Cell Therapy Products… Falling into the HDE Category?
MedCity News released an article about Nanofiber Solutions development of scaffolds used for organ transplants. The company, a spinoff from The Ohio State University, boasts the first synthetic nanofiber transplant in the world, and hopes to grow in its market of artificial organ transplants.
The technology, as described in the news-release, utilizes artificial organ scaffolds in the shape of the intended organ. Using stem cells that are harvested from the patient, the scaffold is then seeded then cultured for a few days. The result? The patient’s own tissue grows on the scaffold, creating the artificial grown organ, which can be implanted into the patient.
Currently the company is using this technology for trachea transplants. The technology is not FDA approved, but was able to be used in patients because it fit the criteria for Humanitarian Device Exemption. According to the FDA overview of the HDE, this means that the device is intended to benefit patients by treating or diagnosing a disease or condition that affects or is manifested in fewer then 4,000 individuals in the US per year. The HDE is similar in both form and content to a premarket approval (PMA) application, but is exempt from the effectiveness requirements of a PMA. An HDE application is not required to contain the results of scientifically valid clinical investigations demonstrating that the device is effective for its intended purpose.
When used properly the HDE program can provide an incentive for the development of devices for the treatment or diagnosis of rare diseases or conditions. Typically patients who receive humanitarian device exemption devices have no other comparable device available to diagnose or treat their disease or condition. With the expected innovation from the Cell Therapy Industry, it’s easy to see how many of the therapies developed may fit into this category. However, there has been much discussion about the misuse of the program to avoid the time and intensive efforts of conducting appropriately sized and statistically powered clinical trials.
Do you have any experience with the HDE or PMA processes? Share you experiences with us!