Where do the perspectives of patient advocacy and the pharmaceutical industry meet at a common point? Exactly at the point where patient participation, orientation and benefit are important. In the double interview by “Monitor Health Services Research” Dr. Martin Danner, Federal Managing Director of BAG Selbsthilfe, and Dr. Holger Bartz, Managing Director of Medical & Scientific Affairs at Janssen Germany, entered the discourse. “One could assume that the income situation of pharmaceutical companies is not an issue for patients,” explains Danner, for example, and adds: “But it is, because changes to the regulations through the GKV Financial Stabilization Act inevitably change the companies’ strategies become."

>> Mr. Dr. Danner, you have been with BAG Selbsthilfe since 2001 and have been working as Federal Managing Director since 2008. This gives you an overview of a good two decades of health policy with several dozen reforms and reforms. If we leave aside the pure scientific and medical progress - this point would deserve a separate interview, even a white paper -: Is today's healthcare system more patient-oriented than that of 2010 or 2000? In your opinion, what has changed in terms of patient orientation – for the better or for the worse?
Danner: Let's start with the positive. If you compare the state of patient orientation today with the situation 20 years ago, a lot has improved. Patient-relevant endpoints have become more important, on the one hand in the scientific discourse, but on the other hand in today's benefit assessment. I am primarily thinking about the methodological approach with which health technologies are evaluated. It was a very important step for us as patient representatives to give the issue of patient benefit a completely different priority than was the case 20 or even 10 years ago. In addition, the patient perspective is included much more in many healthcare committees than before - whether this happens through a single person or in the form of representative patient representatives. These two achievements, which we fought for as patient representatives, represent a significant change in favor of patient orientation in society.
healthcare. Of course there is also the other side of the coin, because the economization of the entire healthcare system is constantly progressing. The ever-increasing economic pressure means that the actors at the federal and state level are inclined - to put it carefully - to give their own interests a higher priority than the well-being of patients. This aspect runs counter to patient orientation and certainly concerns us patient representatives.

You are certainly right in saying that patients are being heard more than before. But listening and having a say does not mean having a say in Germany. Would it be a demand that you would support that patient representatives should not only have a say, but also a right to co-determination, if the patients were to be provided with appropriate monetary resources?
Danner: If you look at the legislation of the last few years, patient participation has already been implemented in many areas. But so far there is nothing more than having a say and sitting at the table, which has certainly changed the culture of debate. In order to be able to really exercise a right of co-determination and also to make applications, patients lack the structural requirements to be able to incorporate the knowledge gained from the patient's perspective into the care process through evidence-based decision-making processes. If the necessary structural requirements in terms of budgets and know-how are not strengthened, voting rights cannot be exercised effectively. However, I have more and more the impression that this is not wanted at all. A current example of this is the way politicians deal with independent patient advice. What is currently happening counteracts the idea of the UPD.

That would certainly be worth further discussion, because it wouldn't be wrong in itself if the UPD was perpetuated by a foundation under civil law. But as always, the devil is in the details. Let's stick to patient orientation: Dr. Bartz, you are a specialist in medical microbiology and infection epidemiology and have been managing director of Medical & Scientific Affairs at Janssen Germany since 2022. Before that, you were Country Medical Director of Janssen Austria. What distinguishes patient orientation in Germany from patient orientation in Austria? What does patient-centered care mean to you – on the one hand as a doctor and – previously – as a practicing doctor for many years and now as the medical director of a large pharmaceutical company?
Bartz: Having been socialized in the German healthcare system, I worked in Austria for a while and learned that it is currently much easier for patients in our neighboring country to get innovative therapies than it is here. Although historically the differences between the two health systems are not that great, very different assessments are made in different places in Austria.

Here you are talking about the benefit assessment, which in our case is regulated by the AMNOG, while our neighbors have recommendations on reimbursement to the Main Association of Austrian Social Insurance Institutions, which is drawing up a drug evaluation commission.
Bartz: Exactly. There are even more assessment levels in the Austrian system than in ours. But here as there, the pharmaceutical industry, for which I have been working for a year, has the self-defined goal of working and researching in such a way that every patient can receive the best possible therapy for each individual case at the right time. This is exactly where a big difference between the two systems becomes apparent: While in this country the active stakeholders decide - based on evidence as much as possible - on a macro level what the healthcare system should look like, in Austria really good networks have been formed. These strive to think about care for and with the patient population. This also includes creating good access to innovative therapies that are introduced through clever mechanisms in such a way that the system as such remains intact and capable of action. There may often only be gradual differences at certain points, but in the end they have a significant impact on the quality of care. Since in Austria we think from the perspective of the individual patient, there is also a closer relationship - and therefore trust - between practitioners and patients, even in comprehensive care institutions.

Danner: I am occasionally invited to Austria to talk about patient participation, as this is not yet as widespread in the Austrian committees as it is here. I can therefore absolutely confirm that our manifested camp mentality and conflictual struggle over what care can and should provide and what not is much less pronounced in Austria. In our neighboring country, I finally experienced very open health insurance representatives who, together with other stakeholders, tried to build patient-oriented care structures, although monetary considerations were not directly in the foreground.

In preparation for our conversation, I spoke to an acquaintance who has been a chronic patient for decades. She says: “When I go to the doctor, which unfortunately I have to do very often a year, I want to be treated as well as possible.” Even though she has studied, she cannot imagine anything under the term patient orientation. Are we thinking too much from a system and stakeholder perspective without asking patients what they want from the healthcare system?
Danner: It is important that you can go to your doctor without having the feeling that therapy decisions are possibly being controlled by any monetary considerations, by payment methods or contracts with health insurance companies. This is the only way trust can be created. Beyond this, there is another level of knowledge that varies greatly between patients and doctors, although more and more patients are getting information before and after visiting the doctor. In addition, treatment decisions should be made on an evidence-based basis, although this is not always the case in reality. I have the impression that the medical profession often no longer understands which therapeutic options are available for a statutory health insurance patient and how care situations should be optimally structured. The lack of transparency that emerges here almost inevitably leads to errors, which no one monitors.

Isn't it the case that, despite the undisputed successes of evidence-based medicine (EBM), the strict EBM-based approach has distanced patients somewhat from their treating doctors?
Bartz: EBM has achieved what it should: a much higher level of evidence. Nobody denies that, because this movement is essential for medicine and indispensable for the quality of care. On the other hand, EBM has the long-discussed disadvantage that the basic idea is to compare populations with one another. The individual is not the focus of consideration. And that is exactly the basic problem: a treatment decision developed by institutions that may well be optimal for a population may, under certain circumstances, only be correct to a limited extent for the individual within this population.
If it weren't for healthcare research.
Bartz: Exactly. Thanks to the science of health services research, we now have various options for analyzing how this population-individual gap can be described and, in the best case, resolved. In this respect, health services research is always patient-oriented. Through the possibilities of digitalization, which is constantly generating new data streams, and through the addition of real-world data, this science can manage to bring the EBM concept to the patient level. To do this, however, it must first be understood what a patient wants to achieve with his or her therapy. While for a clinical doctor the focus is on maximum clinical improvement in outcome parameters, from the patient's perspective this does not necessarily have to be the case. Quality of life, extensive freedom from pain or social participation may be more important here. What this means and reflected on EBM: health services research can help to further develop EBM in the sense of more individual patient care. Because if you treat patients based on decisions based on population data, you may be providing suboptimal care to the individual patient. The supply can be good - but that is more of a coincidence.

Danner: Mr. Bartz rightly mentioned that a purely study-oriented EBM proceeds in a schematized manner and does not sufficiently take the individual into account. In addition, although randomized studies on inclusion and exclusion criteria have a high level of internal evidence regarding the clearly defined subpopulation, patients who are seen by doctors often do not belong to this narrowly defined and researched group. This means nothing other than that the study results obtained in this way may not be transferable to the individual patient at all - or only to a limited, unknown extent. Here, too, health services research can help to make the study results obtained in RCTs more transferable by using real-word data on the one hand and data collection accompanying the application on the other. Ultimately, however, these are all risk-benefit considerations that the treating doctor can discuss together with the patient being treated, but which ultimately have to be made by the patient according to individual preferences. That's why evidence-based medicine - no matter how good it may be - always carries the risk that individual considerations can no longer take place because these have already been anticipated at an upstream macro or meso level. We have to move away from that a little bit.

A question for both of you: How do we get out of this? If you read through the definition of patient orientation that the federal government follows, chapter 5.9 “Patient orientation” (Health in Germany, 2015) states: “Patient orientation means that the health system and the professionals working within it focus on the wishes, expectations and satisfaction of patients. Patient orientation is examined at different organizational levels - the macro level with a view to socio-political developments and legal framework conditions, the meso level with a focus on institutions and associations, and the micro level, where the focus is on the relationships between professionals and patients. “ This means that even at the micro level with patient orientation, only the relationship between professional practitioners and those being treated is understood, but not the patient’s perspective. Which is no surprise, because the patient's gaze is a black box. Would you agree with that?
Danner: I would put that into perspective a little. The individual doctor-patient relationship is different. As more and better information is available, patients are empowered to not only discuss treatment options with their doctors, but also to confront them with their findings, which means that doctors have to argue more and more. It is clear that this is not yet the case across the board, but it is a clear trend. However, this requires good – and of course as evidence-based as possible – patient information that is understandable to laypeople. Patient participation has also long since arrived on a more abstract level, for example in the design of medical guidelines. This is particularly important because the guidelines are an important forum for making weighing decisions from the patient's perspective. In the best case scenario, therapy corridors are created that are relevant and important in the real doctor-patient relationship. Incidentally, this also sometimes happens with disease management programs and other forms of care in which the patient's perspective should be consistently included.

Will it be done then?
Danner: Not enough yet. We still have room for improvement.

Bartz: The treat-to-target principle has been around for a long time in the field of medical research. This refers to a strategic approach that is intended to enable doctors and patients to be as involved as possible in the therapy process in various indications. To do this, it must be defined in advance which goal should be achieved. The patient’s perspective is crucial here! In the area of rheumatology, for example, it must be discussed and decided on a case-by-case basis whether every joint should be pain-free or whether the goal is to completely stop the progression. Depending on how you decide, you don't have to administer a quadruple combination of synthetic medications, some of which have high rates of side effects, but you can choose a more targeted form of therapy. However, there is always an individual value decision behind this: Do you accept one or two tender joints but don't feel sick in the morning? This decision is highly individual. That's why, in my opinion, it makes sense to involve those affected in the creation of guidelines, disease management programs or even study protocols and treat-to-target concepts. But if you want that, you also have to cushion it in terms of infrastructure. Patient involvement at this level takes time, resources and manpower; especially on the part of the patients, who will only be able to contribute adequately in this way. Patient participation without sufficient funding will therefore always remain a fig leaf initiative.

Danner: I would also like to point out that we still have a lot of untapped potential here. Digitalization can mean that patient organizations will be able to aggregate patients' perspectives much better and more easily in the future because they can use digital offerings to collect and aggregate perspectives on therapy situations among their members and then incorporate them into a decision-making process in a structured manner. This is also why it is important that the resources of patient organizations are strengthened. However, if it's just a matter of sending someone somewhere, patient involvement will never be effective. In order to collect and incorporate aggregated patient experiences, appropriately qualified people are needed who, for example, know the methodology of creating guidelines and are familiar with the work required. But that doesn't happen by itself. This requires political impetus.

Does this impulse exist?
Danner: Unfortunately, it seems that patient involvement is often given lip service. In the current coalition agreement, the further development of patient participation was set as a goal, but no concrete steps have yet been taken as to how this should take place in this legislative period. Since the legislative period has already progressed quite far, a massive final push would be required if we wanted to make adequate progress at this point.

Where does patient participation appear, for example in the Statutory Health Insurance Stabilization Act or in the federal hospital reform? Of course, more quality is always good for the patient, but here again I am missing the perspective of the patient population.
Danner: When developing the hospital reform, it is obvious that no patient representatives were represented in the government commission. So it's not surprising that this is reflected in the recommendations. It can and will make sense to focus more on outpatient operations, but it is noticeable that the issue of travel costs has not been taken into account at all. This would not have happened if there had been a patient representative on the commission, because scientists and doctors who are represented on the commission usually do not have any opinions about, for example, how a patient gets to the hospital in the first place To worry about. To me, this shows very clearly that it leads to blind spots when there is no patient representation in such commissions.

Bartz: From our point of view, the Statutory Health Insurance Stabilization Act is not one that really helps patients. This is not surprising because it only serves to improve the financial problems of the health insurance companies. There is no rationale why patient care should improve just because the pharmaceutical industry is given less money. At this point, the patient's desire for the best possible care collides with the wall of economization. In addition, the law was drawn up in a short time, was only passed in October 2022 and came into force on January 1, 2023 - this hits even an industry with well-functioning basic business models extremely hard and is likely to have negative effects on the supply of therapeutic options .

Please explain this with an example.
Bartz: Let's take combination preparations, on which a flat rate of an additional 20 percent will be charged in the future. Quite apart from the legal justification: This cannot be in the interests of the patients. For them, as many combination preparations as possible – including cheaper ones than single-dose preparations – should be available on the market. But in the long term, which company can afford to develop products that inevitably lead to a steep drop in revenue? Another example is the reduction of the sales threshold for orphan drugs.

What will this do?
Bartz: It's fatal: it's becoming increasingly difficult to research innovative drugs in Germany and then bring them onto the market.

A narrative that is often heard.
Bartz: Politicians must become aware that changes to a set of regulations endanger companies' planning security on the one hand and Germany as a research location as a whole on the other. You may call this a narrative, but it follows a market economy rationale, the effects of which have been observed for a long time. Important location factors for pharmaceutical companies are market and access. If both worsen, fewer and fewer studies will be carried out in Germany. This is bad for patients and those treating them: the former are deprived of early access to innovative medicines and research funds and know-how.

Danner: One might assume that the income situation of pharmaceutical companies is not an issue for patients. But it is because changes to the regulations through the GKV Financial Stabilization Act will inevitably change companies' strategies. We also fear that in some indication areas there are fewer and fewer reasons to advance the necessary research. Second keyword: step innovations: There are indication areas such as oncology in which it is easier to show major differences. For others – such as diabetes – the endpoints are usually designed in such a way that it is much more difficult to show large differences. Our position as patient representatives is clear: All patients have a right to an improvement in their situation. This means that regulations that create negative incentives to no longer invest in certain indications are not in the interests of patients.

Bartz: The AMNOG has basically worked for years – leaving aside detailed questions. The basic idea is simple and understandable: equivalent drugs are paid the same, better drugs are paid better - in different gradations, depending on the level of evaluation and price negotiation. Of course, a system that is getting old needs to be readjusted. But now we are in a situation in which we are hardly able to predict whether our years-long development programs will make it through AMNOG or not. If this doesn't have any consequences this year, it will in two or three years at the latest.

We have now moved a little far away from patient orientation, even if an adequate supply of innovative medicines is certainly in the interests of patients. What does the pharmaceutical industry have to do with patient orientation, which mainly takes place at the doctor/patient level?
Bartz: We are part of the supply landscape. Our task in this complex healthcare system is to develop and produce innovative medications so that they can reach patients promptly. It is our natural mission and our raison d'être to develop innovations based on the needs of patients that interrupt, alleviate or even cure the disease burden of those affected as early as possible. Our top priority is that what we produce has the greatest possible benefit in the individual care of patients, but must also be affordable for the system. That means: Basically, our work is absolutely patient-centered because we think very early on about how we can use the findings from phase 1 studies to eventually turn them into innovative medicines for the benefit of patients.

Danner: Ultimately it's about improving care. To do this, we need good active ingredients in sufficient quantities - keyword supply bottlenecks - and the broadest possible selection of alternatives in order to open up treatment corridors. But we also need to understand that endpoints such as mortality, overall survival and life extension are important, but the improvement in quality of life cannot be neglected. For this purpose, an extremely sophisticated methodology has been developed over the past two decades in order to be able to objectively represent this absolutely patient-relevant endpoint. However, when it comes to the concept of quality of life, we have so far been dealing with an aggregated patient-reported outcome. Now we still have to pay more attention to the specific patient-reported outcome. It is worthwhile for companies to think further in this direction because they can then develop new drug candidates into beneficial innovations from the patient perspective.

Bartz: You mentioned previously the different levels of patient involvement. I see the pharmaceutical industry at all levels: At the macro level, we are committed to the further development of the healthcare system and the political framework in the interests of patients. At the meso level, we work with all stakeholders as best we can, including, of course, patient organizations. At the micro level, however, in Germany we are limited to exchanges with the medical profession, as we are not allowed to communicate directly with patients. What we do, however, is to provide the medical profession with the best possible study data and training materials about our products as well as materials with which they can support the health literacy of their patients.

However, I still lack the patient's perspective. I conducted an interview with the congress president of the 22nd DKVF for the last issue of “Monitor Healthcare Research”. Professor Dr. Geraedts calls for a structured recording of the patient's perspective in order to be able to represent real care: a kind of care cohort for Germany, which previously existed with the Bertelsmann Health Monitor. What do you think of it?
Danner: That is definitely an interesting idea if it is possible to present the wishes, experiences and needs of the population by surveying a representative group. However, I fear that the needs of patients are, on the one hand, very complex and, on the other hand, very heterogeneous. Then there is the regional and local situation, whereby the care structures and the availability and qualifications of the treating people vary greatly. I therefore doubt whether this complexity can be adequately described with such a cohort. Although it would be absolutely desirable to consistently research the patient's perspective and to implement the resulting results about under- and incorrect care in the further development of our healthcare system.

Bartz: In my opinion, a Germany care cohort would be a really useful tool to be able to represent relevant parts of care from the patient's perspective. This is – apart from smaller studies – a black box so far. If we can use such a data set to better understand an extremely important part of the reality of care, we could identify adjustments to better design our care system from the patient's perspective. However, such data only shows the current situation and gaps in the system; new possibilities are not described. That is again the task of health services research. If you do that, there must also be regulations and mechanisms that ensure that whatever opportunities for improvement are possible are transferred and introduced into the system.

Danner: You can think even bigger in terms of a future-oriented vision. At some point we will have a national data room that could also help detect gaps in the system. In addition, pay-for-performance contracts could be used to observe the effects of supply operations over a longer period of time and create an even better knowledge base. The crux of the matter is that we don't have this infrastructure available yet.

Bartz: There are plenty of pay-for-performance models in other European countries. They create the most robust data possible because they require the collection of clinical data to ensure reimbursement. It would be an interesting idea to also incorporate this data into an overall data model. There really is a lot of potential in it, which ultimately saves a lot of money for the system and also helps to provide better care for each individual person.

At the beginning of our conversation, we looked back up to 20 years. What does patient orientation look like in 2033?
Danner: You can't predict how the two antagonists, digitalization and data protection, will develop and how an opportunity may at some point arise to actually develop patient-oriented care. Ultimately, it is always a problem of knowledge to create the best possible care for patients. Otherwise, I am confident that the development process of the last 20 years is progressing and that we will take the patient's perspective into account at all levels. This is already the case today when determining research work and study designs as well as when evaluating endpoints. However, the patient's perspective should also be taken into account when developing new care structures. Who should know better how to design an optimal relationship between outpatient and inpatient care than the patient himself?

Bartz: We will achieve our goals with regard to consistently patient-centered care much faster and better if we involve patients in all processes earlier. In the next five to ten years we will all have to work together to make sure that this is actually done.

Ultimately, the concept behind this is value-based care, although the values are mainly determined by the stakeholders - hopefully with the patient's say, but not from the perspective of the patient population. How do we move from value-based to patient-centered care?
Danner: Ultimately, it's always about setting beneficial targets for those involved in the healthcare system. The first impulses have already been set with goals for the benefit assessment of drugs and medical devices. I now see the need to make concrete patient orientation clear as a goal for all other actors. But be careful: patient participation must not be used as a means of preventing all those involved in the healthcare system from behaving in a patient-oriented manner. This target must apply to everyone.

Are you on board with this?
Bartz: Value-based healthcare means achieving the best possible result with the resources available, i.e. H. to achieve the maximum health-related value (“value”) for those affected. The former is influenced by how quickly a patient gets to the right doctor and whether the doctor then prescribes the right therapy for them. If a chronic patient has been treated incorrectly for over 20 years, it not only creates suffering and an unnecessarily reduced quality of life. It also costs the healthcare system a lot of money. The right treatment can never, ever be that expensive. However, if the patient is treated correctly as early as possible, the costs for the entire system will also decrease. Unfortunately, we use this perspective far too rarely because health is always viewed as a cost factor and never viewed holistically.
Danner: Of course, it is always important that the patient gets to the best possible place for their care as quickly as possible. When I think about personalized medicine, for example, the right diagnostics also play a very important role because it is no longer about the patient as a patient, but about individual predispositions and biomarkers that can contribute to a Disease is discovered earlier, can be treated better or possibly does not break out at all. What is needed here is a change in meaning and a social consensus that does not accept illness as God-given.

You just mentioned the VBHC formula: quality of care divided by cost of care. The GKV patient wants the best treatment, but she doesn't even see the treatment costs.
Danner: That is the entitlement in the compulsory insurance system. I cannot deprive the population of a large part of their income and then withhold supplies. That's why I react allergically when people talk too hastily about rationing in the statutory health insurance system. The strictest possible benefit orientation must therefore ensure that unnecessary treatments and those that do not have the optimal benefit are excluded. But it is a misunderstanding that patient representatives always want to have everything for everyone. We don't want to because we are well aware of the system's limited resources. For us, it's more about competing for the best possible care. VBHC is a benchmark for this because ultimately patient-centeredness must be the benchmark for everything.

So we need to bring about a cultural change from looking at the cost of illness to investing in health.
Bartz: That's right. You see this increasingly in medicine. For a long time, traditional treatment paths started with the cheapest active ingredient and then slowly escalated, instead of immediately using the innovative, usually more expensive active ingredient in order to stop chronic disease or erosion processes as much as possible. For some time now, the mindset of “hit hard and early” or, today, “hit smart and early” has become more and more prevalent in many disciplines. With innovative medications, treatment costs increase on the one hand, but on the other hand the costs for the consequences of illness, treatment times and intangible follow-up costs decrease. Unfortunately, calculations are rarely made in such models. However, I am convinced that this school of thought will prevail in many therapies in terms of patient benefit.

Danner: Which is good if it is clearly proven by proof of benefits. But only then. <<

Messrs. Bartz and Danner, thank you for the interview.