Adrienne Martin Therapeutic Group Manager PHARMAC PO Box 10-254 Wellington 6143 27 August 2014 Dear Ms Martin RE: PHARMAC Consultation Document Multiple Sclerosis treatments funding proposal Joint Submission from The Multiple Sclerosis Society of New Zealand (Inc) and The Multiple Sclerosis Society of Auckland and North Shore (Inc) This is a joint submission by The Multiple Sclerosis Society of New Zealand (Inc) and The Multiple Sclerosis Society of Auckland and North Shore (Inc). The Societies (The Societies) representing the MS Community have endeavoured to consult as widely as possible in the time allowed, with a number of groups to best understand the effect that the current document will have. This includes two consultation meetings held by MS Auckland with over 30 attendees representing their individual or family member s interests. We are very pleased that PHARMAC has agreed to consider funding these two new drugs under the terms proposed and making changes to those treatments currently funded. Nothing in these comments is intended to cause any delay in the implementation of PHARMAC s decision in anyway. If the effect of considering any of these points was to cause a delay we would prefer to proceed on the basis proposed subject to our ability to have ongoing discussions with PHARMAC to make what we believe would be valuable improvements. We are aware that there are a number of People with MS (PwMS) for whom further delay would be detrimental to their health and potential future acceptance for treatment if left longer. We urge that the following points are addressed and considered to ensure that the proposal is clear and beneficial to PwMS. The main problem that we see is the lack of a PwMS centred approach for these services and treatments. Not only the proposal but also the process, care and treatments should be accessible and not restrictive. We believe there are simple solutions to address these issues. 1) Proposed Process a) Clarity - The current process is very clinically focussed and it is difficult for lay people and PwMS to contribute to the debate effectively. It is not people friendly. (Note the PHARMAC document refers to patients we believe this is inappropriate because the bulk on MS treatments including the new ones occur outside of a hospital in-patient environment, accordingly in this document we have used People or Person with MS [PwMS]). The process and criteria needs should be more accessible as many are currently confused and we have heard a number of different interpretations due to the lack of clarity
b) MSTAC Step - The process and number of stages that the neurologist and PwMS must go through to begin treatment as detailed in the consultation document is extremely lengthy and restrictive. We are not aware (although there may be) any other drug for neurological conditions where approval from a body like MSTAC is required. A competent Neurologist should be able to prescribe treatments within the guidelines eliminating the further delay imposed by the MSTAC approval step. Beginning treatment as soon as possible will prevent delays during a time of very high anxiety and stress. Research shows that these treatments must be given as soon as possible to be most effective. Extended waiting times have the potential to increase a person s chance of not fitting the criteria. Guidelines for the prescribing and monitoring treatments should be sufficient for neurologists to adhere to. MSTAC approval should only be required If neurologists are uncertain about the eligibility of the PwMS for treatment, they do not fully fit the criteria but there are valid clinical reasons, or there is doubt in the case. Number of treatment options - It is proven that there is no one treatment that works for every PwMS and until trialled the most effective is unlikely to be known for certain. It is not clear if PwMS can be prescribed another drug if the first, second or third drug goes not work. During our consultation, two PwMS currently on Copaxone reported they are doing well but experience major issues including needle fatigue and extensive body bruising. They both would be keen to try one or other of the new treatments but only on the basis that if they did not work they would have the option of resuming treatment on Copaxone. 2) Terminology The terminology used in the Consultation Document is confusing and is leaving many PwMS wondering if they will be eligible for treatment. a) Relapse i. We believe the use of the word relapse in the document is unclear and appears to be used interchangeably with first event or first attack. ii. There is confusion with the current wording with many reading it that if they have not had a recent relapse or attack they will not be able to access treatment. Thank you for previously confirming for us that the intention of PHARMAC is that a PwMS fitting the criteria can access these drugs after one recorded episode/attack and recorded MRI activity. If a clinical diagnosis of Relapsing Remitting MS, whether or not an experience of symptoms has been recent, is required, it should be specified with more clarity in the document.
For clarity we suggest that PHARMAC uses the following terminology. Clinically Diagnosed MS to mean a confirmed diagnosis of MS and a relapse to mean a further episode or attack. While we understand that this is a clinical document there needs to be a version in plain English which a PwMS and lay people can read, clearly setting out when and how access to these treatments occur. PwMS should not have to go to their neurologist to have the criteria interpreted. The information and process needs to be PwMS centred. b) 4 weeks waiting period after a relapse Item on entry criteria 7 for Natalizumab and 6 for Fingolimod essentially mean the same; that no application can be made until 4 weeks after a relapse. The different wordings are confusing. There also needs to be more clarification on this point about how long following the 4 week period applications can be made. If, as hoped, a recent relapse is not necessary, it should be further specified that there is no time limit for applications if the other criteria are fulfilled. 3) Prescribing of current treatments to PwMS People diagnosed with MS after 1 November 2014 should still be able to access interferon-beta s and glatiramer if their neurologist believes that one of these drugs is the most appropriate. Some PwMS will prefer to begin treatment with the older drugs, which are known to reduce relapse rates by 30%. If a PwMS decides not to proceed with these newly funded first-line treatments they should not be left vulnerable and therefore unable to access treatment. There should be a level of choice between the PwMS and the neurologist regarding the most appropriate treatment. This point is particularly important to review for PwMS in outlying areas who are potentially facing further delays and inequitable access to treatments. The interferon-beta s and glatiramer may be more accessible and helpful to these PwMS who may not be able to access hospital based treatments due to specific circumstances. 4) Criteria a) Entry and Exit Criteria - While we are extremely pleased to see the entry criteria has been lowered to 0 for all treatments we are still concerned the overall entry and exit criteria is too tight. There needs to be some flexibility. b) MSTAC authority outside of the criteria - There needs to be ability for special cases to access treatment. A number of PwMS would see benefit from the new and current treatments but do not quite fit the prescribed criteria. For example; a PwMS may not have fully recovered from an episode of optic neuritis and may be blind in one eye, or incurred a spinal injury from a relapse.
These potentially irreversible after-effects of an episode may push their EDSS score over 4. However they may not have Clinically Diagnosed Progressive MS but are still Relapsing Remitting. These treatments may still have an extremely positive effect on other factors affecting the persons MS. Significant disability in one area should not clinically make someone ineligible. MSTAC should have the authority to review these cases on an individual basis, working around the currently prescribed parameters. 5) Rural PwMS and access to treatments Wherever you are in New Zealand we believe should have the same access to treatment. This is not clear in the proposal as it does not seem that those living in communities away from those larger DHBs, without neurologists, have been fully considered. It is foreseeable that they will face long waiting times for appointments and for many the travel times and monthly infusions of Natalizumab would be extremely difficult if not impossible. People living in Kaitaia will need to travel 5-6 hours to Auckland Hospital. PwMS on the West Coast will have to travel 3-5 hours to Christchurch DHB for monthly infusions or appointments. It is imperative that this is re-considered. We would propose that following a neurologist s prescription of these new treatments, local DHBs are licensed to administer the treatments or monitoring in the absence of a neurologist through the correct clinical protocols. Outlying hospitals or properly equipped and staffed GP practices should be set up immediately to handle these treatments and monitoring. We must take a PwMS centred approach for these services making the treatments accessible and not restrictive. 6) Self funded PwMS a) Self funding and fitting the criteria It is not clear what the criteria and process is for access for people who are currently funding one of these treatments privately to move to the public funding. Appendix 1 of this submission is the personal account of Mellanie Ullrich, the only self-funding Fingolimod PwMS in New Zealand. Her account details the positive effects she has experienced over the past two years on the treatment, with no relapses, and is able to fulfil a full, positive life. However the financial burden that Michelle and her family have balanced with her quality of life is $60,000 a year ($120,000 over the two years on treatment). Will PwMS such as Mellanie and those self funding Natalizumab have to undergo again the proposed criteria process as currently prescribed or will they be able to access funded treatment immediately?
We want to limit the $5000 a month cost that Mellanie, her family and others like them, are currently paying as soon as possible rather than prolonging the wait. This is especially the case where results already prove the success and viability for treatment. b) Currently self-funding PwMS falling outside the criteria - Will PwMS who are currently funding the drug privately and doing well on the drug, but fall outside the current strict guidelines, be able to access the drug on a case by case basis? c) Non-qualifying PwMS but potential to self-fund - It is not clear in the document If a PwMS who does not qualify for PHARMAC funding, can they purchase the drugs at the PHARMAC listed prices? 7) Overseas Returning PwMS a) Citizens or Residents - The document is not clear on the conditions on which PwMS who have been overseas and on the drugs there can access the drugs in NZ when they return to live here permanently. Does their diagnosis and experience with the condition overseas count or do they start from scratch again? b) Visitors Will a PwMS on a Visitors or Work Visa be able to access these treatments with confirmation from their neurologist from abroad? Will they be able to access treatment at the PHARMAC listed prices and what will the process be? We understand that they will have to pay for them. We ask this question as we regularly receive queries from people travelling to NZ as tourists enquiring about the availability of their prescription medicines here. 8) CUAs and QALY Calculations The CUAs and QALY calculations that PHARMAC have made in respect of these drugs have not been made available to us in time for us to analyse them or to submit them to independent scrutiny and analysis. It is not possible for us to comment fully on PHARMAC s decision or process without them. We wish to engage with PHARMAC again when this information is available and once we and our advisors have had the chance to understand the analysis and its implications. 9) MSTAC Panel The MS Societies believe it would aid the process if the Medical Director of MS New Zealand or their nominee was represented on the panel. This would ensure MSNZ was kept close to the PHARMAC process and have influence at a much earlier stage of the PHARMAC process.
Closing Remarks As an overall observation of the experience, the PHARMAC process is far too long and complicated and it has meant that valuable drugs have been denied to a number of PwMS to their detriment; to the extent that some PwMS who would have benefited from the drugs if they had been available earlier, are now unable to access treatment on the current criteria. We would like to further discuss this with PHARMAC to develop more expeditious processes in the future. We do not want to further delay the listing of these new treatments, and any changes to the currently funded interferon-beta s and glatiramer. With listing hopefully by 1 st November it is unlikely that most PwMS will begin treatment before early 2015 following the proposed process as outlined in the consultation document. This means that many PwMS have the potential to have a relapse that may exclude them from these new treatments if we do not proceed with haste in pushing through the funding and listing of these treatments. We do however want to engage with PHARMAC after listing to create a working environment that addresses some of the issues we have raised. We are well aware that it will not be an easy process setting up DHBs for the changes that these new treatments will bring. However we must ensure that we are taking a PwMS centred approach, making treatments available and not unnecessarily restricted. This will not be achieved by restricting neurologists abilities to prescribe treatments quickly, or limiting treatment options. We thank you for the openness at which you have been willing to consult with us on this document and hope we can work together to bring the best results to people with Multiple Sclerosis in New Zealand, bringing their opportunities for treatment closer to what is available in the rest of the western world. Sincerely, Malcolm Rickerby President The Multiple Sclerosis Society of New Zealand Neil Woodhams President The Multiple Sclerosis Society of Auckland & North Shore
Appendix 1 Mellanie Ullrich s personal experience with Fingolimod (Gilenya) To whom it may concern, I am writing this email to let you know my personal experience with Gilenya and its effect on my Multiple Sclerosis. My name is Mellanie Ullrich. I am 40 years old and I was diagnosed with Multiple Sclerosis in 2006 following a second optic neuritis, confirmed by MRI scan. As no medication was currently available I continued without medication. In 2008 I experienced a significant relapse that left me without sensation below my chest and left me with residual weakness on my left side and tingling sensations in my hands. I also progressively developed fatigue to the point that I needed to go to sleep early in the evenings and often felt drained and without energy. With two young children the fatigue in particular made my quality of life challenging and my husband and I began looking into privately funding treatment. Under the care of Ernie Willoughby, in 2009 we privately paid for Tysabri treatment. Unfortunately I developed neutralising antibodies very quickly and had to stop treatment. I continued to look at alternatives and after reading about the effectiveness of Gilenya, my husband and I decided to import the medicine personally from Australia in 2011. I cannot express enough the significant personal financial sacrifice we made to purchase the drug. One packet that lasts four weeks costs us AUD$ 3,200 plus freight plus gst. Effectively costing us nearly NZ$60,000 per annum. Whilst the financial burden has been great, I also cannot express in words how profound the impact has been on my health and wellbeing from the drug. Within weeks of starting on Gilenya, my fatigue and sleepiness reduced to the point I no longer feel they affect my quality of life. I realise this is not a benefit most people associate with Gilenya but this increase in energy has been noticed not only by myself but my family, friends and neurologist. For a mother to two children and also as a CYFS caregiver to foster babies, this ability to function and have the energy to look after my family has been an incredible change to my life. At the same time I also appreciate the fact that I have had no significant relapses since starting on Gilenya three years ago. Nor have I experienced any side effects from the medication. Myself and my family are incredibly appreciative of this and the fact that eight years after my diagnosis I am living a life free from the pain and fatigue that multiple sclerosis can inflict. As far as I am aware I am the only privately funded and indeed only person on Gilenya in New Zealand. I hope my personal, and extremely positive experience, on Gilenya helps PHARMAC confirm that the decision to fund the drug for patients in New Zealand is the right one. Mellanie Ullrich