Technology Could Enable Personal Medicine Whether We Like It Or Not
The past several months have been like an extended Christmas for those interested in science and technology, especially if you have high hopes that technology will eventually improve our lives in diverse ways. It seems like discoveries are made almost weekly that have the potential to disrupt major industries (think artificial intelligence and autonomous vehicles).
Recently, there have also been several technological developments that could disrupt another major industry, pharmaceuticals, and encourage those patients who hope that one day they will be able to recreate expensive pharmaceutical drugs right in their garage. These developments are only a beginning, but as the technology involved advances and becomes cheaper and easier to use, the possibility of home-manufactured drugs will become increasingly realistic.
Giving the “Disenfranchised” Access to Medicine
Bringing that future of garage-based, personal pharmacies to fruition is one of the passions of Dr. Michael Laufer, a math professor at Menlo College. Laufer gained some fame last year when controversy over the rising price of the EpiPen was raging. He introduced his homemade EpiPencil, which could be constructed for about $30 using materials easily accessible to most people, and released the plans online.
To promote his hacker approach to pharmaceutical drugs, Laufer started a website where other “pharmahackers” could congregate as they attempt to spark a DIY movement. Among other things, the website contains downloadable instructions for creating the EpiPencil and the drug Daraprim, made famous when Martin Shkreli’s Turing Pharmaceuticals significantly increased the price of the drug for no apparent reason.
According to the website, Laufer wants to help those “disenfranchised from access to medicine” for reasons including “price, legality, and lack of infrastructure.” He explains that many cannot afford treatment, are legally barred from receiving treatment (e.g. abortion drugs in countries where abortion is illegal), or live in a remote region that doctors cannot reach. Underlying this desire to help the disenfranchised, is a firm belief in patient autonomy. “People should be able to take control of their own health choices,” he says.
To equip patients to take control of their health, Laufer envisions a device simple enough for patients to operate out of their homes that can manufacture pharmaceutical drugs like aspirin and Daraprim. The math professor has already uploaded plans for the device, which he calls the Apothecary MicroLab. The MicroLab is basically an automated lab reactor that uses fairly easy to acquire parts in its design. Once built, the pharmahacker can program the machine to create the drug that they want using instructions like those Laufer released for Daraprim.
Though he is confident his idea will work, Laufer cautions that everything is still in the testing and development stage. He soon hopes to unveil the beta version of the MicroLab for customers to begin using.
Extremely Easy to Endanger Yourself
Concerns about safety are naturally going to arise when untrained and unsupervised enthusiasts start concocting their own pills. While Laufer says that he has tried some of his own creations without any ill effects, the Food and Drug Administration (FDA) emphasizes that creating your own medication is a dangerous idea. According to the agency, without the FDA’s seal of approval the patient has no way of knowing if their creation is safe or effective. They simply have to trust that they followed Laufer’s instructions exactly, and that those instructions are reliable.
Jeremiah Johnson, of MIT’s chemistry department, further explained that, for someone with little knowledge of chemistry, it would be extremely easy to endanger themselves while making medications. They could end up creating a completely different drug than what they had intended, or take an overdose of the correct drug, or accidentally mix two chemicals and create something toxic. Really any number of things could go wrong. He simply could not envision a method that would protect the patient enough for him to endorse DIY pharmaceuticals.
Laufer counters these cautions by using the argument that some have employed for quite some time: “If someone is dying of a chronic disease, there’s already not much worse they can get.” Additionally, he again affirms his belief that patients have the right to make their own choices, even dangerous ones, about their health.
A Life-Saving “Cup of Noodles”
Though Laufer’s vision seems a little far-fetched, other scientists are working on slightly more believable, and safer, technology that could have similar democratizing effects. At Harvard, biochemist Peter Nguyen is creating a process that he hopes will make preparing life-saving drugs more like cooking a “Cup of Noodles,” as Gizmodo’s Kristen Brown put it. He recently published a paper explaining his process that enables “on-site, on-demand manufacturing of therapeutics and biomolecules.” Nguyen combines special freeze-dried “reaction pellets,” freeze-dried “DNA templates,” and plain old water to spark the chemical process that leads to the creation of vaccines and drugs.
Nguyen intended the process to be used to make it easier and cheaper to deploy drugs to remote corners of the globe, since the elements wouldn’t need to remain cold to retain their potency. However, he admits that “there is also talk of using this for democratizing biotechnology.” Anyone who can purchase or access the pellets and DNA templates some other way could build their own medicine.
Obviously, there are safety risks that are associated with this technology as well. Anyone who has tried to follow a recipe knows that things can quickly go wrong and the product that comes out of the oven can look nothing like the beautiful picture in the cookbook. However, unlike Nguyen’s cup of chemicals, failed baking projects won’t often kill you.
To minimize user error as much as possible, Nguyen looked to the simplicity of ramen noodles for inspiration. Few people mess up the food popular with broke college students because the number of ingredients and steps have been reduced to the fewest possible. The biologist wants his mix-it-yourself medicine to be like that, something simple that “the average consumer truly can’t screw up.”
It Can’t Be Legal
Even if Laufer or Nguyen succeeds in perfecting technology that allows patients to safely create their own pharmaceuticals in their garage, these processes are likely to still run into legal trouble.
The FDA tightly regulates pharmaceutical drugs and acts as the gatekeeper to the marketplace for any company or individual who wants to sell drugs to patients. Congress empowered the agency with this authority mainly through the Federal Food, Drug, and Cosmetic Act.
In Laufer’s vision, patients would be equipped with a machine that could be programed to manufacture drugs like aspirin or Daraprim. They would also have access to the molecular plans for these drugs provided on the Four Thieves website by hobby chemists from around the world. As Professor Patricia Zettler, an expert on medical regulation and the FDA at Georgia State University, explained to me, the agency possesses no authority over the act of publishing the chemical blueprint. However, once patients begin using these instructions to create their own drugs, they will almost assuredly break the law.
Professor Zettler noted that the most relevant pieces of the law were prohibitions on “the introduction … into interstate commerce of any … drug … that is adulterated or misbranded” (21 U.S.C. 331 (a)), and tampering with a drug’s label while that product is being offered for sale after traveling through interstate commerce. Furthermore, the law prohibits anyone from even manufacturing an adulterated or misbranded drug. And since any product created by a nonregistered individual counts as a misbranded drug and not following the FDA’s Good Manufacturing Practices is enough to make a drug adulterated, it is difficult to see how any hobby pharmacist could escape breaking the law even if they are producing drugs for their own use.
It’s the Enforcement That Counts
However, the law on the books matters much less than the law that is actually enforced. And enforcement takes both will and ability. It seems that recently, when it comes to certain aspects of medicine, both of these may be weakening.
Will To Enforce
Just last week, the Senate passed a “right to try” bill introduced by Senator Ron Johnson of Wisconsin. The bill opens a new route for terminally ill patients to gain access to drugs that have passed the Phase I trials but are still in development provided that the patient has exhausted all other treatment options, is unable to participate in a clinical trial, and provides their physician with a written statement of informed consent. Importantly, the bill also shields manufacturers and prescribers from some liability related to allowing patients access to unapproved drugs.
While the bill must still pass the House, it passed the Senate by unanimous consent and has already gained the support of several dozen members in the House. Also David Gorski, an oncologist at Wayne State University, admits that opposing right to try legislation has become politically untenable. “Opposing right-to-try laws is akin to opposing motherhood, apple pie, and the American flag; you just don’t do it and expect to be re-elected,” he said.
Perhaps this is due to how the arguments of individual autonomy that underlie the right to try movement have increasingly resonated with patients. Victor Riches, president of a legal advocacy organization called the Goldwater Institute, explains that “There’s no more fundamental freedom than the right to save your own life.” Patients seem to be embracing that idea and rebelling against the deep-seated paternalism that exists in the medical profession.
As this principle continues to take root and technology continues to enable patients to safely take more control over their health decisions, the legislative will to expand legal access to more medicine may spread to areas like DIY pharmaceuticals. The passage of Senator Johnson’s bill indicates that even Washington is questioning the wisdom of keeping medical authority centralized.
Ability To Enforce
At the same time, technology is making it vastly more difficult to enforce, or even update, old restrictive laws. This phenomenon has been termed the pacing problem and is often discussed by scholars. In his book A Dangerous Master, Yale bioethicist Wendall Wallach explains the pacing problem as “the gap between the introduction of a new technology and the establishment of laws, regulations, and oversight mechanisms for shaping its safe development.” While scientists are genetically modifying human embryos, Congress continues to prohibit the FDA from even acknowledging any applications that involve this new technology. Lawmakers have no time to consider the difficult ethical and practical questions that accompany CRISPR technology. Their attention is devoted to age-old issues like health insurance and taxes.
This in turn places the FDA in an awkward position because they are tasked with upholding laws that basically render many technologies currently being explored by synthetic biologists illegal. It is extremely difficult for the FDA to counter act the pace of technological change, especially in a world, perhaps only several years away, in which a machine akin to the one envisioned by Laufer is safe enough and cheap enough to be used by many patients.
At the point when people can safely create pharmaceutical drugs in their garage or bedroom, there will exist what my colleague Adam Thierer has called a de facto right to try for personal medicine. Machines like those discussed here will continue to decentralize medical authority and empower patients to play an ever growing part in the treatment process to the extent that central government agencies like the FDA will have little say in what patients can and cannot do. At that point the agency must make a decision: use its scare resources to track down and prosecute every garage-based pharmahacker making their own medicine, or prioritize infractions and only pursue those that truly pose catastrophic, widespread, irreversible harm to the public.
Possible Solutions
Considering the large cost of enforcing the current restrictive laws in a world of technology-equipped pharmahackers, the enforcement agencies like the FDA ought to begin considering what Thierer likes to call the “corner cases.” These are situations in which there is a broad consensus that such behavior is harmful to a large portion of the public and ought to be stopped. For example, using genetic modification to intentionally harm a developing child. Most can agree that such a thing would be terrible and ought to be prevented. Focusing resources and enforcement power on such corner cases will help agencies to effectively protect public health and not get sidetracked on less pressing situations.
Adapting the principles of products liability law that currently helps to guide the pharmaceutical industry could also provide a way to promote the safety of patients in a world with distributed medical technology. Most courts agree that many useful prescription drugs are “unavoidably unsafe,” which causes them to generally apply negligence standards as opposed to strict liability. Consider thalidomide, the drug that was widely prescribed for morning sickness and ended up causing babies to be born with physical deformities, as an example of an unavoidably unsafe drug. Despite having these side effects for pregnant mothers, doctors discovered that the drug was effective in treating leprously. If the drug’s chemical makeup was altered to prevent the bad side effects, its effectiveness against leprosy would also vanish. The danger is unavoidable and must be prevented by only giving it to leprosy patients.
This has led to warning requirements that both doctors and manufacturers must meet in order to avoid liability. In most cases, drug manufacturers must provide “reasonable instructions or warnings regarding foreseeable risks of harm” to doctors or the manufacturer can be held liable. And since doctors are the primary point of contact with the patient, they must relay the warnings and instructions from the manufacturer to the patient and can be held liable if they fail to do so under the “learned intermediary doctrine.”
Adapting these principles that have guided the industry for some time and holding pharmahackers to the learned intermediary standard could protect the public and deter many chemistry enthusiasts who have inadequate knowledge of what exactly they have created from distributing it to their neighbors.
A third possible solution is for those scientists like Laufer and Nguyen who are undertaking these projects to begin engaging with interested stakeholders to develop norms to govern what could be a burgeoning industry. These “soft law” mechanisms include codes of conduct, best practices, and multistakeholder processes and have been pursued in other emerging fields like drones, cybersecurity, and the internet of things.
I offer a range of possibilities because there are no silver bullet solutions to the difficult questions that confront the policy makers, scientists, and citizens grappling with quickly advancing technology. Dr. George Church of Harvard says it well in his recent book on synthetic biology Regenesis: there are “no magic bullets for absolutely preventing worst-case scenarios [and] no fail-safe fail-safes.” Instead, utilizing a layered approach and pursuing various solutions will provide the most protection while also allowing for the great benefits that technology can bring.
Conclusion
In light of the pacing problem and projects like those of Laufer and Nguyen, it seems likely that technology will usher in a world where medicine is democratized and decentralized whether those in Congress and at the FDA like it or not. And we should not dread this future. There are many good things that will come with it: greater access to life-saving drugs, novel ways to eliminate debilitating diseases, and more individual liberty. Still, the skeptics do raise some valid concerns that ought to be debated and mitigated. It is for that very reason that regulators should stop trying to halt technological progress and instead engage in candid conversations about how to maximize benefits and minimize risks. Openness and foresight will shepherd in the best possible version of these exciting new technologies that promise better health for more people at a lower cost.
