Re: Ibrance and Kadcyla Petitions

Transcription

1 8 February 2019 Ms Louisa Wall Chairperson Health Select Committee By Dear Ms Wall Re: Ibrance and Kadcyla Petitions Thank you for the opportunity to provide a written submission on the petition of Terre Maize, on behalf of the Metavivors, requesting: The Committee consider better access to life-extending drugs for breast cancer (Ibrance and Kadcyla). Summary PHARMAC has seen the petition submitted by Terre Maize - Fund Kadcyla and Palbociclib for breast cancer sufferers - and has assessed their evidence presented in person to the Health Select Committee. We take very seriously the concerns that have been raised and have carefully listened to what was said. We hope that our written response and appearance at the Health Select Committee provides more information that will enable the Health Select Committee to consider the petition. In this paper we will discuss PHARMAC s role in funding medicines in New Zealand. In particular we will cover access to cancer medicines in New Zealand and will also respond to questions raised by those petitioning for funding for breast cancer medicines. To follow is detailed information on how, as an organisation, we make funding decisions, why decisions are made and how we determine whether PHARMAC is delivering on its objectives of making funded medicines available to New Zealanders within a fixed budget. We also provide information on why decision-making timeframes can vary. A

2 Background PHARMAC was created in 1993 to ensure that New Zealanders get the best possible health outcomes from the money the Government invests in medicines used in the community. Since then, our role has expanded to include managing the comparative assessment, procurement and decision-making on funding for all cancer medicines, all medicines whether used in the community or in DHB hospitals, vaccines and certain haemophilia treatments, all of which are funded by District Health Boards (DHBs) through the Combined Pharmaceutical Budget (CPB). PHARMAC also negotiates national contracts for medical devices used in DHB hospitals. Following a Government decision in 2012, we re working towards budget management of medical devices in the longer term. Since its establishment, PHARMAC has increased the range of subsidised medicines available and met the cost of growth in demand for existing medicines within the amount of funding provided each year. PHARMAC must: Secure for eligible people in need of pharmaceuticals, the best health outcomes that are reasonably achievable from pharmaceutical treatment and from within the amount of funding provided. New Zealand Public Health and Disability Act PHARMAC s decision-making processes are deliberately independent of the Government. The amount of funding provided is the CPB, an amount decided by the Minister of Health following a joint budget bid submitted by DHBs and PHARMAC. The CPB is used to fund all pharmaceuticals already listed on the Pharmaceutical Schedule (including growth in demand for those treatments due to population increases or otherwise), any new pharmaceuticals added to the list or widening of the access criteria for already listed items ( new investments ) and any pharmaceuticals funded for individual patients via PHARMAC s exceptional circumstances decision-making processes. Decision-making timelines Funding decisions can take time and we understand the impact that this has on patients, their families and whānau. We take our responsibility to fund the right medicines very seriously. We have a fixed budget, which means we must make careful and considered decisions about spending any available money on medicines to deliver the best health outcomes for New Zealanders. At any point in time PHARMAC are reviewing many funding applications; each of these needs to be fully and carefully assessed. There are several stages in the assessment of a funding application. These ensure that decisions are robust and that we re choosing the right medicines to fund. This includes: seeking expert advice from practicing clinicians and other health professionals; reviewing developing evidence from applicants and other sources; applying our Factors for Consideration decision making framework; and A

3 comparing and ranking the medicine against all other medicines we ve had funding applications for.. PHARMAC uses a prioritisation process to compare and rank funding options. Possible opportunities for funding new medicines will always exceed the budget PHARMAC has available; therefore, prioritisation is an intrinsic part of PHARMAC s decision-making process. A funding application, or proposal is considered ready for prioritisation when sufficient information is available to permit it to be reviewed against PHARMAC's Factors for Consideration and to inform a comparison with other possible funding options. PHARMAC s decisions are made within the context of the Factors for Consideration (see Appendix One) a comprehensive decision-making framework that was established with considerable public input, outlining the relevant factors PHARMAC takes into account when assessing funding applications and making decisions. The Factors cover four aspects in relation to consideration of funding application: Need impact of the disease, condition or illness. Health benefits potential health gain and risks/losses from the medicine/medical device being considered. Suitability non-clinical features of the medicine/ medical device that might impact on health outcomes. Costs and savings fiscal impact of the pharmaceutical. We assess these four aspects across three levels of impact: on the individual; on family, whānau and wider society; and on the health system. This model ensures that decisions are based on the best available evidence and consider a wide range of factors. How PHARMAC seeks to achieve best health outcomes There will always be a greater demand for funded medicines than the available resources allow. PHARMAC s job to assess the potential health outcomes from a medicine and list for funding, is a complex and challenging one. To ensure that the funding of medicines within the budget results in the best health outcomes and is as fair and robust as possible, PHARMAC has an established funding process outlined in our Operating Policies and Procedures. This includes: evaluation of clinical evidence; comparing and ranking the medicine against all other medicines we ve had funding applications for; commercial negotiation; public consultation; decision-making; and notification and implementation. A

4 In 2017/ million New Zealanders accessed funded medicines; an increase of 2.2 percent on the previous year million prescription items were filled. 0.6% (250,000) were for funded cancer prescriptions. $220 million gross on cancer medicines (up from $203 million gross the previous year). 83,000 people accessed cancer medicines. PHARMAC listed four new cancer medicines for people with breast, head and neck cancers, mesothelioma and chronic lymphocytic leukaemia. o Zoledronic acid a treatment for post-menopausal women with early breast cancer that prevents the disease spreading to the bones. Up to 3000 women per year will benefit from this decision. o Cetuximab for people with head and neck cancer, benefitting up to 50 patients per year. o Bendamustine for chronic lymphocytic leukaemia benefitting up to 35 patients per year. This drug is also expected to help people with low-grade lymphoma (approximately 130 patients). o Pemetrexed for people with mesothelioma benefitting 40 to 50 patients. Pemextexed will also benefit around 400 first line patients and 50 second line patients who have non-small cell lung cancer. 13 new medicines were funded, including the shingles vaccine and aflibercept for degenerative eye conditions. 39 medicines had access criteria widened, including zoledronic acid for the adjuvant treatment of early breast cancer in postmenopausal patients. New Zealand was one of the first countries in the world to widen access to an HIV treatment, Truvada for pre exposure prohylaxis (PReP) of HIV infection. In December 2018 PHARMAC also announced approval of funding for Maviret (from 1 February 2019), a potential cure for 99% of the estimated 50,000 people with hepatitis C. This will lead to significantly fewer people developing liver failure, liver cancer and reduce the need for liver transplants. A

5 Specific Questions raised during the presentation of the petition. Does PHARMAC return money to the Government? No. PHARMAC does not return money to the Government. When savings are made, using the bargaining power of PHARMAC, these savings are used to fund more medicines or widen access to already listed medicines. As part of Budget 2018 the Government agreed that PHARAMC would assume budget responsibility for hospital medicines from 1 July 2018 and included in the CPB. PHARMAC s Board determined that the next best investment of available CPB funds in 2017/18 was in hospital medicines and accordingly reimbursed DHBs for their hospital medicines expenditure from 1 May to 30 June The level of the CPB for 2017/18 was $870.8 million and the actual final CPB expenditure was $870.8 million for 2017/18. In 2018/19 the level of the CPB was increased to $985.0 million to reflect the increased budget responsibility for hospital medicines. Should the budget be increased to purchase more cancer medications? PHARMAC has a fixed budget set by the Government and approved by the Minister of Health. This fixed budget means that PHARMAC negotiates as hard as possible to achieve the best price for medicines. As previously mentioned, any savings achieved are put towards funding more medicines or widening access to already listed medicines. In the last 8 years (since PHARMAC took responsibility for managing hospital cancer medicines), the amount spent on cancer medicines has increased by 50 percent. Around 83,000 New Zealanders accessed cancer medicines in 2017/18 at a gross cost of $220 million. This is over $100 million more than in 2011 when approximately 53,000 people were accessing cancer medicines. Of the top 20 medicines (across all medicine groups) by gross spend in 2017/18, five of them were treatments for cancer: Name of medicine Condition it treats Gross spend in 2017/18 Transtuzumab (Herceptin) Breast Cancer $36.7 million Pembrolizumab (Keytruda) Skin Cancer $23.6 million Abiraterone (Zytiga) Prostate Cancer $22.3 million Lenalidomide (Revlimind) Blood Cancer $21.3 million Rituximab (Mabthera) Blood Cancer $18.1 million Funding applications for palbociclib (Ibrance) and trastuzumab emtansine (Kadcyla) The funding application for palbociclib (Ibrance) and the funding application for trastuzumab emtansine (Kadcyla) were assessed by one of our expert clinical advisory groups in September This group, the Cancer Treatments Subcommittee of the Pharmacology and Therapeutics Advisory Committee (CaTSoP), provides PHARMAC with specialist advice on cancer treatments. A

6 Palbociclib (Ibrance) and trastuzumab emtansine (Kadcyla) were two of eight medicines considered by CaTSoP in September. Seven of these were recommended for funding, one was recommended for decline. Link to CaTSoP minutes: Palbociclib (Ibrance) PHARMAC received a funding application for palbociciib (Ibrance) an initial endocrine therapy for patients with locally advanced or metastatic, HER2-negative, HR-positive breast cancer in February In September 2018 CaTSoP advised that, while this treatment looks promising, the evidence only showed modest benefits for patients and the price is prohibitively expensive. The Subcommittee did, however, recommend it for funding for people who haven t already received a hormonal treatment for their metastatic breast cancer, with medium priority. PHARMAC has also recently received a funding application from a consumer group, Breast Cancer Aotearoa Coalition (November 2018), for Palbociclib (Ibrance) to be funded for people who have already received a hormonal treatment for breast cancer. PHARMAC needs more advice from our clinical experts on this second funding application, so we have asked the supplier for more detailed evidence about the use of Palbociclib (Ibrance) for people who have already received a hormonal treatment. Trastuzumab emtansine (Kadcyla) PHARMAC received an application for Trastuzumab emtansine (Kadcyla) for use as a secondline treatment for patients with HER-2 positive metastatic breast cancer who have already been treated with trastuzumab (Herceptin) and a taxane in August In September 2018 CaTSoP advised that the evidence was both weak and poor quality, and not directly relevant to New Zealand patients because the evidence did not assess the use of Kadcyla after treatment with pertuzumab (Perjeta). Pertuzumab has been funded in New Zealand for the same patient group since 1 January However, our clinical advisors considered that there would be a small number of people in New Zealand (100 or less) that will not have been treated with pertuzumab and recommended Trastuzumab emtansine (Kadcyla) for funding for these people. Our primary clinical advisory committee, PTAC, will consider CaTSoP s advice for both medicines in February Once it has been to PTAC, PHARMAC will rank the recommendations made and will assess it internally against any other funding applications that may have also been recommended for funding. Unfortunately we are unable to provide a definitive timeframe for if, or when, a funding decision would be made. This is because the relative priority of funding one medicine compared with other medicines can change over time. Details like the relative health benefits, the amount of funding available, the success of negotiations with suppliers, updated clinical data, and the mix of other funding applications being considered at any one time, are all examples of factors that may change the relative priorities of funding choices. A

7 Has PHARMAC ever been reviewed? Since PHARMAC s establishment in 1993, there have been a number of reviews, both internal and external, into aspects of PHARMAC s operations. These include: September 2000 Review of PHARMAC s Operating Policies & Procedures by Dr Joel Lexchin & Hon David Caygill undertaken at the request of the Minister of Health and the Health Funding Authority Ministerial Review Group Meeting the Challenge set up to recommend how New Zealand might improve the quality and performance of the public health system. Recommended an expansion of PHARMAC s role to include hospital medical devices Review of access to high cost, highly specialised medicines, a three-person Panel chaired by Dr Paul McCormack and including Joy Quigley and Associate Professor Paul Hansen. ecialised_medicines_31_april_2010.pdf 2013/14 PHARMAC carried out a comprehensive review of its decision-making criteria. We consulted widely across New Zealand as part of this review, including a series of 12 community forums at different locations around New Zealand. December 2017 the Ombudsman released the finds of an investigation into PHARMAC s decision making on cancer medicines Opdivo and Keytruda. The investigation found that PHARMAC acted reasonably and in line with its statutory objects of obtaining the best health outcomes within available funding. es/2773/original/case_note_pharmac_keytruda.pdf? Health & Disability System Review chaired by Heather Simpson. While PHARMAC is out of scope of this review, the relationship between the Health & Disability System and PHARMAC is within scope. Equity of access to funded medicines PHARMAC has set a goal of eliminating inequities in access to funded medicines by In 2013, our research told us there were 608,000 lost opportunities for Māori to access medicines and that resulted in 1.1 million medication treatments that Māori did not receive. That number is unacceptable and we will work with the wider health system to address this. The submission claims that Māori survival rates are unacceptably low, with only 5% of Māori with advanced breast cancer surviving 5 years. While we acknowledge that significant inequity in access and adherence to medicines exists for Māori, in the case of breast cancer survival the most important intervention is finding breast cancer at an earlier stage before it becomes advanced. The importance of early detection and making the breast screening programme more accessible for Māori and Pacific women is highlighted in the study 1 Ethnic disparities in breast cancer survival in New Zealand: which factors contribute? (Tin Tin 2018) led by Prof 1 A

8 Lawrenson of University of Waikato. The study found that stage at diagnosis accounted for a substantial proportion of the survival differential in Māori and Pacific women. Following extensive consultation and engagement with communities to identify the health areas they consider most important - Hauora Arotahi - and working with our Whānau Ora Collective partners on initiatives, PHARMAC will this year continue to work with communities and the rest of the health sector on addressing inequities in access to medicines. When we consider funding any medicine, our decision-making framework means we always include the potential benefits for Māori specifically. Comparison with other countries Australia is often cited as a place that funds medicines that aren t funded in New Zealand. A simple count of funded medicines won't show you how successful a health system is. When it comes to having funded access to medicines, it s not about how many you have, but having access to the ones that deliver meaningful benefits. PHARMAC works to ensure that, within a fixed budget, New Zealanders achieve the best outcomes possible from public expenditure on pharmaceuticals. New Zealand must make its own decisions, carefully assessing the available evidence and thinking about medicines use in the New Zealand health context. Research on medicines in Australia compared to New Zealand called Mind the Gap was completed in It showed that New Zealand funded fewer cancer medicines than Australia (102 v 124); however, it also showed that New Zealand had avoided funding a large number of cancer medicines that offered little or no clinically meaningful benefit for patients relative to currently funded options in New Zealand, and in some cases, PHARMAC has avoided funding medicines that deliver health losses. The research concluded that, while New Zealand funds fewer cancer medicines than Australia, most of these additional medicines did not deliver clinically meaningful health gains in terms of extending time to disease progression or death for cancer patients. This suggests that simply funding more cancer medicines to achieve funding parity with Australia would likely not represent good value for money in terms of delivering the best health outcomes for New Zealanders. PHARMAC s method for funding new medicines that demonstrate clear clinical benefit and are cost-effective and affordable, ensures that health dollars are not directed towards medicines that are unlikely to deliver clinically meaningful health gains to patients and are diverted away from medicines that would deliver benefits Many new medicine approvals are made on the basis of early data that has significant limitations. Later evidence often shows that these medicines do not in fact offer meaningful benefit in overall survival. In the US, a study 2 looked at 36 cancer medicines approved by the FDA between 2008 and 2012 on the basis of surrogate outcome measures (such as reduction in tumor size or progression free survival) and reported that 4 years later only 5 were shown to improve overall survival. The remaining 31 medicines failed to improve overall survival or continued to have unknown survival effects. 2 A

9 Analysis from the European Medicines Agency's approvals for cancer medicines between report stated that most entered the market without evidence of benefit or survival gain. More than three years afterwards, there was often no conclusive evidence that lives were extended or improved for most cancer indications, and where improvements were demonstrated, they were often marginal. Many new medicines are launched and registered for sale in New Zealand without clear evidence that they work as well as the pharmaceutical companies claim. These treatments can look promising, but we need to be absolutely sure they will deliver the benefits claimed and that we spend public money wisely. If we fund a medicine that has high uncertainty about its benefits and risks, the reality is that it would take away funding from other more proven treatments. PHARMAC understands that patients, their families and whānau and clinicians want the latest medicines in the hope they will provide the best possible health result. We too want to fund new medicines to improve the range and effectiveness of medicines available for all New Zealanders and deliver best health outcomes. Thank you for taking the time to consider our response we are happy to answer any additional questions you may have. Yours sincerely Sarah Fitt Chief Executive PHARMAC 3 A

10 Appendix One: Factors for Consideration After meetings across the country to discuss what s relevant when assessing pharmaceuticals, in 2016 we changed the way we make decisions; the most significant change to PHARMAC s decision-making in 23 years. Building off what we previously used decision criteria our review identified that a clearer framework was required to increase transparency of how we make decisions. We also needed to ensure greater visibility of all considerations important to funding decisions. The three circular rings the layers ensure we think carefully and broadly about the pros and cons of funding choices for: 1. people who may benefit from treatment; 2. their family/whānau and wider society; and 3. the wider health system. The four segments the dimensions ensure we group similar considerations together and undertake a robust assessment of everything relevant to a dimension. Some of the factors may be more or less relevant depending on the nature of the decision being made. Judgement is always required to ensure we respond appropriately to different situations. We also always consider one funding option relative to others to ensure we make the best choices for New Zealand. Interactive: Factors for Consideration A

11 Factors for Consideration A

12 In considering need, we focus on how valuable a new or additional treatment would be. In 2016, treatment for Hepatitis C an area without good treatments previously was a good example of addressing a clear health need. We think hard about all the benefits of funding a medicine, whether for an individual, their family and/or the wider health system. Understanding all these benefits, and their likelihood of eventuating, is central to our assessment work. Just as benefits need to be identified, so too do all the costs and savings of a funding option. We often hear that PHARMAC doesn t include costs and savings of its decisions to the wider health sector (including DHBs) but we certainly do factor these into our assessments. We ve all had times when something we wanted to use had potential but wasn t ultimately fitfor-purpose. We avoid such situations by carefully assessing the suitability of pharmaceuticals, including a number of important practical considerations. A