FDA Humanitarian Device Approval for Fetal Surgery

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The FDA approved a device set for fetal surgery from Karl Storz Endoscopy America (KSEA) under its little known humanitarian device exemption (HDE) program.  Interestingly many of the devices approved under this program are artificial hearts or heart assist devices.  It’s worth checking out the link above to see what sort of things fall into this category by the FDA.

First a bit of interesting background on the FDA HDE Program:  As the FDA says

On June 26, 1996, FDA issued a final rule to carry out provisions of the Safe Medical Devices Act of 1990 regarding humanitarian use devices (HUDs). This regulation became effective on October 24, 1996. An HUD is a device that is intended to benefit patients by treating or diagnosing a disease or condition that affects fewer than 4,000 individuals in the United States per year. A device manufacturer’s research and development costs could exceed its market returns for diseases or conditions affecting small patient populations. FDA, therefore, developed and published this regulation to provide an incentive for the development of devices for use in the treatment or diagnosis of diseases affecting these populations.

The regulation provides for the submission of an humanitarian device exemption (HDE) application, which 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. The application, however, must contain sufficient information for FDA to determine that the device does not pose an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use, taking into account the probable risks and benefits of currently available devices or alternative forms of treatment. Additionally, the applicant must demonstrate that no comparable devices are available to treat or diagnose the disease or condition, and that they could not otherwise bring the device to market.

An approved HDE authorizes marketing of the HUD. However, an HUD may only be used after IRB approval has been obtained for the use of the device for the FDA approved indication. The labeling for an HUD must state that the device is an humanitarian use device and that, although the device is authorized by Federal Law, the effectiveness of the device for the specific indication has not been demonstrated.

Why HDE is so important:  In addition to being a fertility specialist I work on and consult with industy on surgical device development.  Countless times I have seen a wonderful device that fills a clinical need to help my patients.  Unfortunately, the cold reality of what it will cost to bring the device to market often prohibits anyone making it!  I am not talking about an idea that is not pursued (that happens every day) but about real devices that exist and have completed trails showing they work– only to be killed off prior to commercialization because the millions it will take would not be recouped by sales.  It can easily cost $5 million to take a product through a clinical trial and FDA approval process. If it’s just an idea in the hands of a start up it can cost $25 million to develop and bring it to market. Hense the “well doc it’s important for the disease you treat but unlessyou can tell me how to sell millions I can’t bring this to market” conversation.  You can read how military research with medical trickle-down overcomes this hurdle often here. 

The approval of this device for twin-to-twin transfusion syndrome:  I myself have performed this exact kind of fetal surgery. Back when I was at Yale I led a team of gyn endoscopic surgerons, general surgery laparoscopy specialists, and maternal fetal medicine specialists trying to save a twin pregnancy that was dying from twin-to-twin transfusion syndrome.  Additional information about the disease is available at the twin-twin transfusion syndrome foundation and in a detailed medical tech post here and a list of fetal therapy centers here and a list of treatment options here.  In this disease, there is an abnormal link in the blood supply between two twins (they are normally totaly separate).  What happens is that the blood from one overwhelms the other twin causing multisystem failure and ultimately death.  Back when we did this there were no approved devices but we used this exact type of equipment. The manufacturer here did not invent somethig new but is getting approval to market and use the device which will hopefully make it easier for doctors to use it. This approval from the FDA is for a special kind of microlaproscope (read about the history and uses of tiny microlaparoscopes here)

How the new set works: 

The Karl Storz Fetoscopy Instrument Sets are surgical tools used in the treatment of twin-to-twin transfusion syndrome (TTTS). TTTS is a rare condition in which there is an imbalance in the amniotic fluid of some identical twins. This imbalance is caused by an uneven flow of blood between the twins through shared blood vessels that are present when the twins share one placenta.

The Karl Storz Fetoscopy Instrument Sets consist of a fetoscope (telescopic camera used to view a fetus) and sheaths which are used to pass other surgical instruments and/or fluid through the entry site. After identifying the shared blood vessels on the placenta with the fetoscope, a laser can be passed through a sheath. The laser, which is already cleared for marketing, is used to photocoagulate (destroy with heat) the shared vessels which should help to normalize the flow of blood between the twins.

How does it work? After a tiny cut in the mother’s abdomen is made, the fetoscope and sheath are inserted through the mother’s abdomen and uterus under ultrasound guidance. Through the fetoscope the surgeon is able to view the placenta and determine which blood vessels are being shared by the twins and which ones are not. The Fetoscopy Instrument Set provides a pathway for a laser that can be used to destroy these shared or shared vessels after they have been identified. After all of the target vessels are identified and treated, the laser, fetoscope and sheath are removed.  

Stay tuned for posts reviewing the FDA device approval process in more detail as well as my thoughts on how profoundly the medical device climate has changed in the last decade.

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