Precision Medicine: The Long Road from Inception to Care

Philipp Grätzel von Grätz|2019-12-19

The age of precision medicine holds the promise of accurate diagnoses, personalized therapies, and fewer treatment errors. While these goals are achievable, obstacles still remain to be cleared in many places.

“The goal of precision medicine is to account as accurately as possible for individual characteristics in prevention, diagnosis, and treatment,” said Prof. Jochen Werner, MD, Medical Director and Chairman of the University Hospital Essen, in remarks delivered at the Hauptstadtkongress event, Germany’s premier conference for medical practitioners, in Berlin at the end of May. The point was not, as he emphasized, to disparage the current state of caregiving. Rather, he argued that advances in the field of molecular biology, the digitization of diagnostics, and increased capabilities for data analysis through artificial intelligence-based algorithms opened up completely new caregiving concepts that had previously not been feasible.

“Precision medicine affects diagnostics and therapy alike,” says Cornelia Baltes, Head of Marketing, Sales Operations & Communications Central Western Europe at Siemens Healthineers. Both fields reinforce one another: In the first step, the aim is often to improve diagnostic accuracy and to reduce unwarranted variation. However, the focus is subsequently on customizing, to the highest possible degree, the treatment of more precisely diagnosed and more clearly defined patient groups. The overarching aim, of course, is to improve therapeutic outcomes for as many patients as possible.

Prof. Christof von Kalle, MD, Member of the Advisory Council on the Assessment of Developments in the Health Care Sector and BIH Chair for Clinical Translational Sciences and Chief Research Officer of Sidra Medicine in Doha, Qatar, believes that medical science still lags far behind other professions in terms of precision and safety: “In areas such as transportation or workplace safety, there is a social consensus that accidental deaths are simply not acceptable, and accordingly, everything is done to prevent them. We’re not at that point yet in the healthcare sector.” He believes this has to change, and change can’t be achieved with cheap talk, but only through very concrete measures in very practical care scenarios.

Von Kalle represents the discipline of oncology. Cancer centers such as the one in Heidelberg have made great advances in precision medicine in recent years, bringing about a convergence of molecular methods and information technology. The molecular tumor boards established in Heidelberg regularly feature not only pathologists and oncologists, but also bioinformaticians. And more and more patients are benefiting from treatment concepts that are tailored to the individual genome of a tumor.

One reason why precision medicine, having established itself at least partially in oncology, has still failed to be implemented more broadly in other areas of healthcare is that the necessary data is not easily accessible, von Kalle notes: “While digitalization now permeates all areas of medical science, we still have trouble condensing, analyzing, and retrieving data, not least for technical reasons.“

In addition to technical shortcomings, notably relating to interoperability, the oncologist believes that precision medicine is being held back by privacy regulations that fail to adopt a patient-centric perspective: “For too long, we have convinced ourselves that our failure to process data constitutes data protection. But patients with life-threatening illnesses have the right to expect that their data should be properly evaluated. That, too, is data protection.” Von Kalle is a member of the Council of Experts, Germany’s most important advisory body for healthcare policy. In 2020, he says, this body will publish a report on digitalization that will also argue in favor of improved access to medical data in Germany.

There is no doubt that patients and holders of health insurance are in favor of precision medicine, says Hans Unterhuber, PhD, CEO of the Siemens Health Insurance Fund: “We already employ precision medicine in a number of areas. We have received very positive feedback from the patients who benefited from such procedures. After all, the question of whether chemotherapy is necessary or even effective in a patient is a highly relevant one for their quality of life. We therefore have high hopes that precision medicine will help us achieve significant progress, especially from the patient’s perspective.”

As Unterhuber sees it, interoperability and data availability are the two main points that need to be resolved if precision medicine is to be introduced across the board in healthcare. But evidence assessment, which also relates to the issue of compensation, needs to be addressed, too, he believes: “We have allowed an incredible amount of bureaucracy to build up in recent years. We need to come up with more efficient and straightforward procedures.” As far as the use of precision medicine is concerned, Unterhuber supports new performance-based compensation models, such as the ones now being introduced in connection with CAR-T cell therapy in Germany, which are also being considered in other countries: “The idea of value-based payment models may sound brutal at first, but such models are a good way of determining whether a manufacturer is genuinely convinced that their approach works.”

Prof. Christoph Herborn, MD, Medical Director and Member of the Executive Board at Asklepios Kliniken GmbH und Co. KG, thinks there is a somewhat exaggerated hype surrounding precision medicine. In his view, the role of precision medicine is currently essentially limited to the fields of laboratory diagnostics and pathology: “We should take care not to overuse the term. At the same time, we should not abandon our goal of giving personalized medical care tailored to genotype and phenotype where there is a benefit to the patients.”

Prof. Michael Albrecht, MD, is the Medical Director of Dresden University Hospital, home to one of Germany’s proton therapy centers. He, too, warns that the complexity of precision medicine should not be underestimated. Most recently, he notes, his center together with Siemens Healthineers developed methods that further improved the already high degree of therapeutic precision, ensuring that even less healthy tissue is affected by irradiation. Contrary to popular belief, precision oncology does not consist simply of a blood test, but is based on an enormous amount of data generated at various times. Moreover, Albrecht explains, the individual tumor pattern that this data reflects is not constant, but changes over the course of the disease.

Understanding this dynamic and drawing the right conclusions cannot be achieved overnight, Albrecht says: “Precision medicine will only advance if we collect large amounts of data from many patients in as many locations as possible, recognize correlations, and review the resulting therapies in clinical trials. This can only be done in a joint effort. We should use Germany’s National Decade against Cancer to join forces instead of instigating a rat race between various institutions.”

Gunther K. Weiß, MD, Board Member of RHÖN-KLINIKUM AG, emphasizes that precision medicine and evidence-based medicine are not mutually exclusive and should not be played off against each other. He argues that certain approaches in precision medicine, including new highly stratified study designs, should be seen as a further development of evidence-based medicine.

As an example of a concept for successful, evidence-based precision medicine, Weiß cited the ion therapy center in Marburg (Marburger Ionenstrahl-Therapiezentrum MIT), which was launched with support from Siemens Healthineers, and in which the latter is a service partner. The MIT is one of only two centers in Germany that offers both proton therapy and heavy ion therapy. These treatments, Weiß points out, undergo rigorous evaluation in clinical trials, where they must hold their own against competing approaches such as intensity-modulated radiotherapy, before being introduced into standard healthcare practice via the Federal Joint Committee.

Clinical evaluation of a precision medical radiation approach is a tiresome process, but a very rewarding one for the patient groups who benefit from its very real added value. Conventional radiotherapy and particle therapy are also increasingly being combined into a boost therapy, so that the number of patients benefiting from particle therapy is likely to increase. “All in all, our patients are very happy that we offer this therapy. But they also want their case to be discussed in depth beforehand,” says Weiß. Accordingly, only those patients are referred to MIT whose course of disease was discussed at an interdisciplinary particle tumor conference and for whom particle therapy is the optimal treatment strategy.

Philipp Grätzel von Grätz is a freelance medical and healthcare journalist based in Berlin. His special areas of expertise are digitalization, technology, and cardiovascular therapy.