by Lisa Jenkins VanLuvanee

I believe that people are special. They are a unique combination of both intrinsic factors and external experiences. Even identical twins who share the same genetic compliment are different from one another in personality, desires, goals, objectives, and lifestyles. Like a snowflake, no two people on this earth are alike. Thank goodness!

Similarly, I believe that all companies are special. Companies are comprised of a group of people (who we have already established are special) who band together under a unique leader to work toward a specific, and likely innovative, goal. After all, a company that doesn’t provide a unique and valuable contribution of some sort is not going to survive for long.

Extending this into the pharmaceutical space, I believe that every non-generic “product” (e.g., drug, biologic, device), is also special. The product could be a new dosage form, new chemical entity, a novel combination of two already approved products for a new indication, or countless other variants on a theme. Each “product” is developed because it provides something different that no other product provides. One could even argue that generic products are special because they add value by giving physicians, patients, and payers a lower-cost option that was not available before.

Although people, companies, and products are special, the basic requirements for getting a product to market and keeping it there are remarkably consistent, predictable, and, perhaps most importantly, driven by the laws of each country!

For every product, a company must:

  • Establish and maintain the identity, strength, quality and purity of the product.
  • Demonstrate the product is efficacious for its intended use.
  • Demonstrate the product is safe for its intended use.

For each one of these 3 requirements, health authorities and governments have set minimum standards that must be met. For example, in the United States, section 505(d) of the Food, Drug, and Cosmetics Act states that to adequately demonstrate that a product is efficacious for its intended use, one must provide:

…evidence consisting of adequate and well-controlled investigations, including clinical investigations, by experts qualified by scientific training and experience to evaluate the effectiveness of the drug involved, on the basis of which it could fairly and responsibly be concluded by such experts that the drug will have the effect it purports or is represented to have under the conditions of use prescribed, recommended, or suggested in the labeling or proposed labeling thereof.

Although the US FDA has some leeway within the limits imposed by Congress (e.g., Congress permits FDA to accept data from one adequate and well-controlled clinical investigation if FDA determines that such data are compelling enough), US regulation requires that all companies must meet the same basic standards for establishing product efficacy.

Now, how a company meets the basic standards for these 3 requirements can differ depending on the product. For example, the 505(b)(2) regulatory route was designed specifically for those companies who can rely on published literature or other publicly available sources of data to support some aspect (efficacy, safety, and/or quality) of their own application. In this case, they use other data in addition to, or entirely in place of, collecting their own data on their product to meet one or more of the minimum regulatory requirements for FDA. However, even in this case; it is still necessary for the company to meet all 3 of FDA’s basic requirements (quality, efficacy and safety) to get their product approved and keep it on the market. Regulatory health authorities in other geographies have similar basic regulatory requirements by which they evaluate medical products.

This may sound obvious, but there are still developers who think they, their company, and/or their product are special, so the drug development, approval, and/or post-marketing regulatory requirements should be different. (I have found this particularly interesting and wonder if these same developers think that the regulatory requirements should be different for just them or if it should be different for everyone, including their competitors!)

For example, I have heard companies say that their product should be approved because it has exceptional efficacy in a population of patients for which there is no currently available treatment even though their product could not be reliably manufactured. I’ve heard other companies say that their drug is safe and should be approved even though there was no clear demonstration of efficacy. Finally, I’ve heard several companies discuss approaching government officials, patient advocacy groups or others to try to get regulators to approve their product or keep it on the market when safety issues arise.

I’ve not seen any of these approaches work. The drug approval process is not about who you know. It is not about the uniqueness of the product inventor, developer, company or even product! It’s about providing regulators with adequate and compelling quality, efficacy, and safety information based on pre-established criteria.

Special is a wonderful thing. Be special and celebrate in it. Work for unique and innovative companies! In fact, this is how I believe new, life-changing, and truly ingenious therapies are discovered. But remember: among all the “specialness”, drug development, approval, and post-marketing regulatory requirements are dictated by law and are standard for all.

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