Analysis of consumer comments into PBAC decision-making (2014-9).

OBJECTIVES: The Pharmaceutical Benefits Advisory Committee (PBAC) is an independent expert body that recommends new technologies for listing on the Pharmaceutical Benefits Scheme. Its decision-making process is evidence-based and considers a technology's clinical effectiveness, safety, and cost-effectiveness compared with other technologies. Since 2014, the PBAC has formally taken into account input from those impacted by the technology via an online consumer comments portal and has also reported on received comments in the Public Summary Documents (PSDs). Comments are welcomed from those whose health the technology is trying to improve, as well as carers, clinicians, and organizations. Our objective was to analyze and review consumer comments in the PBAC's decision-making process. METHODS: We extracted information about consumer comments from the PBAC PSDs from 2014-9. We conducted simple descriptive analyses. RESULTS: Our findings reveal that two thirds of all submissions did not receive a single consumer comment. Of the remaining third, eight submissions (less than 1 percent) had a substantial number of consumer comments (>500). For these technologies, multiple submissions were required before a recommendation was issued. Submissions spanned multiple therapeutic areas, the therapeutic areas with the most consumer comments were genetic disease, pediatrics, and oncology. CONCLUSIONS: In the light of our review, we have identified limitations to the current consumer comments process, and after an examination of the processes of other comparable health technology assessment agencies, we have identified a number of improvements that could be made to the PBAC's process to increase consumer engagement.

Are Australians able to access new medicines on the pharmaceutical benefits scheme in a more or less timely manner? An analysis of pharmaceutical benefits advisory committee recommendations, 1999-2003.

OBJECTIVE: Timely access to necessary medicines that Australians need is one of the four pillars of the Australian Government's National Medicines Policy. We were interested to determine whether there was a change in the time taken for medicines to be listed once recommended by the Pharmaceutical Benefits Advisory Committee (PBAC). METHODS: Descriptive statistics were used to show the pattern of recommendations for PBAC meetings from 1999 to 2003. For successful recommendations, we developed a linear regression model to analyze the time to list from the PBAC meeting to date of listing (time to list). The model determined whether this time had changed over the 4-year period, and the reasons for any changes. RESULTS: The PBAC made 307 positive recommendations at its 17 meetings over the study period. Ninety percent resulted in a Pharmaceutical Benefits Scheme (PBS) listing on or before April 1, 2005. Eighty-two percent of the recommendations made in 1999 and 2000 resulted in early or on-time listings. In 2001, 2002, and 2003, the comparable proportions were 67%, 68%, and 75%. Mean times to list for the years from 1999 to 2003 were similar (approximately 23 weeks), except in 2001 where it was 30 weeks. CONCLUSIONS: Over the study period, 90% of all PBAC recommendations resulted in a PBS listing. In 2001 there was a statistically significant increase in the mean time to list. In addition, it appears that recommendations for new listings and new indications (medicines that are likely to result in substantial Government expenditure) were associated with a longer time to list.

Development of a predictive population survival model according to the cytogenetic response rate for patients with chronic myeloid leukemia in the chronic phase.

This study sets out to investigate whether the proportion of patients with chronic myeloid leukemia (CML) in the chronic phase who achieve a major cytogenetic response (MCR) can be used as the basis for estimating long-term survival through the use of modeling. Data from seven randomized controlled trials of drugs to treat patients with CML in the chronic phase were used to explore the association between MCR and survival by way of regression analysis. The estimated weighted odds ratio for the survival of those who achieved an MCR when compared with those who did not was 7 (95% CI 5 - 11) at 2 years and 5 (95% CI 3 - 8) at 4 years. Four long-term survival models were subsequently constructed. All models were found to be robust to variations in the data included. Model D was favored using the 'Ockham's razor' principle; it suggests that the median survival may be increased by 1.8 years for every 25 percentage point increase in MCR rate. The results support the use of the proportion of patients with CML in the chronic phase with an MCR to estimate overall long-term survival.