option.

  • - If you’d rather not be part of a clinical trial and want something that the FDA has already granted full approval, you can search for your condition here. This is a constantly updated list of newly approved treatments. Simply search for your condition, and you can read the press release (or show it to your doctor) about the new drug.

If you feel nervous about presenting an idea to your doctor, just remember: the worst they can do is say, “No, I’m sorry. That’s not a good option for you.”

Doctors typically stay on top of clinical research better than we do, but that’s not to say they aren’t busy people. You might find something that they haven’t had the chance to see yet. Remember: no harm ever came from advocating for yourself or asking a question.

Casey Head had Acute Lymphoblastic Leukemia. She was going to be part of a clinical trial, and it fell through.

Another became available, but she had to fight to get in it. She sent a letter of compassion to the drug company to allow her to get in it.

She got in and ultimately, it was that immunotherapy (Inotuzumab) that put her in remission.

“The immunotherapy worked. It was the Inotuzumab. I went through four rounds of that, and it worked like a freaking lucky charm. I’ve been in remission ever since.”

Don’t lose hope. Researchers are constantly coming up with new treatments, and doctors are always looking into new ways to treat you safely and effectively. Reach out to The Patient Story and other resources if you have any questions. Most importantly, always consult with your doctor(s).