Abstract
The objective of this study was to develop the benefit package for diagnosis, treatment/rehabilitation, and prevention for MPS patients under universal coverage scheme. According to the medical record review from 7 rare disease centers, in November 2564 there were 81 MPS patients in Thailand (both alive and unsure status). Of these 10 were MPS I, 35 were MPS II, 11 were MPS III, 13 were MPS IV, 10 were MPS VI, and 2 were MPS VII. Proposed benefit package after stakeholder meetings include the followings; 1) Benefits for diagnosis a. Diagnosis with enzyme activity assay and molecular testing b. Carrier-testing for at-risk family members c. Prenatal diagnosis (in case that the couple desire to have another child) The 5-year budget impact for diagnosis with enzyme activity assay was estimated at 85,714 – 214,286 Baht (Average approximately 17, 143 – 42, 857 Bath per year) while those of molecular testing, carrier testing for at-risk family members, and pre-natal diagnosis were 450,000 Baht (average 90,000 baht per year), 300,000 Baht (average 60,000 baht per year), and 75,000 Baht (average 15,000 baht per year), respectively. 2) Benefit for supportive treatment. We proposed continuous Positive Airway Pressure (CPAP) or Bilevel Positive Airway Pressure (BiPAP) for MPS patients who were in medical need. This is based on the evidences that MPS patients are at risk of severe respiratory insufficiency and these patients will get benefit from CPAP and BiPAP. This was also consistent with our interview, which found that MPS patients were in need for CPAP/BiPAP. However, both CPAP/BiPAP are not included in the benefit package. The estimated budget for inclusion of CPAP/BiPAP for MPS patients is approximately 300,000-750,000 Baht for the first year (estimate budget for year 2 onwards is approximately 40,000 to 100,000 Baht annually). 3) Benefit related to treatment system. We proposed that affected patients can receive treatment and monitoring at rare disease centers without the need for referral for every visit. We also proposed for the development of co-ordinate care so that patients could receive routine and continuous care at the local hospitals. This was based on the fact that patients with MPS requires continuous care while the number of geneticists were limited and mostly available at rare disease centers. According to the interviews, patients were facing with difficulty in transporting to get treatment. 4) Benefit related to specific treatment with HSCT: We proposed the development of the payment mechanism that reimbursed for HLA Matching cost before translation. The new payment mechanism is expected to improve patients access to HSCT care. 5) Benefit related to specific treatment with ERT Our rapid review indicated that the efficacy of ERT in terms of mortality reduction, quality of life, and important clinical outcomes were unclear and that most studies were of moderate to low quality due to small sample size. Due to the challenges in conducting studies in very limited number of patients, most studies had no comparison group and emphasized on the change on surrogate outcome after short term use of ERT. While MPS is a rare disease that profoundly affects socio-economic status, and quality of life of patients and family and that ERT is the only specific treatment available (besides HSCT) treatment with ERT is very costly. During the 2nd and 3rd stakeholder meetings, it was suggested to firstly propose ERT for MPS II into the benefit package as ERT for MPS II is currently under consideration of National List of Essential medicine committee and that there is a proposed criteria for starting, stopping, and monitoring ERT treatment for MPS II. On the other hand, ERT for MPS VI and VII are not currently approved in Thailand. Based on the expert opinion, the use of ERT among MPS II can delay disease progression and improve quality of life of the patients. Therefore, ERT for MPS II is proposed to be included in the benefit package. Nevertheless, there should be an expert committee to effectively monitor its use. If government could manage the budget in the future, the stakeholders propose to include ERT for treatment of MPS I, IV, VI, and VII, respectively.
