NICE has published its Final Draft Guidance in relation to ruxolitinib (Opzelura) here:
Please see the Final Draft Guidance in this list dated 18 July for further information.
The decision is based first on the fact that the original trial compared ruxolitinib with a placebo and not a treatment that is currently used for vitiligo, such as a topical immunomodulator like tacrolimus (Protopic). NICE say "An indirect comparison is too uncertain to show how well ruxolitinib cream works compared with phototherapy."
If the trial had compared ruxoliitinib with phototherapy or a current treatment, then NICE could have drawn a direct comparison.
Secondly, NICE say that "the cost-effectiveness estimates are very uncertain because of limitations in the economic model, which does not reflect how vitiligo is treated in the NHS. The most likely cost-effectiveness estimate is higher than what NICE considers an acceptable use of NHS resources.".
Therefore, the decision is still based on cost, so remains open, we believe, to further negotiation.
Finally, NICE state that it "is also uncertain whether treatment with ruxolitinib cream would improve people’s quality of life".
So, the treatment was declined because there's no effective existing treatment for vitiligo that could have been used in the trial and for global applicability, so the trial chose a placebo. The EMA and the FDA were able to use this placebo-based trial to recommend the drug, but NICE were not able to.
In addition, the uncertainty about a positive impact on people's quality of life does not fully take into account either the evidence of the trial, or the evidence of the significant impact of vitiligo provided in our patient evidence to NICE, or the multiple research studies that indicate the impact of vitiligo on patients' QoL.
We are continuing to work hard to get this drug approved for patients' use.
One way in which you can help, if you are resident in England, is by writing to your MP. This draft letter will help:
Use this link to find the MP for your area:
You can continue to follow the results here and in our Facebook group at https://www.facebook.com/groups/VitiligoSupportUK
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