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A significant change is coming to the way the U.S. Food and Drug Administration (FDA) enforces its regulations for certain human cell, tissue, and cellular and tissue-based products (HCT/Ps).

Come May 31, 2021, FDA’s enforcement discretion policy for HCT/Ps will expire, opening up life sciences companies and many physician practices involved in manufacturing, selling, or using these products to increased regulatory scrutiny.

Although the Agency has had regulations for cell and tissue products for more than 20 years, FDA further developed and explained its regenerative medicine policy framework in November 2017 to help facilitate and support the innovation of regenerative medicine therapies. The Agency gave the industry until November 2020 to ensure that its HCT/Ps met the appropriate legal requirements, stating that it would exercise limited enforcement discretion against these products during this three year period. This November deadline was delayed until May 2021 due to the COVID-19 pandemic, and now companies have just a few months left to make sure they fully comprehend the regulatory requirements and policy framework, and how it applies to their HCT/Ps.

The regenerative medicine policy framework is complex, and many grey areas exist as to where certain rules apply. It can be difficult for any life sciences company with deep experience in FDA regulations – let alone a physician practice or healthcare provider – to fully understand the nuances of how they and their HCT/Ps will be regulated.

Thankfully, there’s still time left to engage with FDA and implement necessary changes. For any organizations manufacturing, selling, or using HCT/Ps, now is the time to work with regulatory experts to assess their risks and compliance with the framework to prepare for the May 2021 deadline.

Below are five steps every organization that works with cells and tissues — whether a company developing a new product, an organization offering HCT/Ps for sale to other parties, or healthcare professionals that use cells or tissues to treat a patient — must take to understand this regulatory framework before FDA’s enforcement discretion period expires.

1. Understand that the scope of FDA’s reach is wide and complex

It can be difficult for many healthcare organizations and life sciences companies to decipher exactly whether and how they’re regulated by FDA when it comes to HCT/Ps under the Agency’s framework. Although FDA regulates cells and tissues that are intended for implantation, transplantation, infusion, or transfer into a human recipient, there are times when these products fall outside the scope of FDA’s reach, especially when it comes to certain procedures conducted by physicians.

FDA utilizes a risk-based framework to determine how an HCT/P is regulated. Organizations should therefore think of FDA’s oversight as a continuum. At one extreme are cells, tissues, and people exempt from complying with FDA regulations, and on the other hand are HCT/Ps that require FDA approval. In the middle lay HCT/Ps that must meet some FDA requirements but do not need to be approved. As the risk to a patient receiving an HCT/P increases, so does FDA’s oversight. All of this means that there are many grey areas, and organizations are best served when they understand this framework – and better yet, have someone on their side who knows this area in great detail.

2. Determine where you fall on FDA’s regulatory oversight continuum

Once a life sciences company or physician practice understands the scope of FDA’s reach, the organization must then look at its own procedures and processes to see where they might be subject to oversight.

Considering FDA operates under a risk-based framework, organizations should start by asking themselves three questions: How do I obtain my cells and tissues? How do I manipulate or process the cells and tissues? And how do I intend to use these cells and tissues? Answering these three questions will help determine the level of regulation. For example, removing cells or tissues from an individual and then reimplanting them into the same person — in certain limited cases — is outside the scope of FDA regulation. However, removing cells or tissues, processing them to change their characteristics, and then transplanting them for an entirely different use is likely to mean that they will require more regulation. Physicians and life sciences companies must therefore take a close look at FDA’s regenerative medicine regulations and policy framework, including how their own operations and products match up to that framework. Again, expertise here is key.

3. Look at how FDA has previously approached similar products

FDA provides a wealth of resources to help life sciences companies and healthcare providers understand the HCT/P regulations and remain compliant. In addition to guidance documents and other policy statements, FDA publishes warning letters that describe how a company failed to comply with the law, how and why FDA intervened, and what corrections the organization had to take to become compliant.

These materials are available for the public to view and study. It is important for any organization that works with HCT/Ps to look at these examples and understand FDA’s past enforcement actions. Doing so will help the organization assess how FDA may view their own products and actions. These resources also provide insight on how to potentially correct any issues before the enforcement discretion period expires.

4. Maintain an open and active dialogue with FDA

Since November 2017, FDA has encouraged its stakeholders to speak directly with the Agency, ask questions, and begin a dialogue about the stakeholder’s HCT/Ps. For example, under FDA’s TRG Rapid Inquiry Program (TRIP), manufacturers of HCT/Ps can send FDA the answers to the “how” questions noted above and then receive a nonbinding response with guidance. TRIP expires on March 31, 2021, ahead of the enforcement discretion policy expiration for HCT/Ps, but there is still time to use this pathway to receive advice from FDA about HCT/P products and practices.

There are other avenues to work with FDA as well, including submitting a request for designation (RFD) or a pre-RFD. These means of communicating with the Agency are ongoing and will not expire. While the Agency remains focused on examining and approving COVID-19 vaccines and other medical countermeasures, it still has bandwidth to accommodate requests for information around its HCT/P regulatory framework.

5. Leverage outside regulatory expertise

Even after a thorough review of FDA’s HCT/P framework and an organization’s own procedures and products, it is critical to leverage a third party’s perspective, especially one with decades of regulatory experience in the life sciences and healthcare fields. Compliance experts can provide an unbiased and thorough assessment of an organization’s practices and benchmark them against historical examples and emerging FDA positions. This outside perspective is invaluable when attempting to become compliant with complicated regulations. Thankfully, there are many attorneys and compliance professionals at Buchanan Ingersoll & Rooney with the expertise and wherewithal to identify potential gaps in compliance and recommend strategies to remedy them.

There’s still time

While the May 31 deadline for the expiration of FDA’s HCT/P enforcement discretion policy is rapidly approaching, there’s still time for life sciences companies and healthcare providers to prepare. They must start by fully understanding the framework and how it applies to their products and procedures. But doing so can be difficult. It requires not only open and ongoing dialogue with FDA, but also expert counsel from professionals at Buchanan who have experience in both regulatory compliance and the life sciences and healthcare industries.