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Principles of Health: Occupational Therapy & Physical Therapy

Clinical Trials

by Glenn Ellen Starr Stilling

Specialties and related fields: All

Definition: research studies that test new drugs or treatments on human subjects to determine whether and at what dosage they are safe, effective, and better than similar products already in use

KEY TERMS

blinded or single-blind: a study in which the patients are not told whether they are in an experimental or control group

control group: a group of patients receiving either a standard treatment or a placebo, allowing comparison with the experimental treatment

double-blind: refers to a study in which neither the patients nor the research staff knows which patients are receiving which treatment

informed consent: consent for treatment by a patient who has been educated fully about the purpose, benefits, and risks of a clinical trial

Institutional Review Board (IRB): a committee that oversees informed consent, reviews the progress of clinical trials and safeguards participants’ rights

placebo: an inactive substance resembling the experimental drug that might be given to a control group, especially when no standard treatment exists

protocol: a lengthy, technical document outlining the rules for inclusion in, the scientific rationale of, and procedures for a clinical trial

randomization: assigning, by chance, patients with similar characteristics to either the experimental or the control group in a clinical trial

INDICATIONS AND PROCEDURES

Clinical trials offer the most reliable process for bringing new drugs and medical treatments into public use. The process has features that can protect human participants; avoid biases; ensure that patient improvements are due to the experimental treatment and not other factors; and allow accurate comparison of the experimental treatment with others on the market. Clinical trials are usually initiated and managed by academic institutions (often with grant funding), pharmaceutical companies, or government research agencies, such as the National Cancer Institute.

In 1998, it was estimated that the cost of developing a new drug was, on average, $500 million. The process could take twelve to fifteen years—from discovery and laboratory testing, through clinical trials, Food and Drug Administration (FDA) approval, and finally getting the drug to market. By the late 1990s, a new drug might undergo sixty-eight clinical trials. The average number of patients enrolled in a trial was 3,800.

Clinical trials fit into one of four types. Phase I trials, which usually involve only twenty to one hundred seriously ill patients, try to determine how to administer a new drug, the maximally tolerated dose (MTD), how the human body processes the drug, and any significant side effects. Phase II trials, which are usually randomized, treat up to several hundred patients with measurable disease rates. These trials study the effectiveness of the drug. Phase III trials, which are usually randomized and blinded and treat hundreds or thousands of patients, have more relaxed criteria for inclusion and are usually multicenter (held simultaneously at more than one site). These trials determine whether the new drug is better than current, standard ones. Phase IV trials are often informal and conducted once a drug is on the market. Pharmaceutical companies may ask physicians to submit reports on how their patients respond to the drug.

USES AND COMPLICATIONS

The 1979 Belmont Report detailed three ethical principles to guide clinical trials. They include respect for persons (abiding by their opinions and choices as autonomous agents), beneficence (doing no harm and maximizing the possible benefits while minimizing possible harm), and justice (distributing the benefits and burdens of research fairly).

A clinical trial participant receives an injection.

POHTherapy_0504_0001.jpg

Photo by NIAID, via Wikimedia Commons.

Two standard features of clinical trials help ensure ethical principles are followed. First, all clinical trials in the United States must be approved and monitored by an Institutional Review Board (IRB), including scientists and laypersons. Multicenter trials must also have a data safety and monitoring board composed of independent experts. This group monitors data from the trial regarding the treatment’s effectiveness and any adverse reactions. Second, the detailed informed consent document that patients must carefully consider and sign provides many categories of information. Most important, anticipated physical risks and discomforts are explained, as are financial risks. Similar practices are followed in countries other than the United States as well.

PERSPECTIVE AND PROSPECTS

In October 1948, the British Medical Journal published an article reporting on what was probably the first study using all the methodological features of the randomized clinical trial. Since then, the randomized clinical trial has come to be regarded as perhaps the most important medical achievement of the twentieth century. It transformed biomedical research and allowed physicians to make treatment choices based on scientific evidence rather than personal opinion and experience.

The National Cancer Institute (NCI) and other sources reported a low participation rate in clinical trials—ranging in the late 1990s, from 3 to 20 percent of patients. One of many causes was that insurance companies and managed care providers frequently refused payment for experimental treatments. Their concerns were that they might be liable for adverse reactions or additional care after the trial ends and that clinical trials are more costly than conventional treatments. Because so many insurers would not cover the costs of clinical trials, researchers had trouble finding patients willing to participate, thus slowing the development of more effective drugs and treatments. Insurers gradually realized that more widespread coverage of the costs of trials might speed the development of better drugs, which could ultimately save them money. In 1998, US states began to pass laws requiring insurers to cover the routine medical costs (such as tests and office visits) of treatment in clinical trials of drugs for life-threatening diseases.

Criticism has been leveled at clinical trials for insufficient inclusion of women, children, people of color, and older adults. When these groups are underrepresented, there is no certainty that a drug will be effective or without side effects for them.

In June 2000, the FDA added a regulation that would place a clinical hold on a phase I trial of a drug or treatment for a life-threatening disease affecting both women and men if either gender was excluded because of risk to their reproductive potential. That same month, President Bill Clinton signed an executive memorandum directing Medicare to reimburse senior citizens for routine medical costs incurred in clinical trials. A major impetus for this change came from reports that only 33 percent of cancer clinical trial participants were over sixty-five, while 63 percent of all cancer patients were over sixty-five.

SIGNIFICANCE

Like other healthcare providers, occupational therapists (OTs) use techniques whose efficacy is established by rigorous, placebo-controlled, double-blinded clinical trials. Consequently, they participate in clinical trials to increase the effectiveness of their treatment strategies.

OTs can play a valuable role in clinical trials, particularly when evaluating new treatments or interventions.

An occupational therapist can help develop and select outcome measures when designing the clinical trial. Outcome measures capture the potential benefits of new treatments or interventions on individuals’ functional abilities, participation in daily activities, and quality of life. OTs provide expertise in assessing and quantifying changes in occupational performance and help identify relevant and meaningful outcome measures.

Once the clinical trial commences, OTs can educate participants about the purpose of the clinical trial, the potential benefits and risks, and their rights as participants. They can provide ongoing support and counseling to address concerns, answer questions, and help participants cope with the physical, emotional, and occupational challenges associated with the trial.

During the clinical trial, OTs can comprehensively assess participants’ functional abilities, including their physical, cognitive, and psychosocial skills. These assessments can establish baselines, identify eligibility criteria, and measure the impact of the new treatment or intervention throughout the trial.

Also, during the clinical trial, OTs can monitor participants’ adherence to the treatment or intervention protocols outlined in the clinical trial. They can provide education and support to help participants understand and follow the prescribed interventions, ensuring consistency and accuracy in implementing the new treatment or intervention.

Throughout the trial, OTs can contribute to adverse event reporting by monitoring and documenting any negative effects or unexpected outcomes related to the new treatment or intervention. They can provide valuable insight into the potential impact on participants’ functional abilities, safety, and overall well-being.

OTs collaborate with the research team, including principal investigators, researchers, and other healthcare professionals involved in the clinical trial. They can contribute their expertise in occupational therapy (OT) practice and work together to ensure the trial’s smooth implementation and successful completion.

After the clinical trial is published, OTs can use the data from the trial to develop treatment plans and interventions based on the specific goals of the clinical trial. They can design interventions targeting motor skills, cognitive abilities, sensory processing, self-care, work-related skills, or community integration. OTs can also adapt interventions to meet the unique needs of each participant in the trial.

It is important to note that the involvement of OT in clinical trials may vary depending on the nature of the trial, the specific research question being investigated, and the target population. OTs are valuable research team members, bringing their unique perspectives on functional abilities, occupational performance, and quality of life to enhance the evaluation of new treatments or interventions.

Citation Types

Type

Format

MLA 9th

Starr Stilling, Glenn Ellen. "Clinical Trials." Principles of Health: Occupational Therapy & Physical Therapy, edited by Michael A. Buratovich, Salem Press, 2023. Salem Online, online.salempress.com/articleDetails.do?articleName=POHTherapy_0113.

APA 7th

Starr Stilling, G. E. (2023). Clinical Trials. In M. A. Buratovich (Ed.), Principles of Health: Occupational Therapy & Physical Therapy. Salem Press. online.salempress.com.

CMOS 17th

Starr Stilling, Glenn Ellen. "Clinical Trials." Edited by Michael A. Buratovich. Principles of Health: Occupational Therapy & Physical Therapy. Hackensack: Salem Press, 2023. Accessed December 14, 2025. online.salempress.com.

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