Abstract
Introduction. Autoimmune musculoskeletal diseases such as rheumatoid arthritis, ankylosing spondylitis, and psoriatic arthritis and inflammatory bowel diseases (IBD) have a considerable impact on patient by reducing the ability to work and perform everyday tasks; there is also substantial expenditure by the government on medicines. Biological disease modifying anti-rheumatic drugs (bDMARDs) are a class of medicines used to treat these conditions. Use is increasing with huge coasts to the Australian government via subsidy on the national Pharmaceutical Benefits Schedule (PBS). Medicines are subsidised on the PBS based on evidence of efficacy in clinical trials. However, the quality and strength of the trials supporting the approval on the PBS is still yet to be evaluated.
Aims. This study aims to examine strength of evidence of trials of bDMARDs used to support decisions for subsidy in Australia as provided in PSDs: effectiveness, safety, quality of trials, and risk of bias. Methods. PBS Public summary documents for relevant bDMARDs were extracted from the PBS website. Clinical trials used in PBS applications for subsidy were extracted from PSDs. Information from each trial was extracted: patient cohort, patient numbers, randomisation, concealment, type of trial etc. Each trial was assessed for risk of bias using the Cochrane risk of bias tool.
Results. Between 2006 to 2018 across 5 indications and 9 bDMARD medicines, 53 trials were identified. Across those trials, the median number of patients enrolled for the trials was 368, with interquartile range being from 200 to 619 patients. Around 50% of the trials were performed under an intention to treat statistical analysis. Majority of trials had a high risk of bias particularly when considering role of pharmaceutical companies for funding and sponsorship.
Discussion. The characteristics of the trials varies greatly across both specific medicines but also across within indications. This can have important implications for decision making by physicians and patients, and also if methods for evaluating the quality of trials should be put into consideration during the PBS approval process.
Aims. This study aims to examine strength of evidence of trials of bDMARDs used to support decisions for subsidy in Australia as provided in PSDs: effectiveness, safety, quality of trials, and risk of bias. Methods. PBS Public summary documents for relevant bDMARDs were extracted from the PBS website. Clinical trials used in PBS applications for subsidy were extracted from PSDs. Information from each trial was extracted: patient cohort, patient numbers, randomisation, concealment, type of trial etc. Each trial was assessed for risk of bias using the Cochrane risk of bias tool.
Results. Between 2006 to 2018 across 5 indications and 9 bDMARD medicines, 53 trials were identified. Across those trials, the median number of patients enrolled for the trials was 368, with interquartile range being from 200 to 619 patients. Around 50% of the trials were performed under an intention to treat statistical analysis. Majority of trials had a high risk of bias particularly when considering role of pharmaceutical companies for funding and sponsorship.
Discussion. The characteristics of the trials varies greatly across both specific medicines but also across within indications. This can have important implications for decision making by physicians and patients, and also if methods for evaluating the quality of trials should be put into consideration during the PBS approval process.
| Original language | English |
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| Pages | 12 |
| Publication status | Published - Dec 2019 |
| Event | Australasian Pharmaceutical Science Association (APSA) 2019 Annual Conference - Monash University, Melbourne, Australia Duration: 1 Dec 2019 → 4 Dec 2019 http://apsa-online.org/conferences.html |
Conference
| Conference | Australasian Pharmaceutical Science Association (APSA) 2019 Annual Conference |
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| Abbreviated title | APSA |
| Country/Territory | Australia |
| City | Melbourne |
| Period | 1/12/19 → 4/12/19 |
| Other | The Association hosts an annual conference that provides a forum for presentation of research and development applications from all branches of pharmaceutical science, pharmacy practice and pharmacy education, with an emphasis on participation by postgraduate students. The Annual Conference is open to members and non-members from related disciplines, and is relevant for: - academic and industrial scientists associated with drug discovery, drug formulation and drug delivery research and development - regulatory and clinical scientists involved in bioavailability and bioequivalence study design and assessment, preparation and review of regulatory dossiers - start-up company representatives faced with implementing strategies for rapid drug discovery and development - researchers in the clinical and practice areas of pharmacy - academics with an interest in pharmacy education - students and postdoctoral fellows from Australia and New Zealand in all areas of pharmaceutical science and pharmacy |
| Internet address |