In this study, 9 types of RDs with extremely high expenditure burdens were used as research objects and patient data was collected by a field survey. The estimated costs of these 9 diseases and the budget impact under different scenarios in Chengdu in 2019 were estimated, and the budget for the next three years was calculated. To our knowledge, this was the first study in China to investigate the budgets needed for RDs treatment as well as the distribution characteristics of studied RDs based on the field survey instead of using reported data. Budgets estimated through policy scenarios were expected to provide implications for budget as well as potential overdraft risks induced by high-cost OMPs from health care payers’ perspectives. The findings would facilitate the initiation of policy support for RDs.
Coverage of OMPs by social health insurance improved their affordability as well as accessibility, largely reduced the financial burden on RDs patients. However, there are still some high-priced OMPs not being considered in the reimbursement policies 36. In our study, the annual per capita cost for the 9 selected RD types engaged for analysis was found to be CNY 1,890,400, which was 55 times the annual per capita income of Chengdu residents in 2019. Residents were not considered to be in poverty when their annual household income remains higher than CNY 4,726,000. This benchmark value was found to be equivalent to 87.75 years of income for urban households in China, which is even higher than the 69.34 years analyzed in other Chinese studies19. However, it is noteworthy that the lack of epidemiological data had posed huge obstacles in facilitating sustainable financial support for RDs patients. Despite the current healthcare system in China has established a rare disease registry system, it is still at its initial stage, where a standardized reporting protocol for rare disease cases has not been developed. Moreover, rare disease patients could not adhere to long-term treatment plans due to unaffordable costs. This has led to a considerable amount of data being absent from the rare disease registry system. To overcome this limitation, the budget impact analysis was conducted using patient data collected from multiple sources of data. Despite the improved credibility for estimation, the diagnostic yield of RDs engaged in the survey was found to be merely 16.08% in sharp contrast with the international diagnostic yield of 25–50% 27. This suggested there was still huge space for improvement in the diagnostic procedure for RDs in China. The implementation of policies also means healthcare professionals could accumulate knowledge and experiences from treating an increased number of patients, which was expected to improve the diagnostic yield of RDs from a long-term perspective. Based on these considerations, estimations for RDs budgets were conducted under different number of patients, namely prevalence and diagnostic yield 27,28,37,38.
Based on the number of patients engaged in the survey, the estimated budget needed for supporting high-priced OMPs treatments in Chengdu was found to range from CNY 32 million to CNY 156 million in 2019, which accounted for 0.88–4.04% of the total financing of CII. This indicated that the studied RDs merely posed a limited impact on the total CII funding. This could largely alleviate the concerns of the payer that the implementation of such a budget for OMPs for RDs might potentially pose negative impacts on the overall CII financing system. About market analysis, the large population in China has made its nationwide drug market larger than other countries. Meanwhile, the Chinese government has approved the release of OMPs into the drug market which was seen as an essential strategy for attracting more OMPs to China’s drug market while largely facilitating the negotiation between the government and other global pharmaceutical companies. This would reduce the OMPs prices by the participation of more and more pharmaceutical companies in the drug market and through the exchange of market share for price reductions performed at the governmental level39. This would ultimately alleviate the financial burden posed on rare disease patients40. It was beyond controversy that the list of OMPs covered by the budget will be constantly modified with the release of newly authorized OMPs into the drug market. According to the long-term studies in the United States and European countries, the emergence of newly approved drugs was very much unlikely to pose surge pressure on the budgets, while the proportion of OMPs costs to total drug costs was found to be essentially proportional to the proportion of rare disease patients to all patients12,16,41. Based on all this evidence, it could be predicted that from a long-term perspective, the implementation of policy for supporting RDs is very much unlikely to pose a heavy financial burden on the payers. The results of the budget for the next three years indicated that the annual growth rate of the estimated budget was about 19.5%. This evidence alleviated governmental concerns about the heavy financial burden that OMPs for RDs might potentially pose on the budget. Moreover, analysis of the budget required for supporting RDs patients was of significant value to benefit the whole society.
Strictly speaking, if patients need to pay for their treatments out of pocket, there would inevitably be some patients who would have to give up treatments due to financial burden either directly or indirectly. Nevertheless, in this study, the budget required for supporting RDs patients was estimated without considering the potential impacts of drug utilization rates. The main reason was that the actual diagnostic yield for RDs in Chengdu was found to be very low based on our studied population. Therefore, the drug utilization rate has been excluded from the analysis, which has underestimated the risk of overdraft issues induced by underestimation. On the other hand, ideally, shreds of evidence collected from multiple aspects would be needed for budget estimation such as the incidence of adverse drug reactions and patients’ medical service utilization behaviors across regional boundaries. However, all these parameters failed to be included in the analysis due to the lack of clinical evidence collected for RDs and the unavailability of data related to cross-regional medical service utilization.
From a long-term perspective, the diagnostic yield of rare disease cases is expected to be improved by the increased OMPs utilization rates among various rare disease patient groups, while drug price fluctuations will be induced by newly authorized homogeneous OMPs constantly released into the drug market. Therefore, it is highly recommended that longitudinal data should be collected in further studies to investigate how all these parameters would pose potential impacts on the budget of high-priced OMPs for RDs.