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Treatment of CML with dasatinib in Scotland

The Scottish Medicines Consortium (SMC) which, like NICE in England, assesses the clinical and cost effectiveness of drugs for use in the NHS in Scotland contacted CML Support last week to invite us to participate in an assessment of dasatinib (Sprycel®) as a first treatment for adults newly diagnosed with CML in chronic phase (CP).

The SMC also wants to assess dasatinib treatment for adults in any of the three phases of CML who have exhibited either resistance or intolerance to a previous treatment which more often than not would be with imatinib (Glivec).  

The procedures for us registering to enter the process and the submission form we are required to complete have recently been changed. What has not changed however is the role of patient groups as the interface for patients experience of CML and treatments they receive following diagnosis.

We have now completed the registration process and would like to ask if any Forum members are willing to assist us with their accounts of their personal experience of dasatinib, either as a first or subsequent line of treatment.

For obvious reasons we would particularly like to hear from patients in Scotland, but we also welcome contributions from patients elsewhere in the UK. The deadline for our submission is June 6th so would like to receive contributions by May 20th to allow us time to collate them into a coherent set of responses.

If you would prefer not to get in touch via this Forum please email directly to cmlsupportgroup@gmail.com

The part of the revised submission form we would like your assistance in completing consists of five questions plus a summary statement which is an improvement on the previous submission form.

The questions, as paraphrased and personalised below, are:

1. How does having CML affect your day to day life?

2. How well are you able to manage your CML with the NHS treatment you currently receive?

3. Has treatment with dasatinib improved your quality of life and experience of care? 

4. What impact has receiving dasatinib treatment had on your family members and/or those close to you?   

5. Do you think there are any disadvantages to your being treated with dasatinib?

Finally, what are the key points you would like to highlight about your treatment with dasatinib.  

Thanks for your help,

David

Just a short post to inform those with an interest that CMLSg submitted two responses to the SMC appraisal within the Monday (6th) timeline.

Its a rather unusual appraisal because it covers two very different indications. One is for first line treatment of newly diagnosed patients in chronic phase only and the other is for second line treatment for patients in any of the three phases who have already been treated with a TKI, including imatinib, and have experienced either intolerance or resistance based failure on treatment.  

I'll update as soon as I hear anything from the SMC.

A very big thanks to the four patients who provided excellent and varied descriptions of their experience with dasatinib and, for second line, with another TKI and dasatinib.  

We were advised by the SMC last week that a PACE meeting for 2nd line treatment is to be convened and we have now completed the paperwork that will allow us to attend the meeting in Glasgow on the 12th July.

A PACE (Patient & Clinician Engagement) meeting is a relatively new addition to the SMC drug appraisal process and is available as an option to pharmaceutical companies provided their drug meets one or more criteria. These are that the condition (or more technically: an indication) for which the drug is being used as a treatment is rare (with the drug qualifying for orphan status) or very rare (with the drug qualifying for ultra orphan status). Another criteria is that the drug should meet a set of requirments that qualify it as what's called an 'End of Life' drug where treatment is able to extend a patient's  already shortened life.

The option can also only be exercised by the company should the assessment process conclude there is uncertainty present about a drug's clinical effectiveness following treatment.

Although the company manufacturing the drug can trigger the option to hold a PACE meeting they are not able to attend the meeting since this is restricted to patient group representatives and specialist clinicians plus the SMC.     

There is no discussion of the cost effectiveness of a drug at the meeting rather the focus is on clinical effectiveness and, importantly, any gain in quality of life conferred by the treatment. There is very considerable flexibility granted as to what is able to be included within a discussion of quality of life with the impact of a condition and the benefits conferred by a treatment expanded beyond the patient to include others signficant in their lives.

Comments made at the meeting and the supporting paperwork provided to attendees are confidential with external participants being required to sign a confidentilaity agreement before attending the meeting.

Following the meeting, a document summarizing the meeting discussion is circulated to attendees for comment and agreement. Once this stage is completed it is then presented at the key SMC meeting to assist the Committee in reaching a decision as to whether to recommend, in this case, dasatinib for 2nd line use in the NHS in Scotland. 

We feel very confident that the eventual outcome will be that the SMC approve dasatinib for first line use in Scotland and fairly confident they will do so for 2nd line use. 

 

   

 

 

We have been informed that the SMC will publish their decisions on 1st and 2nd line dasatinib treatment on the 12th September. We remain optimistic that their decision will be to recommend dasatinib for routine use in the NHS in Scotland in 1st and 2nd line. 

On schedule., the SMC have published their recommendations for dasatinib treatment. We are very pleased to see that dasatinib is recommended as a treatment for CML for:

(i) newly diagnosed adult patients in chronic phase

(ii) for adults patients in either chronic, accelerated or blast phase who have proved either intolerant of, or resistant to, prior therapy where that might include imatinib.

The former is generally referred to as 1st line and the latter 2nd line.

The manufacturer, Bristol Myers Squibb or BMS, made a Patient Access Scheme (PAS) available in each case which proved acceptable and the recommendations are dependent on these staying in place. The PAS variant in both cases would almost certainly be a (confidential) discounted price.

We remain optimistic that similar recommendations will be forthcoming from NICE early next year.  

The link to the details of the 1st line decision is:  

http://www.scottishmedicines.org/files/advice/dasatinib_Sprycel_FINAL_Au...

And 2nd line is:

http://www.scottishmedicines.org/files/advice/dasatinib_Sprycel_Resub_FI...

 

 

Great news David, thanks for the update and help in getting this though, England to follow soon we all hope, and expect !