Introduction

This primer will provide you with an overview of the different regulatory pathways provided by the United States Federal Drug Administration (FDA). The FDA has two separate approval pathways, depending on whether you have a:

Medical Device or Diagnostic

Drug (Small Molecule or biological)

 

Our hope is to help you understand what kind of approval you may require in different situations. When possible, we provide estimates for time and cost of the requirements to aid in your planning.

Combination Products

Some products will combine together two of more categories to result in a combination product. This may include:

Two or more regulated components combined or mixed and produced as a single entity

Two or more separate products packaged together in a single package or as a unit.

A product packaged separately that is intended for use only with another approved product.

In the case of a combination product, the combination needs review, even if all components are approved separately. The combination product will be reviewed based on the product’s primary jurisdiction. This is determined by the product’s primary mode of action (PMOA), or which component provides the most important therapeutic action. If it’s unclear, you can file a Request for Designation (RFD) with the FDA.

„Examples of Non-Obvious FDA Pathways

1. Regranex

Regranex is a topical treatment for the prevention of lower extremity diabetic ulcers, containing platelet-derived growth factor. Prevention of diabetic ulcers required metabolization of the active ingredient, platelet-derived growth factor. Because the primary therapeutic means was achieved through an enzyme that needs to be metabolized, it was classified as a biological. As a result „Regranex was approved with a Biologics License Application (BLA).

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2. Gene Therapy

Gene therapy is a treatment that alters the genetic composition of the cells in a wound to promote healing. „It involves any introduction of genetic material to affect a change, which can be seen as needing to be “metabolized” to produce its effect. Thus, gene therapy is regulated as a biological using a Biologics License Application (BLA). Note that to date no gene therapy product has been approved for sale.

More on CBER, the FDA's cell and gene therapy division.

 

3. „Infused Bone Graft

This product is a synthetic bone graft consisting of a resorbable collagen sponge with a genetically-engineered human protein. This has both a device (the resorbable collagen sponge) and a biological not metabolized in the body (the genetically-engineered human protein, rhBMP-2, that promotes bone growth). The biological was determined as the primary mode of action. Since it is not classified as a drug and since it had no predicate, this product was approved as a combination product using a PMA pathway

 

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We discuss the two pathways separately, since they are distinct. You can review both, or select the one that is relevant to your product.


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