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  • [Reporter’s View]Raising the voice of rare disease patients
  • by Eo, Yun-Ho | translator Byun Kyung A | May 20, 2020 06:11am
Seems like rare diseases put patients in utmost pain, because of its ‘rarity.’ Rare disease treatments can hardly prove cost-effectiveness and predictability with limited patient size for the reimbursement listing approval.

Regardless, the Korean government is aware of the struggle. The revised drug pricing system the Ministry of Health and Welfare (MOHW) unveiled is in process of legislative preannouncement until June 11.

The key revision in the new drug pricing system is to expand eligibility in pharmacoeconomic evaluation (PE) exemption and RSA. The revision would stipulate the Korean government to sign risk sharing agreement (RSA) not only with first-in-class drugs, but also with follow-on drugs. And also the revision would expand subjects for PE exemption and coverage with phase III clinical evidence in development. Even the PE exemption, previously restricted to anticancer and rare disease treatments, would be applicable on National Essential Drugs, such as tuberculosis treatment, antibiotics and emergency antidotes.

However, many of the public claim rare disease treatments would still face various obstacles before receiving RSA and PE exemption benefits.

In Korea, rare disease is defined as a disease with patient size less than 20,000 with difficulties in diagnosis to survey accurate number of patients. The affected patients are desperate to get access to life-saving treatment as the diseases are difficult to diagnose and treat, but many of the diseases cannot even start a clinical study with barely sufficient number of patients.

Pharmaceutical companies are hesitant to develop a novel drug for rare disease, as the limited number of patients means limited marketability, while the chances of successfully proving the cost-effectiveness of the drug through PE would be thin.

Many countries around the world are introducing regulatory exceptions in legislation, special approval or reimbursement listing for better access to rare disease treatments. Korea has also provided regulatory exceptions like medically essential drug management, PE exemption system and RSA in reimbursement listing or drug pricing. Regardless, the limitations to treatment access still exist.

In fact, data comparing listing rate by each drug type before and after implementing RSA and PE exemption systems for bettering the access to high-cost treatments showed a significant improvement in general drug (79.6 percent to 98.6 percent) and anticancer treatment (77.1 percent to 91.7 percent), but the improvement in rare disease treatment (71.1 percent to 71.4 percent) was rather unnoticeable.

As a solution to the much needed access to rare disease treatment, some argue the PE exemption eligibility should be expanded for the rare disease treatment.

They say the reimbursement review system should be flexible to exempt PE or adjust the number of PE sample patients similar to the special case reimbursement standard, when a drug has no other alternative option or has been approved with placebo-controlled study results.

For instance, the U.S. Food and Drug Administration’s (FDA) Breakthrough Therapy Designation (BTD) or European Medicines Agency’s (EMA) Priority Medicine (PRIME) designation could be referred as a review standard. The PE exemption system aims to provide patients the access to rare disease and anticancer treatments unable to produce PE data, while protecting the initial purpose of positive listing system. So to serve its purpose, the Korean health authority should contemplate on the flexibility of the healthcare reimbursement listing procedure.

Same goes for RSA. Compared to numerous anticancer treatments that received coverage through RSA, many of rare disease treatments have failed in passing the barrier to RSA due to lack of PE data. As far as quality-adjusted life-year (QALY) goes, the majority of rare diseases results in severe physical damage in patient’s body, lowers quality of life, and shortens life expectancy. And because of extremely small number of users, rare disease treatments are inevitably high-priced and show poor cost-effectiveness.

The rare disease treatments can hardly prove cost-effectiveness, when applied with incremental cost-effectiveness ratio (ICER) thresholds on par with other general drugs. The regulation stipulates the health authorities to flexibly set ICER thresholds on rare disease treatments, but the drugs are challenged with PE barriers higher than that of anticancer treatments due to their practices so far. This is why many urge more adaptable ICER thresholds should be given to rare disease treatments for their PE.

When the Rare Disease Management Act came in effect in 2015, the Korean government designated May 23 as the Rare Disease Day to raise awareness and understanding of patients fighting against rare diseases. Rare diseases have a handful of treatments for a handful of patients, which is why the voice to demand their healthcare benefit is so easily lost. Hopefully in the near future, the government and pharmaceutical companies would pay a closer attention to those patients, as much as they do for cancer patients.
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