Literature review

EVALUATION OF TECHNICAL NOTES ON THE SUPPLY OF MIGLUSTATE FOR TREATMENT OF NIEMANN PICK TYPE C DISEASE

How to cite: Zuza RS, Miziara CSMG, Miziara ID. Evaluation of technical notes on the supply of miglustate for treatment of Niemann Pick type C disease. Persp Med Legal Pericias Med. 2022; 7: e221220

https://dx.doi.org/10.47005/221220

Submitted 11/01/2022
Accepted 12/17/2022

The authors inform no conflict of interest.

AVALIAÇÃO DAS NOTAS TÉCNICAS SOBRE O FORNECIMENTO DE MIGLUSTATE PARA TRATAMENTO DE DOENÇA DE NIEMANN PICK TIPO C

Ricardo dos Santos Zuza (1)

https://orcid.org/0000-0001-5543-8104

Carmen Silvia Molleis Galego Miziara (2)

https://orcid.org/0000-0002-4266-0117

Ivan Dieb Miziara (2)

https://orcid.org/0000-0001-7180-8873

(1) Residente do 3º ano de Medicina Legal e Perícias Médicas na USP

(2) Coordenador da residência de Medicina Legal e Perícias Médicas na USP

ABSTRACT

Introduction: This article aims to survey the Technical Notes on the obligation to supply the drug Miglustat for Niemann Pick type C disease. The authors evaluate the bibliography and whether there is mention of the document issued by CONITEC (National Comission on the Incorporation of Technology to Brazil’s Unified Health System), the issuing body, and whether the urgency of the request was considered justifiable. Method: Search at Natjus websites of the term ‘’Miglustat’’. Duplicate notes or notes that addressed other diseases were excluded. Results: 23 Notes were found, 14 of which were favorable and 9 were not favorable. As for urgency, 9 notes considered that there was urgency and 9 concluded there was none, while 4 did not approach urgency. In 18 notes the CONITEC document was mentioned, while it was not mentioned in 5. We found 2 articles with a high degree of evidence, published after 2019, on the topic. Discussion: It was to be expected that the technical notes favorable to the release would present studies not evaluated by CONITEC, with new information. When reading the bibliography, only note 01/2021 cites these articles. None of the other 20 notes, favorable or not to the release, cite the articles, while 2 notes do not present a bibliography. Conclusion: This survey indicates a lack of standardization in the preparation of technical notes, with notes on the same subject altered according to the issuing entity. The bibliographic survey of the notes appears to be flawed by not discussing works with a high level of evidence regarding survival and therapeutic gains. Further work on technical notes is needed.

Keywords: Technical notes, miglustat, Niemann Pick disease type C.


Bibliographical references

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