MOHW's view of ‘few’ and ‘pediatric’ for PE exemptions

“Will flexibly apply the system in drugs used by around 200 people”

“Difficult to apply PE exemptions only to pediatric patients for all-age indications”

Chang-Hyun Oh, Director of Pharmaceutical Benefits at the Ministry of Health and Welfare

Although yet to be finalized, the PE exemption criteria will not be amended' in the direction desired by pharmaceutical companies.

 

In particular, companies are likely to have little room in utilizing the special exemption of the pharmacoeconomic evaluation system due to the restrictions set, such as treatments that are used by ‘few’ patients and those with ‘pediatric’ indications.

 

The period for industry opinion collection still remains, with the deadline being the 30th.

 

But as the government is determined to implement the amendments to the system, to what extent the opinions will be reflected remains to be seen.

 

Chang-Hyun Oh, Director of Pharmaceutical Benefits at the Ministry of Health and Welfare, said so regarding the “Measures to Improve Patient Access and Manage Reimbursement of High-Priced Severe Disease Treatments" at a recent meeting with the multinational pharmaceutical company press gallery.

 

The ‘Few’ patients standard to be maintained at a 200-patient level The government's improvement plan raised industry concerns because it changes the major premise set for PE exemptions.

 

The new change adds ‘few numbers of subject patients’ as a prerequisite for PE exemptions.

 

Originally, the condition was a ‘OR’ clause included with other optional clauses such as ‘if a single-arm study was conducted,’ under Subparagraph 2(c) of Regulation for Drugs Subject to PE Exemptions.

 

The ‘few’ was defined to be in the 200 range.

 

Although the exact number has not been specified in the condition, the PE exemption evaluations has been made at that level.

 

Therefore, there were many opinions in the industry that the number defined as a ‘few’ should be adjusted upward if the condition was to become a prerequisite.

 

However, Director Oh said, “We don’t plan to change the standards we set to define the ‘few’ just because of the amendment.

 

However, no specific number is universally applied to all drugs, therefore, the number may be flexibly adjusted to a small extent for each drug in the future." In other words, if the amendment is applied as is, all drugs that seek PE exemptions will first have to satisfy the 'few numbers of patient' (around 200) requirement, then receive recognition from the committee for their difficulty to generate PE data.

 

What about drugs that have broader indications that are applied to pediatric to adult patients? The main point of the system improvement being made was to improve coverage for pediatric patients.

 

The government had added the clause 'drugs used to treat pediatric patients that are therapeutically equivalent or has no available treatment option, and demonstrates improvement in quality of life or is otherwise approved by the committee.' In essence, the added clause excluded the 'life-threatening disease' condition for PE exemptions just for pediatric patients.

 

In other words, if drugs used for pediatric patients satisfy the remaining conditions, the PE exemption may be applied even if the condition is not life-threatening.

 

On the surface, it seems like an encouraging improvement.

 

However, there were wild speculations in the industry on what the scope of 'drugs used in pediatric patients' will be.

 

The government had not specified whether the 'drugs used to treat pediatric patients' clause applies only to diseases that occur only in children, drugs that only have indications for children, or drugs that can be prescribed broadly to children and adults.

 

However, it is likely that this clause will also not be widely applied.

 

Director Oh said, “It is difficult to provide a clear answer on that part.

 

However, the system was not intended to limit the indication of drugs that can be used for even the adults to only children so that they can take the PE exemption track."