What are innovative treatments and why should I consider them?

An innovative treatment is a new treatment approach that aims to improve outcomes for cancer patients. What once seemed impossible is quickly becoming a reality thanks to a number of technological breakthroughs in how we find, visualize, understand, and treat cancer. These innovative treatments are mainly found in clinical trials and not yet approved for widespread use.

Clinical trials are how all cancer drugs are approved. Through years of various studies, drugs are first shown to be safe enough and then they must prove they offer better results than the currently approved standard treatments in a given cancer type. After this long process—frequently over a decade—new cancer drugs become available (and, then, are usually covered by health insurance).

The Keytruda Story: A Major Advance and a High Profile Patient

The story of pembrolizumab (Keytruda^Ⓡ) is an exciting illustration of the enormous patient gains from innovative treatments. In 2014, the FDA approved the drug for treatment of advanced cutaneous melanoma (a high-risk skin cancer). In most cases of cutaneous melanoma, and in several other types of cancer including lung cancer, head and neck cancer, and bladder cancer, cancer cells hide themselves from the body’s immune system. Pembrolizumab was the first in a new class of drug that was able to “uncloak” these tumors, allowing the patient’s immune system to see the cancer and attack it. This was a game-changer as previous standard treatment in these cases offered little hope.

Former president Jimmy Carter suffered from metastatic melanoma that had spread to his liver and brain. In December 2015, after receiving a combination of treatments including pembrolizumab, it was announced that he was free of cancer.

Pembrolizumab has been a major advance in the treatment of cancer and is now widely available. Studies have shown that patients with metastatic melanoma treated with pembrolizumab (and other drugs like it) improve their chances of being alive 5 years past their diagnosis by at least 500% compared to chemotherapy historically.¹ There are now more than 20 different drugs that work in a similar way, some of which are also already approved for widespread use and others that are available only in clinical trials.

Today, over 1,000 different clinical trials are continuing to explore how this class of drugs can best be used and what other similar approaches might someday offer even greater results. Innovative treatments as exemplified by pembrolizumab represent the best hope for longer and healthier lives for many cancer patients.

Understanding Clinical Trials

Clinical trials are often viewed—by patients and doctors—as a last resort after all standard treatments have been exhausted. We believe for high-risk cancer patients, this is backwards. We believe clinical trial opportunities should be considered at every point and balanced with standard treatments. We also believe when patients are matched to clinical trials based on a scientific rationale instead of the luck of where a clinical trial is offered, the benefits can be remarkable.

While trials might have incidental costs such as travel, in nearly all cases, the costs of clinical trials are covered by a patient’s health insurance and/or the trial sponsor.

In non-cancer-related clinical trials, subjects of a study’s placebo group will typically receive a sugar pill of sorts, rather than any actual treatment. With cancer patients, it would be unethical to not provide treatment, so in most cases, if a patient does not receive the more promising innovative treatment, they would still receive a standard therapy, meaning the same treatment they would likely have received outside the trial, anyway. In many cancer trials, patients know what group they are in and can always voluntarily withdraw at any time.

Early Access to the Drugs of Tomorrow

Over a decade ago, the first clinical trial of pembrolizumab kicked off, enrolling 1,260 patients.

It would be another 8 years before pembrolizumab was approved. During those 8 years, it is estimated that over 70,000 new cases of advanced melanoma were diagnosed in the US alone. Nearly all of these patients received standard chemotherapy, with its limited effectiveness in keeping patients alive.

As cancer treatment becomes more complex and personalized, innovative treatments offered via clinical trials often offer early access to tomorrow’s more advanced treatments. Pembrolizumab is just one story—one treatment approach among thousands being pursued currently.

At any given time, there are over 10,000 ongoing clinical trials in cancer, each at various stages and they often involve complicated patient requirements. There are many types of trials and each can have varying benefits for any given patient. Some trials are early, but promising. Others are long established and have more predictable outcomes.

The majority of cancer patients face overwhelming odds. Over 60 percent of cancer patients have high-risk cancers and are not expected to be alive in 5 years, and yet only 3-5 percent of adult cancer patients participate in clinical trials and receive innovative therapies.

Why would this be?

Why Most Patients Are Not Getting Innovative Treatments

Most cancer patients are not offered a clinical trial and virtually none are offered a trial that would transfer their care to another medical center. Oncologists work long hours and lack the tools and time to keep up on thousands of clinical trials and advances across the entire oncology research field. Innovative treatments only reach the prescription pad after they’ve been approved as a standard therapy, despite being available for years via clinical trials.

Just as cancer patients are unique in their type of cancer, tumor location(s), treatment history, and genetic make-up, the army of oncologists and researchers in the fight against cancer are highly specialized. Often a single researcher or group of researchers at a particular cancer center will have deep expertise and knowledge about a particular cancer type, treatment approach, or specific gene mutation.

For most patients, the only chance they have to be offered a clinical trial is when they happen to walk into the expert’s office and are a perfect match for the trial that the researching doctor is already running.  The low chances of this happening is probably the reason only 3-5% of adult cancer patients participate in clinical trials today.

Without a deep understanding of the entire drug development landscape, patients are powerless to reach the right care team.

What Else You Need to Know

The health care system does not offer incentives for oncologists to send their patients to other cancer centers where the treatment options might be different.

With the current limitations of the system, many cancer patients and their families are in a race against time to do research on their own, but the complexities of cancer make searching and understanding publicly available information nearly impossible.

This is why it matters which medical center and oncologist provides your care and why you need to know how to talk to your oncologist about innovative treatments.

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1 https://pubmed.ncbi.nlm.nih.gov/34509219/

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