The availability of bosutinib and ponatinib for CML patients in England: an update

Update on bosutinib: I went to a meeting on Monday (7th) at the Department of Health where the Chair of the CDF Panel, Peter Clark, briefed patient groups on the current delist and the Panel’s decision making process. The core problem was that data at the end of month two (ie May) of the current 2015/16 financial year indicated that the CDF was forecasting an overspend of £70 million on the £340 million already allocated for its budget over the year. This forecast promoted a review of drugs on the CDF list and other activity in late June/early July, with the Panel announcing the list of drugs to be delisted on the 4th September. The outcome for the drugs for blood cancers reviewed was particularly bad with two thirds of the treatments with these drugs being delisted. Apart from bosutinib these included drugs for the treatment of lymphoma, myeloma and other leukaemia conditions, chronic lymphocytic leukaemia (CLL) in particular I asked why, apart from the treatment of patients positive for the T315i mutation with ponatinib, neither bosutinib, dasatinib or ponatinib were available, via the CDF, to treat CML patients who were resistant to nilotinib who had either been treated with imatinib 1st line followed by nilotinib or nilotinib 1st line . Peter Clark’s answer was that, although there was some evidence that certain TKIs were effective against particular mutations, there was not enough evidence to support a CDF listing and that the issue of lack of evidence applies more generally to resistance. Peter also said, since the cost of a TKI now forms part of the CDF evaluation process, the situation was further complicated by all five TKIs having different prices. If they were all the same price, he said this would make evaluation much easier and might persuade the Panel to extend access by including more TKIs in the CDF in addition to extending access to patients who were resistant to nilotinib, as well as, in some cases, other TKIs. In addition Peter said the Panel were waiting for the CML Working Group of leading clinicians to submit an algorithm to the Panel that built on the one submitted last year (an algorithm is a set of rules governing the circumstances under which a specific TKI should be used for a particular group of patients after a defined previous treatment, one example would be the ELN Guidelines). Peter also agreed that the CDF had not been designed with TKIs for CML in mind. This applied even more since a cost component had been introduced into the evaluation process because the success of TKIs resulted in long term survival for most patients and this in turn has had an impact on the finite CDF budget. He confirmed that the public consultation on the ‘new model’ that will be used as part of the assessment of all new cancer drugs will begin very soon (we think next week). The problem for bosutinib is that its already in the CDF and, as such, it falls in the ‘old drugs‘ category and would not be eligible for entry into the new model. The only other way to achieve access for resistant patients would be for bosutinib’s manufacturer to attempt to directly re-enter the NICE evaluation process. We, at the CML Support Group, have grave doubts that, if permitted, this would prove successful and would like to see the new model made available to bosutinib. We will of course make a submission to the consultation where we will, amongst other issues, stress the points made above. Update on ponatinib: We understand that the small company that manufactures ponatinib, would like to apply to the CDF to increase access to patients beyond those exhibiting the T315i mutation. Their reason for doing so is because they have an EMA license that captures a broader patient population. Their application also has the support of some key CML clinicians as well as the CML Support Group. Unfortunately the CDF is now closed to all new applications and will almost certainly remain closed for the remainder of 2015/16 (ie until the end of March next year). Assuming the CDF, or a successor Fund, becomes operative at that point; our and others fear is that the larger companies will displace the smaller to ensure their drugs enter the new evaluation process first, pushing other drugs to the back of what will probably be a long queue, given the current pause in the new applications process. We will attempt to prevent that. Recent and future activity: After last Monday’s meeting we had a short meeting with a number of blood cancer charities about the delist, the CDF, the new model and the broader healthcare landscape. It was proposed that we meet again as a group in October to discuss these issues in much greater depth so that we can take a collective stance to enable the many small blood cancer patient populations to speak with a single, and therefore much larger, voice. We will update you on that after the meeting late next month. This will add to the activity we were involved in over late July and August where we wrote to David Cameron, health minister Jeremy Hunt and Simon Stevens (the CEO of NHS England) to highlight the possible consequences of a delist of bosutinib and inability of ponatinib to enter the CDF. We asked Leukaemia Care, Leukaemia & Lymphoma Research, Nigel Deekes’ CML UK Facebook Group and CML Blogger Kris Griffin to be signatories to all the letters and were very pleased that they all agreed to sign the letters. The best way of describing the replies we received were that they were holding replies. That is, since this was before the announcement of which drugs would be delisted, they simply said they could not comment at this stage but there would be announcements that would hopefully answer the issues we raised. We were not expecting anything other than that, but we now have that on record as a resource for future activity, especially that with the other blood cancer charities who will participate in the meeting in October, but also, we hope, with the other coalitions we are members of such as Cancer 52 and the Rarer Cancers Foundation. We will keep you posted.