ReviewsRare cancers—Rarity as a cost and value argument
Section snippets
Rare diseases are quite frequent
About 7000–8000 of the 30,000 known diseases are rare diseases, also commonly known as orphan diseases [1]. The European Union (EU) defines a rare disease as having a prevalence of less than 500 cases per million people [2]. In contrast, rare diseases in other countries are defined through different prevalence rates: e.g., in United States (US) 750, in Japan 400, and in Australia 120 patients per million people [3].
It seems paradoxical that, while the patient population for each rare condition
Rare diseases from an economic point of view
In the well-known article “The Voluntary Exchange Theory of Public Economy” [11], published in 1939 by Richard Musgrave, the responsibilities of a government are structured into three major “branches”: the stabilization of the economy, (re)distribution, and achievement of an efficient allocation of resources. This conceptual division of the responsibilities of governments can also be transferred to health care systems. From an economic viewpoint, we can identify three major tasks for health
Challenges for the health care system posed by rare diseases in general
Rare diseases pose a number of challenges for health care systems. From a dual economic and organizational point of view, the following six issues can be formulated:
- 1.
In many cases, the diagnosis of rare diseases is very difficult due to their rarity and heterogeneity. The majority of physicians have little or no experience with these disorders, e.g. rare childhood or rare eye cancer. Educational efforts and better information systems can help both physicians be more sensitive towards rare
Challenges for the health care system posed by rare cancers
In addition to the above challenges presented by rare diseases in general, rare cancers pose additional challenges for the health care system.
- 1.
Rare cancers are difficult to study due to the low patient numbers, poor diagnostic precision, and therapeutic mismanagement [8], [22]. If available, historical and non-controlled studies are the basis for many of the standard treatments for rare cancers [23]. Hence, scientific societies and research networks in the area of rare cancers are essential for
Challenges for orphan drugs’ price setting
A highly debated question is, if prices for innovative drugs to treat rare cancer patients are too high or too low. If prices are too high, payers pay too much relatively to other health services and give an incentive to pharmaceutical industry to spend too much for research and development (R&D). If reimbursement prices are too low, there are no incentives to develop drugs for the treatment of patient with rare diseases.
Prices in general have different functions, among others an allocative and
Patient and physicians reported outcomes about rare diseases
In a research project for the Federal Government of Germany, we asked patients suffering from rare diseases what factors they considered important for an appropriate treatment of their disease. The competence of the physician was identified as the most important factor, followed by excellent information, and easy access to medical aid. The importance of different health care aspects for patients with rare diseases is shown in Table 1. Surprisingly, it was not important for patients whether the
Conclusion
From an economic viewpoint, health care systems should strive to achieve three goals, which are extremely difficult particularly in relation to rare diseases:
- a)
efficient production of health care
- b)
just distribution of resources
- c)
stable financing.
Because the number of patients suffering from rare diseases is large and the treatments are costly, these conditions pose an extraordinary financial and organizational challenge for health care payers. Furthermore, the treatment of these patients can be
Acknowledgements
The author had used no funding to conduct this study or prepare this manuscript. They have no conflicts of interest that are directly relevant to this article.
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