Evidence map›Paper›PMID 39614329›Full record

ReviewOrphanet journal of rare diseases2024

Off-label use of medicines in South Africa: a review.

N N Ngcobo, L J Mathibe

Abstract readReview
In one paragraph

Review 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 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Optimizing Pediatric Drug Therapy: Pediatric Pharmacokinetics and Administration Routes.The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG · 2026
    Article
  3. 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

2 authors.

N N NgcoboDiscipline of Pharmaceutical Sciences, School of Health Sciences, University of KwaZulu-Natal, Durban, South Africa. ngcobon11@ukzn.ac.za.ORCID 0000-0003-0513-991X
L J MathibeDivision of Pharmacology (Therapeutics), Discipline of Pharmaceutical Sciences, Nelson R. Mandela School of Medicine, University of KwaZulu-Natal, Durban, South Africa.ORCID 0000-0003-4739-8944

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundOff-label use of medicinal products has become an important part of mainstream and legitimate medical practice worldwide. This practice is common in oncology, obstetrics, paediatrics, and in the management of infectious diseases (notably HIV), and inflammatory conditions as well as in rare and/or orphan diseases. However, the off-label use of medicines recently-raised many clinical and legal difficulties, not only among medical practitioners but also among pharmacists and other healthcare professionals.

aimThis paper, therefore, highlights the advantages (such as cost saving for both the patient and the country/insurance that is paying for the medication) and disadvantages (insufficient evidence available) of the use of medicines to treat specific conditions or indications for which they are currently not registered.

conclusionOff-label drug use can be likened to a double-edged sword, offering valuable opportunities for medical practitioners while carrying potential risks. When the scientific basis for off-label use is unclear, it may place patients at risk of unapproved experimentation, unforeseen health hazards, and ineffective treatments. Hence, there is a pressing need in South Africa for clear regulatory guidelines on off-label drug use. Additionally, the timely review and approval of new indications for medicines, backed by robust scientific evidence, are essential. This would reduce the significant burden and inherent risks faced by medical practitioners when using medicines off-label to provide compassionate care.

Indexed as

Off-Label UseHumansSouth AfricaEvidence-based medicineFDAOff-labelRare diseasesRegulationsSAHPRA

Identifiers

PMID39614329
PMCPMC11607944

What Socratic holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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 Socratic graph.