Did Mooncare truly reinforce coverage for rare diseases?

entering 4th year of implementation of Mooncare

“Reimbursement rate 100% in 2020”…”does not include rejected or voluntary withdrawal items”

KPBMA·KRPIA submits statement stating a “Need to expand the pharmacoeconomic evaluation exemption system”

The voice requesting expanded coverage for patients with rare diseases had been exceptionally high this year in the 4th year of Mooncare.

 

Starting with NA discussions held to enhance coverage of innovative new drugs for rare genetic disorders in May by the NA Health and Welfare Committee member Sunwoo Kang, NA members Byungwon Kang, Woni Kim, Young Seok Seo, Hyunyoung Shin held a public hearing to discuss ways to resolve the medically unattended areas, continuing on the effort to foster a policy environment that in which patients with rare diseases are not marginalized.

 

The main contents that were discussed included the need to apply special exemption of calculation to diseases that are not being covered due to non-designation as a rare disease, and the strong proposal on the need to expand patient access to new rare disease treatments.

 

The discussion continued to the NA Audit, where the NA Health and Welfare Committee member Sunwoo Kang, and members of the ruling and parties including JaeKeun In and Jongseong Lee all unilaterally urged improvement.

 

◆100% reimbursement rate for rare disease treatments in 2020? However, the public hearing revealed the different views held by the Health Insurance Review and Assessment Service.

 

At the public hearing in May, HIRA presented that the reimbursement rate for rare disease treatments was 85.3%(2016~2020) and 100% in 2020.

 

The numbers indicate that patient access to rare disease treatments is perfect.

 

But if this is the case, why is the voice to expand reimbursement for rare disease treatments continuing to rise?

 

출처: 희귀유전질환 혁신신약 접근성강화를 위한 국회 토론회

The results announced by HIRA were the reimbursement rate of drugs that went through the review and assessment process, not the actual reimbursement rate among all rare disease drugs.

 

In other words, HIRA’s result excluded various factors including rejected and voluntarily withdrawn items.

 

Data studied by the Korean Research-based Pharma Industry Association and the Korea Pharmaceutical and Bio-Pharma Manufacturers Association showed that only 50% of the pharmaceuticals that were designated orphan drugs over the past decade were listed on the reimbursement list.

 

◆Industry "Need to expand the pharmacoeconomic evaluation exemption system" The two associations joined forces to address the issue.

 

Dailpharm found that KPBMA and KRPIA saw consensus on the need to expand accessibility to rare disease treatments and submitted a statement on the need to expand the pharmacoeconomic evaluation exemption system for rare disease treatments.

 

In the statement, the two associations pointed out the existence of many rare diseases that greatly affect households with indirect medical costs and deteriorate the quality of life of patients but are not qualified for the ‘life-threatening (less than 2 years of life expectancy)’ condition that needs to be fulfilled to be able to utilize the current special exemption system (RSA and pharmacoeconomic evaluation exemption system) Therefore, the associations insisted that evaluating the ‘clinical need’ only with life expectancy does not take into account the characteristics of rare diseases, and diseases that do not meet the ‘less than 2-year life expectancy’ part of the clinical need requirement should also be allowed PE exemption if it is authorized through expedited approval processes abroad (US BTD, EU PRIME).

 

The government had said that it would make efforts to improve access to rare disease treatments in the several public hearing and NA audits, but no specific plan or goal seems to be in place.

 

An industry official said, “The government had recently adjusted the price evaluation criteria of PE exemption drugs by 20% from the lowest A7-adjusted price to improve the price transparency of PE exemption drugs.

 

If the authorities decided to reduce the risk factors in price as such, they also should increase its scope of benefits.”