Indication Expansion & Off-Label Uses for Cancer Therapies

With the rapid advances in technology and research techniques achieved over the past several decades, the healthcare community has made enormous strides in understanding how cancer develops and spreads, and continues to make progress in improving patient treatment outcomes. However, there is still a great deal of work to be done. We’ve learned, for example, that cancer is a moving target and its growth is driven by many complex factors, including genetic changes within tumors. As researchers untangle the multifaceted processes by which malignancies develop and progress, they are becoming increasingly capable of developing therapies that target the genetic underpinnings of an individual patient’s cancer. Indeed, this type of precision medicine is beginning to bear fruit and is revolutionizing cancer care.

Of course, researchers and physicians aren’t simply waiting for a new paradigm of cancer treatment to emerge. Oncologists around the world are doing everything they can to ensure their patients benefit from the latest research advancements right now, and this often means relying on off-label uses of approved cancer therapies. These real-world applications of off-label drugs complement research into precision cancer care, as the outcomes can provide valuable insights into everything from the genetic features of tumors that impact treatment efficacy to the propensity of certain types of cancer to develop resistance to specific drugs. As an oncology-focused bioinformatics company, M2GEN’s mission is to move precision medicine forward by collecting, organizing, and contextualizing such data, making it more useful to our research partners.

Understanding Indication Expansion & Off-Label Drug Usage

To provide the highest quality cancer care, physicians must leverage the collective knowledge of oncologists and cancer researchers around the globe. As research advances, new evidence frequently emerges that therapies approved for a certain disease might be effective in fighting another. A new indication means there is evidence that a drug is effective for use in an application other than what it was originally developed for.

In oncology, indication expansion is key in not only helping improve treatment outcomes for countless patients but also in advancing researchers’ understanding of existing therapies and the behavior of specific types of cancer. Patients who are diagnosed with cancer deserve access to the latest treatment advances, and indication expansion is frequently used to make this a reality. Most often, this manifests in the prescription of off-label drugs.

FDA approval of a new cancer therapy is based on a comprehensive assessment of its risks and requires strong scientific evidence of its benefits. This approval process also involves thorough labeling that dictates how the drug is intended to be used. However, healthcare providers aren’t entirely constrained by such labeling, and prescribing off-label drugs based on new indications is a common practice. This is especially true in cancer treatment, where combination therapy plans are often developed based on a wide array of factors, including the type and stage of a patient’s cancer as well as the genetic features of the tumor, such as the presence of specific biomarkers.

Off-label drug uses can include everything from dosage adjustments to prescribing a drug for a type of cancer other than what it was originally developed to treat. However, off-label drug use is not without obstacles and risks. Physicians base their decisions to prescribe off-label drugs on strong evidence that they are safe and effective within a given treatment plan. However, this evidence will never be as strong as that generated through the rigorous testing required for FDA approval. For this reason, indication expansion and data collected from the outcomes of off-label drug usage are effectively utilized in the development of new clinical trials for existing therapies.

How M2GEN Helps Expand the Use of Existing Cancer Therapies

As part of our mission to accelerate the discovery and development of precision medicine in cancer care, M2GEN is committed to facilitating indication expansion as well as the safe and effective use of off-label drugs. We have leveraged the patented Total Cancer Care^® protocol to provide a never-before-seen data generation pipeline, providing researchers with contextualized longitudinal clinical and molecular information for hundreds of thousands of consenting cancer patients.

Our continuously expanding TCC Active Clinical Registry includes data ranging from patient demographic, clinical, and diagnostic information to molecular and genetic sequencing results and analyses based on clinical treatment attributes. This enables researchers to accurately track the efficacy of treatments over the course of many patients’ lifetimes, efficiently assess pharmacovigilance, and build treatment-protocol-focused expanded access programs (EAPs). Additionally, with access to this robust, highly structured data, it’s easier than ever for researchers to perform indication expansion and off-label drug usage analysis to effectively and efficiently expand market opportunities from existing cancer therapies.

M2GEN also utilizes our unprecedented data pipeline to provide a full suite of custom and ready-made bioinformatics solutions. For example, our signature ORIEN AVATAR platform can be used to create robust comparator cohorts as well as in silico modeling and trial design, helping to rapidly identify promising new targets for existing cancer therapies. Beyond providing real-world evidence for off-label drug usage that can help improve treatment outcomes right away, new indications gleaned from our bioinformatics solutions can be used to develop in vivo clinical trials needed to submit Supplemental New Drug Applications (sNDA) for FDA approval. M2GEN makes this easier as well since patients within the TCC Active Clinical Registry agree to be recontacted, allowing our research partners to quickly identify excellent candidates based on highly specific sets of criteria, such as patient treatment history and the presence of genetic biomarkers.

At M2GEN, we are helping to shape the future of precision cancer medicine while using every tool at our disposal to ensure cancer patients today are receiving the best care. Contact us to learn more about how we help researchers unlock the full potential of existing cancer therapies.