Elsevier

Journal of Cancer Policy

Volume 11, March 2017, Pages 54-59
Journal of Cancer Policy

Reviews
Rare cancers—Rarity as a cost and value argument

https://doi.org/10.1016/j.jcpo.2016.09.004Get rights and content

Highlights

  • The overarching goal is to provide high-quality care and meet patients’ needs.

  • New concepts for defining the prices for orphan drugs have to be developed.

  • There are public health and economic challenges posed by rare diseases.

  • A main task is the generation of evidence-based information about rare cancers.

Abstract

Here is a paradox in medicine: rare diseases are unusual, but developing a rare disease is quite frequent. This is also true for rare forms of cancer. Almost every 20th person in the world suffers from a rare disease, and about one quarter of all new diagnosed cancers belong to rare cancers. The downside of rare diseases for patients is the difficulty to find the right institution for their treatment, for health care payers it is the costly treatments, and for medical professionals their limited knowledge if they are not specialized in the disease in question. On the other hand, the upside for clinical researchers is that rare diseases are beneficial for their scientific careers, as many clinical and scientific questions are still open. The advantages for the pharmaceutical industry are the premium prices, the special governmental programs to stimulate research, and achieving a dominant position in a small market. To sum up, rare cancers are important for all stakeholders in medical care and deserve more attention from public health research.

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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