[Report: recommendations concerning the information required to monitor nusinersen use in real-world settings]

Tremblay É
Record ID 32018000903
French
Original Title: Avis: recommandations sur les informations requises pour le suivi de l’usage du nusinersen en contexte de vie réelle
Authors' objectives: The Ministère de la Santé et des Services sociaux asked INESSS to determine the minimum information required to monitor nusinersen use and the minimum frequency at which this information should be gathered for presymptomatic individuals and those with type I, II or III. Ultimately, clinical monitoring will make it possible, among other things, to document the impact of nusinersen on their ability to maintain a functional status deemed clinically meaningful, their quality of life and their life expectancy.
Authors' results and conclusions: RESULTS: The minimum information required to assess the impact of nusinersen on motor function should be obtained using one or more of the following motor function measurement tools, depending on the patient’s age and functional status: the HINE Section 2, the CHOP INTEND (or the CHOP ATEND), the HFMSE, the RULM, or the 6MWT. However, more subtle questioning of the patient or his/her caregiver will be used to record, in the space marked "Other motor skills", information not obtained with one of the recommended measurement tools. The minimum information required to assess the impact of nusinersen on quality of life should be obtained using the tools PedsQL 4.0 and 3.0 for patients 24 months of age or older, along with the SMAFRS, a measurement tool for assessing functional capacity, and qualitative data on patients’ (or a caregiver’s) and health professionals’ perceptions. The minimum information required to assess the impact of nusinersen on motor function, quality of life, functional capacity, patients’ and health professionals’ perceptions, and perceptions of respiratory function and of previous or current treatments administered to modify the course of the disease should be gathered at least at the start of treatment and then every 12 months. Additional information regarding quality of life and functional capacity, such as the use of ventilation support or a gastrointestinal tube, scoliosis surgery or, simply, hospitalization, should be gathered every 12 months. Information for documenting the impact of nusinersen on the life expectancy of SMA patients should be gathered using the date of death, if applicable. The nusinersen stop date should also be recorded, if applicable. Information on nusinersen administration and tolerance of this drug should be gathered at each administration. Additional procedures performed during its administration should be recorded at the time of each administration in order to document the organization of care and services. Lastly, information for profiling each nusinersen user should be gathered at the time of the first dose of the drug.
Authors' recomendations: In December 2018, INESSS recommended to the Minister that a recognized indication for nusinersen be added to the List of Medications – Institutions for the treatment of children with presymptomatic 5q SMA and that the recognized indication for the treatment of patients with 5q SMA type I be modified to include types II and III of the disease, if its use is guided on a recognized indication that promotes appropriate use, if clinical monitoring is put in place and if the manufacturer helps mitigate the economic burden. The recommendations in this report are intended to fulfill one of the conditions set out, namely, providing clinical monitoring of patients with SMA 5q of all types. Thus, recommendations aimed at developing an approach to evaluation in a real-world setting have been drawn up. These recommendations concern the minimum information required for documenting the use of nusinersen in a real-world setting, particularly with regard to certain efficacy and safety parameters, and to its administration and the repercussions on the organization of intrahospital services. The frequency at which health-care institutions should share the information and the measures to be taken to implement this clinical monitoring remain to be defined with the Ministère de la Santé et des Services sociaux.
Authors' methods: For the purposes of this request, we gathered data from the scientific literature, contextual information, and experiential knowledge obtained from consultations with stakeholders, SMA patients and caregivers. A literature review was conducted to glean the data that were used in clinical studies to assess the impact of nusinersen on SMA patients’ motor function and quality of life. To gather contextual information and experiential knowledge, an advisory committee consisting of health professionals and representatives from the Programme de gestion thérapeutique des médicaments (PGTM) was formed. Consultations with SMA patients and caregivers completed the gathering of experiential knowledge. The Canadian Neuromuscular Disease Registry was consulted as well. Triangulating the data from the scientific literature, the contextual information and the experiential knowledge enabled us to develop preliminary recommendations, which were then submitted to the members of the Comité scientifique d'évaluation des médicaments aux fins d'inscription (CSEMI) for deliberation.
Details
Project Status: Completed
Year Published: 2020
Requestor: Minister of Health
English language abstract: An English language summary is available
Publication Type: Full HTA
Country: Canada
Province: Quebec
MeSH Terms
  • Muscular Atrophy, Spinal
  • Spinal Muscular Atrophies of Childhood
  • Oligonucleotides
  • Oligonucleotides, Antisense
  • Adolescent
  • Child
  • Infant
  • Drug Therapy
  • Data Collection
  • Research Design
Keywords
  • Progressive muscle atrophy
  • Oligonucleotides
Contact
Organisation Name: Institut national d'excellence en sante et en services sociaux
Contact Address: L'Institut national d'excellence en sante et en services sociaux (INESSS) , 2021, avenue Union, bureau 10.083, Montreal, Quebec, Canada, H3A 2S9;Tel: 1+514-873-2563, Fax: 1+514-873-1369
Contact Name: demande@inesss.qc.ca
Contact Email: demande@inesss.qc.ca
Copyright: Gouvernement du Québec
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.