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Monitoring the FDA Pipeline

By August 27, 2020January 15th, 2021Employers Pharmacy Spotlight
Test tubes and a microscope. Science!

The U.S. Food and Drug Administration (FDA) must approve medications and other therapies as safe and effective before they can be sold in the U.S. Back in the late 1980s, it took about three years for a drug to go from submission to FDA approval. Fast forward about 30 years, and the timeline is much more accelerated. In 2018, the average FDA review time was 10 months.

A shorter review process may bring drugs to market faster, but it presents more challenges for health plans. With the high costs of many new therapies, it is critical for Independence Blue Cross (Independence) to determine management strategies before a drug hits the market. Otherwise, there is a window of time when doctors can prescribe the new drug without the health plan approving and monitoring its use.

Our solution: Careful monitoring of the FDA drug pipeline.

What is the FDA drug pipeline?

The pipeline refers to an inventory of drugs in the FDA review process. This list includes all brand, generic, biologic, and biosimilar drugs.

Our dedicated team of pharmacists, physicians, nurses, and business analysts tracks drugs through the FDA process. We analyze various features of each pipeline drug, including:

  • Treatment indication and coverage criteria (that is, how is it supposed to be used and who is eligible to use it?)
  • Similarity to other drugs on the market
  • Likelihood of off‑label usage or abuse (that is, will this drug be used for something other than what it’s approved for?)
  • Cost (estimated, if drug cost isn’t available yet)

Once we target drugs in the pipeline, we can determine our benefits and coverage positions before the FDA approves the drugs. This process also gives us time to develop medical policies and determine if any utilization management strategies are needed, such as precertification, prior authorization, step therapy, and tiering. These strategies help manage cost and utilization and ensure that the drugs are being used appropriately.

Looking Ahead to Gene Therapies

In no drug class is it more important for Independence to watch the drug pipeline than for gene therapies. Medical technology is moving at a dynamic pace, and gene therapies have emerged as promising treatment options for those with certain inherited conditions or genetic mutations. There are currently more than 150 gene therapies in the FDA pipeline. Many replace a missing or nonfunctioning gene with a new, corrected copy. Others edit a gene so that it can start doing its job. Some deactivate a mutated gene that is causing problems.

Gene therapies can be life-changing, but their costs are significant. Millions, in fact! Two recently FDA-approved gene therapies are Luxturna™ and Zolgensma®. Luxturna treats a rare form of blindness, and a one-time dose costs nearly $1 million. Zolgensma treats spinal muscular atrophy, a rare but lethal disorder that causes motor skill deterioration, so things like walking, eating, and breathing become impossible. A one-time dose of Zolgensma costs $2.1 million.

As more gene therapies get approved, each one is likely to be more expensive than the last. These initial gene therapies treat ultra-rare conditions, but the gene therapies in the FDA pipeline treat conditions that affect more people, such as hemophilia and sickle cell disease. Independence is watching the movement of these therapies through the pipeline, as they will likely impact more members.

Remaining Focused on Gene Therapies

Our dedicated team must remain vigilant when it comes to managing these unique therapies for many reasons:

  • Unlike clinical trials for most drugs, where hundreds or even thousands of people participate, clinical trials for gene therapies have much lower enrollment because the conditions they treat are rare.
  • Long-term data on gene therapies is often unavailable. This is especially true for conditions that have no existing treatment options. In those cases, the pressure to get a therapy to market is significant.
  • As mentioned, the costs associated with gene therapies are extremely high. Their extraordinary price tag can impact everyone from the member, to employers who provide health insurance for their employees, to the health plan.

Independence continues to establish guidelines for gene therapies to ensure these innovative treatments are available to those who are most likely to benefit from them. We also work with the drug manufacturers, the Blue and Cross Blue Shield Association, and our network providers to ensure gene therapies are given in facilities that are appropriately staffed to prepare and administer them. All gene therapies require precertification approval from Independence, and coverage criteria is detailed in our medical policies.

We believe that our strong network relationships and effective management strategies will allow Independence to continue providing access to these life-changing therapies, while improving member outcomes and controlling overall spend. To learn more about our cost-saving strategies, please reach out to us.


Gregory Gambescia

Gregory Gambescia is the Director for Specialty Pharmacy at Independence Blue Cross. He has over 15 years of Managed Care experience and specializes in Medical Specialty Pharmacy management. In his current role at Independence, Greg develops and implements management strategies to save on the rising drug costs on drugs covered under the Medical Benefit. He is an integral member of the Medical Cost Council and provides strategic direction on new initiatives to save on medical costs.