There are continually new discoveries in the fight against diseases. However, getting a novel agent from the laboratory to approval for use as a treatment in patients may take ten years or more. Once researchers identify a potential new therapeutic, it often takes three to six years to develop and test that treatment in animal models. It will then take another six to seven years, on average, to complete clinical trials in humans. This can frustrate patients seeking access to new treatments, who often ask, “Why do clinical trials take so long?”
Designing clinical trials and protocols is complex, requiring rigid standards and the time to ensure the safety and efficacy of a particular treatment approach. In addition, investigators must be highly selective about whom to enroll, even though enrollment in clinical trials often falls short. In summary, there are various answers to the question, “Why do clinical trials take so long?”
Patient enrollment is a challenge
When a trial is designed, certain protocols are created, and investigators need a statistically significant number of participants to validate the results. Often, clinical trials do not meet enrollment targets, causing trial delays or cancellations. At the same time, many of these trials exclude patients because they don’t meet those specific requirements. Many patients aspire to join a clinical trial to try a new treatment approach but are ultimately excluded because they do not meet the criteria.
Eligibility criteria are essential in defining the patient population being studied to best determine the effectiveness of an investigational treatment and ensure the safety of participants. Before enrolling, a participant must provide investigators with a range of information, such as their gender, age, and type and stage of disease, along with details about previous treatments and other medical conditions.
Inclusion criteria allow a participant into a study. Exclusion criteria disqualify a person from participating. For instance, having a specific type of tumor may qualify a patient for a trial, while having a particular medical condition may ultimately disqualify that patient.
Eligibility criteria are critical to the validity and outcome of a study. However, there are concerns that criteria that are too rigid may negatively impact enrollment, restrict access to novel treatments, and limit knowledge of how an investigational treatment may ultimately affect a more diverse population.
While eligibility criteria do impact enrollment, there are many reasons for low patient enrollment rates. Patients may not enroll in clinical trials because they don’t fully understand how the process works, they fear receiving a placebo or the potential side effects of an investigational agent, they are concerned that it will take too much time, or they simply aren’t asked and are not aware that they are eligible. One study showed that 86% of clinical trials are delayed due to lack of patients, while another reported that 25% of cancer trials failed to enroll enough patients.
FDA approval is done in phases
The main answer to the question, “Why do clinical trials take so long?”, is that ensuring the safety and efficacy of a new treatment takes time. There are cases of patients gaining access to investigational drugs based on compassionate use approval or accelerated approval in certain scenarios. However, most investigational treatment approvals must undergo a lengthy Food and Drug Administration (FDA) development process.
Once an investigational drug or treatment makes it past the discovery phase in the laboratory, also known as phase 0 or a pilot study, there are four steps in the clinical trial process:
- Phase I clinical trials
- This marks the first evaluation of an investigational drug or treatment in a small group of people (typically twenty to eighty), with the goal of evaluating safety and side effects.
- Phase II clinical trials
- After a successful Phase I, the investigational treatment is given to a larger group (100-300) to determine effectiveness and further evaluate safety.
- Phase III clinical trials
- After a successful Phase II trial, this phase compares a new treatment with the standard treatment and typically involves hundreds or thousands of patients.
- Success may result in FDA approval.
- Phase IV (post-marketing surveillance)
- After the treatment is in use, this surveillance checks a treatment’s performance and optimal use and evaluates any long-term benefits and risks.
Expanding enrollment and improving matches
There are a variety of efforts designed to educate patients about the benefits of enrolling in clinical trials. There are also efforts to boost diversity in trials and expand criteria to include more patients who may have previously been excluded, like cancer survivors or those with other medical conditions. Each is done based on the specific requirements of a particular trial.
Many patients find that complete medical records play a significant role in getting second opinions and identifying the clinical trials available to them. Incomplete records or delays in submitting records can lead to a missed treatment opportunity. For example, the eligibility criteria for breast cancer trials can be extremely specific and are increasingly based on gene expression profiling of a tumor. All information must be readily accessible on short notice.
A free clinical trial-matching service called Ciitizen is alleviating the burden that patients feel when forced to collect their own records, which can be challenging due to missing documents and incompatible formats. With one click, patients can enroll and let the service compile their medical records and identify the best clinical trials based on their profile.
Ciitizen is a free service that helps patients get more out of their health records. Our platform enables patients to find better treatment options and gives them the opportunity to advance the research for cures.
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