Rather bizarrely for an area of activity with such high media visibility, NHS England (NHSE) did not put out, as they usually do, a media release in its news section of the website. Instead a very short summary of the new process was posted on the website's Cancer Drugs Fund (CDF) page. The link to the website page is here: https://www.england.nhs.uk/ourwork/cancer/cdf/ and you can click on a link on the page that will take you to the publication.
Last Tuesday NHSE hosted a webinar where they talked participants through the new process using a power point presentation to set out its key features. This is available here:https://healthsector.webex.com/healthsector/ldr.php?RCID=42ec4f3fe8845ac...
It takes a little while to load up so you will have to be patient if you want to hear what was said. On my Mac, the power point slides and controls for the webinar sit under a blank screen for some reason although this may be something particular to my rather old and very sluggish computer.
The talk through of the slide set should be understandable to those of you that are interested and its worth replaying the voice over a few times if you miss the details the first time round. Very usefully you can of course halt the commentary and scroll back. The rather lengthy and sometimes scrappy Q & A that followed the presentation is mostly rather technical but for those with an more professional interest its revealing for the questions mainly asked by the pharmaceutical company representatives who participated and the responses form NHSE and NICE, who were also involved in the webinar.
I don't want to repeat what's in the slide set and also set out in the Executive Summary of the publication which also seems to act as an easy read version of the remainder of the document including the much more technical Appendices. If you want an even quicker read, I'd look at the summary on the CDF website page.
A key question, touched on in a rather round about way in the webinar Q & A, is whether the new process will result in more 'yes' recommendations for the drugs like those used to treat CML. These are drugs that are usually, and increasingly, developed to treat small patient populations that can be identified by some key characteristic(s) with data about their clinical effectiveness often being limited for a variety of reasons.
For example the total number of patients likely to require treatment with bosutinib each year in England is 75, see the table in this link https://www.nice.org.uk/guidance/ta299/resources/costing-statement-42680... and for patients with the T315i mutation requiring ponatinib treatment, the number is much smaller.
One of the reasons for establishing the CDF was to provide an avenue to access for drugs like this although its worth pointing out that drugs for CML are also very different from most drugs in the CDF in that they have proved so phenomenally successful in allowing most patients to look forward to a normal life expectancy.
In theory the new process should secure the availability of a new drug for CML if there were questions (in technical language, uncertainties) about exactly how effective it was by providing a breathing space where its real world performance in the NHS could be studied before a final evaluation was made. Provided the drug was judged to be likely to be cost effective, the drug company would receive some reduced payment whilst this exercise to gather information about its performance was ongoing.
An everyday parallel would be those free sachets of shampoo or washing liquid that sometimes arrive with the post for you to try. The manufacturer hopes you'll be so impressed with the results when you use them that you'll go out and buy the larger container version. When you do so, one part of your payment will be paying for the cost of delivering the free sachets.
I say in theory because although the process is much more clearly thought through than when initially presented last year, its difficult to judge how activating the process details will pan out or how the committee that makes the 'yes', 'maybe yes' or 'no' decisions will act.
My particular worry concerns the origin, gathering and analysis of real world data on side effects and the experiences of patients (technically, quality of life) and whether the eventual output would be granted the status currently given to clinical trial data.
An additional worry would be that, if there were doubts about the feasibility of gathering such data or its likely quality, would this breathing space option simply be abandoned with the only other option being a 'no' decision to recommend the use of a drug in the NHS even though there was no argument about it having some degree of clinical effectiveness.
We will have to wait and see but the good news is that the situation with dasatinib especially but also ponatinib to some degree is rather different because these are not considered 'new cancer drugs'. Although the evaluation environment for them will be exacting, there are more levers that can be pulled that will advantage them in that situation although I do retain some concern about ponatinib.