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).

Analysis of PBAC submissions and outcomes for medicines (2010-2018).

Objectives: The Pharmaceutical Benefits Scheme (PBS) provides timely, reliable, and affordable access to necessary medicines for Australians. We reviewed the Pharmaceutical Benefits Advisory Committee (PBAC) submissions and their related outcomes and timelines since 2010.

Methods: We examined the PBS Website to identify submissions and their related PBAC outcomes for new medicines, new indications, and new combination products that had been considered by the PBAC since 2010.

Analysis of sponsor hearings on health technology assessment decision making.

Objective The aim of this study was to get a better understanding of the frequency of Pharmaceutical Benefits Advisory Committee (PBAC) hearings, the factors that influence a sponsor's decision to proceed with a hearing and to assess the impact hearings may have had on PBAC decision making. Methods All public summary documents (PSDs) from March 2014 to November 2016 PBAC meetings, obtained from the Pharmaceutical Benefits Scheme (PBS) website, were examined to identify major submissions for which sponsor hearings were conducted. Each PSD was analysed to determine the topics discussed at the sponsor hearing and the 'usefulness' of a sponsor hearing from the PBAC's perspective.

Subsidised access to new melanoma drugs: in need of further innovation?

Aims: Melanoma is the most serious of the three common forms of skin cancer. New Zealand and Australia have the highest melanoma incidence rate in the world. A number of new treatments for melanoma with different modes of action have recently become available.

Assessment of the Quality of the Clinical Evidence in Submissions to the Australian Pharmaceutical Benefits Advisory Committee: Fit for Purpose?

Background: Assessments of the comparative clinical (and cost) effectiveness of new medicines are increasingly being used to inform decisions on their reimbursement. Assessments of added clinical benefit are invariably based on evidence generated to support registration.

Objective: Our objective was to identify and characterize significant problems relating to the quality of the clinical evidence in submissions to the Australian Pharmaceutical Benefits Advisory Committee (PBAC) seeking subsidy on the Pharmaceutical Benefits Scheme and thus determine whether the evidence presented to the committee was "fit for purpose.

What impact does 'conventional' economic evaluation have on patient access to new orphan medicines? A comparative study of their reimbursement in Australia (2005-2012).

Background: Orphan medicines used to treat patients with rare diseases often come at high costs with lower levels of clinical evidence. We compared the likelihood and timeliness of reimbursement for orphan medicines with non-orphan medicines in Australia between 2005 and 2012.

Methods: We developed two key assessment metrics to compare submissions and outcomes for new orphan medicines with those for new non-orphan medicines, viz.

Exploring the implications of a fixed budget for new medicines: a study of reimbursement of new medicines in Australia and New Zealand.

Objective: Spending on medicines under the Pharmaceutical Benefits Scheme (PBS) represents the ninth largest expense to the Federal Government. A recent report by the Commission of Audit to the Federal Government suggested spending on the PBS is unsustainable and a capped budget, similar to New Zealand's PHARMAC model, may be required to contain costs. The objective of the present study was to compare listing outcomes between Australia and New Zealand, thereby exploring the opportunity cost of a capped budget for new medicines.

What can be gained from increased early-stage interaction between regulators, payers and the pharmaceutical industry?

New medicines are the lifeblood of the global innovative pharmaceutical industry. Developments in genomics, proteomics, immunology and cellular biology are set to promise a plethora of novel targets for the industry to create and develop innovative new medicines. For a new medicine to fulfill its therapeutic and commercial potential (i.

Early scientific advice obtained simultaneously from regulators and payers: findings from a pilot study in Australia.

Objective: There is scope for better interaction between regulators, payers/HTA agencies, and medicines developers in their common objective of getting new medicines to patients. This paper reports on a tripartite early scientific advice pilot conducted by a pharmaceutical company (developer), the Therapeutic Goods Administration (TGA: regulator) and the Pharmaceutical Benefit Advisory Committee (PBAC) Secretariat (HTA agency) in Australia. The objective was to explore the practicality, feasibility, and sustainability of means of obtaining simultaneous scientific advice from both a regulatory and reimbursement perspective.

Australian managed entry scheme: a new manageable process for the reimbursement of new medicines?

The global prescription medicines industry argues that it needs high prices for new medicines to meet ever-increasing development costs. While many payers are prepared to pay high prices if they represent good value for money, they first need to feel assured that the value for money estimates are robust. Insofar as new medicines enter the market with limited and uncertain data relating to their performance in normal clinical practice, the value for money case for some medicines may well be driven largely by assumptions than by empirical evidence.

Access to new medicines in New Zealand compared to Australia.

Aim: To compare access to new prescription-only medicines in New Zealand (NZ) with that in Australia.

Method: The range of new prescription medicines and the timing of their regulatory approval and reimbursement in NZ and Australia in the period 2000 to 2009 were compared.

Results: 136 new prescription medicines were first listed in the Australian Schedule of Pharmaceutical Benefits in the study period and 59 (43%) of these were listed in the NZ Pharmaceutical Schedule.

Early dialogue between the developers of new technologies and pricing and reimbursement agencies: a pilot study.

It is common practice for developers of new health care technologies to engage in early dialogue with the major regulatory agencies; such discussions frequently center around the proposed clinical trial designs to support the registration of new interventions and suggestions on their improvement. Pricing and reimbursement agencies are increasingly using the results from health technology assessments to inform their decision making for new technologies. Such assessments are invariably underpinned by the phase 3 clinical trial evidence which may not provide answers to the key questions.

Good research practices for measuring drug costs in cost effectiveness analyses: an industry perspective: the ISPOR Drug Cost Task Force report--Part V.

Objectives: The industry perspective on drug costs should be framed by the need for decision-makers to use actual and relevant costs, and to inform real-world decisions regarding medication selection and use. The objective of this report is to provide guidance and recommendations on how manufacturers should approach the use of drug costs.

Methods: The Task Force was appointed with the advice and consent of the ISPOR Board of Directors.

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.

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.