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FDA’s Pilot Clinical Outcome Assessment Compendium – A Valuable Drug‑Development Resource

April 28, 2016 | Jacquie Powell, PhD, Clinical Research Scientist II | Regulatory Affairs, Drug Development Consulting

The three R’s (reduce, reuse, and recycle) typically conjure up ideas about conserving natural resources but they are also a great illustration for how FDA’s newly-available pilot clinical outcome assessment (COA) compendium can increase the efficiency of the drug-development process.

By collating and summarizing COAs across many different disease states and indications, the publically-available COA compendium should allow drug developers to reduce the amount of time spent on the development of new COAs, reuse existing COAs as appropriate, and potentially recycle elements of existing COAs to create meaningful measures in clinical areas where no COAs currently exist.

But maybe we should take a step back and start at the beginning…

rec

What is a COA anyway?

As you know, new drugs are approved by the FDA based on a demonstrated treatment effect on a clinical endpoint; that is, on a measure of how patients feel, function, or survive. Survival as a clinical endpoint is easy to measure, but may not always be practical. A COA is a measure of how a patient feels or functions in daily life.

Using COAs, investigational drugs may be considered efficacious if a patient’s health-related quality of life, or the way he or she functions in daily life, improves. This means that a new drug doesn’t necessarily need to demonstrate a treatment effect on survival or a biomarker‑related measurement in order to be considered clinically relevant.

There are 4 general types of COA measures:

  • Patient-reported outcomes (PROs) – based on reports that come from patients
    Example: Evaluating Respiratory Symptoms in Chronic Obstructive Pulmonary Disease* (E-RS: COPD) to measure respiratory symptoms of stable COPD
  • Clinician-reported outcomes (ClinRO) – based on reports that come from trained healthcare professionals
    Example: Psoriasis Area and Severity Index (PASI) for adult subjects with moderate to severe plaque psoriasis
  • Observer-reported outcomes (ObsRO) – based on observations made by someone other than the patient or healthcare professional
    Example: Diaries maintained by caretakers used to assess seizure frequency in subjects with partial‑onset seizures
  • Performance outcomes (PerfO) – based on patient performance on one or more tasks
    Example: Abilities Captured Through Interactive Video Evaluation (ACTIVE)-seated* to measure upper extremity and trunk movement in subjects with Duchenne muscular dystrophy.

* Recently qualified by the FDA for exploratory use! Stay tuned for our upcoming blog post on the COA qualification process.

COAs generally rely on assessments that can be influenced by human choice, judgement, or motivation and can be used to directly or indirectly support a drug’s treatment benefit.

Many drug-development programs already use COAs, so why make a COA compendium?

Up until now, most COAs have been developed on a case-by-case basis, as part of individual drug-development programs. In an effort to streamline this process, the FDA has created a COA compendium listing the majority of COAs that have either been used to support labeling claims for new drugs approved between 2003 and 2014 or are currently under development within the existing Drug Development Tools (DDT) Qualification Program. Keep in mind that some COAs were considered too complex or out-of-date to include in the pilot compendium.

The COA compendium is currently in a pilot stage of development and consists of a 6-column table that summarizes COAs by providing the following information:

Column 1: Disease/Condition

Column 2: Indication and/or Claim(s) Description

Column 3: Outcome of Interest

Column 4: COA Type

Column 5: COA Context of Use

Column 6: COA Qualification Information

Representative text from the COA Compendium is provided below.

chart
Source

The goal is to create an easy-to-use, comprehensive resource to assist drug developers in selecting (and hopefully reusing) appropriate COAs to evaluate the treatment effects of new drugs. Once a COA is qualified for use in the COA compendium, it can be used in future development programs without the need for the Agency to reconsider or reconfirm its suitability; however, this assumes that the COA is used to measure the “thing” that it was qualified to measure (ie, the “Concept of Interest”) and is used in the appropriate drug‑development context.

After the pilot phase, the COA compendium will move into an expansion phase. How this stage will take shape is still a work in progress but it may involve including COAs from efficacy supplements and statements about gaps in currently existing COAs (eg, concepts highlighted as important in patient-focused drug-development meetings but for which no tools exist).

What does this mean for drug development?

Producing the COA compendium reflects the FDA’s desire to see patient-focused outcomes factor more prominently in the drug-development process. By improving communication, removing barriers, and increasing transparency, the goal is to increase awareness of available COAs so that they might be used more efficiently and potentially earlier in the drug‑development process (before the end of Phase 2 trials).

While COAs allow the efficacy of a drug to be more broadly defined, they can be less reliable than other outcome measures and may lack adequate sensitivity to detect results. Therefore, the use of COAs should be carefully considered in any drug-development program. Also, qualification of a COA doesn’t mean it’s globally endorsed by the FDA for all drug‑development programs. Make sure to get direct guidance from the FDA before using any COA.

Let us know what you think

Do you know how to effectively integrate COAs into your clinical development program? IMPACT has experienced regulatory affairs professionals ready to consult with you. If you would like to discuss your specific needs, please contact us or give IMPACT a call today at (919) 899-9248.

Recycling image courtesy of Keerati from FreeDigitalPhotos.net.

Category: Regulatory Affairs, Drug Development Consulting
Keywords: Clinical outcomes assessment, COA compendium, FDA, PRO, ClinRO, ObsRO, PerfO

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