Dr Michael Silverman, principal at BioStrategics Consulting Ltd, talks about an important strategic element of clinical trial design and pharmaceutical development in this blog, and that is, the crafting of the product label. This is the defining document for how the product will be used in the market place.
In the United States and other Western countries, the product label or package insert, characterizes the market for a drug by defining the indications and the appropriate patients population, informing prescribers, and serving as the basis for promotional material. Therefore, it should be self-evident that the package insert also drives development. In its most strategic context, clinical development is the process of generating the human data that populate the final package insert.
Recognizing the label’s importance, many biopharmaceutical companies have instituted formal procedures for crafting and approving, early in development, a prospective patient package insert (PPI) for every new product. This is a key planning document that articulates those developmental goals that must be realized to maximize product value. It serves as the corporate statement of those attributes a new drug must possess for it to be considered a value-adding component of the overall portfolio. Selected practical considerations and characteristics of a prospective PPI are:
The prospective PPI serves many audiences and addresses many important functions. For the development team, it provides a series of goals, raises new questions, and crystallizes thinking around central themes of development for a given product. For management, it initially serves to ensure that the development philosophy harmonizes with strategic goals; subsequently, it provides both a framework for capturing new knowledge and yardstick against which developmental progress and results can be measured. For the organization as a whole, it provides an invaluable communication and decision-making tool; at every step of the way, actual results can be compared with aspirations, and reassessment can be made regarding the wisdom of continuing investment in the project.
Central to the process of developing the prospective PPI is determining the nature of the Indications Statement as specifically as possible. No drug is ever approved with an indication to “treat infection”; instead, it is approved with a label that specifies the site(s) of infection, the causative organism(s), and the characteristics of the patient population. A crisp articulation of the Indications Statement must be made at the outset of development to provide focus and direction to the clinical effort.
Two points about the prospective PPI are worth highlighting. First, not all the new data that accrue during development and that influence decisions, emanate from clinical trials. Results are also generated by toxicology studies, manufacturing processes, competitors’ programs, and the like. Certainly, negative information about a compound’s chronic toxicity or carcinogenicity, high cost of goods, or inferior profile relative to a competitive compound will affect a company’s view of that compound’s value. Second, nota all label components lend themselves to being designed into a clinical trial but, rather, emerge from observations that are made during the course of a trial. In general, label data relating to pharmacology, efficacy, and dosing are subject to hypothesis formulation and therefore are directly designed into a protocol, whereas information relating to safety is obtained not by hypothesis testing but by careful empiric observation.
Dr Michael Silverman is the principal clinical consultant at BioStrategics Consulting Ltd., a clinical development consulting company based in Marblehead, MA, with business interests around the globe. Dr Silverman would be delighted to hear your thoughts on today’s blog, your experiences, or anecdotes and of course, your questions. To make comments or ask questions, click on this link.
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