How Does the FDA View the World of Regenerative Medicine?

A major focus of the Regenerative Medicine Guru Blog is the development of new medical interventions based on regenerative medicine.

Therefore, it is important to understand how the ‘gatekeepers’ view this field. In the U.S., the Food and Drug Administration (FDA) is the key gatekeeper. You may be surprised to learn that there is not a specific regulatory pathway spelled out for regenerative medicine products.

Instead, the FDA’s approach is to examine each specific regenerative medicine product and determine the regulatory pathway based on the most important component(s) of the regenerative medicine product. To accomplish this, the FDA essentially conducts a thought experiment in which the regenerative medicine product is ‘de-constructed’ in order to identify the key components and ask the question, which component(s) are most important in providing benefit to the patient.

Some simple examples should help clarify the FDA’s approach:


Examples of Regulatory Pathway For Regenerative Medicine Products

If the regenerative medicine product:

  • Consists of living cells that are delivered to the patient, it would be regulated as a cellular therapy.
  • Consists of cells that are genetically modified outside the body and given to the patient, it would be regulated as gene therapy
  • Consists of genetic material (DNA or RNA) that is directly delivered to the patient it is regulated as gene therapy
  • Consists of cells that are combined with a natural or synthetic biomaterial it will be regulated as a biologic-device combination product. Depending on the primary mode of action (PMOA) this type of regenerative medicine product could be regulated by the FDA as either a biologic or medical device. This will be discussed in a future post.
  • Consists of proteins or peptides or a small molecule that is given to a patient to stimulate the body’s endogenous repair processes, it is likely to be regulated as biotechnology drug.
  • Consists of minimally manipulated human tissue for homologous use, it may not require FDA approval. Instead, these ’tissue products’ must comply with the FDA’s good tissue practices (GTPs).
  • Consists of human tissue that is manipulated and/or used in non-homologous manner, it will likely require FDA approval as either a biological product or a medical device.

This is not an exhaustive list of the types of regenerative medicine products that are under development, but it should give you an idea of how the FDA may regulate a given regenerative medicine product.

As you may imagine, there are numerous variations on the theme for the exact mechanism of action for how these products may act within or on the body. When the ‘primary mode of action’ of the regenerative medicine product is not clear, it is wise to submit a request for designation (RFD) to the FDA. You may be surprised how the FDA views your regenerative medicine product and could end up wasting a lot of time and money due to an erroneous assumption. The FDA has recently issued a guidance document on how to write an RFD.

In summary, the FDA’s current regulatory approach to the field of regenerative medicine is to ‘de-construct’ the medical product and identify the component(s) responsible for the primary mode of action in the patient. Usually the key component is analogous to other regulated products such as cellular therapy, gene therapy, combination products, human tissues, drugs or medical devices, which allows the FDA identify what it believes to be the most appropriate set of regulations for the regenerative medicine product under development.

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#1 What the Heck is a Regenerative Medicine Therapy? — Regenerative Medicine Guru on 07.29.08 at 12:25 am

[…] How Does the FDA View the World of Regenerative Medicine? […]

#2 IXA Statement on Xenotransplants for Type 1 Diabetes — Regenerative Medicine Guru on 08.07.09 at 4:09 pm

[…] Since it is clear that there continues to be  large unmet medical needs that are not yet met by any existing medical products, there has been a small but dedicated group of individuals who are attempting to determine if animal-derived cells and tissues can help.  In my view, xenotransplantation, as a cell replacement therapy,  fits within the scope of regenerative medicine. […]

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