Earlier this summer in June, I set out on a mission, it was something I had a little understanding of at the time but, I knew it was something that needed to be done, and had been suggested to me at ASCO by an Irish Oncologist as something we as patients should be not just involved in but actually driving the change needed. after all our lives depend on it.
So armed with the knowledge I had acquired throughout the last year since I once again got thrown in at the deep end of Melanoma for the second time in my life, I set about assembling a patients group submission on behalf of Irish Melanoma patients for the approval of the Nivo/Ipi combination treatment for un-resectable disease (can no longer be removed by surgery). This treatment has been hailed as the most effective treatment to date for treatment of late stage melanoma it is FDA and EMA approved and has been trialed now extensively. For a patient who has been diagnosed at stage 4 with Malignant Melanoma it is now something that is an achievable dream for many to have a long-term durable response to treatment, where before you were destined to a certain early grave, there is now hope but it’s not just that there is proof to back up the hopes.
The first signs that something exciting was happening came at ASCO 13 when according to the Cancer Research Institute Jedd D. Wolchok presented results from phase 1 trial showing that patients had experienced “rapid and deep regression” of their tumours. There followed a Phase 2 trial after which the FDA announced accelerated approval of the combination the results of the Phase 2 trial had proven that the combination worked better than Ipi alone. Even though it had accelerated approval from the FDA a third Phase 3 trial Checkmate 067 went ahead with three treatment arms patients received either Nivo+Ipi or Nivo mono or Ipi mono (remember they already know combo works better than this by now) This trial was also available in Ireland at that time but because it was a double-blind trial you or your treatment team were not aware of which treatment you were receiving, so it could have been Ipi which had a Progression Free Survival (PFS) which means the length of time a patient sees no worsening of disease of 2.9 months or Nivo mono 6.9 months or the combo at 11.5 months. The rate of complete response to treatment stands at Ipi 2.2% Nivo 8.9% and combination is 11.5% and the response rate overall stands at 57.6% for the combo 43.7% for Nivo and 19% for Ipi. (Dr.Wolchok ASCO 16).
So as you can see there is good reason to be excited about this combination as it has had the highest PFS rate to date of any treatment. This was a very large trial and had 945 participants. There are of course still some unknowns such as the PD-L1 presence in the tumour and what it means in terms of take up by the drug it may be for some patients that Nivo mono is just as effective for them and they can avoid the extra toxicity associated with the combination.
So with all this evidence mounting up for the benefit of the combination its approval by the European Medicines Agency (EMA) as safe for use its fast tracking through NICE in England its approval in many other European countries including Greece (who are supposedly bankrupt) and also Scotland earlier this week it was a devastating blow to Irish patients that it did not get a recommendation from our HTA body the National Centre for Pharmoeconomics (NCPE) last Wednesday.
Of course this needs to be looked at with a logical eye and an understanding of whats going on in the world of drug reimbursement. These treatments are not cheap and Ireland has like many countries a limited budget for new medications. This said we have heard of multi million Euro investments in Irish Water, wasted spending of epic proportions and now a new house building program announced in the same week that the reimbursement was denied, will this money be wisely spent or will we again have tribunals of enquiry in years to come about corruption and money wasted!!
This is not the first disappointment we have had this year in terms of access to new medication as Nivo Mono was also rejected in June this year. We are also anxiously awaiting the outcome of a submission for the approval of Mekinist which is also desperately needed by advanced stage melanoma patients.
This is a problem which will not go away, and is only set to get worse as these drugs are now showing promise in other cancer types, there will be new developments we want access to in the future, do we deny all these patients a chance at life-like we are doing to Melanoma patients, life changing decisions are being made that affect all of us experiencing a diagnosis of cancer, it is a game of how high or low can you go on price between Pharma and the HSE, and the patients are the pawns stuck in the center with the highest stake to lose.