What Is the Main Idea?

Patient-reported outcomes are a valuable aspect of clinical trials. Regulatory authorities and research groups are encouraging the inclusion of these insights earlier in the clinical trial process, even as early as phase I. An interesting open access clinical study titled “The Use of Patient-Reported Outcome Measures in Phase I Clinical Trials”, published in the journal Oncology, goes into detail on the importance of this type of information as well as studying how often such information is used. This blog post summarizes the main points.

What Else Can You Learn?

Get a straightforward explanation of the phases of a clinical trial so you can better understand what is meant when a drug is being trialed, approved, and released. You can also see some suggestions for how you can share your experiences with a drug or treatment.

Refresh Your Memory on Clinical Trials

In 2020, we were all strongly invested in the clinical trials of the COVID-19 vaccines. These expedited trials were frequently in the news, and many people became familiar with the phases of clinical trials and the questions researchers try to answer in each one.

Nevertheless, it’s always handy to collect information in one place, so here’s a quick refresher. Clinical trials are studies focused on testing if a drug, medical device, treatment, or diagnostic will function in the broader population.

What Are the Phases of a Clinical Trial?

If preclinical studies suggest that a drug should be safe and might be effective, an application is made to a regulatory body (e.g., the European Medicines Agency (EMA) or the Food and Drug Administration (FDA)) to approve the clinical trial. Then it can begin.

  • Phase I looks to establish whether the drug is safe. They try to establish the highest safe dose by giving subjects increasing doses and closely observing any effects and side effects. They involve small numbers of participants.
  • Phase II asks whether the drug works. A larger group of people receive the highest safe dose from phase I. The drug is given until the goal of the treatment is achieved (improved quality of life, reduced pain, tumor shrinkage, etc.).
  • Phase III asks whether the drug is better than existing ones. For drugs, this involves giving several hundred patients an existing drug or the new drug and comparing the effects.
  • After phase III, the drug may be approved. However, it is generally still subject to an ongoing phase IV trial, which looks at its behavior over a longer period of time in the general population. This helps to establish more safety, efficacy, and treatment information. An example of something found in this way is the extremely rare blood clots seen with the Pfizer–BioNTech COVID-19 vaccine.

What’s Changing about Phase I Trials?

As described above, phase I clinical trials traditionally focused on quantifiable parameters such as the maximum tolerated dose (the highest dose a patient can tolerate safely). However, in recent years, regulatory and research bodies have begun to request or even require the inclusion of certain qualitative measures even in these very early phases.

What Are Patient-Reported Outcomes?

One such qualitative measure is the patient-reported outcome, which is defined by the FDA as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” Examples of patient-reported outcomes are pain level, fatigue, changes in mood, and changes in concentration.

Asking for information of this type during a phase I clinical trial means that the pros and cons of a treatment are visible right from the start. It could mean that changes are made to phase II and III or a revised phase I is designed. This increase in the adaptiveness of trial design might mean effective treatments are made available sooner.

Including patient-reported outcomes is important because it puts the patients’ experiences in greater focus, reflecting a commitment to improved patient care and quality of life. If healthcare workers and patients are more aware of the qualitative impact of a drug, they can make better-informed decisions.

Are Patient-Reported Outcomes in Common Use?

Even though the value of this information is increasingly widely recognized, adoption of this approach is low. There is an expectation that they will be included more frequently in the future. There is also hope that patient-reported outcomes will start to be included on pharmaceutical product labels, providing patients with direct access to these insights.

The article discusses some of the barriers to the inclusion of patient-reported outcomes in phase I clinical trials, on product labels, and in other stages of drug development. You may be interested in diving deeper into this information, which is towards the end of the discussion.

Is There a Place for My Experiences?

If you’re ever involved in a clinical trial, consider asking if patient-reported outcomes are being incorporated and during which phase. Also, if your doctor is prescribing you a new medication, ask them what the clinical trial patients reported. They may be able to retrieve this information for you. Finally, if you know a drug is new, you can share your experiences with your doctor or directly with the pharmaceutical company as part of a phase IV trial that may be ongoing.

It’s important that we share our experiences with drugs and other treatments to further the understanding of their impact and improve patient-centric medical care.

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