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 life—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.
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."
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
Dose Benefit Model
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.