Need-to-Know Laws and Regulations for Regenerative Medicine

Researchers, medical companies, and health providers need to understand which healthcare laws apply to regenerative medicine. The FDA has the authority to seize unapproved drugs and stop treatments.

Regenerative medicine is an exciting new medical field that is bringing physicians, biologists, chemists, engineers, professionals in computer science and robotics, and others together to help increase the healing process.

While the human body is designed to heal itself with time, regenerative medicine aims to accelerate the healing process and add new ways of healing. Regenerative medicines works to replace tissue or organs that have been damaged by disease, trauma, or congenital issues. Instead of focusing on symptoms, regenerative medicine uses cellular therapies, tissue engineering, medical devices, and artificial organs to help people get healthy.

Types of regenerative medicine

The McGowan Institute for Regenerative Medicine classifies three types of regenerative medicine:

1. Tissue Engineering and Biomaterials. Tissue engineering is a strategy where biologically compatible scaffolds are implanted in the body at the site where new tissue is to be formed. If the scaffold is in the geometric shape of the tissue that needs to be generated, and the scaffold attracts cells the outcome is new tissue in the shape desired. If the newly forming tissue is subjected to exercise as it forms, the outcome can be new functional engineered issue. This field, still in its infancy, has helped people mostly with the regeneration of soft tissue.
2. Stem cell therapies. Many millions of adult stem cells are found in every human. Our body uses stem cells as one way of repairing itself. Studies have illustrated that if adult stem cells are harvested and then injected at the site of diseased or damaged tissue, reconstruction of the tissue is feasible under the right circumstances. These cells can be collected from blood, fat, bone marrow, dental pulp, skeletal muscle and other sources. Cord blood provides yet another source of adult stem cells. This field of medicine is quickly advancing as researchers and practitioners work to study and use it.
3. Medical Devices and Artificial Organs. Current practice when an organ fails is to look for a suitable donor and arrange a transplant. The recipient of the transplanted organ needs to take immune-suppression medications which have uncomfortable side effects. While the patient is waiting for the donation, advanced efforts need to be taken to keep the patient alive.

Using circulatory support as an example, there are technologies in various stages of maturity, initially using ventricular assist devices (VADs) as a bridge to a heart transplant, and now there are VADs that are used for long-term circulatory support (destination therapy).

Scientists and clinicians around the world are developing and evaluation devices to supplement or to replace the function of many organ systems including the heart, lung, liver and kidney.

Many companies and organizations are investing in clinical studies and research to address some of the most deadly and debilitating diseases – such as cancer, diabetes, and Parkinson’s disease.

The Food and Drug Administration (FDA) is working to govern the research and treatments that are based on regenerative medicine. The FDA is the main regulator of these new products and treatments. The Federal Trade Commission (FTC) monitors the truth of claims that health companies and health companies are making, sometimes falsely, to entice other doctors and patients to use their devices and services.

New FDA regulations

The FDA, part of the US Department of Health and Human Services, is responsible for protecting the public from dangerous products and working to ensure their safety. Products include medications for humans and animals, vaccines, and other biological products. The Food and Drug Administration also regulates our food, dietary supplements, cosmetics, tobacco products, and other items.

In August, 2017, the FDA announced new oversight regulations for regenerative medicine. The policies are meant to balance the good these new treatments can do with the concerns of patient safety and unscrupulous practices.

• Premarket approval versus the practice of medicine. The FDA recognizes that there is a fine line between medical products which can be regulated by the FDA and individualized treatments done by a physician during his medical practice. In the field of regenerative medicine, the line is fine because science and clinical developments are quickly changing the products and the treatments.

Experienced regenerative healthcare medicine lawyers understand how to address the dividing line between FDA governance and state medical board governance.

From FDA’s Website:

“For example, sometimes when cells or tissues are taken from and given back to the same individual or when the cells or tissues do not undergo significant manufacturing, are intended to perform the same basic functions, and are not combined with another drug or device, among other factors; their benefits and risks are well understood. In these circumstances, the products may not require premarket review under current law.

However, when significant manufacturing is performed on the cells or tissues, or when the cells or tissues are not intended to perform the same basic functions, far greater uncertainty exists as to the benefits and risks involved. In these cases, it’s necessary to understand the benefits and risks in clinical trials prior to widespread use of the products. Therefore, premarket review is required.”

• FDA enforcement. The FDA is increasing its enforcement actions against health providers and companies who try to deceive patients by implying that their products and services can deliver miracles. One example of aggressive FDA enforcement was a recent court request to seize product components of a vaccinia virus vaccine which was supposed to help treat cancer but instead put the patients at risk.
• Encouraging innovation. The FDA is changing its policy framework so that product developers (including people working in hospitals and clinics) will be able to more efficiently gain FDA approval for their products through a process that is minimally burdensome and less costly. Innovation should be encouraged provided patient safety is paramount. The polices will work to incorporate the 21st Century Cures Act – as it relates to regenerative medicine.
• Compliance policy. The policy framework will also include a compliance policy that will help legitimate product developers interact with the FDA in a reasonable time to determine if they need to submit an application for marketing authorization and to come into the agency and work on a path toward approval.
• Regenerative medicine advanced therapy. The FDA will also work to fully implement the Regenerative Medicine Advanced Therapy (RMAT) designation.

As described in Section 3033 of the 21st Century Cures Act, a drug is eligible for regenerative medicine advanced therapy (RMAT) designation if:

• The drug is a regenerative medicine therapy, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, except for those regulated solely under Section 361 of the Public Health Service Act and part 1271 of Title 21, Code of Federal Regulations;
• The drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and
• Preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.

FDA, Regenerative Medicine Advanced Therapy Designation

The FDA plans to include certain gene therapy products that permanently alter tissue and produce a sustained therapeutic benefit as part of the products that will meet the definition of being eligible to come under the pathway enabled by RMAT.

21st Century Cures Act

 The Cures Act implements measures to:

• Help expedite clinical trials
• Help researchers attend conferences so they can network with colleagues
• Help NIH-supported researchers share data
• Improve research privacy
• Help young investigators get access to NIH funding
• Encourage inclusion of diverse populations represented in clinical research

Supporters of the Cures Act says that the law helps bring drugs, devices, and treatments to market faster. Opponents say the rush to market makes these items more dangerous.

HCT/P

The FDA regulates human cells, tissues, and cellular and tissue-based products (HCT/Ps). They may either be regulated by the Center for Devices and Radiological Health (CDRH) as medical devices or by the Center for Biologics Evaluation and Research (CBER). The aim of regulations is to prevent communicable diseases.

As we wrote in our article on the ambiguity of stem cell therapies and regenerative clinics, there are some key considerations an experienced regenerative medicine lawyer will review. These include:

The FDA HCT/P regulations:

• Create a unified registration and listing system for establishments that manufacture HCT/Ps
• Manufactures of HCT/P must comply with the FDA’s regulations.
  • Manufacture means, but is not limited to, any or all steps in the recovery, processing, storage, labeling, packaging, or distribution of any human cell or tissue, and the screening or testing of the cell or tissue donor.

Critically, the following conditions must be met:

• HCT/P’s must be minimally manipulated.
• HCT/P’s must be intended for homologous use only, as reflected in the labeling, advertising, or other indications of the manufacturer’s objective intent.
• The manufacture of the HCT/P does not involve the combination of the cells or tissues with another article, with certain stated exceptions.
• Either: (i) The HCT/P does not have a systemic effect and is not dependent upon the metabolic activity of living cells for its primary function; or (ii) The HCT/P has a systemic effect or is dependent upon the metabolic activity of living cells for its primary function, and: (a ) Is for autologous use; (b ) Is for allogeneic use in a first-degree or second-degree blood relative; or (c ) Is for reproductive use.

Two key definitions:

• Homologous use means the repair, reconstruction, replacement, or supplementation of a recipient’s cells or tissues with an HCT/P that performs the same basic function or functions in the recipient as in the donor….
• Minimal manipulation means:
  • For structural tissue, processing that does not alter the original relevant characteristics of the tissue relating to the tissue’s utility for reconstruction, repair, or replacement; and
  • For cells or nonstructural tissues, processing that does not alter the relevant biological characteristics of cells or tissues.

The following items are not considered HCT/Ps (by the FDA stem cell regulations):

• Ancillary products used in manufacture
• Blood products
• Blood vessels recovered with an organ
• In-vitro diagnostic products
• Minimally manipulated bone marrow
• Secreted or extracted products; e.g., human milk, collagen, cell factors
• Tissues intended for educational or non-clinical research use (not intended for transplantation)
• Vascularized organs
• Xenografts

Health care researchers, companies, and practitioners need to understand when FDA regulations apply to their products and services and when the treatments are governed by state medical practice laws. If the FDA regulations govern their products and treatments, they need to understand what steps such as premarket approval they must take and how to comply with the FDA rules.

Failure to comply with FDA warning letters can result in warning letters, product seizures, injunctions, fines, and criminal prosecution. To understand how to best comply with the FDA rules and regulations, you need the help of an experienced regenerative medicine attorney – one with a strong record of working with healthcare companies and physicians. Make the call to Cohen Healthcare Law Group PC today at 310-844-3173 or fill out our online contact form to make an appointment.