Japan’s Ministry of Health, Labour and Welfare (MHLW) is considering adopting cost-effectiveness focused health-technology assessments (HTA) to re-price a range of existing pharmaceutical treatments. Chuikyo (the Central Social Insurance Medical Council) have been tasked with developing a list of pharmaceutical products suitable for trial HTA evaluations by March 2016.
The selection criteria will focus on products with larger than expected peak sales projections that were priced either by the cost-calculation method (for drugs with no relevant comparator) or those who earned additional premiums over comparators at launch. Orphan drug treatments and state designated diseases (haemophilia, HIV) are likely to be excluded from these trial HTA evaluations. The cost-effectiveness HTA evaluations will be initiated in April 2016, with the entire assessment process expected to last at least two years. These trial HTA evaluations follow-on from an earlier HTA trial in 2014 which investigated the cost-effectiveness of five listed treatments, the results of which have yet to be made public.
The methodology to be used within the cost-effectiveness HTA evaluations is still unclear. It is likely to be
based on a cost per QALY (quality-adjusted life years) as the main efficacy measure, but other criteria,
such as life year; quality-of-life values; cure rate and mortality rate are also likely to be considered
depending on the characteristics of specific diseases and treatments. It is unclear if lost productivity or
public spending on medicines or nursing will be incorporated into the health economic analysis.
The HTA evaluation process will be divided into four phases:
- Company data submission
Pharmaceutical companies will be obliged to submit an HTA dossiers for treatments that are
selected according to pre-determined criteria (criteria have yet to be announced). - Analysis of company data
The company data will then be analysed to ensure that the submission is robust. There are
currently three potential bodies have been identified for conducting this analysis namely: the
MHLW; a dedicated body within an existing organisation or the establishment of a new entity. - Appraisals
The HTA body who will conduct the appraisal of HTA submissions (yet to be determined) will be
made up of healthcare professionals, insurers, patient representatives, and health economists.
Chuikyo representatives may also be included in the HTA organisation with a specific focus on
drug costs. The HTA appraisal meetings will closed to the public. It is also likely that the findings
from the trial HTA evaluations will not be made public, similar to that of the 2014 trial HTA
findings. - Decision-making on appraisal results
Whilst the HTA trial is focused on re-pricing of listed medicines, the same process could be used
to set reimbursed prices for new products in the future.
Pharmaceutical companies have raised a number of concerns over these proposed cost-effectiveness HTA
assessments. The major concern is related to expense and expertise required to develop such HTA
dossiers. It has been estimated that under 5% of pharmaceutical firms currently include health economic
data for reimbursement price negotiations in Japan. This indicates that additional staff and significant
training would be required to ensure companies can develop and submit health economic focused HTA
submissions.
Another concern relates to the difficulties in collecting relevant data for the health economic
analysis from the Japanese population. To address this, the MHLW has announced the establishment of a
patient data collection programme with the aim to make clinical trial processes more efficient and to
collect relative data for HTA submissions.
The introduction of HTA within Japan has been long-anticipated, and whilst there is still much debate over
the exact HTA process and cost-effectiveness criteria that will be used, it has been confirmed a new HTA
trial will be initiated in 2016. The announcement of another HTA trial within Japan is a clear indicator that
the Japanese authorities now consider HTA as the most appropriate way to determine the repricing of
launched products and potentially pricing of new drugs. It is likely that these HTAs will be closed to the
public, unlike the NICE process, and it is uncertain if the results will be published or how they will be used
in price negotiations.
This will make it difficult for pharmaceutical companies to interact in the HTA evaluation and to learn from previous companies HTA evaluations. It will be important for pharmaceutical companies operating in Japan to ensure that they have the internal expertise and resources to develop these HTA submission documents to ensure that the price of their product reflects their value within the
Japanese setting.