Transparent Trade-Offs

A clinical utility index (CUI) openly evaluates a product's attributes—and chance of success
Mar 01, 2005

CUI Model
In designing clinical trials, the pharma industry has been primarily concerned with a drug's efficacy, and therefore, modeling and simulation technologies used for those designs have also focused on efficacy. But the clinical utility and economic worth of a compound depends on many dimensions beyond efficacy, including important influences such as target population, product formulation, and patient compliance. To enhance market share and value to patients, pharma companies must consider those dimensions earlier in the drug development process.

One way to do that is with the use of a clinical utility index (CUI), which quantifies factors like a product's efficacy, safety, cost, and contribution to quality of lifeā€”and makes trade-offs transparent to decision makers. A CUI provides a single metric for multiple dimensions of benefit and risk and captures expert opinion on the therapeutic importance of various product characteristics. It also enhances communication between discovery, development, and the commercial side.

Multiple Dimensions Getting the right information to physicians about the best clinical use of a drug becomes more challenging as the number of available therapies grows. In his article "Choosing Which Drug to Prescribe" (Medscape General Medicine, 6 August 2003), Tomas Kramer, MD pens: "Recently, a great deal has been written urging clinicians to practice 'evidence-based medicine,' in which clinical decision making is based on scientifically derived data. The problem with this, of course, is there is very little evidence upon which to practice evidence-based medicine."

Dose Benefit Model
Treatment choices A prescribing physician must choose a therapy that gives the best balance of efficacy, side effects, safety, ease-of-use, and quality-of-life benefits for a given patient. Analytic tools developed in pharmacological science and operations/market research can organize knowledge from different sources into a decision making framework. (See "CUI Model" below.) First, modeling is used to predict, design, and learn. A drug candidate's likely clinical performance is modeled using information from related therapies as well as pre-clinical and early clinical data. Next, the predicted product profile(s) are evaluated using the CUI.

Clinical utility Every medical therapy has benefits and risks. The relative importance of these characteristics depends on the disease, patient, and how the drug is used (for example, dosage or concomitant therapy). A CUI is a transparent means of weighing and quantifying a compound's trade-offs. It provides a scale to compare product profiles and measure the impact of scientific and market assumptions. It can be used at low cost (a small amount of time and effort on the part of the drug development team) and early in a clinical program (Phases I and II)."

Clinical utility is defined as the net benefit of treatment to the patient as perceived by the prescribing physician or a surrogate decision maker such as an HMO making formulary decisions. A CUI quantifies the contribution of each attribute in the product profile (label) using physician preference data, if available, or by relying on clinical advisors and other internal expertise. The latter is less expensive, and may be more appropriate early in a development program.