I’ve been covering oncology market access trends for three years now, and I’ve been in the oncology world for longer still. At the close of every year I review the annual highlights, and I tend to do so with great expectations since it is usually such a busy market.
This year is no different, except that I looked back at 2011 with more hopeful, yet more critical, eyes than usual as this was the 40 year anniversary of the signing of the National Cancer Act of 1971, the unofficial start to the ‘War on Cancer’. My expectations may have been slightly different, but what I found is largely in keeping with tradition, and I had the same impression as always: progress is incremental.
Progress in Melanoma Treatment
Although progress didn’t grow in leaps and bounds this year, there were some notable events. The most significant happened in melanoma, with two new drugs hitting the market after a dearth of more than two decades.
The first one, Yervoy, approved for unresectable or metastatic melanoma in the US in March, and in the EU in July, was approved based on results which demonstrated a 10 month extension in median overall survival (OS).
Then in August, Zelboraf was approved for metastatic melanoma in the US. At the time of approval, OS has not been reached in those patients on the Zelboraf arm (OS in the chemo arm was 7.9 months), however risk of death was reduced by 56%. This figure shouldn’t be belittled, but it’s a far cry from 100%.
And 40 years after the war on cancer fired its first shots, should we really be measuring success, even in metastatic disease, in months? Shouldn’t the yardstick be years by now?
Shifts in Regulatory Approval Criteria
There is one area in which the yardstick was permanently changed this year: the measure by which regulators are assessing success in clinical trials. When once proof of a progression free survival (PFS) advantage was often enough for approval, it is no longer sufficient — now you must show an OS advantage.
Need convincing? Just look at the example of Avastin in breast cancer in the US. Conditionally approved on the basis of strong PFS evidence, it was revoked based on Roche and Genentech’s inability to prove a survival basis.
But it’s Not Just Approvers Who Need Proof
It’s not only approvers who are demanding proof of a survival advantage, but reimbursers as well. This is particularly true in Germany, where the introduction of AMNOG has drastically changed the pharma landscape from that of ‘anything goes’, to that of ‘restrictions and conditions’. If a company wants free pricing for a new innovative drug, they must prove that it has an advantage in terms of patient-related outcomes over existing, marketed treatments.
As an extension in OS is one significant way to meet the patient-related outcomes requirement, those looking for advantageous pricing in Germany must ensure that they have solid OS data or, if OS data is not mature at the time of submission, they must prove a correlation between PFS data and OS data.
Drug manufacturers are already taking heed of the emphasis on OS data. Just recently, AstraZeneca announced that it was scrapping its Olaparib development programme in Ovarian cancer as results showed that promising progression-free survival (PFS) data will not translate to positive OS data.
What are the ramifications of this emphasis on survival? Will it increase innovation? Will it keep subpar medicines from entering the market? Will we finally assess achievement in years and not months? In any case, I know when 2012 comes to an end, I will look back at the year with even greater anticipation than normal.