At the simplest level, most readers would answer that they are developing a drug, medical device, biological, or diagnostic product. While these designations are literally correct, the premise of this chapter is that they are often not the best way to refer to an investigational (or even marketed) product. In paying more careful attention to what you call the type of product you are working on, a greater sense of the product’s value may be created, potentially yielding great commercial dividends. The author stresses that this chapter is not referring to the trade name or generic name you choose subject to negotiation with the relevant agencies for a product, but rather to the function of the product.

To illustrate this point, here are examples of terms other than “drug” that could be used to describe your product. Obviously, the terms that could be chosen for a specific product will depend on its function and properties.

There are several reasons why this approach often makes sense. First, ask yourself if you, as a patient, physician, regulatory agency, reimbursement agency, or pharmaceutical marketing department, would rather use, prescribe, approve, pay for, or market a drug or a product that is described both functionally and accurately as previously listed. Functional descriptors give a more positive feeling about the need and value of the product than does the bare term drug. Even the term medicine has much better connotations than “drug,” given the widespread use of the term drug, referring to illegal use and abuse. It is rather surprising that the term medicine is not more widely used.

If you worked in a regulatory agency, wouldn’t you expect to want to facilitate the development and approval of an enzyme replacement more than a “drug” to treat the same disease? The term replacement suggests physiological dosing to replace what is missing, and the author thinks that most of us would tend to accept a smaller dossier for that type of product compared with a “drug” to treat the same problem.

When a marketing department promotes a product through medical literature, advertisements, or exhibits at a professional meeting, they usually describe the product in terms of its functions (i.e., reduces X or improves Y). The marketing message to healthcare professionals would be enhanced if it included such phrases as “X (brand name) acts as an enzyme replacement,” or “X is an essential nutrient,” or “X is part of the chemopreparative regimen.” Of course, some drugs are approved with those terms, but many more could be, and this would both improve the image of the product and possibly lead to increased revenue.

Interactions with regulatory authorities are also likely to be facilitated if the medical value of a product is established early in development and the company refers to its product with one of the functional terms rather than as a drug. In addition, it is useful to refer to the product by its functional term each time it is mentioned in communications between the agency and company. In these ways, by using the specific functional name, it is possible that the connotations of the functional name may facilitate interactions with the agency, which is always a highly desirable goal.

A company mantra one often hears is “We are creating a lean-and-mean development plan to get this product to the market as soon as possible.” In other words, the company seeks to have its regulatory submission completed as rapidly as possible by submitting the absolute minimum quantity of data.

Unfortunately, that approach often fails, particularly if the magnitude of the development plan has not been agreed to by the regulatory agency. One would think that senior company executives would not make unilateral decisions to bypass the critical step of seeking regulatory endorsement of a lean plan, but that is exactly what often happens. This may result from executive naiveté or ego, or it may reflect pressures from more senior staff or boards of directors to push an ill-conceived and unrealistic plan.

The author has seen cases where a company, having had their “lean-and-mean” New Drug Application (NDA) plan rejected at a pre-NDA meeting, immediately adopted the opposite strategy for developing the next drug. In the latter plan, the company tried to collect enough data to address almost any question that a regulatory agency could possibly throw at them. This “fat” NDA approach is also fraught with many problems. First, there are many opportunities to uncover issues that may not be real but that must then be examined in additional clinical trials. This leads to long delays in the program on top of the delays caused by conducting a program that contains unnecessary studies in the first place. In addition, the mountain of data collected must be entered into computers, analyzed, interpreted, and then written up as final study reports, adding further delays to development.

After one has read a number of meta-analyses in respected medical journals, it is easy to think that there is a single standard for these analyses, or at least a single gold standard. In this type of analysis, publications of clinical trials that meet certain standards and criteria are identified in the literature using standardized search methods, and then the data from those articles (along with any relevant unpublished reports or other data obtained) are combined and analyzed using standard statistical tests. Using confidence intervals as a guide to help understand statistical significance of the individual trials, it is often possible to gain insight from the combination of trials in order to address an important research question. This is the most common type of meta-analysis performed, but other types are also possible. This section focuses on some of these other types of meta-analyses and some of their regulatory uses.