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Reimbursement bar too high for rare cancers: MA
Posted 9 June 2017
The reimbursement system isn't flexible enough when it comes to rare cancers and hasn't kept pace with the challenges of innovative trial design, Medicines Australia's Elizabeth de Somer has told the Senate committee looking at research for low survival rate cancers.
Asked by committee deputy chair Senator David Bushby to expand on elements in MA's submission touching on so-called 'basket' trials and more "out there" yardsticks, she said the small number of rare cancer patients made the case for reimbursement difficult.
"Inherently in rare cancers, there are very small numbers of patients. With small numbers...he statistical validity of the evidence is reduced and that sometimes doesn't meet our current standards for reimbursement", she said.
While developers and researchers were calling for innovative trial designs in these diseases "the assessment process on whether to fund them has not caught up or kept pace," she said.
While the Sansom Review recommendations had introduced some potential pathways in the registration process there was still "limited progress on how we adjust the reimbursement decision-making to accommodate these innovative trial designs and different sources of data".
The issue is one of two key points in the MA submission with it calling for "changes to the current access model, specific for medicines for rare and low survival conditions" which would require "agreeing and accepting flexible evidentiary requirements which acknowledge the specific challenges of clinical trials in this area".
When two of the senators said other submitters had said that pharma wouldn't do trials for rare cancers because it was unprofitable, she replied that "the difficulty in rare diseases is not so much that they are not profitable but the evidentiary requirements make it very difficult to achieve access through the reimbursement process".
"They never make any money if you can't sell them," he said.
"You really need to be flexible in the evidentiary requirements so you can accept different levels or types of evidence for something that has a very small population so it can achieve access..and entry into the market," she said.
De Somer, together with Helen Aunedi from Roche and Melissa Grady from AstraZeneca, also told the committee that MA's proposal for a national office of clinical trials - the other lynch-pin of its submission - was an attempt to stop Australia's stagnation in the sector, as a result of vast number of regulations and the contractual discussions involved in coordinating up to 200 accredited research sites.
Countries like China and South Korea were now able to mount clinical trials in 30 days compared to around 130 days in Australia, the committee heard.
And all three agreed with Bushby's observation that such a central office would probably need Federal legislation to make it work, potentially with "coercive powers", De Somer noting that in spite of general willingness across states and territories for more streamlined processes, a decade on there was still "no outcome".
David Rowley
david.rowley@lushmedia.com.au
Posted 9 June 2017
The reimbursement system isn't flexible enough when it comes to rare cancers and hasn't kept pace with the challenges of innovative trial design, Medicines Australia's Elizabeth de Somer has told the Senate committee looking at research for low survival rate cancers.
Asked by committee deputy chair Senator David Bushby to expand on elements in MA's submission touching on so-called 'basket' trials and more "out there" yardsticks, she said the small number of rare cancer patients made the case for reimbursement difficult.
"Inherently in rare cancers, there are very small numbers of patients. With small numbers...he statistical validity of the evidence is reduced and that sometimes doesn't meet our current standards for reimbursement", she said.
While developers and researchers were calling for innovative trial designs in these diseases "the assessment process on whether to fund them has not caught up or kept pace," she said.
While the Sansom Review recommendations had introduced some potential pathways in the registration process there was still "limited progress on how we adjust the reimbursement decision-making to accommodate these innovative trial designs and different sources of data".
The issue is one of two key points in the MA submission with it calling for "changes to the current access model, specific for medicines for rare and low survival conditions" which would require "agreeing and accepting flexible evidentiary requirements which acknowledge the specific challenges of clinical trials in this area".
When two of the senators said other submitters had said that pharma wouldn't do trials for rare cancers because it was unprofitable, she replied that "the difficulty in rare diseases is not so much that they are not profitable but the evidentiary requirements make it very difficult to achieve access through the reimbursement process".
"They never make any money if you can't sell them," he said.
"You really need to be flexible in the evidentiary requirements so you can accept different levels or types of evidence for something that has a very small population so it can achieve access..and entry into the market," she said.
De Somer, together with Helen Aunedi from Roche and Melissa Grady from AstraZeneca, also told the committee that MA's proposal for a national office of clinical trials - the other lynch-pin of its submission - was an attempt to stop Australia's stagnation in the sector, as a result of vast number of regulations and the contractual discussions involved in coordinating up to 200 accredited research sites.
Countries like China and South Korea were now able to mount clinical trials in 30 days compared to around 130 days in Australia, the committee heard.
And all three agreed with Bushby's observation that such a central office would probably need Federal legislation to make it work, potentially with "coercive powers", De Somer noting that in spite of general willingness across states and territories for more streamlined processes, a decade on there was still "no outcome".
David Rowley
david.rowley@lushmedia.com.au
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