Evidence map›Paper›PMID 39736792›Full record

ArticleOrphanet journal of rare diseases2024

Risdiplam utilization, adherence, and associated health care costs for patients with spinal muscular atrophy: a United States retrospective claims database analysis.

Anish Patel, Walter Toro, Min Yang, Wei Song, Raj Desai, Mingchen Ye, Nadia Tabatabaeepour, Omar Dabbous

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Trial
  3. Review
  4. Article
  5. Article
  6. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Anish PatelNovartis Gene Therapies, Inc., 2275 Half Day Road, Suite 200, Bannockburn, IL, 60015, USA. abs1@kaysquare-sci.com.
Walter ToroNovartis Gene Therapies, Inc., 2275 Half Day Road, Suite 200, Bannockburn, IL, 60015, USA.
Min YangAnalysis Group, Inc., 111 Huntington Ave, Floor 13, Boston, MA, 02199, USA.
Wei SongAnalysis Group, Inc., 111 Huntington Ave, Floor 13, Boston, MA, 02199, USA.
Raj DesaiAnalysis Group, Inc., 111 Huntington Ave, Floor 13, Boston, MA, 02199, USA.
Mingchen YeAnalysis Group, Inc., 111 Huntington Ave, Floor 13, Boston, MA, 02199, USA.
Nadia TabatabaeepourAnalysis Group, Inc., 650 California Street, Floor 23, San Francisco, CA, 94108, USA.
Omar DabbousNovartis Gene Therapies, Inc., 2275 Half Day Road, Suite 200, Bannockburn, IL, 60015, USA.

Funding

Novartis Gene Therapies Novartis Gene Therapies
6 · The paper itself

Abstract

backgroundSpinal muscular atrophy (SMA) is a genetic neuromuscular disease associated with progressive loss of motor function. Risdiplam, a daily oral therapy, was approved in the United States for the treatment of SMA. Risdiplam's effectiveness depends on patient adherence to the treatment regimen. This retrospective claims database analysis assessed real-world treatment adherence and persistence, and all-cause health care costs by adherence status, for patients with SMA receiving risdiplam. Outcomes were summarized by SMA types (1-4) and age groups (0-2, 3-5, 6-17, and ≥ 18 years).

results86 patients with ≥ 1 SMA diagnosis, risdiplam treatment, and ≥ 6 months of continuous enrollment after the index date (SMA diagnosis) were identified in the IQVIA PharMetrics

conclusionsNonadherence to risdiplam was observed in the first year of treatment, especially for patients with SMA type 4 and patients aged 6-12 years. Nonadherence was associated with higher all-cause health care costs, with the most pronounced cost difference for SMA type 2. For adherent patients, those who were highly adherent incurred lower health care costs. These findings underscore the importance of treatment adherence and persistence for patients with SMA receiving risdiplam, particularly for younger children and those with greater disease severity.

Indexed as

Health Care CostsMuscular Atrophy, SpinalAdolescentAdultAzo CompoundsChildChild, PreschoolDatabases, FactualFemaleHumansInfantInfant, NewbornMalePyrimidinesRetrospective StudiesUnited StatesAzo CompoundsPyrimidinesRisdiplamAdherenceClaims database analysisHealth care costsHealth care utilizationPersistenceRisdiplamSpinal muscular atrophyUnited States

Identifiers

PMID39736792
PMCPMC11684252

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.