Evidence map›Paper›PMID 38796677›Full record

ArticleClinical drug investigation2024

An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom.

Pablo E Bretos-Azcona, Matthew Wallace, Murvin Jootun, Guanyi Jin, Ion Agirrezabal, Agota Szende

Abstract read
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In one paragraph

Article in Clinical drug investigation, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–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. Review
  2. Propionic acidemia in Mexico: Clinical and genotypic spectrum.Molecular genetics and metabolism reports · 2026
    Article
  3. Review
  4. Review
  5. Article
  6. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors.

Pablo E Bretos-AzconaProduct Development and Market Access Consulting, Fortrea, 4 Maguire Street, London, SE1 2NQ, UK.
Matthew WallaceProduct Development and Market Access Consulting, Fortrea, 4 Maguire Street, London, SE1 2NQ, UK.
Murvin JootunProduct Development and Market Access Consulting, Fortrea, 4 Maguire Street, London, SE1 2NQ, UK.
Guanyi JinProduct Development and Market Access Consulting, Fortrea, 4 Maguire Street, London, SE1 2NQ, UK.
Ion AgirrezabalProduct Development and Market Access Consulting, Fortrea, 4 Maguire Street, London, SE1 2NQ, UK.
Agota SzendeProduct Development and Market Access Consulting, Fortrea, 4 Maguire Street, London, SE1 2NQ, UK. agota_szende@hotmail.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND AND

objectiveNovel messenger RNA (mRNA)-based therapies, currently in development, are emerging as a promising potential treatment modality for a broad range of life-threatening and life-limiting inherited liver diseases, including methylmalonic acidemia (MMA) and propionic acidemia (PA). However, owing in part to their complexity, they are likely to come at considerable financial cost to healthcare systems. The objective of this research was to synthesize available evidence on the costs and clinical consequences associated with MMA and PA for the purpose of exploratory economic evaluation of novel mRNA-based therapies using an early cost-utility model from the United Kingdom payer perspective.

methodsA Markov model was constructed to simulate the costs and outcomes associated with novel mRNA therapies, compared with a combination of dietary management and organ transplantation (standard of care) among hypothetical cohorts of new-born patients with MMA and PA. Key model drivers were identified, and a price threshold analysis was performed to estimate value-based price ranges for future mRNA therapies given willingness-to-pay thresholds for orphan diseases.

resultsmRNA therapy was associated with an additional 5.7 and 1.3 quality-adjusted life-years (QALYs) gained per patient lifetime among patients with MMA and PA, respectively. Key drivers of cost-effectiveness were relative improvement in utility among patients who receive mRNA-based therapy and transplantation, and the cost of mRNA therapy. Assuming a willingness to pay range of £100,000-£300,000 per QALY gained, the model demonstrated mRNA therapy to be cost-effective in MMA and PA at an annual treatment cost of £70,452-£94,575 and £31,313-£36,695, respectively.

conclusionsDespite the lack of a strong evidence base in MMA and PA, this model provides a useful tool to estimate the cost-effectiveness, and inform value-based pricing, of new mRNA-based therapies. Our analyses also identified areas for research that will have the greatest value in reducing uncertainty in future health economic evaluations of such treatments.

Indexed as

Amino Acid Metabolism, Inborn ErrorsCost-Benefit AnalysisMarkov ChainsPropionic AcidemiaQuality-Adjusted Life YearsRNA, MessengerGenetic TherapyHumansModels, EconomicUnited KingdomRNA, Messenger

Identifiers

PMID38796677

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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.