patient report and clinical benefits fda

4 Benefits of financial and clinical reporting Avoidance of medical conditions – Healthcare reporting and analytics use past and current data to spot any medical issues in patients before they occur.

Full Answer

What is the FDA doing to improve patient-reported outcomes in cancer trials?

However, achieving sufficient consistency and quality of these data in cancer drug applications submitted to the FDA has been a challenge. The FDA’s Oncology Center of Excellence has undertaken a sustained effort to identify methods to rigorously collect patient-reported outcomes in cancer clinical trials.

Does the FDA endorse pro measures in clinical trials?

FDA does not endorse any specific PRO measure and examples within this document are illustrative and should not be construed as endorsements. This guidance is specific to registration trials for anti-cancer therapies intended to demonstrate an effect on survival, tumor response, or delay in the progression of a malignancy.

When to use Patient-Reported Outcome measures (pro) in medical product development?

Refer to the guidance for industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims for situations where the PRO endpoint will be used as the primary evidence of effectiveness.

What does the FDA do?

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

What are patient-reported outcomes used for?

Defining Patient-Reported Outcomes PRO data are used to inform and guide patient-centered care, clinical decision-making, and health policy decisions and are an important component in learning healthcare systems [1]. PROs are also used to measure risks and benefits of treatments.

What are patient-reported outcomes in clinical trials?

Patient-reported outcomes (PROs) are any report of the patients' perspectives about the impact of disease and treatment on their health status, for example quality of life and symptoms, without the interpretation of a clinician, or anyone else [1, 2].

What are some of the advantages of using patient-reported outcomes?

The greatest perceived benefits were the ability to track changes in clinical symptoms over time, improved quality of care, and better disease control among patients, providers, and administrators, respectively.

Why is PFDD important?

Patient-focused drug development (PFDD) is a systematic approach to help ensure that patients' experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation.

How do you include patient reported outcome measures in clinical trials?

How to Collect Patient-Reported Outcomes in Clinical TrialsStep 1: Research existing PRO instruments for your condition area. ... Step 2: Conduct patient interviews or focus groups. ... Step 3: Decide whether you'll use electronic patient-reported outcomes or paper forms. ... Step 4: Formulate your questions and timing.More items...•

How do you collect patient reported outcomes?

Strategies for Collecting High-Quality Patient-Reported OutcomesSet Clear Goals. ... Use Validated Questions When Possible. ... Avoid Multiple Interpretations of Questions. ... Keep It Short and Sweet. ... Ensure Patient Understanding. ... Choose the Best Method for the Target Population. ... Protect a Patient's Right to Refuse. ... Conclusion.

Why are patient outcomes important in healthcare?

Use of patient-reported outcomes is an essential aspect for improving clinical care, because it enhances the connections among doctors and with patients.

What are patient-reported measures and why are they important?

Patient Reported Outcome Measures (PROMs) assess the quality of care delivered to NHS patients from the patient perspective. Currently covering two clinical procedures, PROMs calculate the health gains after surgical treatment using pre- and post-operative surveys.

Are patient-reported outcomes reliable?

Research shows a high correlation between patient-reported data and clinically documented chart abstracted data. This means data collected from patients is accurate and trustworthy.

What is the primary focus of Phase 3 clinical testing?

The main focus of phase 3 trials is to demonstrate and confirm the prelimary evidence gathered in the previous trials that the drug is, a safe, beneficial and effective treatment for the intended indication.

What is non clinical drug development?

Nonclinical studies define the relevant efficacy and/or safety characteristics of drugs, biologics, and medical devices. They begin well before a Sponsor's first interaction with the Food and Drug Administration (FDA) and continue throughout product development.

What is provision for doubtful debts?

The provision for doubtful debts, which is also referred to as the provision for bad debts or the provision for losses on accounts receivable, is an estimation of the amount of doubtful debt that will need to be written off during a given period.

What is the FDA's priority?

FDA’s priority is your health and safety. While everyone’s daily lives are impacted during the coronavirus disease 2019 (COVID-19) pandemic, the impact may be even greater on older adults and people of any age who have chronic medical conditions. If you are not feeling well or have any questions about your health, ...

What is the FDA's program for reporting medical devices?

Report a Drug or Medical Device Issue to FDA. Report a safety concern or negative event with an FDA-regulated medical product using FDA’s information and event reporting program, MedWatch.

What is the FDA?

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices.

Who is the director of the FDA's Oncology Center of Excellence?

The following quote is attributed to Richard Pazdur, M.D. , director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research

What is PRO measure in cancer?

Although this guidance focuses on patient-reported outcome (PRO) measures, some of these recommendations may be relevant to other clinical outcome assessments (i.e., clinician-reported outcome, observer-reported outcome, performance outcome) in cancer clinical trials. Recommendations supplement previous guidance on use of PRO measures in clinical trials by providing additional considerations specific to the cancer clinical trial setting. Guidance specific to PRO endpoints and details of analytic methods are not comprehensively covered. FDA does not endorse any specific PRO measure and examples within this document are illustrative and should not be construed as endorsements.

Does the FDA endorse pro measures?

FDA does not en dorse any specific PRO measure and examples within this document are illustrative and should not be construed as endorsements. This guidance is specific to registration trials for anti-cancer therapies intended to demonstrate an effect on survival, tumor response, or delay in the progression of a malignancy.

What is the purpose of PROs?

The use of PROs to evaluate functional ability and similar concepts provides multiple benefits when used in conjunction with other measures, such as imaging or performance assessments. Patient reported function can provide a broader view of function than can be assessed in a clinical setting. For instance, physical function, including activities of daily living, may be best assessed from the patient’s perspective, without the need for interpretation by a trained professional. Additionally, physical function can play in important role in many phases of a trial. In the DOD, loss of function can be among the indicators for inclusion in a trial. Functional status, as measured by the Neck Disability Index (NDI),23 was used as a part of the inclusion criteria for the trial supporting the submission P140019.24 Similarly, the physical function scale of the Zurich Claudication Questionnaire (ZCQ),25 a disease-specific measure for patients with lumbar spinal stenosis, was used in P14000426 as one of the clinical components used to define moderate degenerative lumbar spinal stenosis, part of the inclusion criteria for the trial. How the patient is affected by the disease or condition plays an important role in determining the need and course of treatment and that information is often best capture directly from the patient through a standardized set of questions.

What is the scale used to assess aesthetic improvement?

common scale used to assess improvement in aesthetic procedures is the Global Aesthetic Improvement Scale (GAIS), which is often filled out by the clinician or investigator, as seen in 510(k) submission K161885.14 However, the scale has limits as clinical evidence to inform regulatory decision making. First, it lacks standardization, as can be seen in the variation in response options and descriptions from study to study.e.g.15,16 Second, despite widespread use, the GAIS has a lack of methodological or developmental evidence supporting the reliability and validity of scores obtained from its use. These deficiencies limit the use of the scores obtained from the measure in regulatory decision making.

What is the goal of aesthetic surgery?

goal of aesthetic and reconstructive procedures or plastic surgery is patient satisfaction with the results. In the absence of objective measures, the success of a treatment utilizing an aesthetic device can be difficult to define and even more difficult to measure. Therefore, scientific assessments from the clinician’s or patient’s perspective are essential for device evaluations. The clinician’s expertise is necessary to evaluate certain aspects of success and help evaluate clinical effectiveness. Thus, clinician evaluation will remain an integral part of evaluating aesthetic devices. However, how the patient feels about the results of the procedure are also important, but often overlooked. Information directly from the patient will be necessary to fully capture the effectiveness of the procedure, such as satisfaction or the impact on quality of life.

What is a bridging study?

Bridging studies provide an opportunity to reduce the burden of using PROs in regulatory studies by building on existing PRO measures. For new device technologies, PRO measures can be used to assess the device’s novel functions, outcomes, adverse events, etc. In some cases, a bridging study can be used to adapt and/or modify an existing PRO measure for a new context of use, rather than creating a new instrument. Some examples of bridging studies are:

What is a DCD?

For example, the Division of Cardiovascular Devices (DCD) worked with sponsors, academics, and the Centers for Medicare and Medicaid Services (CMS) through the Mitral Valve Academic Research Consortium to highlight the value of four patient-centered outcomes including symptoms, function, and quality of life one year following surgery10.

What is a PRO measure?

PRO measures can be used to capture a patient’s everyday experience with a medical device, including experience outside of the clinician’s office, and the effects of a treatment on a patient’s activities of daily living. Furthermore, in some cases, PRO measures enable us to measure important health status information that cannot yet be detected by other measures, such as pain.

What is CDRH in medical?

The Center for Devices and Radiological Health (CDRH or Center) is committed to our vision that patients have access to high-quality, safe, and effective medical devices and safe radiation-emitting products first in the world. We strive to ensure that patients and their care-partners stay at the center of our regulatory decision-making process. As part of our 2016-2017 Strategic Priorities 1, we made a commitment to Partner with Patients to successfully achieve our mission and vision2 in service of patients. One way we are partnering with patients is by incorporating the patient perspective as evidence in our decisions, including both patient preference information (PPI) and patient-reported outcomes (PROs).3

Examples of Clinical Benefits

Direct clinical benefits are fairly straightforward and usually easier to associate with specific clinical outcome parameters. For example, a total hip replacement has clear and direct clinical benefits to the patient: reduction of pain and improved hip function. These can be measured using various clinical measures, such as Harris Hip Scores.

Practical Tips for Defining Clinical Benefits

The most important tip for defining clinical benefits is simply to do it. If you ignore this part of the regulation, notified bodies will ask why it’s missing.

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