More work on access to high-cost, highly specialised medicines

NZORD has worked with a large number of support groups for over 5 years through the Access to Medicines Coalition, to win improvements to medicine policy and funding arrangements. Establishment of the Medicines Strategy in 2007 gave some reasons for cautious optimism that a number of important issues would be addressed by the Ministry of Health, DHBs and Pharmac, but subsequent responses from those agencies left us very disappointed, with no real change in sight.

Promises made by the new government formed in 2008 did lead to extra funding specifically for medicines in the 2009 budget, resulting in a number of new medicines being funded and wider access for many more. This positive step forward on medicine policy was matched by Health Minister Tony Ryall’s appointment of a panel to review access to high-cost, highly specialised medicines.  In December 2009  the panel produced an interim report to elicit discussion and feedback. Here is the submission in response to that interim report, submitted by the Access to Medicines Coalition in February 2010 and endorsed by NZORD.A response from the Minister to the panel’s final report to him, is expected in May 2010.

Submission to the Ministerial Review Panel on high-cost, highly specialised medicines:

  1. The review panel has approached the job of improving access to high-cost, highly-specialised medicines by looking at the big picture of the medicines system, and suggesting that improved access to HCHS medicines is most likely to come from improved efficiencies across the board.
  2. Such an approach might work in the long term in releasing more funds to target HCHS medicines, but it is unlikely to produce any improvement to access in the short to medium term. To produce the outcome which our groups want, and which the Minister has asked for a report on, there needs to be specific comment on:
    • Addressing the chronic underfunding of medicines that has occurred in the past
    • Recognising that specific new investment will need to be made to fund HCHS medicines
    • Re-focusing the draft report onto those medicines which are high-cost AND highly-specialised, (Top right box in diagram p7 of the interim report) rather than the much wider perspective taken to date by the panel
    • Providing more explicitly for the exceptions (that HCHS medicines inevitably are) and which provide major challenges for existing frameworks and decision-making. An overhaul of the Exceptional Circumstances scheme is needed if that is intended as the vehicle to deal with HCHS medicines.
  3. In concentrating on the big picture of the medicine system the panel has not given sufficient weight to some critical parts of the system, including how the medicines budget gets set, and the serious problems in that area which make things extra difficult for those in need of HCHS medicines. This is stated as an action point flowing from the Medicines Strategy but there has been no effective work on that since the strategy was published over two years ago.
  4. Ethical considerations of equity, community values and fairness are not adequately addressed. These are essential for all medicines decisions but especially so for HCHS medicines, and are specifically included in the medicines strategy. The panel’s comments about the wide variety of ethical perspectives is too general. Applied ethics in NZ’s health system should give priority for urgency and seriousness, and give fair consideration to small groups of patients who would be underserved by focusing on whole populations.
  5. Given that the panel does not recommend a separate decision-making process and budget for HCHS medicines, we support the alternative approach of suggesting the Exceptional Circumstances scheme as the method of addressing the needs for HCHS medicines. However we urge the panel to note the existing Community EC scheme is not appropriately structured and will need significant revision to make it suitable for dealing with HCHS medicines. The scheme must be made to work towards the intention of providing access to HCHS meds. We ask the panel to note:
    • The unsatisfactory experience of many patient groups trying to access a number of HCHS medicines through the existing Community EC scheme
    • The restrictive criteria and limited budget that together would not be effective in addressing access to HCHS medicines
    • The risk of unintended consequences of combining the three existing Exceptional Circumstances schemes
    The panel notes the Community EC scheme budget is $3 million and that it is mainly under-spent. In the absence of specific data available to indicate appropriate criteria and budget that would effectively improve access to HCHS medicines through Community Exceptional Circumstances, we suggest a threshold of less than 100 and a substantially increased budget, may be much more appropriate than the existing criteria and budget.
  6. The panel should note and advise the Minister that a suitable “entry” criteria for consideration as a HCHS medicine include one or more of the following factors, rather than just price alone:
    • Rarity of the disease and/or size of likely pool of candidates for treatment (reviewed upwards significantly from the existing “fewer than 10” guidance)
    • Complexity of the disease
    • Novel aspects/complexity of the medicine discovery and manufacture
    • Initial review under CUA puts it well outside the currently accepted range
    • Uncertainty of the evidence base for effectiveness of the medicine (which will often be the case for many years for rare orphan diseases), combined with expert opinion on the potential for benefit, both of which may suggest accepting less than ideal evidence in the absence of substantial controlled trials
  7. Other considerations

    We support the panel noting that decision making criteria for pharmaceuticals is far more stringent than the decision criteria used for other health interventions such as equipment and/or operations.  Given the importance of access to medications in improving the quality of life for those with chronic conditions the need to have decisions made on an equitable basis is paramount.

    We reinforce points made in earlier submissions that there needs to be greater transparency of certain criteria used by Pharmac in its decision-making. For example without compromising commercial considerations:

    • it should be a requirement to disclose more details about the assessment of such matters as downstream savings in the health sector, so this is transparent and its validity can be contested
    • there should be more specific opportunities for clinicians and patient groups to participate in Pharmac processes, including the exceptional circumstances scheme
    • there should be greater distinguishing of clinical recommendations from financial recommendations, such as PTAC or specialist committee clinical advice being distinguished from budget constraints

Yours sincerely,

for the ATM coalition

John Forman
Executive Director, NZORD