The Medical Futurist | January 15, 2019

Globalization reached healthcare big time: a French patient can easily ask a Belgian company to sequence the DNA of his tumor tissue, then convince a Swiss pharma company to sign them up for a relevant clinical trial on a Spanish island. Not only that but also the recent controversies around gene editing in babies in China, as well as the operation of direct-to-consumer genetic testing companies, the online medication scene, health technology companies expanding their services to other countries highlight the need for a global “FDA” day by day.

We are not waiting for regulators

The availability of know-how, raw materials, rapidly developing methods and technology entails the exponential growth of innovation everywhere – that also means the rigorously regulated healthcare and life sciences. Previously, the traditional “ivory tower of medical knowledge” hindered the appearance of out-of-the-box solutions – individuals outside the medical community didn’t have access to knowledge, means or materials for innovation. However, 21st-century sharing economy enables peoples, groups or companies to access information, tools or raw materials in fields never available before – such as healthcare.

Also, as the sector is (over)regulated in certain geographical and scientific areas, and regulatory bodies react too slowly to issues raised by innovation, many disruptors are by-passing the nods for a new method or product from regulatory agencies and just target the right population on online channels. Also, numerous empowered patients act similarly: they are seeking out the treatment most appropriate for them – regardless of boundaries – and they are knocking on the doctors’ door with well-articulated plans regarding their care.

My favorite example is the #wearenotwaiting Twitter-movement for patients suffering from diabetes. The initiator of the community, Dana Lewis, and her husband built a so-called artificial pancreas at home and started to spread the blueprints and know-how on Twitter to other diabetes patients – without waiting for the approval of the FDA or any other agency. Why? Because it works and patients needed it. Dana had been using the device for almost two years by the time the US Food and Drug Administration finally approved it. Currently, the big fish of the diabetes market, Medtronic and Dexcom are both developing similar closed-loop systems as the “garage artificial pancreas” created by the #wearenotwaiting community.

The FDA shows the way forward for regulatory bodies

Regulations slowly following the tracks of innovation is a regular concept, but the FDA managed to catch up with the signs of times. However, it took some years for the agency to realize that they need another (faster) approach for digital health technologies.

For example, look at the case of direct-to-consumer (DTC) genetic testing companies. They appeared approximately ten years ago on the market – it developed too fast, and the FDA “recoiled”. In 2014, the FDA decided to restrict 23andme’s operations saying that the health information services were not clear or complete enough for customers. So, the genetic testing company scaled back its activity mostly to provide data about ancestry. The organization took its time to scrutinize the technology, but when it figured that DTC genetic testing companies can be regulated in a way that protects patients but allows these services to be available, it gave it the green light – and even significantly changed its attitude. Three years later, in April 2017, the FDA re-approved 23andme’s services, and in November 2018, the first pharmacogenetic test of 23andme.

Not only DTC genetic testing companies could sense the support of the FDA lately. The agency supports various digital health trends starting from artificial intelligence through 3D printing up until digital pills with its guidelines, workshops, and regulations. The FDA approved the first deep learning algorithm for cardiac imaging built by Arterys in 2017. Already a year later, the agency cleared another 12 smart algorithms in healthcare.

The FDA has been actively supporting the adoption of 3D printing technology in the biomedical and dental industry. In 2017, the agency made a statement regarding its commitment to the technology and released guidelines for manufacturers submitting 3D printed implants and other medical devices. Numerous 3D printing companies reacted positively to the document, and there was a noticeable upsurge in the field. At the end of last year, the FDA also announced it is reviewing the regulatory issues related to the bioprinting of biological, cellular and tissue-based products to determine whether additional guidance is needed. During the year, the agency organized workshops and meetings to prepare the regulatory body as well as the players of the industry for the coming changes.

Shaping up national health regulatory bodies is the first step. What comes next?

The digitization of healthcare as well as other parts of society entails transnationalization and the disappearance of boundaries. Cross-border cooperation in healthcare through technologies could enable treatments outside the scope of the national healthcare systems.

Individuals realizing the existing therapeutic options in other countries travel outside their national healthcare systems to receive better or cheaper treatment – or care without restrictions. For example, many women go from Poland or Ireland to the UK for abortion as both countries have restrictive abortion laws. From Western European countries, numerous people travel to Eastern Europe for dental treatments as the quality of care is high, but prices are much lower than in France or the Benelux states. The EU estimates that the demand for cross-border healthcare represents only around 1 percent of public spending on healthcare, which is currently about €10 billion. Some Americans also migrate to Europe in the hope of receiving more accessible care – just as the editor of The New York Times ended up in France.

And of course, my favorite example: imagine that a patient living in France asks a Belgian company to sequence the tissue of his tumor. Afterward, another enterprise in precision diagnostics using the cloud computing services of Amazon (AWS) establishes that a Swiss pharma company has precisely that type of clinical trial on a Spanish island, which the patient could use – for free. In case the treatment proves to be effective, the person will be healed without the expertise of French doctors.

But that’s just the beginning. The American ECG sensor, AliveCor could tell me in the morning in Hungary that I have atrial fibrillation. An Australian company, MyDNA, could send me the same day my pharmacogenomic review and advised me on which medications I’m allergic to. While my Withings smartwatch produced by a French company could warn me about my low blood oxygen level in the evening. Hell, that would be a rough day! And an example of how multinational medical technology or pharma companies serve their customers globally and how our medical care will by-pass the traditional national healthcare systems – and insurance systems – more and more in the future. That’s what technological revolution, globalization and sharing economy brought to us.

Where are the global regulatory efforts?

It’s somewhat unambiguous that if you have a global phenomenon, you can only regulate it right with concerted efforts from all over the world.

The WHO has the Global Benchmarking Tool (GBT) for the evaluation of national regulatory systems, which aims for the convergence of various schemes in the long run. Besides, efforts are also underway to accelerate the harmonization and convergence of medical device regulations. Members of the International Medical Device Regulators Forum (IMDRF) meet regularly to produce harmonized principles that act as guidance for individual jurisdictions. However, this guidance extends only to the regulation of medical devices, leaving the categories of health-related apps or programs seen as non-devices, subject to the regulatory and enforcement discretion of individual national jurisdictions. Not to speak about various emerging medical treatment methods, such as gene editing and gene therapy.

The FDA already realized the need to “go global” more than a decade ago. The regulatory body expanded its domestic and international product quality and safety efforts, including global collaboration, global data-sharing, development and harmonization of standards, field operations, compliance, and enforcement activities. Naturally, though, these programs and initiatives operate along the lines of the interests of the United States and their citizens, and not as elements of a value-neutral global health institution.

Setting up a global health regulatory body

I think we should respond together to the challenges of globalization, digitalization and the transformation of healthcare into digital health. That necessitates the establishment of a global regulatory agency – a “global FDA” – which would support the players of individual countries as an independent body in solving global problems related to medicine and healthcare. No matter whether it’s about health technologies, gadgets, smartphone apps, genetic testing, clinical trials, medical research or ethical issues.

I recommend that

  1. the leaders of the major national regulatory agencies should set up a governing body filled with digital health experts,
  2. the FDA should show the direction in regulating digital health,
  3. the global regulatory body should take up the task of creating regulations for countries to adopt in their own healthcare systems (g., the FDA’s Pre-Cert Program),
  4. health policy researchers and bioethicists should become members of the regulatory agency to help raise questions and launch debates about issues long overdue (genomic and genetic research, gene editing, longevity research, bioprinting, artificial intelligence),
  5. and the regulatory body should communicate and conduct its actions clearly, concisely and openly so that it can educate the general public and prepare the companies developing innovative technologies for compliance.

The next step is for you to share what you think!