Ataluren: where the agencies disagree
This page collects every recorded health technology assessment decision for one molecule across the agencies in our archive, and compares them. The comparison is the point: each agency publishes its own verdict, none publishes how that verdict sits against the others.
What the record shows
AWMSG (Wales) recommended or restricted ataluren, while AOTMIT (Poland), DMC (Denmark) and NYE METODER (Norway), among others, did not recommend it.
On the same population the two systems disagree outright: AWMSG (Wales) recorded recommended for “Treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patien…”, while SMC (Scotland) recorded not recommended for “Treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. The presence of a nonse…”.
Assessment has run over 11.4 years, from 21 Jan 2015 at HAS (France) to 8 Jun 2026 at SMC (Scotland).
TLV (Sweden) changed its position: restricted on 29 Apr 2019, not recommended by 16 Jun 2022.
No assessment is on file from PBAC (Australia), PHARMAC (New Zealand), CIPM (Spain) and G-BA (Germany), each among the eight highest-volume agencies here.
The file is active: 2 of 13 records date from 2023 or later.
Against its own class, ataluren is an outlier: 42% of its decisive outcomes were positive, where molecules sharing ATC class M09A average 73%.
HAS (France) was first to assess ataluren, on 21 Jan 2015; SMC (Scotland) did not open a file until 8 Jun 2026 — an unusually long tail of 11.4 years between the earliest and latest system to look at it.
The record arrives in waves: 3 separate years each carry two or more decisions, spread across 8 years in total.
3 of the 8 agencies here assessed ataluren more than once, HAS (France) most often at 4 entries — repeat review is the norm for this molecule rather than the exception.
8 of the 18 agencies in this archive hold a record for ataluren.
The full ledger for ataluren reads 7 not recommended, 4 recommended, 1 restricted and 1 superseded.
Decisions on file
| Agency | Date | Indication as recorded | Outcome | Source |
|---|---|---|---|---|
| HAS (France) | 23 Oct 2024 | Re-assessment following post-listing study results | SMR insufficient | source |
| NYE METODER (Norway) | 28 Aug 2022 | Behandling av Duchennes muskeldystrofi hos ambulatoriske pasienter | not introduced | source |
| AOTMIT (Poland) | 28 Aug 2019 | Translarna reimbursement recommendation | not recommended | source |
| TLV (Sweden) | 29 Apr 2019 | Translarna ingår i högkostnadsskyddet till och med 30 juni 2022 | restricted subsidy | source |
| AWMSG (Wales) | 28 Mar 2019 | Treatment of Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene, in ambulatory … | superseded (limited) | source |
8 further decisions for ataluren, across 6 agencies, are not shown here. They include the most recent record on file, 8 Jun 2026 from SMC (Scotland); its outcome is in the subscriber dataset. They link to 5 further source documents.
Related molecules
- risdiplam — same ATC class M09A — Other musculoskeletal
- nusinersen — same ATC class M09A — Other musculoskeletal
- onasemnogene abeparvovec — same ATC class M09A — Other musculoskeletal
- givinostat — same ATC class M09A — Other musculoskeletal
- osimertinib — also decided by SMC in 2026Q2
- brentuximab — also decided by SMC in 2026Q2
Coverage: 13 decisions for ataluren from 8 agencies, 2015 to 2026. Data to 7 Sep 2026. ATC class M09A — Other musculoskeletal. How this page is produced. Compiled by Altima Partners; queries: sales@altimapartners.co.uk.