One of the goals of CDER (The FDA’s Center for Drug Evaluation and Research) is to foster patient-focused drug development. As part of that effort, the agency has published a compendium of clinical outcome assessments (COAs) to promote the use of patient-focused outcome measurement in the drug development process. The pilot version of the Clinical Outcome Assessment Compendium (COA Compendium), which is now available on the FDA website, could potentially be used as a communication tool to achieve improved outcomes, while providing clarity and transparency to drug developers and the research community. This will be done by summarizing COA information for different diseases into a single resource, which can then be used as a starting point when considering how outcome assessments might be used in trials and early drug development.
The information on the site includes purpose, key considerations, how assessments are selected for the pilot COA Compendium, and limitations. CDER is currently seeking public comment on the pilot version.
In guidance provided by Dr. Elektra Papadopoulos, Acting Associate Director, Clinical Outcome Assessments Staff, Office of New Drugs, CDER, FDA, she notes patient-focused outcome measurement starts with an understanding of the impact of a disease or condition on the people who have it, and what they value most in terms of alleviating symptoms. “This information can then be used to develop tools that assess the things that patients most care about,” she says. “Patients and prescribers need meaningful information about a drug’s benefits and risks to make important treatment decisions. Patient-focused outcome measurement is really about measuring things that are important to patients, such as how a treatment will affect their symptoms, day-to-day functioning, and overall survival.”
How Will It Work?
Patient input can influence the choice of outcome measures, and selecting the right outcomes is clearly an important aspect of drug development. One of the best ways to ensure the patient voice is heard is to select those outcomes that you know are important to them. When questionnaires and diaries are used to collect patient information, that input can also help to verify the right information is being collected and measured in a proper manner. Later, clear and accurate information can also be included in product labeling.
“COAs are simply the patient-focused tools that we use as part of our assessments of clinical outcomes, such as how patients feel or function in their daily lives,” notes Papadopoulos. “These measurements of patient outcomes provide essential information about benefits and risks of a drug.”
COAs are captured using four types of COA measures:
In each case, the type of COA used depends on the particular research question and the context in which it will be used. According to Papadopoulos, COAs can be used to support product approval and claims of treatment benefit in drug labeling. A COA measure must also be fit for its intended purpose in drug development. FDA regulations state that assessments used to support labeling claims of treatment benefit must be well-defined and reliable, so that changes seen in the outcome assessment can be interpreted and described as a clear treatment benefit.
“The COA Compendium is a table that describes how COAs have been used in drug development to measure the patient’s experience (such as disease-related symptoms) and support labeling claims,” says Papadopoulos. “It also identifies COAs that have been qualified for potential use in multiple drug development programs and recognizes ongoing COA qualification projects to encourage community collaboration in the development of COAs for unmet measurement needs. Ultimately it will serve as a living document that will be updated on a regular basis. It is designed to be used as a starting point when considering the use of COAs in clinical trials and to promote early engagement and discussions with the FDA.”
What Does It Mean For Patients?
Before a product can be developed to treat a disease or condition, researchers need to know how it will affect the patient. Once information is gathered from patients, it can be used to define the specific aspects of the disease that should be treated. Only then can appropriate outcome measures be selected.
The hope is that having a COA Compendium to collect and list outcome measures will encourage the use and development of patient-focused outcomes in future trials. CDER plans to continue to engage with the public and scientific community to support future program and policy development activities.
Papadopoulos notes the compendium will not be a comprehensive list of all COAs, and that is not a goal of the project. It is also not meant to replace existing communication channels with CDER review divisions. Rather, it will be a way to encourage development and use of clinical trial endpoints that provide meaningful information to a patient taking a drug. Drug developers are also encouraged to consider incorporating adequate, well-defined, and reliable outcome measures, and to discuss those measures with CDER as early as possible in product development.