Exclusive: Thousands could benefit from new PBAC process

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Patient access to some new cancer medicines could be accelerated by around a year based on a proposal considered by PBAC at its August meeting.

One of the most anticipated outcomes from August's meeting was the committee's response to a proposed new process designed to avoid resubmissions and accelerate access to breakthrough cancer medicines.

The Rapid Resolution Process, developed by the Cancer Drugs Access Committee (CDAC) of the Cancer Drugs Alliance (CDA), was developed over several months. Its development has involved input from patients, industry and Department of Health officials.

CDAC is chaired by former Victorian premier, John Brumby AO.

According to a supporting analysis prepared by PwC, "Thousands of patients could benefit from a mechanism to achieve earlier access to priority cancer medications and earlier access could extend people's lives or slow the progression of their cancer." 

The analysis said, "…subsidised access to important cancer drugs through the PBS is delayed in Australia." It partly attributed delays to the PBS-listing process but also acknowledged some of the causes "lie beyond the remit of government."

Representatives of CDAC presented the new process to the PBAC at its August meeting. It is understood the committee discussed it in detail and, while not explicitly endorsing its adoption, said it could be considered as part of potential wider reforms to the current process.  

PBAC chair Professor Andrew Wilson recently called for a review of National Medicines Policy and argued for a system-wide approach to reform.

It is understood the committee was unsure why companies would participate in the process, given the experience with managed access, and ultimately thought any decision on its adoption sits with health minister Greg Hunt.

It is also understood the committee believed many of the issues identified in first major submissions are highly complex, difficult to resolve in the absence of a subsequent submission, and require the type of concessions from companies they are usually reluctant to make. These concessions, particularly in relation to pricing, tend to come after significant discussion on issues like comparators.

The proposed new process

The Rapid Resolution Process (RRP) is designed to apply to a small number of medicines considered a 'priority', in terms addressing an unmet patient need, but where companies are unable to immediately address the 'uncertainty' driving a PBAC rejection.

It would in part complement the existing managed access program and potentially the TGA's recently adopted designation that accelerates regulatory consideration of new 'breakthrough' medicines.

Its key component involves a change to the current post-PBAC meeting, at which companies and PBAC discuss the reasons for a rejection and potential resolution, with the goal of a resubmission.

Under the proposed RRP, this 30-minute meeting would be extended into a workshop to "include discussion of issues requiring resolution and potential approaches to resolve the issues."

The workshop would be followed by a final negotiation meeting, "…to discuss the updated economic analysis," with the goal of agreeing on its presentation to PBAC.

The RRP has been designed for cancer medicines but could eventually be applied to other priority therapies, according to CDAC, which might ease Professor Wilson’s publicly expressed concern over the risks of prioritising treatments for particular patient groups.

The key point is that, for medicines meeting the criteria and where companies agree to participate, the time taken for resubmissions could be avoided. PBAC could make a recommendation within four months of its original consideration. This could accelerate listings by several months and possibly even one year.

Medicines would still be subject to the existing requirements of the PBAC, specifically in relation to cost-effectiveness, but the process is simply designed to create an opportunity to accelerate resolution of identified issues.

According to the analysis provided to PBAC, based on the past five years, around three medicines would have been considered under the process each year, with 1.2 major and 0.6 minor submissions avoided per medicine. The 15 medicines would have been listed on the PBS around one year sooner at a first-year cost to government of around $42 million.

An average of 355 patients would have been impacted by each medicine with 220 life years gained. 

Going forward, if the process was adopted, the analysis found between 5,800 and 7,100 patients would gain earlier access to 18 medicines over the next five years. Approximately $83 million in PBS spending would be brought forward by one year.