The Cancer Patient’s Guide to Clinical Trials

Introduction

In the realm of medical research, clinical trials play a pivotal role in advancing our understanding of cancer and developing innovative treatments. Clinical trials are carefully designed studies conducted to evaluate the safety, efficacy, and potential side effects of new medical interventions. By participating in these trials, patients can gain access to cutting-edge therapies while contributing to the overall progress in cancer research. In this article, we delve into the world of clinical trials, shedding light on their significance and offering valuable insights to those seeking comprehensive information about this crucial aspect of cancer treatment.

The NCCN Treatment Guidelines

Nearly every oncologist uses the same guidelines to treat the 1.7 million US citizens diagnosed with cancer each year. These guidelines are produced by the National Comprehensive Cancer Network (NCCN), a non-profit alliance of 33 cancer centers in the United States.

The NCCN guidelines represent the collective wisdom of how cancer should be treated in any given situation, selecting from current FDA-approved cancer treatments. While oncologists have the flexibility to prescribe any cancer drug that’s been approved for any type of cancer, they widely follow these guidelines, knowing these treatments are acceptable and will be covered by health insurance.

NCCN Recommendation of Clinical Trials

Given the complexity of cancer, the NCCN guidelines are invaluable. They help oncologists choose appropriate treatments. In addition to standard treatments, the NCCN guidelines also emphasize the significance of clinical trials. In fact, on every page of the NCCN guidelines, you'll find the statement:

Clinical Trials: NCCN believes that the best management of any patient with cancer is in a clinical trial. Participation in clinical trials is especially encouraged.

However, most oncologists skip over this repeatedly because they have no clinical trial to offer. They likely have a standard therapy to offer. And if they don’t, they lack the tools and time to scour the country for clinical trials that match the patient's profile. And, unfortunately, they have no incentive to send the patient elsewhere for their care.

What is a Clinical Trial?

Clinical trials serve as a means to improve upon standard treatments. Through years of rigorous study, drugs undergo multiple stages to demonstrate their safety and efficacy compared to the currently approved standard treatments for specific cancer types. Once this lengthy process, often spanning over a decade, is complete, new cancer drugs become available and are typically covered by health insurance.

The FDA approves drugs for particular cancer types. Once a drug is approved for a single cancer type, it can technically be prescribed for any cancer patient. This is what is meant by accessible therapy. However, the accessible drug may not be covered by insurance or might not be available through a cancer center’s pharmacy. 

Drugs that are not approved by the FDA are only available through clinical trials.

At any given moment, there are over 13,000 clinical trials recruiting cancer patients, spread across the United States at various locations. Only 3-5% of adult cancer patients are participating in trials, despite over 70% expressing interest. The NCCN, the country’s foremost authority on cancer treatments, suggests that 100% of patients should consider clinical trials. We agree.

Participating in clinical trials can offer early access to promising drugs and the opportunity to receive care from oncologists with specialized expertise and in-depth knowledge of the science behind your specific cancer.‍

Phases of Clinical Trials‍

Clinical trials are typically conducted in several phases, each serving a specific purpose in the research process. This is the traditional understanding of clinical trial phases in cancer:‍

Phase I: Safety and Dosage‍

Phase I trials are the initial step in evaluating a new treatment. They primarily focus on assessing the safety and determining the appropriate dosage of the intervention. A small group of patients is enrolled in these trials, and the treatment's effects and any potential side effects are closely monitored. These trials help researchers establish the maximum tolerated dose and provide valuable insights for subsequent phases.‍

Phase II: Efficacy and Side Effects‍

During Phase II trials, the treatment's efficacy is evaluated in a larger group of patients. These trials aim to gather more extensive data on the intervention's effectiveness and further explore potential side effects. The results of Phase II trials help researchers determine whether the treatment should progress to the next phase.‍

Phase III: Comparison and Validation‍

Phase III trials involve comparing the new treatment with the current standard of care. A larger patient population is enrolled, and the study aims to determine whether the new intervention is superior, equivalent, or inferior to existing treatments. These trials play a critical role in establishing the new treatment as a standard option for cancer patients.‍

Phase IV: Post-Market Surveillance‍

Phase IV trials occur after the treatment has been approved and made available to the general population. These trials continue to monitor the intervention's long-term safety, effectiveness, and any rare side effects that may not have been apparent in previous phases. Phase IV trials provide ongoing data to ensure the treatment's continued safety and efficacy.‍

The Evolution of Clinical Trial Phases

Over time, the phases of clinical trials for cancer have shifted, allowing for more efficient and expedited evaluation of new treatments. Traditionally, clinical trials followed a sequential progression, with each phase building upon the results of the previous one. However, with advancements in technology, data analysis, and a deeper understanding of cancer biology, the boundaries between phases have become more fluid.

The traditional approach involved starting with Phase I trials, which primarily focused on assessing the safety and determining the appropriate dosage of the intervention. Phase II trials followed, evaluating the efficacy and potential side effects in a larger group of patients. Finally, Phase III trials compared the new treatment with the standard of care to establish its superiority, equivalence, or inferiority.

In recent years, a shift towards more innovative trial designs has emerged. Adaptive clinical trials, for example, allow for real-time modifications to the study protocol based on accumulating data. This flexibility enables researchers to refine the treatment approach, explore different dosages, and target specific patient populations more effectively. By incorporating feedback from earlier stages, researchers can make informed decisions and adapt the trial's course to maximize its potential for success.‍

Which Phase is Right for Cancer Patients?‍

Traditionally, the phase of a clinical trial has been a significant consideration for both patients and healthcare providers. However, with the advancements in genetic tumor sequencing, targeted therapies, precision medicine, and immunotherapy, the match between the patient and the existing data supporting the trials has become more important than the trial's specific phase.

In the era of precision medicine, tumor profiling through genetic sequencing allows oncologists to identify specific genetic alterations in a patient's tumor. This knowledge enables them to tailor treatment strategies and match patients to clinical trials investigating targeted therapies that directly address the identified genetic abnormalities. The emphasis is on identifying the most appropriate treatment approach based on the unique characteristics of each patient's cancer.

Targeted therapies, designed to interfere with specific molecules involved in the growth and spread of cancer cells, have revolutionized cancer treatment. By focusing on the molecular drivers of a particular cancer, these therapies offer the potential for improved efficacy and reduced side effects compared to traditional chemotherapy. Clinical trials evaluating targeted therapies often enroll patients based on specific molecular markers, allowing for a more precise and personalized approach to treatment.

Immunotherapy, another breakthrough in cancer treatment, harnesses the body's immune system to recognize and attack cancer cells. The remarkable success of immunotherapy in certain types of cancer has led to the development of numerous clinical trials exploring its potential in different cancer types and settings. Immunotherapy trials often consider various factors such as the expression of specific biomarkers and the overall immune profile of patients to determine eligibility and potential benefit.

The availability of existing data supporting targeted therapies and immunotherapy has shifted the focus from trial phases to finding the most appropriate treatment option for each patient. While each phase of a clinical trial serves a specific purpose in the research process, the potential benefits of participating in a trial that aligns with the patient's genetic profile and treatment needs can outweigh the consideration of the trial's specific phase. ‍

Investment in Clinical Trials‍

Clinical trials require substantial investment from various stakeholders, including pharmaceutical companies, research institutions, and government agencies. The belief that clinical trials hold the promise of proving better than standard therapy has been a driving force behind this investment. While not all trials result in the expected outcomes, the potential for groundbreaking discoveries and advancements in cancer treatment drives the commitment to conducting clinical trials.

Investment in clinical trials has increased significantly over the years due to several factors. Firstly, advancements in technology and scientific knowledge have enabled researchers to identify potential targets and develop innovative therapies with greater precision. This has sparked optimism about the potential benefits these trials can offer to patients.

Secondly, the recognition that early access to promising drugs and treatments can significantly impact patient outcomes has driven the urgency to conduct clinical trials. By involving patients in trials at earlier stages of development, researchers aim to provide access to potentially life-saving interventions before they become widely available.

Moreover, regulatory agencies have streamlined processes to facilitate the initiation and execution of clinical trials. This has reduced barriers and accelerated the timeline from drug development to clinical implementation.‍

Benefits of Clinical Trials

Participating in a clinical trial can offer several benefits to cancer patients and the medical community as a whole. Some key advantages include:

• Access to Cutting-Edge Treatments: Clinical trials provide access to novel treatments that may be more effective than current standard options.
• Contributing to Scientific Knowledge: By participating in a clinical trial, patients contribute to the advancement of cancer research and help shape future treatment strategies.
• Receiving Expert Medical Care: Patients enrolled in clinical trials receive close monitoring and care from a team of specialized healthcare professionals.
  

Placebos and Assignment to Treatment Groups

When it comes to cancer clinical trials, the use of placebos is very rare, particularly in trials involving patients with advanced or life-threatening conditions. Instead, researchers and physicians prioritize providing the best supportive care to all participants. Best supportive care refers to the standard treatments, medications, and interventions that are widely accepted as the current standard of care for a specific cancer type. This ensures that all participants receive a level of care that aligns with the existing medical practices for their condition.

In some clinical trials, there may be multiple treatment groups. Participants are assigned to these groups through a process called randomization. Randomization involves the random assignment of participants to different treatment groups. In some cases, participants may be aware of their assigned treatment group, while in others, the assignment may be blinded, meaning they are unaware of which treatment they are receiving. Blinding helps minimize bias and ensures that the evaluation of the treatment's effectiveness remains objective.

One treatment group in a clinical trial may receive the standard of care, which consists of the established treatments that have been proven effective for a particular cancer. The other treatment group, known as the experimental group, receives the new experimental treatment being investigated in the trial. The comparison between the standard of care and the experimental treatment allows researchers to assess the efficacy, safety, and potential benefits of the new intervention.

It is important to note that the assignment to a particular treatment group is done randomly, which means that participants have an equal chance of being assigned to either group. Randomization helps ensure that any observed differences in outcomes between the groups are due to the treatment itself and not other factors such as patient characteristics or preferences.

‍Conclusion‍

Clinical trials serve as the cornerstone of advancements in cancer treatment and research. By comprehensively understanding the purpose, phases, benefits, and risks of these trials, individuals can make informed decisions about participating in them. Remember, clinical trials provide access to cutting-edge treatments, contribute to scientific knowledge, and offer expert medical care. In the era of precision medicine, the match between the patient and existing data supporting the trials has become crucial. Placebos are rarely used in cancer trials, and instead, best supportive care is provided. Additionally, participants may be assigned to different treatment groups through randomization, with some trials employing blinding to maintain objectivity.

If you or a loved one is interested in clinical trials, you can begin by talking to your oncologist, visiting an NCI-accredited cancer center, or reaching out to an advocacy group. However, it is still incredibly difficult for patients to access and evaluate clinical trials. 

At Sagely Health, we have deep expertise in cancer and clinical trials, and we have built powerful technology to manage and search the over 13,000 current clinical trials for cancer for any patient. We match patients to treatments and then connect them to second-opinion experts that can evaluate them for the most innovative treatments available.

Let us know if you’re interested in a consultation to explore all your treatment options, including clinical trials.