The time is ripe to define national care pathways

Prof. A.M. Pereira Arias is a specialist in internal medicine and endocrinology and was Professor of Endocrinology at the University of Leiden (the Netherlands) from 2012 until he was appointed Professor of Internal Medicine, with a special focus on endocrinology, at the University of Amsterdam in 2022.In 2012 he founded the Centre for Endocrine Tumours in Leiden.  From 2016 to 2018 he was President of the European NeuroEndocrine Association (ENEA). He has been the coordinator of the European Endo-ERN network since its establishment in 2017.

In January of this year, Prof. Alberto Pereira Arias gave his second inaugural lecture, this time as Professor of Internal Medicine, and chair of Endocrinology, at the University of Amsterdam. He had previously been Professor at Leiden University Medical Centre. The endocrinologist is an outspoken advocate for the formation of networks coordinated by centres of expertise. We now need to work out the precise contours of how and who will do what.

‘I enjoyed 22 fantastic years in Leiden, but when the opportunity arose to become head of the largest endocrinology department in the Netherlands, I saw it as a wonderful new opportunity and a logical next step’ is how Pereira explains his move. ‘The new department at Amsterdam UMC is still in the midst of the merger and lateralisation of the two departments at the AMC (University of Amsterdam medical centre) and the VUmc (Free University of Amsterdam medical centre). That offers excellent opportunities to shape the profile of the new department so that we are optimally prepared for the future.’ He entitled his lecture ‘2034’, a reference to the year in which he will probably retire. He considers what the future holds for endocrinology based on a patient who has Cushing’s disease. ‘Hopefully, Cushing patients will then be diagnosed more quickly. This will be made possible thanks to the formation of networks and centres of expertise, a development that has now been set in motion.

I also expect that more attention will then be paid to the long-term consequences of the disease, thus improving both quality of life and life expectancy.’

Non-academic hospitals

In his opinion, the formation of national centres of expertise for rare and complex conditions will be of great benefit for all. Much has already been gained in this area in recent years. ‘As far as I know, pituitary surgeries are now only carried out in eight hospitals in the Netherlands. At the beginning of this century, that was 19.’ Oncology can serve as an example when it comes to concentration of care. ‘In oncology, networks with clear agreements about which part of the care takes place in which reference centre have been common practice for much longer. 

Within endocrinology, specific complex and rare conditions are not yet treated in centres of expertise alone. And there is no need, Pereira believes. ‘Patients with a rare endocrine disease can be cared for – either largely or at least in part – in a non-academic hospital, provided that it is part of a network and agreements have been made about which part of the care will be provided there and which part will be provided in a reference centre. Centralised where necessary, close to home where possible, is a credo that has my full backing. The networks of the NVE (Dutch Endocrine Society) create the ideal conditions for such a policy. The time is now ripe to define national healthcare pathways and the European Journeys for patients with rare endocrine conditions.

In 2017, the European Reference Network on endocrine conditions (Endo-ERN) was installed by the European Commission. Parallel to this, initiatives for national networks emerged in the Netherlands, recalls Pereira; he was himself closely involved in the formation of such networks. ‘The national networks currently fall under the umbrella of the NVE, but they need to be further developed so that they can connect up with the European networks. There is as yet no funding for the formation of these national networks, but a large European grant (the ERDERA project) has now been funded that will enable the ERNs to integrate into the national healthcare systems of all 27 EU member states.’

‘The cortisol excess that is present in patients with Cushing syndrome is a good model to study the exposure to chronic stress in the general population’ 

Patients are increasingly being supported to determine where they can go for “the best” care. Alongside the official designation as ‘centre of expertise’, social media, and patient associations have gained a more influential voice, one that is also heard by the government and health insurers. ‘Healthcare providers therefore really need to do their best to show what they are good at’, noted Pereira. Since 2011, every patient in the European Union has had the right to choose his or her own healthcare provider, but this is not yet common knowledge. When a patient chooses a healthcare provider located in another EU country, the patient’s home country is obliged to cover the costs involved. That fits in with ERN’s mission to reduce disparities in health and the access to care within Europe.’ But at the same time, it is not the intention that all those people who believe they have a rare condition should go travelling criss-cross through Europe. Therefore, the European Commission has promoted the development of the Clinical Patient Management System, CPMS. ‘This telemedicine tool allows healthcare providers who are affiliated to an ERN to discuss their patient’s case with a panel of international experts. If necessary, the patient can then still travel for care, but up to now three-quarters of patients can continue to be treated in their home country after such a virtual consultation. CPMS is the first ICT tool that fully complies with European privacy legislation.’

Blind spots

As coordinator of Endo-ERN, Pereira wants to improve accessibility to care for every patient in the EU who potentially has a rare or difficult-to-treat condition. ‘Too little use is still being made of the possibility of discussing patients within a network, and this applies to the Netherlands as well. And that while everyone suffers from blind spots. I myself experienced that when a colleague took over my consultations for a year while I was working in the laboratory, she picked up on things I had myself overlooked.’ That is why he recommends that everyone should join a regional, national and European network. ‘That will really enrich your field of vision. I am very happy that there is an opportunity to discuss patients with, for example, rare bone disorders, difficult-to-treat pituitary disorders or rare adrenal and thyroid diseases.’ There is still, however, a lack of any formal financial/reimbursement structure for the provided added value of the expert consultation within these networks. ‘The virtual consultations are now done within regular working hours, but are relatively time-consuming. A separate tariff for this is desirable and the first steps are currently being taken to this end.’

Feyenoord-fan in Ajax shirt

Pereira Arias was born in Uruguay to a South American father and a Dutch mother. He grew up in Rotterdam. Football was always an important outlet for him, previously to play and nowadays to watch. Upon his arrival as a professor in Amsterdam, he was hoisted into Ajax-shirt, but he continues to support Feyenoord. ‘I am still a member of a group app for Dutch professors who are Feyenoord fans. At my farewell from Leiden, I received a Feyenoord jersey with my name on it. And I was also given a bag full of Feyenoord stickers with which to decorate the AMC during a quiet night shift, but they haven’t been used yet’, he says with a smile.

Leveraging health data

The Dutch Minister for Health, Welfare and Sport has asked the university medical centres (UMCs) to coordinate the networking of healthcare in their own referral area. ‘The UMCs themselves would also like to be last resort for specific rare disorders and concentrate on complex treatments, but there are as yet no agreements with network partners about which care will be provided where. The effects of networked care on healthcare revenue and the training of doctors and other healthcare staff have not yet been mapped and all these factors need to be part of a national plan,’ Pereira said. In his vision, UMCs would also act as healthcare laboratories, platforms for integration of new care concepts, and focus on a mechanistic approach to research. ‘Rare disorders can serve as models for common conditions. Rare disease research would then not only be important for patients with rare disorders, but also for a much broader group. For example, the cortisol excess that Cushing patients experience is a good model to study the exposure to chronic stress in the general population, just as it is a good model to study the effects of treatment of people receiving corticosteroids for another condition. Exposure to excess stress hormones puts all these groups at higher risk of, for example, cardiovascular disease and depression.’

To facilitate the link from care to research, the ERNs have established European registries for data collection. Pereira is very happy about this. ‘These registries thus serve as a platform, making it possible to link healthcare and research and boost the exchange of knowledge.’ In order to exploit the full potential of health data, a large European project is under way that, among other things, encourages citizens to take control of their own health data. ‘Awareness is crucial in this respect. If people feel they are co-owners of their own data, they are more willing to share it.’ Globally, this European Health Data Space project is being followed with interest because Europe is leading the way.

Expensive medicines

A hot issue Pereira calls the availability of medication. ‘That applies to the whole of healthcare, but applies particularly to endocrinology, because for many hormonal diseases’ medicines are of vital importance. Recently, there have been situations when certain medications have suddenly become unavailable. There are several European and national initiatives to identify what patients with rare and less rare diseases need, and what can be done about these shortages.’ The topic of ‘expensive medication’ is also an issue in endocrinology. In the Integral Care Agreement which was concluded last year in the Netherlands, it was agreed, among other things, to achieve an appropriate use of expensive drugs and orphan drugs. ‘To this end, the so-called Orphan Drug Access Protocol (ODAP) was developed under the leadership of my colleague Prof. Carla Hollak. Crucial within the ODAP is that accessibility of orphan drugs is linked to effective use. The aim is to make new, promising non-oncological drugs for the treatment of rare diseases available as soon as possible, and at an acceptable price. In practice, the effectiveness of that drug should then be evaluated.’

Original interview/DUTCH text:  Raymon Heemskerk
Translation: Alberto Pereira Arias & Peggy van Schaik

   Original was published in the magazine 
“Endocrinologie” (the official NVE magazine) September 2023 edition, pages 09/10/11