More Precision
for Patients

If this ideal is to be achieved, however, a new way of thinking about healthcare is needed. Bergen speaks about having to develop a “holistic take on health.” This approach, which focuses on the patient and all of their data, sounds promising. The hope is to gain a precise picture of the individual effects of any given medical intervention, preferably before trying it out. “This could be done by means of a digital copy of that person, which reflects their biology, genetic material, and other specific features,” she says. She would therefore support the idea of creating “digital twins,” an aim also defined as visionary by Roman Hipp, the life sciences expert. According to Bergen, digital twins would be of major benefit to both patients and physicians. “We could check the effects of a treatment before actually applying it. Afterward, we could also determine how it worked.”

The approach seems revolutionary. And Bergen insists that appearances here do not deceive. “Personalized medicine will fundamentally change the entire healthcare sector,” she predicts. The healthcare industry could conceivably develop into a “hyperlocalized economy” to provide patients with medical treatment tailored to their individual needs. Medications would then be produced directly at the point of care, similar to how CAR T-cell therapy is conducted in part today, or to take an entirely different example, to how providers such as Amazon’s Pillpack operate.

These developments are not a foregone conclusion, however. Healthcare systems need new and different incentives to put visions such as these into practice, explains Bergen. Moreover, further investment in innovations is needed to make personalized and individualized medicine possible in the first place. “Money alone is not enough, because the innovations are based in large part on data,” she says. “In order for us to know exactly what will help whom, the data have to be available in digitalized and standardized form.”

Bergen therefore views current public debate on this topic as an obstacle to achieving more precision-based forms of medicine. “Media reports are often marked by skepticism as soon the question of health data comes up. The emphasis here shouldn’t be on fueling fear but rather on how sharing data can benefit patients.” The expert, who also runs a podcast on healthcare, urges open discussions and a new look at the topic. “When patients realize they’re getting something from sharing their information, they’re willing to do so. We need a positive, future-oriented narrative in our society in order to clear the way for innovations in Germany.”

Boidol therefore prefers to talk about “precision medicine.” For him that would mean the following approach: “The first step is to use the latest technology to gain an extremely precise understanding of the individual condition, and the second is to find and offer a very precise treatment for exactly that condition.” His strong emphasis on understanding the condition is based on very sound reasoning. “The molecular causes of an illness are often underestimated—but we can only develop and successfully apply a precision treatment if we have thoroughly understood the condition.”

Although Boidol may define the matter in somewhat tighter terms, he and many other experts agree on one thing: these new methods have already led to enormous advances in oncology in particular. Chronic myeloid leukemia is one example. A specific genetic mutation is often a causal factor in this type of leukemia. If clinical tests detect the mutation, a new type of medication can be given that selectively binds the genetic variation and suppresses its function—with only minimal side effects. As Boidol notes, “What was a death sentence twenty years ago is now a chronic disease that can be treated for ten or fifteen years.”

Another point on which the Proxygen CEO agrees with most experts is that rapid progress in precision medicine can only be made if more data from patients become available. “The data are there,” he says. “The problem lies in the strict and therefore limiting data protection regulations.” And in fact one might ask why hospitals cannot sell their data at least in anonymized form. After all, the data would be used to develop medications that could help gravely ill individuals and thereby reduce suffering. “I think that should be possible,” he says. The key thereby is to ensure that “the focus is on what benefits patients.”

Boidol does not currently see an acute need for additional measures specifically designed to boost these new methods to a breakthrough. As he explains, “Many things that would advance the pharmaceutical industry in general would also be helpful from a precision medical perspective.” In his view, progress would already be served by leaving the pricing of certain medications such as antibiotics to companies and refraining from imposing price caps. “The rapid and successful development of the Covid vaccines is a good example of the level of medical and competitive benefits that policy can have.”

Promising innovations in the field of personalized medicine are expected to make this possible. “We have to get away from the ‘one pill fits all’ approach and adopt treatments tailored as closely as possible to individual diagnoses and needs,” she says. “We have to move beyond the repair approach to sick people—which is more of an illness than a health system—and establish a healthcare system worthy of the name.”

The good news is that the first steps have already been taken and we’ve entered the new world of precision medicine. Citing current information from the German Association of Research-Based Pharmaceutical Companies (vfa), Brakmann notes that 97 medications authorized in Germany meet the criteria of targeted treatment, most of them in oncology. Common to all is a consideration of individual genetic characteristics. In other words, before one of these medications is administered, specialists test the patient’s genetic material. The biochemical and genetic preliminary assessments show which preparations will work well and which will probably not. “This is a way to give the right substances to the right patients at the right times,” she says.

There’s a good reason for why this approach is being used especially in oncology. “Cancer is a disease of the genes,” says Dorothee Brakmann. It therefore makes sense to look for relevant genetic features and examine them before prescribing treatment. Janssen is working in this area and has developed numerous active agents for various blood, lung, prostate, and bladder cancers. Precision medicine is not restricted to treating cancer, however. As Brakmann explains, “Oncology has made a start, and other areas are following.”

Nearly all the fields of research at Janssen take a personalized approach—“with all the attendant problems,” observes Brakmann. One challenge has to do with the considerably smaller patient cohorts. Participants are selected on the basis of genetic features needed for clinical studies focusing on ever more specific subgroups of a particular disease. Another challenge concerns the high costs required to develop medications for only a few patients, albeit with targeted effects. “Healthcare systems do not command endless resources,” says Brakmann. “We, too, are well aware of this.”

The pharmaceutical expert therefore draws a “fine line” between three determining factors in a health system: patient access to medication, affordability, and incentives for research companies to pursue innovations. “We need to maintain a good level of dialogue among everyone involved,” she says, while noting that’s easier said than done. “When you focus your efforts on one factor in this magic triangle, you always affect the other two as well.”

While Dorothee Brakmann attests to the good work done by regulatory authorities, she sees room for improvement in the reimbursement system—including in Germany. “There are far too many old-school models in operation here,” she says with reference to the extensive randomized clinical studies that funders prefer. “They’re the gold standard of course,” she concedes. “But if you’re developing medications for very small groups of patients, it’s very difficult to set up that type of comprehensive study.” To enable precision medicine to take a quantum leap forward, adjustments are needed to the system itself—over and above the release of anonymized health data that Brakmann and many other experts are calling for.

If we devote our efforts to the research part of the healthcare system’s magic triangle, a clear road stretches on into the future—namely a future in which we stop diseases before they have a chance to set in. At Janssen this approach is called “disease interception.” It functions similarly to that for personalized cancer treatments: by identifying biomarkers such as specific gene mutations that make certain diseases more likely. “If you detect this type of predictor, there’s an especially high likelihood that the disease will be found in that individual even before any symptoms appear. You can therefore take countermeasures at an early point in time to prevent it from breaking out.”

The more biomarkers can be detected with the help of AI-supported analyses and the more anonymized patient data are made available, the easier it will be for companies to develop better treatments. Brakmann is convinced we won’t have to wait too much longer. “The first treatments could come onto the market in four or five years,” she says. Which sounds like science fiction might in fact become reality.

Trained as a radiologist, he became CEO of Bergman Clinics in Germany in August 2022 but gave the interview for this article while still the medical director and one of five managing directors for the Asklepios Group. The diagnostic processes he refers to are part of treatment strategies based on molecular genetic testing for tumors. And they are clearly leading to successful results at Asklepios. “By combining conventional chemotherapy with new antibody therapies we’re able to arrest tumor growth or even reduce the size of primary tumors,” reports Herborn.

Although many patients are still unfamiliar with this type of treatment, they only rarely have reservations about it, says Herborn. “The vast majority are open to the new methods.” Their confidence is reinforced by the fact that Asklepios does not assign them to one department alone. “Cancer medicine at our clinics is an interdisciplinary affair.” The heart of the multidisciplinary team is the “tumor board,” consisting of experts from oncology, radiology, pathology, radiotherapy, and surgery. “They confer to make the best treatment decisions for each individual case.”

When the expert committee selects an individualized treatment, the first question for the former Asklepios managing director has to do with funding. “Personalized treatments are often very costly and generally not included in standard medical coverage,” he notes. He therefore either had to either negotiate with the insurance companies or pursue the treatment as part of a study. “In such cases the pharmaceutical industry assumes the costs.”

The professor’s emphasis on funding might raise some doubts. Does that mean only patients who are privately insured will receive these effective but expensive treatments? Might the rise of precision medicine even lead to two divergent classes of medical care? Herborn, however, sees no reason to fear that the innovative treatments will acquire an elitist character. “Everything that’s available and can be offered as part of good medical practice is in fact on offer. Nothing needs to be democratized or made any more accessible than it already is,” he declares.

With the new forms of treatment already offered at clinics on a regular basis, 
Herborn is looking further into the future. “Over the next ten or fifteen years I expect the new personalized forms of treatment to be applied not only in oncology but also to other diseases such as cardiological and circulatory conditions.” Their treatment currently focuses on risk factors from unhealthy lifestyles that lead to high blood sugar or lipid levels. “A good way to supplement this approach would be to determine the ideal preventive measures on the basis of cardiovascular profiles.” 

“Generally speaking, however, we’re now developing treatments that are considerably more targeted and precise than has ever been possible before.” These recent developments are what ultimately make the difference for the Harvard professor. “Over the short or long term, we can assume that personalized medicine will become the standard treatment.”

That being said, personalization will not proceed at the same pace for all diseases. Stern expects the near future to bring advances for cancer in particular, but also more targeted approaches for ophthalmological and hematological disorders. “Patients will enjoy substantial benefits in these settings where their medications will be increasingly tailored to their respective genetic makeup or the mutations from their specific type of cancer.”

There are a number of hurdles to clear, however, before many personalized treatments can achieve breakthroughs and become widely available. Ariel Dora Stern speaks of an “institutional mismatch” between FDA and EMA regulatory framework conditions on the one hand and research requirements on the other. She advocates for what she calls “regulatory innovation,” noting that “Our current medical product regulations are a product of the previous century. They simply no longer reflect the needs of the next generation of products or cutting edge practices in therapeutic research and development.” A good example is the need to set up platform studies instead of traditional clinical trials. “Platform studies focus on the overall clinical picture,” she explains. “They let us divide groups of patients into different arms of the study based on biomarkers and to test different medications at the same time. Because established regulatory approaches were not designed with such studies in mind, getting them off the ground has been a considerable challenge—despite the fact that they offer clear advantages for both patients and clinical investigators.”

Ariel Dora Stern doesn’t try to downplay the considerable time and funding required to set up a platform study. But she also says the advantages far outweigh the disadvantages. “This type of trail isn’t over after you’ve conducted the first series of tests—the platform structure means that meaningful research can continue even after specific clinical questions have been answered. This means you can keep researching that disease in a sustainable way.” In addition, different statistical models are applied that facilitate dynamic analyses and thereby yield faster and more efficient results in a statistically rigorous manner.

Regulations are not the only factors holding up these studies. The organizations that conduct platform studies are designed as open systems. In other words, they aren’t just a single enterprise but instead can consist of multiple participants. “This type of setup is complicated, of course, when you have competitors working together,” observes Stern, who also currently holds a guest professorship at the Digital Health Center of the Hasso Plattner Institute in Potsdam. Her solution: “Starting a non-profit foundation or identifying another trusted, independent third party to coordinate and run the study has proved to be a good way of building trust. We have seen this, for example, in the work of the Global Coalition for Adaptive Research (GCAR) in the United States.” Once a platform study is established, its infrastructure makes the entry requirements lower than those for launching a traditional clinical trial. “This allows smaller biotech and pharmaceutical companies study diseases and populations that they may not otherwise have investigated, and also creates incentives to develop treatments for rarer diseases.”

Whether and when people can benefit from personalized medicine will depend on more than the research itself. Stern notes that although the new treatments are often very effective, they can also be very expensive. “Each country’s healthcare system will therefore play a key role in providing access to these treatments.” Current approaches to funding the corresponding medications in the USA can look baffling to German eyes. “Payment models there range from ‘drug mortgages’ (where expensive treatments are paid off over time) to having the amount of reimbursement depend on the success of the treatment.” Europe will certainly be taking a different approach here, although innovative payment models are already being piloted.

For Hadjiat, these digital biomarkers provide revolutionary data when it comes to tracking the progression of multiple sclerosis and other neuromuscular diseases. With Cleo and Konectom, he has already launched solutions that make patients’ lives easier using precisely these technologies. Cleo, for example, provides multiple sclerosis patients with daily support, information, tips, symptom tracking, reminders, and other customized programs. The download data shows how highly patients value this app: as of 2022, Cleo has been downloaded over 600,000 times worldwide. The Konectom platform, in contrast, focuses on the improvement of neurological disease measurement. It uses smartphones and wearable sensors to enable a much higher resolution in the way we track, monitor, and measure neurological diseases.
Konectom takes into account the patient’s key neurological functions, from cognition to fine and gross motor skills.

The goal of Hadjiat and his team at BDH: to use technology to address patient needs in a goal-oriented manner and drive the change toward a more patient-centered and better care. Digital biomarkers play a major role in this endeavor: they not only allow people to track and better manage their own disease but also enable their neurologists to monitor progress more accurately and with higher resolution from afar, all thanks to technology.

Central to such solutions is smartphone sensor technology, which helps to continuously gather relevant health data. If critical patterns emerge, neurologists are alerted and can help their patients at an early stage if necessary. According to Hadjiat, the potential of the new technology thus arises from observation of conditions that progress slowly and the possibilities of this technology to predict clinical conditions before they occur.

Yacine Hadjiat is certain that digital health applications will soon be part of the medical standard. But simply transferring solutions from the offline to the online world is not enough, he says. After all, he notes, digital health and technology offer a much greater promise: “Through digital health, for the first time, we have the possibility of translating new technical capabilities into tangible health solutions—that’s something completely new.” Hadjiat notes that he will have reached his goal when the digital health hype subsides, and digital solutions and applications have become an integral component for medical standards of care. “Or, to put it another way, when digital health is the status quo.”

Personalized medicine is a passion for Baars. In her view, it’s neither a niche nor an add-on to established methods of treatment, but rather the essence of medical care. It already helps many patients today, and can offer more effective and less arduous forms of treatment to those diagnosed with cancer and rare conditions. What may sound like a breakthrough to many people is just the beginning for Baars. “We’re talking about a revolution here,” she says. “The tremendous increase in computational capacities and global connectivity is what will really unleash the full power of personalized medicine.”

Predictive bioinformatics and machine-learning models make it possible to gather millions of data points from the real world. These can be patient data, clinical data, laboratory tests, genomics or imaging. Compiling these data is just the first step. “Data alone can’t save a single life,” notes Baars. True medical progress, she adds, lies in analyzing and learning from these comprehensive data points, detecting patterns, and making actionable insights accessible for physicians and patients independent of their geographic location. No two cancer patients are the same. So, no two treatments should be either.

The aim is not only to provide more targeted treatments, but to redesign the healthcare system to make it more intelligent. “Together with Varian, the leading cancer-care company that we acquired last year, we’re leveraging logic and artificial intelligence to build a comprehensive oncology ecosystem that transforms workflows.” For example, the path from diagnosis to treatments can be accelerated from weeks to hours. For patients, hospital providers, and society this makes a huge difference. In Baars’s view, we need to move beyond the current system of “sick care” and finally attain actual “health care.” As she puts it: “We have the technologies and the expertise, but if patients don’t have access to solutions, then it’s missing its purpose. The data we gather in the course of personalized medical care will enable the shift towards early detection and prevent people from developing these conditions in the first place.”

Personalized medicine is not merely a matter of refining a few parts of the old system. The vision inspiring Baars is one of transformation—organizing lifesaving information and making it accessible for everyone to give more patients access to personalized treatments, when and where they need them.

According to Baars, this new approach will reduce illness and suffering—while also saving a great deal of money. For example, many cases of cancer today are not diagnosed until they have reached the most advanced stage or have already spread. Suitable treatments at this point are not only difficult but also likely to need multiple different interventions, increasing side effects for patients. This means longer hospital stays for patients and also higher expenses for healthcare systems. “If we can diagnose cancer at an early stage, we can really cure patients and it doesn’t cost as much,” she explains.

In order to reach patients early on, patient-supporting tools such as digital twins and multi-disciplinary oncology platforms will play an ever greater role. A patient digital twin integrates clinical data from disparate sources—and turns them into a virtual representation of the patient that can be used to free up physicians’ time and run simulations to reveal future health scenarios. When algorithms detect critical patterns, the corresponding tools will not only alert people but also tell them where in the healthcare system to go for a check-up. “Combining a digital twin, AI technology, and personalized care enables us to achieve the best possible outcomes for patients,” says Baars. This is based on the pathological and molecular specifics of their tumors, imaging characteristics, treatment history, and evidence-based treatment options in discussions with physicians and providers. “The challenge here is to redesign our systems around patients,” she says.

Here is where Baars’s arguments come full circle. “I envision a world with globally connected personalized health pathways designed to drive efficiencies and deliver world-class treatment along the cancer continuum to increase impact where it matters most: helping patients,” she says. The huge volumes of data needed to help patients with tailored treatments can only be gathered by holistically connecting patients, physicians, governments, and industry in a true team effort through digital solutions on an international scale, she believes. In short: the better we collaborate and the greater number of people who benefit from precision treatments, the more things will improve for everyone. This, then, is also a powerful impetus for countries with advanced healthcare systems to make precision medicine accessible to everyone on the planet.

One of the key takeaways from the interviews with experts is that medical information and digitalization will both play considerably greater roles in the context of personalized medicine. “We recommend that pharmaceutical and medical technology companies expand their data strategies and invest in relevant new products and services,” says Hipp. If this new market is to offer opportunities for development, transmission of at least (semi-) anonymized data will have to be streamlined. “It’s crucial to have secure data management that also enables innovative breakthroughs,” says Hipp. The aim here has to be a “new balance” that fosters research and innovation to promote human well-being while also providing the highest degree of data privacy. As he notes, “This might not be a convincing approach for absolutely everyone, but we shouldn’t overlook the fact that some innovative health start-ups have already left Germany to pursue research and healthcare innovations in other parts of the world.”

The new methods will bring fundamental changes to the pharmaceutical industry. Medications are currently produced in large numbers for broad groups of patients. By contrast, precision medical approaches require the manufacture of individualized medications that address diagnostic details or even integrate patients’ cell material into the production processes. “These individualized means of production, which in some cases will surely be decentralized, will place new demands on logistics and technical systems that companies will have to meet,” says Hipp. In his view, strategies from the automotive industry—such as the “smart factory” principle—can serve as guides for pharmaceutical companies.

It is by no means clear which companies will be able to take the lead in the new field of personalized medicine. Patients will be the winners in any case, according to Roman Hipp. “They can expect faster and more precise diagnostics and more promising treatments, including the prospect of actual cures.”