Need to specify conditions for postponing PE data submission

Reevaluation of listed PE exemption drugs is necessary

HRIRA discloses research service results...Proposes to exclude 'pediatric drugs' whose indication was extended a year ago

Study on Improving the Pharmacoeconomic Evaluation Data Waiver (PE exemption) System

The results of the research service that was ordered by the Health Insurance Review and Assessment Service to devise measures on improving the pharmacoeconomic data submission waiver system, or the PE exemption system, have been disclosed in full.

 

The improvements suggested by the researchers include redesigning the PE exemption system into a PE deferral system, and establishing a process to demonstrate economic feasibility after listing.

 

It was also suggested that a reevaluation system should be established for drugs that have already been listed through the PE exemption track.

 

Based on the study, HIRA plans to come up with a follow-up and reevaluation plan for the PE exemption drugs.

 

According to the results of the ‘Study on Improving the PE evaluation data waiver system (Seoul National University R&DB Foundation, Professor Tae-jin Lee, Principal Investigator)' that was released on the 3rd, ithe PE exemption system needs to be redesigned into a system that defers proof of economic feasibility for drugs when necessary, rather than a system for waiving submission of PE evaluation data overall.

 

The PE exemption system was introduced in May 2015 and applied to 26 drugs until July 2022.

 

Explaining the background of their proposal, the researchers said, "The introduction of the PE exmpetion system has had some positive effects on improving patient access, such as by improving the rate of new drug listings and shortening the listing period.

 

However, when considering the various characteristics of subject drugs, there seems to be a high need for the PE exemption drugs to be managed within the basic principles of the positive listing system that is based on the demonstration of cost-effectiveness.” In this regard, the research team proposed ▲ redesigning the system into an economic feasibility demonstration deferral system ▲ establishment a post-listing economic feasibility demonstration process through a specific agreements on items that need to be clarified in advance ▲ establishment of a post-marketing and reevaluation system based on cost-effectiveness evaluations ▲ and others, such as increasing the practicality of total expenditures by setting a set amount for each disease unit or an expenditure cap and setting a reasonable baseline for the evaluation amount based on foreign drug price.

 

The researchers also saw the need to reestablish the current PE exemption conditions.

 

More specifically, the condition, ‘anticancer drugs or rare diseases for which no substitute or therapeutically equivalent product or treatment exist,' needs to be specified.

 

On this, the researchers proposed limiting the condition to ‘rare disease drugs or anticancer drugs used for serious conditions that threaten survival, such as those for diseases with a life expectancy of less than 2 years,’ and to set specific requirements such as ‘drugs that bring a significant clinical improvement over nontreatment and has no other alternative treatment,’ or ‘provide a significant clinical improvement over existing treatments, such as a significant prolongation of survival.’ The reseachers also believed that the current grounds for lacking evidence were also inadequate.

 

The current requirement is: drugs for a small number of patients, ▲ that were approved with single-arm clinical data without a control group, ▲ approved with a Phase II clinical trial that has a control group but without a conditional Phase III trial, or ▲ have other difficulties in producing evidence.

 

More specifically, the researchers proposed ▲ cases where no clinical trial with a control group has been conducted and indirect comparison is difficult, ▲cases where PE evaluations have not been conducted in other countries as well, ▲cases where it is difficult to confirm the final effect due to immature clinical data, and it is not appropriate to conduct PE evaluation by estimating the final result through modeling.

 

However, ▲if the size of the required finances is above a certain level, economic feasibility must be demonstrated.

 

Also, the researchers pointed out that the requirement of a ‘small number of patients’ should also be revised to a ‘diseases with a prevalence 200 or less patients per independent condition.' In addition, the researchers claimed that drugs used for pediatric patients and tuberculosis treatments, which were added as PE exemption drugs last year, should be removed again as drugs that are not considered to be rare diseases but are life-threatening were waived PE data submissions even before the revisions were made.

 

The researchers concluded that real-world evidence (RWE) can be recognized as a source of evidence after listing.

 

They also suggested the need for a collective reevaluation of the listed PE exemption drugs.

 

"In the case of PE exemption drugs they have already been listed, all other HTA-based countries have evaluated their economic feasibility after listing.

 

Therefore, it is necessary to reevaluate the drugs even if their control period has been completed to manage the appropriate listing price of latecomers that will be listed using the PE exemption drugs as a comparator."