Welcome to the new Science|Business blog about “future-proofing medicines,” which will follow the EU’s new pharmaceutical strategy, related legislative revisions and the European Health Data Space, among others. Our blog is part of a major Science|Business initiative to inform Europe’s rethinking of its medicines regulatory framework, bringing together experts in a series of workshops to debate how to best develop and assess Europe’s next generation of medicines and health technologies.
The series will kick off in Copenhagen on 27 June with a high-level roundtable on how to harness the power of data. Follow along with us for the latest from Denmark and other countries throughout the next year.
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Analysis of real-world data could bridge the treatment efficacy gap between clinical trials and routine medical practice, as well as enable personalised medicine—but exactly which datasets should be analysed to produce reliable evidence? At the second national roundtable of the Future-Proofing Medicines initiative in Berlin, the panel heard that Germany’s new approval and reimbursement pathway for healthcare apps (DIGA) had expanded the nature of evidence to include “positive healthcare effects”, which can be improvements in patients’ health literacy, treatment adherence or even a reduction of the burden on caregivers. As some participants pointed out, this has created a need to define what types of data are required to measure these effects: hospital billing data, patient-reported outcomes or possibly entirely new datasets.
A task force, initiated by roundtable co-host EIT Health and made up of representatives from European regulatory bodies, including the German Federal Institute for Drugs and Medical Devices (BfArM), has recently set out to do just that in a bid to harmonise the evidence standards for digital health solutions in Europe. The quality indicators already used by hospitals would be a good starting point, according to one panellist, to drive rapid progress for the approval of health apps and the evaluation of new, integrated care concepts.
Closing the German edition of the Science|Business roundtable series with a wish list for the future, the panel called for investments in education on data-driven models of care for patients, doctors and the general public. They highlighted that France has allocated €80 million to support the introduction of a reimbursement framework for digital medical devices similar to the German DIGA pathway.
Just because you could theoretically kill someone with a pen, does that mean we all need a permit to carry one? Nick Schneider, head of division, new technologies and data use at the German Federal Ministry of Health (BMG), used this analogy during the second national roundtable of the Future-Proofing Medicines initiative to highlight a bias in the public debate about sharing and processing health data, which he said is currently dominated by fears surrounding misuse without sufficient consideration of its utility.
Schneider called on all users of health data to speak up and express their wishes clearly in order to bring about a change of mindset in Germany: “Only once we recognise that there is a broad array of meaningful purposes for which we need to allow the use of health data, can we begin to clearly define the risks associated with this and establish strict controls and punishment for its misuse following the example of the Scandinavian countries,” he said.
Another participant contended that the current focus on data privacy can surpass the expectations of patients themselves, failing in the process to address their needs and interests.
Germany’s 30-year-old regulatory framework for medicines is no longer fit to support therapeutic innovation in the modern era, according to participants of the second national roundtable of the Future-Proofing Medicines initiative, staged in Berlin by Science|Business in partnership with EIT Health Germany-Switzerland. Going forward, regulatory bodies will have to adapt to a pharmaceutical landscape that will not be made up of individual products, but increasingly see the emergence of platform solutions that shape the entire patient journey, panellists predicted.
With the European Union’s pharmaceutical legislation currently under review and changes set to be adopted by the end of this year, Karl Broich, president of the German Federal Institute for Drugs and Medical Devices (BfArM) and current chairman of the European Heads of Medicines Agencies (HMA), called for regulatory bodies across the continent to adopt an innovation mindset and make Europe more attractive and competitive in the field of clinical assessment compared to the USA.
The EMA-HMA Network Strategy 2025 includes, among other things, the integration of digital tools and the use of real-world evidence in regulatory decision-making processes. Accordingly, Broich said that, while no compromise could be made on standards for demonstrating drug safety, BfArM is now open to new methods for showing the efficacy and clinical benefits of medicines. The panel agreed that innovative clinical trial designs leveraging real-world data or non-traditional study endpoints, such as quality of life, merit consideration as a complement for traditional randomised controlled trials which remain the gold standard of clinical research.
The European Health Data Space (EHDS) can help deliver many of the objectives in the coalition agreement of the German federal government, from legislating on health registries and health data use to developing a research data infrastructure for health, said Nick Schneider, head of division, new technologies and data use at the German Federal Ministry of Health (BMG), during the second national roundtable of the Future-Proofing Medicines initiative in Berlin yesterday.
One participant pointed out that not using health data—to help patients, advance research, or guide health policymaking and regulation—would be unethical. Yet much of the data that already exists is not usable for these purposes in Germany. This is because secondary use of health data is not legally provided for.
Emphasising that the EU General Data Protection Regulation (GDPR) was originally intended to pursue a risk-based approach to balancing the protection of basic human rights and the free movement of data, Schneider said Germany needs to adapt both its legislation, and its oversight and implementation, to this spirit. He noted that national legislation on health data use, which will support the introduction of the EHDS in Germany, is currently under preparation.
The new legislation will need to be supported by changes in practices: Some panellists regretted that data being collected in various cancer registries and other repositories is not interoperable and often not even accessible.
Large national or even international data-sharing frameworks can take a long time to realise in the healthcare sector. An illustration of how local efforts may bring meaningful benefits to patients in the short term was provided at the second national roundtable of the Future-Proofing Medicines initiative with “Gesundes Kinzigtal”, a regional integrated care network encompassing 32,000 citizens with statutory health insurance in Germany. After launching a digital project to aggregate and analyse data from the medical and administrative systems within the network, Gesundes Kinzigtal now has access to near real time insights that allow it to steer and optimise care provision in a transparent way, according to its CEO and roundtable participant Christoph Löschmann.
Finding the right scale for the right applications is important, the panel agreed, as is developing intermediate structures that can serve as bridges between different data repositories, argued one panellist, who also highlighted the security advantages conferred by decentralised data storage.
Drug approval may be harmonised across Europe, but patients’ access to treatment and care remains highly variable. Discussants at the second national roundtable of the Future-Proofing Medicines initiative shone a light on some of the inequities in health that policymakers are called on to address, beginning with the time it takes for a drug newly licensed by the EMA to become available in individual EU countries: while minimal in Germany, delays of up to three years have been observed in other Member States. Progress on digitalisation in healthcare was also noted to be heterogeneous across the Union, with certain countries like France now following Germany’s example on the introduction of a fast-track regulatory and reimbursement pathway for digital medical devices, and others lagging further behind.
Even within Germany, the panel urged that the health system must get better at making improvements and innovation in healthcare available to all patients faster. One participant cited the example of heart CT scans, a less invasive and less expensive method for diagnosing coronary heart disease than the standard catheter-based angiogram, to which patients’ access is currently conditioned by where they live and whether they have private health insurance. Similarly, the panellist highlighted the missed opportunity to return more people to normal living, and potentially to the labour market, represented by less than 10% of cardiac patients in Germany attending a cardiac rehabilitation programme.
The rolling review process that led to the approval and distribution of COVID-19 vaccines in record time could become an important instrument to accelerate the market entry of breakthrough therapeutical innovations in the future. This was suggested by Karl Broich, president of the German Federal Institute for Drugs and Medical Devices (BfArM), at the opening session of the second national roundtable of the Future-Proofing Medicines initiative taking place in Berlin today. In particular, BfArM expects to conduct clinical assessments on an ongoing basis more frequently for the evaluation of novel anticancer medicines, which it expects to account for 70% of the agency’s approvals in the coming years.
Speed should not come at the cost of safety, however, and roundtable moderator Carlos Härtel, chair of the board at Science|Business, emphasised that Facebook founder Mark Zuckerberg’s famous motto, “move fast and break things”, could never work in the health sector. Panellists agreed that the criticisms and fears expressed during the pandemic about using entire populations as guinea pigs highlighted the importance of building and nurturing public trust in the system. This must involve robust pharmaceutical surveillance, which in the case of the COVID vaccines allowed the identification of rare, but serious side-effects in certain population subgroups.
Yet this effort could and, according to participants, should begin much earlier on, by incorporating patients and the outcomes that matter to them, beyond traditional survival endpoints, in medicine development and approval processes. In the field of oncology, for example, certain new therapies add only a few months on average to aggregate survival gains. Therefore, it is important to consider the individual perspective of the patients who do respond to a medicine when assessing its value, according to one participant. Broich said that BfArM will continue to integrate patient representatives more consistently into its decision-making processes in the future.
In recent years, digital technologies have moved from the margins to the mainstream of healthcare at a rapid pace – a trend which has been turbo-charged by the COVID-19 crisis. In parallel, the health data landscape on which digital solutions depend is itself being transformed, with the emergence of real-world evidence, big data analytics, and – perhaps most importantly – a European Common Health Data Space (EHDS) designed to pool vast amounts of information to support the next generation of health and life science research and innovation.
From wearable devices and online diagnostic tools, to AI-based analytics and the use of advanced computing to identify new drug molecules and process genomic data, national health systems are scrambling to adjust to the new reality. With a flourishing digital health sector, Germany is well positioned to be in the European vanguard of this transformation – but will its regulatory system for new drugs and devices allow it to do so? Can it keep pace with latest technological developments while harmonising its health data systems? And what should policy makers prioritise to enable progress?
This afternoon, at 15:00 CEST, join the public debate that Science|Business is staging in Berlin, with the support of EIT Health, as part of its “Future-Proofing Medicines” initiative. policy makers and digital health entrepreneurs as they chart their way forward. Along with leading voices from industry and academia, Nick Schneider – head of new technologies and data use at the German Federal Ministry of Health – will share his views on the evolution of the field, and whether the EHDS will truly be a game-changer for the new age of digital health.
Follow the discussions live from Berlin, at 15:00 CEST, via livestream on the Science|Business website.
Drug approval may be harmonised across Europe, but patients’ access to treatment and care remains highly variable. The second national roundtable of the Future-Proofing Medicines initiative shone a light on some of the inequities in health that policymakers need to address. These include the time it takes for a drug newly-licensed by the European Medicines Agency to become available in individual EU countries: while minimal in Germany, delays of up to three years have been observed in some Member States.
Progress on digitalisation in healthcare also varies greatly across the Union. Some countries, such as France, are now following Germany’s example on the introduction of a fast-track regulatory and reimbursement pathway for digital medical devices, but other EU countries are lagging some way behind.
Even within Germany, the health system must get better at making improvements and innovation in healthcare available to all patients faster, according to the roundtable participants. One speaker cited the example of uneven access to heart CT scans (a less invasive and less expensive method for diagnosing coronary heart disease than the standard catheter-based angiogram), which depends on where patients live and whether they have private health insurance. Similarly, the panellist noted that less than 10% of cardiac patients in Germany attend a cardiac rehabilitation programme, representing a missed opportunity to return many of these people to normal living, and potentially to the labour market.
One of the key challenges for the leaders of every national health system is to anticipate the longer-term needs of patients and citizens, and the short-term investments required to prepare for a more sustainable and affordable future. An integral part of this, however, is understanding the pace of progress being made today in medical science and laboratory-based innovation, and what potential this holds to tackle infectious and non-communicable diseases alike.
The global response to the COVID crisis demonstrated that – when needs must – health systems can move with speed and agility to tackle urgent health concerns. But what lessons should be taken from the COVID process for developing vaccines, ventilators and other solutions? How can the experience inspire a new set of reflections among regulators, policy makers and other stakeholders on future frameworks that allow breakthrough science and innovation to flourish? Do we need a new system of risk-based approaches to develop and approve modern therapeutics? And what role for patients themselves in mapping out the way forward?
On September 29 in Berlin, Prof. Karl Broich – president of the German Federal Institute for Drugs and Medical Devices (BfArM), and one of the most influential figures in European medicines regulation – will headline the next major event in the Science|Business “Future-Proofing Medicines” initiative, supported by EIT Health, to address these questions and more. Follow the discussions live from Berlin, at 14:15 CEST on September 29, via livestream on the Science|Business website.