Adults in Japan support public funding for rare disease treatments

Patient burden, healthcare costs weigh more heavily than rarity of diseases

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Most adults in the general public in Japan support using public funds to help cover the costs of treatment for people with rare diseases, according to a recent study.

The study, “General public’s understanding of rare diseases and their opinions on medical resource allocation in Japan: a cross-sectional study,” was published in the Orphanet Journal of Rare Diseases.

Because rare diseases like Lambert-Eaton myasthenic syndrome (LEMS) are by definition rare, treatments won’t sell as much as those meant to treat more common diseases. For this reason, pharmaceutical companies usually set higher prices for rare disease treatments.

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Survey in Japan gauges public understanding of rare diseases

Japan is a country that offers universal healthcare, where costs of medical care are generally covered by public funds.

In the study, researchers conducted a survey of adults in Japan to gauge the general public’s understanding of rare diseases and their opinions about financial support for treatments.

The survey was completed by 11,019 people. About one in 10 (9.9%) reported that they or someone close to them had personally been impacted by a rare disease.

When asked about their perceptions of rare diseases, most of the respondents correctly expressed that these disorders often pose a financial burden to patients and their families, and lack effective treatment options.

However, other aspects were less well-understood. For example, when respondents were asked about how uncommon a disease would need be in order to be considered rare, most respondents reported prevalence rates that were much lower than the definitions used by regulatory authorities in the U.S., the European Union, and Japan.

In making funding decisions for RDs [rare diseases], the general public considers burdens on daily living or financial burdens more than the epidemiological characteristics of RDs. Rarity … was less prioritized by the general public as a factor in making funding decisions.

“Rather than scientific discussions such as those regarding disease prevalence, the word ‘rare’ for RDs [rare diseases] in Japan has been defined based on political discussions, such as government financial assistance for patients and promotion of research and development,” the researchers wrote. “Thus, a gap also appears to exist between the definition of RDs in policies and the public perception of a ‘rare disease.’ ”

The researchers called for efforts to improve public awareness of rare diseases, noting that “gaps in understanding may hinder the public from accepting the prioritization of RDs in financial support allocation.”

Since rare diseases such as LEMS can vary widely in their symptoms, the team suggested an informational toolkit that could be used to broadly communicate important information across rare diagnoses.

The majority of survey respondents said they supported using public funds to partially help cover the cost of care for rare diseases, though the rate of support was notably higher for diseases affecting children compared with those involving adults (66.8% vs. 59.5%).

The most commonly noted reasons for favoring this kind of funding included the high costs of care associated with rare diseases, the lack of treatment options, and the fact that these diseases commonly have a substantial impact on patients’ quality of life.

“Our respondents appeared to accept the prioritization of RDs in the allocation of financial support in light of their understanding of RDs,” the researchers wrote.

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Most survey respondents favor using taxes or joint private-public funding

When asked about how the costs of rare disease treatments would be covered, most respondents favored using taxes or joint funding by the government and pharmaceutical companies.

In the last part of the survey, the participants were asked whether they thought the government should prioritize funding research into rare or common diseases. Nearly half of the participants said they weren’t sure. Among those who answered, slightly more than half (56%) favored prioritizing research into rare diseases.

Notably, among those who favored prioritizing rare diseases, the most commonly given reasons were the fact that these diseases are often poorly understood and lack treatment options — not the fact that they are rare in and of itself.

“In making funding decisions for RDs, the general public considers burdens on daily living or financial burdens more than the epidemiological characteristics of RDs,” the researchers wrote. “Rarity … was less prioritized by the general public as a factor in making funding decisions.”