Hemophilia in Poland. Experts identify key directions for change

Hemophilia is a disease that is extremely well protected by the health care system - but it is still not an optimal model. Access to drug therapies is improving, while challenges remain, including comprehensive and coordinated care, especially for comorbidities, access to rehabilitation, and staff shortages.

On October 28, two reports were presented outlining the current state of hemophilia care, key challenges and directions for the hemophilia treatment system in Poland. "Hemophilia. Current state and directions of development" is a report by the Institute of Healthcare Management at Lazarski University, edited by Dr. Malgorzata Galazka-Sobotka, and "System analysis of the organization and functioning of the treatment of patients with hemophilia with implementation" was prepared by EconMed Europe.

- The total cost of hemophilia in Poland far exceeds the funding of drugs and medical procedures. Annual direct costs, borne by the National Health Fund and the Ministry of Health, amount to PLN 521 million. Patients and their families are also financially burdened - their direct expenses are about PLN 10 million (including nearly PLN 2 million in travel costs to treatment centers), and indirect costs are PLN 433 million (consisting of, among other things, absenteeism and presenteeism, i.e. reduced productivity). - Indirect costs make up a significant part of the total burden, and although they are lower than direct costs, they remain at a similar level, explained Dr. Michal Seweryn, president of EconMed Europe, a specialist in public health and epidemiology, and an associate of the Faculty of Medicine and Health Sciences at Krakow's Frycz Modrzewski Academy, during a press conference dedicated to the two reports.

Indirect costs and the need to take them into account when making key decisions about - for example - reimbursing new medical technologies has been one of the leitmotifs of discussions devoted to chronic diseases, including rare diseases, for years. Part of the report prepared by EconMed are the results of a survey in which dozens of patients (or patient caregivers) participated. As Dr. Seweryn emphasized, although the number of respondents is not very impressive, it is worth noting that a similar survey is in vain both in Poland and abroad. - In our analysis, patients reported, among other things, frequent sick leave, the need to reduce working hours or to give up work due to illness. We observed a similar phenomenon among caregivers of children with hemophilia, as half of them declared that they had to quit their jobs and another 30 percent reduced their full-time hours. This shows that the economic consequences of hemophilia affect entire families," the expert said. At the same time, Dr. Seweryn acknowledged that the percentage of active workers - employed or self-employed - was surprisingly high, exceeding 80 percent of respondents.

Active professional and socially active patients are all the more in need of a modern model of care, which should, Dr. Seweryn stressed, be based on the following pillars:

- Individualization of therapy tailored to the patient's needs,

- Development of a network of coordinated reference centers,

- Ensuring home delivery of medicines,

- Digitization of care and education of patients and their families.

These pillars reinforced by psychological support, rehabilitation, vocational activation and social support mean nothing less than an intedisciplinary, cross-silo approach - combining the tasks of the health care system, social policy and education. Only in this way can we achieve both improved clinical outcomes, but also optimize system costs and significantly improve the quality of life of people with hemophilia. - The more integrated the patient pathway and the better the cooperation between centers, the more effective the treatment. This is a universal principle that also applies to other rare diseases. We would like to see tools that support the personalization of therapy function as widely as possible, and we would like patients to benefit from the fact that it is possible to make therapeutic decisions based on their individual needs and preferences.

The report, prepared by the Institute for Healthcare Management at Lazarski University, shows that while the hemophilia treatment system is well-developed in terms of organization and logistics, it needs further modernization to meet global standards, particularly in terms of treatment. Dr. Malgorzata Galazka-Sobotka, dean of the Center for Postgraduate Education and director of the Institute for Healthcare Management at Lazarski University in Warsaw, stressed that a great deal has been achieved in the area of hemophilia treatment, but that does not mean that more cannot be achieved.

Key deficiencies include: limited availability of modern therapies, insufficient comprehensiveness of care, fragmented registries and interruptions in the continuity of treatment for adult patients.

The current organizational and financial arrangements are based on the National Program for the Treatment of Hemophilia (NPLH) and drug program B.15 These form the foundation for the treatment of hemophilia A and B, but against the background of international guidelines - both from the World Federation of Hemophilia and the International Society of Thrombosis and Hemostasis - gaps in patient care are apparent. These particularly relate to the availability of therapy, comprehensiveness of care, and the system for monitoring and reporting treatment effects.

As Dr. Galazka-Sobotka stressed, the Polish system for treating hemophilia patients focuses almost exclusively on monitoring pharmacotherapy and clinical indicators (number of bleedings or laboratory results). What is missing is the use of patient-centered indicators, which should - according to international guidelines - be an integral part of assessing the effectiveness of treatment, as they monitor the impact of the therapies used on the patient's daily life, ability to study, work and social activities. The expert stressed that currently therapeutic goals are not set by the doctor alone - they are (and certainly should be) the result of agreement with the patient.

The treatment of hemophilia in our country is financed through two pathways - the National Program for the Treatment of Hemophilia and Related Hemorrhagic Disorders (NPLH), which is a health policy program and covers all age groups, and the B.15 drug program, dedicated to children with severe hemophilia A and B. The National Hemophilia Treatment Program, which has been in operation in Poland for two decades, has been financed from the state budget since its inception, while the drug program, which (like other drug programs) reimburses expensive and very expensive innovative therapies - by the National Health Fund. As of 2022, both of these programs are financed by the National Health Fund, but drug programs are under the strict supervision of the Fund (exercised by coordinating teams responsible for qualifying patients and monitoring treatment effects, performance indicators). This is necessary because, as the economist reminded, this year spending on drugs made available under drug programs has already exceeded the value of pharmacy reimbursement.

- Around the National Program for the Treatment of Hemophilia and Related Hemorrhagic Disorders, we should build solutions that would allow modern treatment in accordance with current medical knowledge to enter this program, based on tools that are widely available in Europe. Let's look for solutions that would bring the advantages of the drug program to NPLH. Our discussions with the community of clinicians, but also patient representatives, clearly indicate that the NPLH is a very good base to build the potential to modernize care in," Dr. Galazka-Sobotka said.

- I can say that at the moment the care in Poland for patients with hemophilia is really at a good level. This is evidenced, for example, by the number of units of clotting factors VIII and IX that we have available per capita, as well as the number of hemophilia treatment centers. On the other hand, of course, there are some problems," said Prof. Jacek Trelinski, head of the Hemophilia and Other Blood Coagulation Disorders Treatment Center of the Provincial Multispecialty Oncology and Traumatology Center in Lodz.

The expert stressed that the next edition of the NPLH should include a requirement to employ physiotherapists, psychologists and dentists in centers providing care to patients with hemophilia. The home drug delivery program should also be expanded. - And finally, access to modern drugs. As doctors, we are constantly expanding our knowledge, traveling to foreign conventions, reading publications and knowing that the world is moving forward. We are actually already at the threshold of the great revolution that has taken place in the treatment of hemophilia, which is the administration of non-substituted drugs and other medications, and we would like to have access to them," he said, adding that the patient's voice should be crucial in the choice of therapy. - There are those who want to stay with the good old methods, i.e. prophylaxis with clotting factors, and we have nothing against it. If it is effective, and the patient is not discouraged by the fact that he has to give himself clotting factor 150 times a year," he reminded. At the same time, the expert noted that new therapies, clotting factors with prolonged action, which patients in Poland do not have access to at the moment, make it possible to significantly reduce the number of administrations. He called the drugs administered subcutaneously a revolution. - We doctors would like to have access to all of them. And to tell the truth, we don't care much whether it's under the National Hemophilia Treatment Program or under the drug program," he stressed.

Pediatric patients are now fully - until they reach the age of majority - eligible for the drug program. Until September of this year, only the youngest children could benefit from the program. - The change that has taken place since October, i.e. the expansion of the availability of both the subcutaneous drug and the prolonged-acting agents, will allow optimal protection for all pediatric patients up to the age of 18, so that we can have personalized therapy in the full sense of the word, said Olga Zając-Spychała, MD, head of the Regional Center for the Treatment of Hemophilia and Related Hemorrhagic Cases for Children in Poznań. As the specialist emphasized, the youngest child who was qualified for the program was only 5 weeks old. In the youngest children, who are not yet moving, bleeding also occurs, and it is particularly dangerous, because it enters the central nervous system.

And in children and adults, as Prof. Trelinski reminded, the clinical goal is to eliminate bleeding, which would mean a normal quality of life for a patient with hemophilia, who could - like a healthy child, a healthy adult - study, work, but also, for example, play sports. This goal is achievable, the expert said, with the use of modern therapies.