Human digitization has moved closer to reality, with three recent FDA-related developments strongly suggesting that regulatory hurdles to cutting-edge life sciences technologies are less daunting than generally assumed:
#1: Approval of a Digital Pill
The U.S. Food and Drug Administration has approved the first-ever digital pill, produced in collaboration with Otsuka, the medication maker, and Proteus Digital Health, a medical device company. The solution comprises a pill with an embedded sensor that, when activated by stomach acids, sends a signal to a patch worn by the patient, which then communicates with a Bluetooth phone/tablet app that makes the data shareable – with the user’s authorization – with healthcare providers and caregivers. The resulting digital records of medicinal compliance help patients keep track of medications and allow healthcare providers to see whether the prescribed regimen is being followed. (Estimates of the cost of nonadherence to medicinal treatments range up to $300 billion a year – primarily from unnecessary hospitalizations.
The digital pill, Abilify Mycite®, is a controversial choice for the FDA’s nod. Because Abilify is used to treat serious mental illness, it raises the concern that the prevalence of paranoid delusions as a symptom of psychosis and other conditions such as schizophrenia will undermine the purpose of the digital pill system. Such patients may fear the motivations and activities of “the authorities,” and a digital pill that tracks their very ingestion seems highly likely to induce such fear in some (or even many) patients. Even for a broader patient population, it hardly seems overly paranoid to imagine that the wide use of such digital pills would be driven eventually by insurers mandating compliance as a condition of coverage. In general, patients might consider this “internal surveillance” as too much of a privacy incursion. While the delivery mechanism is aimed at improving health outcomes through better visibility into medicinal adherence, there is a potential shadow side stemming from concerns about “Big Brotherly” encroachments.
At the same time, the pilot program will certainly offer key insights into the types of hurdles digital medications will face in terms of wider acceptance. After all, a successful pilot would pave the way for wide-scale implementation with more mundane meds. Chronic mental disorders often require patients to take medications for prolonged periods, and slippage from such regimens is very common. If the digital pill does indeed prove to reduce relapses and hospital stays, we will likely see a slew of similar applications for FDA approval. And digital pills ultimately could be used as preventive and diagnostic tools as well as treatment options.
#2: Approval of Gene Therapy Treatment for One Type of Cancer
The FDA has also approved the first cell-based gene therapy The drug, Kymriah, is for treating recalcitrant cases of b-cell acute lymphoblastic leukemia, the most common childhood cancer in the U.S. The treatment, also called CAR-T cell therapy, requires that immune-system T cells are removed from the patient and genetically modified (or “reprogrammed”) in a lab to attack and kill leukemia cells – in effect, turning the patient’s cells into a “living drug.” The medicine is made by Novartis, which has set the initial price at a whopping $475,000 for a single treatment – but only for patients who respond to the drug within a month. The company says it is taking measures to ensure that anyone who needs the medicine, which carries a significant side-effect risk (including a potentially fatal reaction called cytokine release syndrome, which sometimes follows T-cell infusions) and requires special training for its administration, can obtain it. Novartis is also trying to get the treatment approved for adults.
Meanwhile, a second gene therapy treatment, called Yescarta and made by Kite Pharma, has also been approved by the FDA. The medication is to treat adults with intransigent non-Hodgkin’s lymphoma (i.e., cancer that has not been vanquished after two sequences of chemotherapy). The price tag for this treatment is a cool $373,000.
In fact, the FDA has indicated that the floodgates for approval of gene therapies and related treatments might be about to blow wide open, driven in part by the administration’s intention to dissuade patients from patronizing “rogue clinics” that offer such non-approved treatments. Indeed, “regenerative medicine” has boomed around the world, and the FDA needs to keep up with this trend. As FDA commissioner Scott Gottlieb said, “These concepts are no longer the stuff of science fiction, but rather, real-life science where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones; where new genes can be introduced into the body to combat disease; and where adult stem cells can generate replacements for cells that are lost to injury or illness.”
#3: Guidelines Issued for Software as a Medical Device
Finally, the third recent noteworthy development was the FDA issuing guidelines for software as a medical device (SaMD), a category of technology that is growing in importance as AI and powerful algorithms continue to be deployed in apps that offer diagnostic and treatment assistance in the life sciences and healthcare fields to improve health outcomes and reduce unnecessary costs, redundancies and errors. (Note that the app tied to the digital pills mentioned above is an excellent example of SaMD.)
We have written about the original directive issued October 2016 and its implications. The issuance of final rules is a key development because it enables developers to proceed rapidly, with the certainty that they will not need to drastically modify the solutions for regulatory compliance.
Just the Beginning?
This cascade of activity triggers a question: Is this a floodgate moment? It certainly looks like one. With the recent wave of regulatory permissiveness, life sciences organizations can proceed more confidently with their new-age visionary solutions, concepts and programs by plowing capital into R&D or seeking to acquire smaller entities with cutting-edge technologies.
On the other hand, it’s hard to construe whether these developments represent a temporary political development raising unrealistic expectations. What does not change is the need for rigorous trials and testing, and the fact that only efficacious treatments ultimately will move forward.
All factors considered, this is certainly the time for all life sciences organizations to turn up the heat on their new-age human digitization projects, as the amount of activity in the field is exploding, and the FDA – as we have seen above – is clearly accommodating and even encouraging these developments.
This is the third in a series of blogs on the burgeoning field of human digitization. Part 1 looked at emerging technologies that are ushering in an era of human augmentation, self-regulation, automated diagnosis and even vast intelligence gains. Part 2 details our numerous cutting-edge projects in the life sciences space, illustrating the reinvention of healthcare through AI.