Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China

Li, Xiaoqiao; Peng, Xiaoxia; Gong, Chunxiu

doi:10.4103/0366-6999.241814

Cited by 3 publications

(2 citation statements)

References 8 publications

(8 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…These disadvantages include: the lack of drug development efforts before incentives were installed [ 97 , 117 , 173 , 175 , 229 ]; disadvantages in traditional cost-effectiveness procedures [ 43 ], as Quality-Adjusted Life Years (QALY) measures would disadvantage people with chronic disabilities [ 73 , 210 ]; the lack of public awareness; and difficulties in collecting effectiveness evidence for OMPs [ 165 ]. (5) A final justification for why rare disease patients were worse off is that OMPs were too expensive to be paid by patients themselves and therefore should be reimbursed [ 46 , 51 , 61 , 116 , 118 , 123 , 133 , 138 , 147 , 161 , 163 , 183 , 199 – 201 , 204 , 208 – 210 , 232 – 234 ]. It was also argued that rare disease patients already carried a substantial financial burden as compared to the general population [ 209 , 210 , 233 ], including indirect costs for transportation to specialized facilities or the inability of family caregivers to be in full-time employment [ 123 ].…”

Section: Resultsmentioning

confidence: 99%

A systematic review of moral reasons on orphan drug reimbursement

Zimmermann

Eichinger

Baumgartner

2021

Orphanet J Rare Dis

View full text Add to dashboard Cite

Background The number of market approvals of orphan medicinal products (OMPs) has been increasing steadily in the last 3 decades. While OMPs can offer a unique chance for patients suffering from rare diseases, they are usually very expensive. The growing number of approved OMPs increases their budget impact despite their low prevalence, making it pressing to find solutions to ethical challenges on how to fairly allocate scarce healthcare resources under this context. One potential solution could be to grant OMPs special status when considering them for reimbursement, meaning that they are subject to different, and less stringent criteria than other drugs. This study aims to provide a systematic analysis of moral reasons for and against such a special status for the reimbursement of OMPs in publicly funded healthcare systems from a multidisciplinary perspective. Results With a systematic review of reasons, we identified 39 reasons represented in 243 articles (scientific and grey literature) for and against special status for the reimbursement of OMPs, then categorized them into nine topics. Taking a multidisciplinary perspective, we found that most articles came from health policy (n = 103) and health economics (n = 49). More articles took the position for a special status of OMPs (n = 97) than those against it (n = 31) and there was a larger number of reasons identified in favour (29 reasons) than against (10 reasons) this special status. Conclusion Results suggest that OMP reimbursement issues should be assessed and analysed from a multidisciplinary perspective. Despite the higher occurrence of reasons and articles in favour of a special status, there is no clear-cut solution for this ethical challenge. The binary perspective of whether or not OMPs should be granted special status oversimplifies the issue: both OMPs and rare diseases are too heterogeneous in their characteristics for such a binary perspective. Thus, the scientific debate should focus less on the question of disease prevalence but rather on how the important variability of different OMPs concerning e.g. target population, cost-effectiveness, level of evidence or mechanism of action could be meaningfully addressed and implemented in Health Technology Assessments.

show abstract

Section: Resultsmentioning

confidence: 99%

A systematic review of moral reasons on orphan drug reimbursement

Zimmermann

Eichinger

Baumgartner

2021

Orphanet J Rare Dis

View full text Add to dashboard Cite

show abstract

“…Some papers enlighten persistent difficulties. For example, in 2018, a case report of two brothers with NC living in China and having no access to cysteamine treatment was published, seeking help from international organizations to obtain the drugs [ 20 ]. Since 2018, China seems to have access to cysteamine, as appears from the current survey.…”

Section: Discussionmentioning

confidence: 99%

Worldwide disparities in access to treatment and investigations for nephropathic cystinosis: a 2023 perspective

Regnier,

Flammier,

Boutaba

et al. 2023

Pediatr Nephrol

View full text Add to dashboard Cite

Background Nephropathic cystinosis (NC) is a rare lysosomal disease, leading to early kidney failure and extra-renal comorbidities. Its prognosis strongly relies on early diagnosis and treatment by cysteamine. Developing economies (DEing) face many challenges when treating patients for rare and chronic diseases. The aim here is to evaluate the access to investigations and treatment in DEing, and to assess for potential inequalities with Developed Economies (DEed). Methods In this international cross-sectional study, a questionnaire on access, price and reimbursement of genetic, biological analyses, and treatment was sent to nephrology centers worldwide during 2022. Results A total of 109 centers responded, coming from 49 countries and managing 741 patients: 43 centers from 30 DEing and Economies in transition (TrE), and 66 from 19 DEed. In 2022, genetics availability was 63% in DEing and 100% in DEed, whereas intra leukocytes cystine levels (IL-CL) were available for 30% of DEing patients, and 94% of DEed patients, both increasing over the last decade, as has access to immediate release cysteamine and to cysteamine eye drops in DEing. However, delayed released cysteamine can be delivered to only 7% vs. 74% of patients from DEing and DEed, respectively, and is still poorly reimbursed in DEing. Conclusions Over the last decade, access to investigations (namely genetics and IL-CL) and to cysteamine have improved in DEing and TrE. However, discrepancies remain with DEed: access to delayed released cysteamine is limited, and reimbursement is still profoundly insufficient, therefore limiting their current use. Graphical abstract

show abstract

Analysis on the Marketing Trend and Approval Lag of Imported Orphan Drugs from 2010 to 2021 in China

Peng,

Zheng

2023

Ther Innov Regul Sci

View full text Add to dashboard Cite

Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China

Cited by 3 publications

References 8 publications

A systematic review of moral reasons on orphan drug reimbursement

A systematic review of moral reasons on orphan drug reimbursement

Worldwide disparities in access to treatment and investigations for nephropathic cystinosis: a 2023 perspective

Analysis on the Marketing Trend and Approval Lag of Imported Orphan Drugs from 2010 to 2021 in China

Contact Info

Product

Resources

About