Access Denied: When First Line Treatments Exclude Patients From Clinical Trials

Standards of care, clinical trials, and exclusionary delivery models

The development of new therapies and access to them is a complex and multifaceted process that involves more than just locating a clinical trial. Just because a drug exists doesn’t mean that a patient can access it. That exclusion is strictly related to previous medical treatments, not financial or geographical accessibility, either, which is an entire other dimension.

Imagine that you’ve just had a recurrence of your brain tumor, say, a low grade astrocytoma, and you know that the best treatment available for your recurrence is only accessible through a clinical trial. Then, picture finding out that you are not eligible to receive that drug as a treatment option because of the therapies you received for your tumor the first time around. This is a growing dilemma that patients and physicians are facing as more therapies start to become part of clinical trials and exclude patients if they have had previous treatments outside of their trial arms.

Some might ask, “What’s wrong with the current standard of care?” but it’s those who ask, “Why can’t patients be treated with what might help them the most?” who are on the right track. Patient autonomy in treatment decisions is important, as are the integrity and control factors in clinical trials. Still, it is those who are being excluded from trials who we need to be concerned about.

The history of brain tumor standards of care

Think back to what it must have been like to treat brain tumors before we had temozolomide, otherwise known as Temodar. It’s hard to imagine, seeing as temozolomide in combination with radiation therapy is the standard of care in treating brain tumors today, right? Temozolomide started its first clinical trials in the early 1980s, moving to phase two trials in 1997. It was first approved as a “second line” therapy back in 2001, just as the Optune Device is used as a second line treatment option for more and more patients today. This means that patients with brain tumors had to have had a recurrence in order to receive the drug. The reason for this delay to receive temozolomide was because more clinical trials needed to be conducted. It was only ten years ago, in 2007, that temozolomide made its debut as a frontline treatment for brain tumors. Now, just about every brain tumor patient who receives chemotherapy is prescribed temozolomide.

Problematic standards and trial processes

Just like temozolomide was kept behind clinical trial bars for years, current promising therapies face the same challenge. With some of these drugs only available as second line options, brain tumor patients may never receive them as potential cures or life-extending drugs before they die. Even if patients do have recurrences, the first-line drugs they took may exclude them from the new therapies in the trials. It’s a cycle of inclusion and exclusion where neither the patient nor researchers behind clinical trials win, all because of the regulations in place. But, is there really anyone to blame? Drug regulation and safety precautions take time, and rightfully so.

The problem is that the brain tumor community – or any cancer community, for that matter – doesn’t have time to wait for the current regulation system to complete the job at hand. Perhaps we need more adaptive trials like GBM AGILE to speed things along. Even with the current trials taking place and therapies being discovered, it’s difficult for patients and providers to keep up with them all. For the first time, one singular therapy probably won’t be the standard of care for all brain tumor patients. We haven’t crossed that threshold just yet, but we’re almost there.

A future of possibilities

While I can’t even begin to imagine what the standard of care for treating brain tumors will look like 10 years from now, I can only hope that patients who want to participate in clinical trials to help it get there are able to do so. With so many brain tumor patients taking the same treatments as front line therapies, why not create a trial using some of the treatments considered exclusions from other trials? If we don’t, at this rate we’ll continue to wait nearly 30 years until the next monotherapy standard of care gets approved. Even if a single therapy as a standard of care isn’t the future for treating this disease, it never hurts to have one in our back pocket to fall back on just in case.

Charlie Blotner Precision Medicine Advocate

Charlie Blotner Precision Medicine Advocate

Charlie Blotner is a senior undergraduate student in Family & Human Development at Arizona State University. Charlie has a passion for increasing clinical trial participation, and wants to empower patients to make the most informed medical decisions for themselves possible with the most research information possible. Charlie has spoken at Stanford Medicine X and believes in the power of patient storytelling. As a brain tumor survivor and patient advocate, Charlie co-moderates Brain Tumor Social Media (#BTSM) Chats on Twitter. More than anything, Charlie wants to be able to contribute to the work that helps patients to be able to breathe easier.

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