Considerations in the Assessment of Clinical Benefit with a Focus on Pain: a Regulatory Perspective
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Considerations in the Assessment of Clinical Benefit with a Focus on Pain: a Regulatory Perspective Christopher O. St. Clair 1
&
Elektra J. Papadopoulos 1
# This is a U.S. government work and not under copyright protection in the U.S.; foreign copyright protection may apply 2020
Abstract In the USA, the regulatory standard for demonstration of efficacy of a drug is evidence of clinical benefit from adequate and wellcontrolled clinical trials. Understanding the natural history of disease and how treatment is expected to alter its course, and gathering input from relevant stakeholders, such as patients, caregivers, and clinicians, is essential to understand the best way to measure clinical benefit in a clinical trial. Though pain intensity has been the primary outcome measure in clinical trials for pain, an array of measures assessing clinical outcomes from multiple perspectives can allow for more comprehensive interpretation of how a treatment affects patients’ lives. Careful consideration should be given to how pain affects the feeling and functioning of each distinct patient population and which outcome assessment, or combination of outcome assessments, may be necessary to provide a more comprehensive view of the patient experience. The early stages of medical product development are an important opportunity to engage with regulatory agencies to discuss potential approaches to clinical trial design and outcome measurement strategies. Key Words FDA . outcome . measurement . trials . pain
Introduction A fundamental goal of a clinical trial is to evaluate the clinical benefit of an intervention on the disease or condition of interest. In the context of most clinical trials, FDA’s perspective is that clinical benefit means that the intervention produces a positive, clinically meaningful effect on how a patient feels, functions, or survives. In the USA, the regulatory standard for demonstration of efficacy of a drug is evidence of clinical benefit from adequate and wellcontrolled clinical trials [1]. Generation of evidence is based on the results of trial endpoints, which generally fall into two categories: clinical endpoints and surrogate endpoints. Clinical endpoints measure or reflect how patients feel, function, or survive and rely on use of clinical outcome assessments. Given that pain is a uniquely subjective and individual experience, clinical outcome assessment * Christopher O. St. Clair [email protected] 1
Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring 20993, MD, USA
informed by patient and caregiver input is key to the understanding and evaluation of pain. In this paper, we focus on strategies for assessment of clinical benefit in medical product development highlighting certain considerations in pain.
Laying the Foundation for Assessing Clinical Benefit Understanding the Disease or Condition and Patient Subpopulations At the early stages of planning for clinical studies—even prior to sele
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