Categories
Diagnosis FAQ

Clinical Trials in Cancer

If you want to look into newer treatment options, it can be hard to know where to start. The FDA has several different approval processes. Here’s a guide for you to help you know what questions you can ask your doctor.

Clinical Trials in Cancer

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. Source

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:

  • https://clinicaltrials.gov – 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.
  • https://www.drugs.com/newdrugs.html – 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).


Cancer Treatment Info Articles

Stem Cell Transplant for Cancer

Bone marrow and stem cell transplants, also called BMT and SCT, are sometimes part of treatment used against blood cancers including multiple myeloma, lymphoma (non-Hodgkin and Hodgkin), and leukemia. ...

Immunotherapy | Cancer Treatment FAQ and Patient Stories

Your immune system fights infections and diseases in your body. Immunotherapy is a kind of biological therapy or treatment that uses your immune system to fight cancer. Immunotherapy is still relatively new which makes its success a bit hard to predict. It's also focused on some of the more common cancers. ...

Targeted Therapy | Cancer FAQ, Patient Stories

Targeted therapy is a type of cancer treatment also known as precision medicine and molecularly-targeted drugs/therapies. There's been growing focus on targeted therapy as a major part of precision medicine, which diagnoses and treats individuals based largely on their genes and proteins. It can also be used to prevent disease. ...

Hormone Therapy for Cancer

Hormone therapy is also known as hormonal therapy, hormone treatment, or endocrine therapy. It is used to either slow or stop the growth of cancers that grow using hormones. ...

Cancer Surgery

One of the most common ways of locally treating cancer, in particular solid tumors, is through surgery. Surgeons, medical doctors, are trained to remove cancer from you body during operations. ...
brachytherapy

Radiation Therapy

Radiation therapy, also known as radiotherapy, uses high doses of radiation to destroy cancer cells and to shrink tumors. Find answers to popular radiation therapy questions and experiences of radiation therapy shared by The Patient Story community. ...

Chemotherapy

Chemotherapy or chemo is one of the most common types of cancer treatments that uses drugs to destroy cancer cells. Find answers to popular chemotherapy questions and experiences of chemotherapy shared by The Patient Story community. ...

Newest CLL Treatments: Targeted Therapy Drugs

Treatment of Chronic Lymphocytic Leukemia is moving towards targeted agents. Chemo and transplants are becoming less common. Hear about the newest treatments from real experts. ...

Health Literacy

October is Health Literacy Awareness. Health literacy is when the information and health services you need align with your ability to process, understand, and use that information and those services. Why does it matter? ...

Genetic Testing For Cancer

Read real genetic counselors and patients explain how genetic testing works, how it affects treatment and outcomes, and other important information to know. ...
Favorite0

Leave a Reply

Your email address will not be published.