As the deadline for drug applications in the government's pilot program to cut the health insurance listing period for rare disease treatments to as little as 100 days is four days away, interest is growing among the medical community, patients, and the pharmaceutical industry.
Because the first pilot will select only up to five drugs, attention is on which drugs will make the final list. Since the aim is to supply patients with needed treatments quickly, patients and specialists are on high alert.
◇ Earlier access to treatment opportunities through streamlined procedures
According to the Ministry of Health and Welfare and the Health Insurance Review & Assessment Service (HIRA) on the 27th, applications for drugs in the "pilot program for expedited listing of rare disease treatments" will close at 6 p.m. on the 31st. The ministry said it will complete evaluations and finalize target drugs in September.
The core of the program is to reduce the overall time it takes for rare disease treatments to be listed for health insurance coverage from the current maximum of 240 days to within 100 days.
Health insurance listing is the process that applies health insurance to a drug so patients pay only part of the price and can receive treatment.
Rare disease treatments have been somewhat sidelined by the system. Because patient numbers are small, it is hard to accumulate sufficient evidence through clinical trials, and because prices are high, these drugs often lose out in cost-effectiveness assessments, which are the main criterion for deciding on reimbursement. In light of these limits, the government launched the pilot program.
For health authorities, it is a bold policy shift.
Under the existing system, drugs are listed for health insurance only after a reimbursement appropriateness assessment by the Health Insurance Review & Assessment Service (HIRA) and a price (drug price) negotiation with the National Health Insurance Service.
The expedited listing pilot for rare disease treatments will first supply treatments to patients and then confirm effectiveness and safety in real-world clinical settings—an "list first, evaluate later" approach. HIRA's reimbursement appropriateness review period will be reduced from up to 150 days to one month, and the drug price negotiation process will be skipped.
The ministry explained it will comprehensively evaluate the presence of alternatives, disease severity, the impact on the health insurance budget, and plans to ensure stable patient access, and then select target drugs within a range of five for the pilot.
◇ Wynrevaer, Iqirvo, Viltepso among those mentioned as candidates
In the medical community, Korea MSD's pulmonary arterial hypertension treatment "Wynrevaer," Ipsen Korea's primary biliary cholangitis treatment "Iqirvo," and the Duchenne muscular dystrophy treatment "Viltepso," for which SK Chemicals holds domestic marketing rights, are being cited as major candidate drugs for this pilot.
Pulmonary arterial hypertension (PAH) is a disease in which the pulmonary vessels narrow, raising pulmonary artery pressure and burdening the heart. Wynrevaer (ingredient sotatercept) was shown in clinical trials to improve exercise capacity and reduce the risk of death or clinical worsening, prompting patients to call for swift reimbursement.
Choi Jeong-hyun, a cardiology professor at Pusan National University Hospital and planning director at the Korean Society of Pulmonary Hypertension, said, "Pulmonary arterial hypertension carries a high risk of sudden death, and without proper treatment the average survival is only three years; it is a severe, progressive rare disease," adding, "In Korea, many patients are women in their 30s to 50s who must juggle work and childrearing, and the longer reimbursement is delayed, the more they can miss the 'golden time' for treatment."
The Korean Society of Pulmonary Hypertension and the Korea Pulmonary Hypertension Patient Association issued a statement urging health authorities and Korea MSD to grant reimbursement for Wynrevaer and to join the expedited listing pilot. Korea MSD said, "We are reviewing an application to participate in the pilot."
Primary biliary cholangitis (PBC), the indication for Iqirvo (ingredient elafibranor), is a disease in which small bile ducts in the liver are gradually damaged. Because a fair number of patients do not respond adequately to the standard therapy ursodeoxycholic acid (UDCA), the field is seen as in need of additional treatments.
Duchenne muscular dystrophy (DMD), the target of Viltepso (ingredient ataluren), is a genetic disease in which abnormalities in the production of the muscle protein dystrophin lead to progressively declining muscle strength. Viltepso works by helping restore normal protein production in patients whose protein synthesis is halted by specific gene mutations.
◇ Drugmakers weighing pros and cons; effectiveness of the system remains a task
For patients and physicians, expectations are high for the pilot because it could move up the start of treatment. Drugmakers, however, have more complex considerations.
An employee at a pharmaceutical company said, "The biggest appeal of the expedited listing pilot is the ability to supply treatments to patients quickly and bring forward market entry, but there are also burdens, so there is keen interest among industry workers in how many companies will apply."
Corporations cited drug price setting and post-listing evaluation requirements as burdens in the pilot.
The government has also put in place safeguards to protect the health insurance budget while improving patient access. To qualify for the pilot, a drug must already be listed in at least three of eight foreign countries (A8: the United States, the United Kingdom, Germany, France, Italy, Switzerland, Japan, and Canada). Selected drugs will have their prices set without negotiation at 90% of the adjusted lowest price among the A8. After listing, real-world treatment outcomes can be assessed to adjust prices and reimbursement conditions.
An employee at a pharmaceutical company said, "Given the nature of rare diseases, the small number of patients can make it hard to secure sufficient domestic clinical data," adding, "There can be pressure because the authorities can judge the results inadequate in a post-listing review and adjust prices or reimbursement conditions."
There is also skepticism in the industry about how effective the system will be. A previous "parallel approval-review-negotiation pilot" introduced to shorten the time for new drugs to enter health insurance coverage took considerably longer than intended to reach listing and reimbursement, making it crucial to see how well the new pilot actually works.
As a result, drugmakers are gauging the pros and cons and watching one another until the last minute before applying. An executive at the Korean unit of a global pharmaceutical company said, "We are busy conducting internal reviews and consulting with global headquarters ahead of the application deadline for the pilot."
Jung Eun-kyeong, Minister of Health and Welfare, said, "The purpose of this pilot is to streamline listing procedures for rare disease treatments and supply patients with needed therapies quickly," adding, "We plan to verify effectiveness and operating methods through the pilot and then move to institutionalize it."