The US Food & Drug Administration’s (FDA) main purpose is to protect and promote public health. It does this by regulating drugs, cosmetics, and other products available to consumers in the US. Marketed drugs in the US undergo a lengthy regulatory process in hopes of getting approval for a particular disease indication. The FDA in 2013 only approved 27 of 81 drugs approved by the EMA according to Thompson Reuters (2014). A drug’s FDA labeled indication has been the cornerstone of what shapes national therapeutic guidelines. National therapeutic guidelines usually tend to be the holy grail of which drugs are appropriate for what conditions. This is very important because 3^rd party payers use therapeutic guidelines to help shape their formulary decisions. Frankly, if the FDA doesn’t grant approval for a certain labeled indication for a drug, the drug will not be included on formularies on a preferred tier, which ultimately will effect a drug’s potential access to market share.

Are FDA approvals of a drug for a particular disease indication and therapeutic guidelines mutually exclusive? No, consider Eliquis, Xarelto, and Pradaxa for example. All are FDA labeled for various conditions that require anticoagulation and prevention of systemic clots. All these drugs went through the FDA approval process and were granted labeled indication at various times during the life-cycle of the drug. The formulary placement of the drug and placement on therapeutic guidelines depended on the indication for the drug and when the approval took place. A drug’s placement on treatment guidelines has a direct impact on weather 3^rd party payers will cover the drug.

There have been plenty of drugs approved in Europe and Canada that have not been approved in the US for certain indications. Can the drug still be marketed for the indication not FDA labeled? No, and 3^rd party payers prefer drugs that are FDA labeled for the conditions they used for. Historically, 3^rd party payers have a formulary with tiers that determine what drugs a patient should receive. The impact of labeled indications extends far beyond initially getting a drug approved.

Post marketing trials are essential in establishing new indications for a drug already on the market. This is extremely important for patients. With new FDA labeled indications 3^rd party payers are now compelled to reevaluate their formulary, allowing patients to have greater access to more appropriate medicines. Big Pharma invests a substantial amount of dollars into post marketing research. A new FDA labeled indication for a drug opens up the possibility to expand a drug’s potential market share in a new therapeutic category.

Drugs that are not FDA labeled for certain indications can be a problem for patients needing access to innovative medicines. This is certainly true of Vertex’s miracle drug Kalydeco, which was initially FDA approved in 2012 to treat patients with Cystic Fibrosis with at least one copy of the G551D mutation. Just recently, in February 2014, the FDA approved 8 additional mutations that can be treated with Kalydeco. Now patients who have Cystic Fibrosis with any of these mutations G178R, S549N, S549R, G551S, G1244E, S1251N, and S1255P have access to Kalydeco. Even 3^rd party payers can face problems with high drug prices because of lack of competition in a particular therapeutic category. It’s no secret that the FDA regulatory process has a direct impact on insurance companies, national therapeutic guidelines, and more importantly the overall patient experience.

Patients are affected greatly by the decisions of the FDA. Pharmaceutical companies should use new regulatory strategies and up to date regulatory intelligence to overcome potential obstacles in bringing drugs to markets or expanding indications. Lately, government agencies, such as EMA and FDA, are approving drugs much faster. If potential market access for a drug is highly based on it is FDA label indication, then the FDA greatly influences how well a drug will perform in today’s market?