PHARMAC's responses to submitter's views (text extract)

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Pharmaceutical Management Agency

Review of Exceptional Circumstances

Responses to Submitters’ Views

June 2011

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Introduction This Responses to Submitters’ views document provides PHARMAC’s responses to key points raised in submissions on proposed changes to the way PHARMAC assesses applications for treatments for patients with exceptional circumstances. Changes to the Named Patient Pharmaceutical Assessment (Exceptional Circumstances) Policy made as a result of submitter comments are described. We have also endeavoured to explain in this document our rationale for retaining aspects of the Policy that submitters suggested should be changed. For the purposes of this summary table, respondents’ comments have been shortened and paraphrased. A more comprehensive summary of submissions is available on PHARMAC’s website (http://www.pharmac.govt.nz/healthpros/haveyoursay/ecreview). All individual submissions, along with the summary of submissions and this document, were provided to and considered by the PHARMAC Board, as part of the decision making process. Please note the purpose of this document is to provide short responses to the issues raised by submitters for those who do not necessarily wish to read through the new NPPA Policy. The responses in this document outline some of PHARMAC's considerations in developing the NPPA Policy. The NPPA Policy itself sets out PHARMAC's final decisions on the Policy and the basis on which PHARMAC will consider applications under the Policy.

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SUMMARY OF SUBMITTERS’ VIEWS AND PHARMAC’S RESPONSE

We have organised the comments into the following 5 categories:      General comments (including those in respect of the principles behind the proposal), Comments relating to Decision Criteria, Comments on the Unique Clinical circumstances Scheme, Comments on the Urgent Assessment Scheme, and Comments on the Hospital Pharmaceuticals in the Community Scheme.

Changes to the Policy as a result of feedback are highlighted in the PHAMAC Response box.

General Comments SUBMISSION COMMENT

A large number of submitters agreed the purpose of the EC scheme is to provide funding in situations where the Schedule process is not appropriate. Some submitters considered the scheme as detailed in the consultation was better defined and its intent was clearer. A number of submitters supported the focus on the clinical circumstances of the patient.

PHARMAC RESPONSE AND POLICY ACTION

PHARMAC has reflected the majority view about the purpose of the EC scheme in the NPPA Policy, which states (Section 3) that:

The provision for exceptional circumstances is an acknowledgement that there are situations in which consideration of an application for a treatment for an individual, outside of the Schedule decision making process used to consider treatments for patient populations, is warranted.

As discussed in the consultation document, we seriously considered in developing the draft proposal for consultation whether we could target the prerequisites for individual assessment to specifically cover rare diseases. We concluded, however, that we would be unable to do this without significantly undermining the Schedule process, chiefly because there is no robust rationale for treating pharmaceuticals for rare diseases differently from other medicines. Submitter comments on this topic did not change our view. In response to submitter comments, we have modified the proposal to allow a named patient that has a set of clinical circumstances that is so unusual that PHARMAC is unlikely, for administrative reasons, to consider using the Schedule listing process to assess these, to be considered through the Unusual Clinical Circumstances pathway (originally proposed as ‘Unique Clinical Circumstances’ in the consultation document).

A number of submitters considered the proposal did not adequately address the issue of rare diseases.

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SUBMISSION COMMENT

A few submitters considered equity and fairness were key elements of an EC scheme, and a subset of these considered the proposal did not meet PHARMAC’s obligations with respect to fairness. One submitter considered that fairness would mean patients who need them should have access to newly developed innovative medicines. One submitter considered that funding decisions should reflect societal values and take account of ethical implications.

PHARMAC RESPONSE AND POLICY ACTION

PHARMAC agrees that ideally all patients would have access to the medicines that may provide them some benefit, however there will always be more interventions in medicines that we can afford to buy, so hard choices will always need to be made. We consider fairness in process is very important, and we endeavour to fairly account for people’s clinical circumstances in all our decisions while still meeting our objective of providing the best health outcomes from within limited funding. PHARMAC considers that the criteria in the Policy are fair. PHARMAC’s role is to provide the best health outcomes within available funding. Societal values are reflected in the legislative framework. Pharmac operates ethically within that framework Therefore, PHARMAC considers there is no need to expressly include societal values in the NPPA Policy. The focus of the scheme is to allow consideration of patients’ individual clinical circumstances that have not been, or are unlikely to be considered in the normal schedule listing process. One category of these patients is those with very unusual conditions, however patients with urgent clinical circumstances who may be part of a group are also explicitly included in the scheme. PHARMAC also has discretion to consider patients that do not fit neatly into the described pathways. PHARMAC’s discretion outside of the situations described in the Policy is clearly described (in Sections 1 and 4(b) of the Policy. The prerequisites for some of the NPPA pathways, and the criteria against which all NPPA applications will be assessed require PHARMAC to take into account the named patient’s clinical circumstances.

Some submitters considered the scope of the scheme was too limited, and it appeared patients needed to be unique to qualify.

A few submitters considered the patient’s clinical and social circumstances should be taken into account.

We expressed the view in the consultation document that the ethical, legal and practical issues associated with consideration of a named patient’s non-clinical circumstances means it is not appropriate that PHARMAC take them into account in decision-making. Submitter comments on this point did not change our view. PHARMAC considers, as stated in the NPPA Policy (in Section 3) that:

Some submitters were concerned that the proposed scheme did not account for all patients.

Together the Schedule decision making process and the exercise of PHARMAC’s discretion to consider funding in exceptional circumstances ensure that there is a pathway for consideration of an individual’s clinical circumstances. If an individual has a 2

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set of clinical circumstances not covered by the NPPA Policy, the Schedule decision making process is available.

A few submitters considered that the scheme should allow patients to apply for treatments not funded on the Schedule despite being assessed. One of these submitters considered that the scheme should consider the individual patient’s circumstances, and therefore the group decision is less important.

Under both the Unusual Clinical Circumstances pathway and the Urgent Assessment pathways, PHARMAC can consider funding for a named patient where their circumstances differ to those considered when a Schedule assessment has been undertaken. In addition, PHARMAC will consider applications made under the Urgent Assessment (UA) pathway for treatments that it is considering for listing on the Schedule if, before starting that consideration, we have approved funding for any patient under UA and the persons who apply subsequently are in the same clinical circumstances.

A few submitter considered innovative medicines should be covered in the proposal, particularly when this is the only viable or best treatment option for the patient in question. The Group considered that these medicines would inevitably fail in the Schedule assessment process where they are for small groups of patients due to weakness in supporting evidence. The Submitter considered the scheme could be used to trial treatments where evidence was limited. A number of submitters considered it was unclear whether PHARMAC would consider unregistered medicines, or unapproved indications for medicines as part of the Scheme. Most of these submitters considered applications involving these medicines should be considered. A few submitters considered that palliative care may not be covered by the proposal. A submitter considered that patients who were unable to tolerate alternatives or for whom these alternatives were ineffective, but whose condition may not be deemed ‘urgent’ effectively have no avenue under the

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PHARMAC does not require that every medicine we fund is supported by the highest grade of clinical evidence, and takes clinical advice on the merits of all applications. PHARMAC has in the past approved funding for patients for a period of time with renewal criteria, effectively funding a trial for the patient, and we will continue to maintain this as an option. We consider the test of whether a medicine is innovative or not is in how much benefit it provides to the patient.

PHARMAC currently does, and will continue to consider medicines for unapproved indications, or that are unregistered, provided the registered alternatives have been considered.

Patients receiving palliative care are not excluded from the proposal, and treatments in this area would be considered on the merits of each application. While the individual clinical circumstances would be considered, in general we consider Schedule Assessment would be appropriate in these cases, although we note the UCC pathway now applies to unusual rather than unique conditions.

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proposal unless they are unique.

PHARMAC RESPONSE AND POLICY ACTION

The NPPA Policy states (Section 4(d)) that One submitter asked if hospital treatments would be considered under the UCC or UA schemes.

PHARMAC is in a transition phase with respect to the management of non-cancer hospital medicines. While PHARMAC will generally not consider in-hospital treatments under the NPPA Policy, PHARMAC may consider applications for therapies for chronic conditions which are delivered outside the community setting.

A few submitters considered that alternative formulations should be able to be considered, for example a non-registered liquid form where stability data for a compounded suspension is not available. Many submitters supported the principle of assessing applications for Schedule listing, however a number of submitters considered there would be practical difficulties with this, particularly where no one wanted to register the medicine here, or there was less evidence available. There were also some concerns that these applications may displace assessment of other funding applications. There was also concern that this would lead to a gap in funding for patients with the proposed end to NPPA applications once the Schedule application is prioritised. Submitters were split on whether earlier wider access at the expense of consistency was better than narrower access initially with greater consistency. Some considered that as long as decisions were fair and transparent it was okay for PHARMAC to approve an application and decline one that might appear similar. Others felt consistency in decision making was very important to the credibility of the process, and acknowledged that this may come at the expense of earlier approvals.

Where a patient’s clinical circumstances meet the prerequisites of the NPPA Policy, these kinds of treatments would be considered.

We agree that there are often significant issues to address to progress a product for Schedule listing. More recently PHARMAC has been reviewing items going through Exceptional Circumstances (particularly Hospital Exceptional Circumstances) to see if a listing is appropriate, and the proposal was an expansion of this. Ideally every medicine listed on the Pharmaceutical Schedule would have a clear, registered supply source that provides some long term certainty to patients on the medicine that it will be there as long as they need it, however we will have to balance this ideal with the practical situation in each case, and are open to the possibility of listing a product that does not meet all these criteria where the only foreseeable alternative is not funding it at all. PHARMAC recognises that there is discomfort with the possibility that similar applications may have different results under the process. In particular, we noted submitter concerns about the proposal that PHARMAC would not consider named patient applications under the Urgent Assessment (UA) pathway for a treatment that had been prioritised even if PHARMAC had approved NPPA applications for named patients in the same clinical circumstances prior to prioritisation. In response to this feedback, we have specified in the NPPA Policy (Section 4(a)) that: The UA pathway will be available for named patient applications received after PHARMAC has started to consider the treatment for listing on the Schedule if, before starting that

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consideration, PHARMAC has funded any patient under this pathway and the named patient applications received subsequently are for the same clinical circumstances. We are still, however, required to consider each application on the information available at the time. We have clarified the implications of this in Section 4(h) of the Policy as follows:

PHARMAC recognises there is a public expectation that people experiencing the same clinical circumstances should have the same outcome from the application process. PHARMAC will endeavour to take an approach to approving NPPA applications which will achieve consistency over time to the greatest extent possible. However, in considering the Decision Criteria, relevant factors other than the clinical circumstances of the named patient (including evidence of the effectiveness of the treatment and the available budget) may differ over time. It is therefore possible that, due to such factors, PHARMAC may make different decisions for patients with the same or similar clinical circumstances.

We have addressed this by including the following, in section (4(j)) of the Policy: Some submitters considered that clinicians should be able to reapply if an application was initially declined where evidence has developed further.

Declined applications can be resubmitted at any time if relevant new clinical circumstances arise or new evidence becomes available. PHARMAC will treat resubmitted applications as new applications, but will report on new applications and resubmitted applications separately so that demand is not overstated..

We agree that increasing the budget for the NPPA scheme would change the mix of treatments funded rather than improve access to treatment overall. PHARMAC will be discussing with DHBs the possibility of increasing the allocation for the NPPA scheme in order to readjust the mix of treatments provided in favour of patients that might meet the NPPA criteria. This discussion will take account of any pressure on other funding applications that may arise. We agree with most submitters, in that cancer treatments should be considered alongside other treatments. While cancer is a particularly threatening disease, there are certainly other conditions that can be as deadly or debilitating which are equally worthy of consideration. PHARMAC does consider health need as part of each funding decision, and people affected by serious illness have a higher health need.

Some submitters were concerned that increasing the budget for exceptions would put pressure on other funding applications. Others acknowledged this impact, but considered it would result in a better balance. Most submitters that commented supported considering cancer treatments in the same way as other treatments. A small number of submitters considered these should remain separate, with one suggesting society appeared willing to pay for cancer treatments that were less cost-effective.

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SUBMISSION COMMENT

There were differing views between a few submitters that commented about the budget arrangements. A few submitters clearly stated support for the position that funding should not be separated from the community pharmaceutical budget, while a few submitters considered a separate pool was the better way to go.

PHARMAC RESPONSE AND POLICY ACTION

We acknowledge the differing views around this point, but agree with the High Cost Highly Specialised Review Panel’s recommendation which is to not have a separate pool. Whether or not the funding for these medicines is separate or part of the pharmaceutical budget as a whole does not remove the requirement to make allocation decisions between them, and these decisions are less responsive when artificial silos have been created. Additionally, the success of the NPPA scheme would not be measured by the amount of funding allocated from within it, given PHARMAC’s intention to consider NPPA applications made under the UA pathway for Schedule listing. If successfully listed, funding would shift from NPPA to the Schedule. We have addressed this with the inclusion of the following in Section 7 of the NPPA Policy:

Some submitters sought clarification on how existing EC patients would be handled, with one stating clearly that PHARMAC should ‘grandparent’ those patients.

Any individuals receiving funding for treatments under Community Exceptional Circumstances or Cancer Exceptional Circumstances will continue to receive this. PHARMAC cannot guarantee continued funding of treatments approved under Hospital Exceptional Circumstances as District Health Boards are directly responsible for the provision of such funding. Applications for renewal of funding for treatments approved under Community Exceptional Circumstances, Cancer Exceptional Circumstances and Hospital Exceptional Circumstances will continue to be assessed against the criteria for these schemes.

A few submitters suggested PHARMAC consider international models such as those used in Australia and the UK for funding small populations and/or innovative medicines. A number of submitters considered there needed to be smooth transition from the current scheme to any new scheme. Some submitters considered an implementation plan needed to be clearly described. Some submitters considered the clinical advisors for EC should include a specialist relevant to the condition

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PHARMAC is aware of international models for medicines funding. The Schemes cited by these submitters all result in consideration of defined populations in isolation. We note that the High Cost Highly Specialised Medicines Panel review recommended against separate consideration. PHARMAC agrees that there should be a smooth transition to the new scheme. We intend to begin using the new scheme from 1 March 2012. This will give us the time we need to make sure the needs of current patients will continue to be met, put in place the administrative processes required for the new scheme, and bring stakeholders and applicants up to speed with the new processes. PHARMAC will take these comments into account as we finalise details on the

SUBMISSION COMMENT

being considered. There was considerable support for an electronic application system, although some submitters indicated the support was conditional on the system being user friendly. A number of submitters considered an appeal or review process was very important. One respondent considered it was important the review involved an independent arbiter. A few submitters considered periodic audits should be conducted to ensure the system is working appropriately. One submitter considered these audits should include follow up of outcome for the patient. Several submitters considered the Scheme needed to be associated with good communication, particularly around reasons for accepting and declining applications. One submitter was concerned about this publication being misleading if it was not accompanied by why the application was accepted or declined. Submitters generally supported the proposal to fund some section 29 medicines via the Schedule rather than EC in future where the use was not exceptional. A few submitters were concerned this might result in funding for some treatments being withdrawn, and one submitter considered that off label usage should remain under the Exceptions Scheme as it was special usage. Another submitter considered PHARMAC should be careful about funding unregistered medicines as it may discourage registration of the medicine with Medsafe.

PHARMAC RESPONSE AND POLICY ACTION

implementation of the NPPA scheme before 1 March 2012.

We welcome this feedback, and will be working towards launching an electronic system in time for the 1 March 2012 implementation date.

The NPPA Policy specifies (in Section 4(k)) that PHARMAC will establish a review process and submitter comments on this will be taken into account as PHARMAC determines the detail of this.

PHARMAC agrees that it will be important to evaluate the performance of the NPPA scheme over time.

We agree that good communication is important, and will be making available publicly the outcome of applications. However, we do not consider it appropriate to publish reasons for decline or acceptance.

Ideally every medicine listed on the Pharmaceutical Schedule would have a clear, registered supply source that provides some long term certainty to patients on the medicine that it will be there as long as they need it, however we will have to balance this ideal with the practical situation in each case, and are open to the possibility of listing a product that does not meet all these criteria where the only alternative at the time is not funding it at all. In some cases it may be appropriate for PHARMAC to keep some section 29 medicines under close review by a panel of clinicians, however in cases where these applications are almost always approved, these medicines are effectively being funded anyway, and a listing on the Schedule would reduce paperwork for clinicians.

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Use of Decision Criteria SUBMISSION COMMENT

Some submitters supported using PHARMAC’s Decision Criteria in considering NPPA applications, as it would provide consistency, equity and transparency while others expressed reservations about the use of decision criteria as in their view these did not apply well to individual patients. Some of these responses suggested aspects that may be considered under the criteria that would be relevant to individual patients. One respondent considered that the use of the decision criteria would be inequitable to small patient groups as these treatments would be unlikely to be able to be assessed against the cost-effectiveness criterion.

PHARMAC RESPONSE

PHARMAC agrees with those submitters who supported the Decision Criteria. The NPPA Policy states (section 4(f)) that:

PHARMAC will use the Decision Criteria to assess both the individual clinical circumstances of each NPPA applicant, and the implications of each NPPA funding decision on PHARMAC's ability to meet its objective for the population as a whole.

Cost-utility analysis (CUA) estimates the additional benefit of medicines being considered for funding on a per patient basis so the size of the patient population does not affect the CUA result. Details of PHARMAC’s CUA process can be found online at (http://www.pharmac.govt.nz/suppliers/EconomicAnalysis/pharmacoeconomics). It is important to note that the standard of evidence is different in all cases, even between different Schedule listing applications, and that not everything that gets funded has been the subject of a randomised control trial. Under the decision criteria, PHARMAC considers the potential for benefit against the ‘risk’ that greater benefit elsewhere would be forgone, and the criteria themselves do not require a particular level of evidence. They are merely a framework under which to focus the judgements required on the relevant factors in that judgement. PHARMAC has decided to retain its policy on this issue, as stated in Section 4(g) NPPA Policy:

Some submitters expressed concern about the level of evidence that is available for some treatments, suggesting that if the same standard is applied as for Schedule decisions, very little would be funded via this mechanism.

Several submitters disagreed with PHARMAC’s proposal not to consider results from self funded trials. In their view this was ignoring relevant information.

PHARMAC will consider applications for named patients who have already received the treatment being applied for, where this treatment has not been funded under a PHARMAC approval. However, in considering such applications, PHARMAC seeks to ensure that applicants who have not received NPAFT have the same opportunity to obtain publicly funded pharmaceuticals as those who have. PHARMAC will therefore not consider information obtained from NPAFT about the effectiveness of the treatment for the applicant specifically, unless PHARMAC is satisfied that to do so would not 8

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undermine equity of opportunity for all applicants, whether or not they have received NPAFT.

Submitter comments on this issue did not change our view for the reasons stated in Policy provision.

Unique Clinical Circumstances (UCC) SUBMISSION COMMENT

A few submitters noted that while it was difficult to meet the test described for UCC, its purpose was clear, in that these patients would be very unlikely to be considered for a Schedule listing. Many submitters considered the criteria for UCC were too restrictive. In particular a number of submitters considered while it was appropriate to restrict this pathway to unusual or rare circumstances, unique was so unlikely that anyone would meet the criteria. A range of alternative names including ‘Unusual Clinical Circumstances’ was suggested. One clinician queried why unique circumstances should warrant a separate pathway, and considered that the patient’s failure on current treatment or underlying disease progression should be the more important factors. One respondent considered PHARMAC should provide some examples of circumstances that might meet the requirements of the proposed scheme.

PHARMAC RESPONSE

Taking account of the many responses on this point, PHARMAC has amended the NPPA Policy to allow for a wider group of patients under this category, and renamed it Unusual Clinical Circumstances as suggested. The intent of this category is still to consider patients whose circumstances are so unusual that PHARMAC is unlikely to consider a funding application for Schedule listing for their particular circumstances, however we have removed the requirement that it be unlikely there is another patient in those circumstances

We consider it is appropriate to have a process for patients whose condition is so unusual that a Schedule application is unlikely to be considered to ensure these patients have a pathway to request funding. That process would naturally still consider what alternatives exist for those patients, and the degree of benefit the patient might receive from the requested treatment. PHARMAC intends to begin publishing results of applications under the NPPA scheme from 1 March 2012, which should assist clinicians and patients in deciding what types of applications are successful. While this should form a useful guide, decisions will be made on an individual basis, and clinical circumstances vary from patient to patient. Where a patient’s clinical circumstances vary considerably from the norm, it may still be worthwhile making an

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application for a treatment that has been declined previously in a high number of cases.

Several submitters considered it was a positive point that UCC would consider the clinical circumstances of the patient, and not simply the prevalence of the particular disease. Some submitters suggested rewording the prerequisites for UCC in a number of ways including removing the requirement to trial “all” funded alternatives, and including patients who have intolerable side effects to treatment, or are likely to experience intolerable side effects A few submitters considered there should not be a requirement that the patient has ceased treatment with all alternatives, as while these treatments may be less than fully effective, or have very unpleasant side effects, not treating at all may be worse for the patient. This was also suggested for UA. A submitter suggested that patients be able to apply for treatments without trying all alternatives if using the alternative would remove the treatment as an option (for example if the treatment was only indicated for first line treatment). This was also suggested for UA.

We also consider this to be an important part of the NPPA Policy.

We have taken these comments into account and have amended the policy to say the patient has “reasonably” tried all alternatives. Please note our comment regarding side effects below, also.

PHARMAC has amended the section (4(a)) in the Policy to take these circumstances into account (additional wording is in bold):

The patient has reasonably tried and failed all alternative funded treatments (or alternative treatments have been contraindicated, or there are no other treatments available), or has experienced such serious side effects with all other relevant funded treatments that treatment has been ceased or cannot reasonably be continued.

We have amended the Policy wording to reflect that all reasonable alternatives have been tried. We note that this change has been made for UA too.

Some submitters queried what would happen under UCC in the event an approval is given for one patient, and then another patient appears.

We have amended UCC to allow consideration of patients with unusual, rather than unique clinical circumstances where PHARMAC is unlikely to assess the treatment through the Schedule process. However, where it becomes apparent that the population has been dramatically underestimated PHARMAC will need to review the most appropriate place for consideration of these patients. Should the condition and clinical circumstances meet the requirements of Urgent Assessment, this may be the most appropriate pathway, or alternatively Schedule assessment may be appropriate.

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SUBMISSION COMMENT

One submitter considered applications should be permitted for treatments that had been considered for listing on the Schedule, as clinical circumstances vary, and the patient may have a unique set of circumstances relative to what was considered.

PHARMAC RESPONSE

The individual clinical circumstances of any patient will be considered, so where these vary significantly from what was considered for Schedule listing, we consider the patient would be eligible for consideration under UCC (subject to meeting other criteria).

Urgent Assessment (UA) SUBMISSION COMMENT PHARMAC RESPONSE

We are pleased with the support for the UA pathway. We consider this is perhaps the most significant change to the process as a result of this review, and will provide a more transparent mechanism for considering funding for patients whose condition may not be very unusual, but who may be particularly disadvantaged if required to wait until a medicine is considered via normal processes. Obviously there can be no certainty that all applications made under this pathway will be funded. As with all NPPA applications, decisions on applications under this pathway will be assessed in accordance with the Decision Criteria. As with most decisions PHARMAC is required to make, a degree of judgement needs to be exercised with EC decisions, and this will continue under the NPPA scheme. Part of this judgement is to assess the strength of the evidence against the possibility the benefit will not be realised, or is not as great as suggested taking account of the impact on the patient’s circumstances if the benefit does occur. These are often difficult decisions, but clinicians should not be discouraged from submitting applications simply because they consider the evidence is not as good as other applications, particularly where the capacity for the patient to benefit is very high. Patients funded via the existing EC scheme will continue to receive that funding provided any conditions of that funding continue to be met. Section 7 of the NPPA Policy states:

Many submitters supported the proposed purpose of the UA pathway. Some respondents considered the pathway would allow patients who currently are not considered under EC to obtain access to pharmaceuticals.

Some submitters considered flexibility in applying the criteria would be required to achieve any benefits from this process. Another submitter considered that it may be beneficial where evidence is available, but there may be difficulties where data is harder to come by.

There was some concern that cases that are currently receiving funding via EC may not qualify under proposed criteria.

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PHARMAC RESPONSE

Some submitters were uncomfortable with the possibility that patients in similar clinical circumstances may not have the same outcome.

This has been discussed in detail above and the proposal has been amended to respond to this concern. Please refer to our comments on page 6.

Many submitters sought further clarification on what might be classed as “urgent” or “serious deterioration” or “significant decline”. A number of submitters considered interpretation of these terms was critical to the Scheme, while one submitter considered an implementation guideline would be useful to assist applicants.

PHARMAC notes first that it has decided to use the words "significant deterioration" in the NPPA Policy rather than "significant decline". We have clarified this prerequisite by noting that not receiving the treatment would lead to significant deterioration in a serious clinical condition or the patient would miss the opportunity for significant improvement in clinical outcome (length or quality of life). We note also that when determining the appropriate level of detail to include in the NPPA Policy, we have been mindful of the need to strike a balance between providing sufficient clarity and specificity to enable potential applicants to determine whether they should apply, and retaining the flexibility for PHARMAC to exercise its discretion. The more prescriptive the Policy, the less it serves the purpose of providing a flexible framework within which PHARMAC can consider the wide and potentially unanticipated range of circumstances that may arise. A degree of judgement will always need to be exercised around these terms. Clinical advice, both from the applying clinician and from PHARMAC’s clinical advisors will be important to this consideration.

We agree this is a risk, and we will work to minimise this. We acknowledge there is a need to strike a balance between considering funding quickly for individuals with a pressing need, and balancing population benefit across all treatments. PHARMAC is not able to require suppliers to make an application. It is our intention to invite

A few submitters were concerned that the relationship between Schedule assessment and UA might lead to the shortcutting of the Schedule review process. Some submitters queried whether the consideration of

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a product for Schedule listing following an application under UA would require the supplier to submit an application for funding.

PHARMAC RESPONSE

a supplier to make an application once a decision has been made consider a treatment for which applications have been made under UA. However, if the supplier is unable or unwilling to do so in a reasonable time period, PHARMAC would look to consider listing without a supplier application. We have amended the original proposed prerequisites based on submitter feedback and consider these to be reasonable. PHARMAC has expressed the prerequisites as "and" statements. We consider that requiring patients to only meet one criterion would not be consistent with the Policy intent of the NPPA scheme.

A few submitters considered the proposed prerequisites should be expressed as ‘or’ statements rather than ‘and’ statements (ie the patient would be required to meet one prerequisite rather than all of them). One submitter queried whether a patient would be considered if they had not tried all alternatives, but doing so would likely lead to significant deterioration compared to the treatment applied for.

We have amended the criteria to allow a patient in these circumstances to be considered provided they have tried all reasonable alternatives.

Hospital Pharmaceuticals in the Community (HPC) SUBMISSION COMMENT

One submitter considered it was unclear where funding would be sourced from under the proposal, and considered it should be made clear that the DHB Hospital is still responsible for the funding. A number of submitters expressed some concern that long term patients were currently being funded via HEC, and were confused as to where these patients would sit if treatment was for longer than the proposed 3 month limit. One submitter considered that there should not be a limit as the savings to the DHB are not time limited. Others expressed concern that patients are managed via clinics often on a 6 monthly basis,

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PHARMAC RESPONSE

The DHB Hospital would continue to be responsible for the funding under the new scheme, and we have amended the Policy to reflect this.

We consider that HPC has never been intended to fund chronic treatment, which is generally meant to be funded from primary care sources such as the Pharmaceutical Budget. Indeed many treatments are listed on the Pharmaceutical Schedule on the basis that they reduce hospitalisations longer term. Long-term funding of community treatments for chronic conditions by DHBs outside the Schedule could give rise to inequities in access and could undermine PHARMAC’s ability to negotiate for listing for the community. Patients with urgent and serious conditions may be able to seek funding via the Urgent Assessment pathway. Those pharmaceuticals for patients with more stable chronic illness

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and the timeframe would not allow for renewals. A few submitters considered it was unclear how PHARMAC would exercise its discretion around applications for more than 3 months treatment under HPC.

PHARMAC RESPONSE

would be assessed via Schedule assessment.

PHARMAC’s discretion enables us to consider an argument put forward by the applying clinician as to why such a treatment should be funded despite the three month limit.

A number of submitters considered the scheme would likely be affected by PHARMAC’s work to create a National Preferred Medicines List for DHB Hospitals.

PHARMAC will be reviewing the Named Patient Pharmaceutical Assessment (exceptional Circumstances) Policy arrangements fit with the proposed National Preferred Medicines List before any list is put into effect. It may be that the NPPA scheme will need to remain in some form until budget arrangements for hospital pharmaceuticals are altered, which is likely to be a number of years away. This would be considered on a case-by-case basis. Generally offsets might include reduced days in hospitals, reduced interventions (including potentially other pharmaceuticals), but any legitimate cost to the DHB would be considered. We agree that the best place for any treatment that is truly cost saving should be listed on the Pharmaceutical Schedule. In some cases however the saving would not accrue to the Combined Pharmaceutical Budget (CPB) and if there was insufficient reserves to fund a treatment from the CPB we consider it important that DHBs are not prohibited from realising that saving through use of other funds. In the longer term we would work with DHBs to move these treatments to the Pharmaceutical Schedule as appropriate. Our intention is not to block legitimate applications, and we agree that admission prevention should be considered as an offset in general. We have removed the provision from the NPPA Policy but continue to be unlikely to approve the minority of applications that indicate that if PHARMAC doesn’t agree to fund a treatment the patient will be admitted solely for the purposes of administering the same treatment, and in the absence of any other reason to admit the patient.

A few submitters queried how cost saving to the DHB would be determined.

A few submitters expressed some discomfort with the concept of HPC, with one indicating it did not understand how PHARMAC would justify not listing a treatment that is cost saving to DHBs.

Some submitters were concerned about PHARMAC’s statement that it would not generally take into account the cost of admission solely to administer the treatment into account, as they considered admission prevention to be a valid cost offset.

Some submitters queried why the applying clinician needed to be a DHB clinician, noting that in some cases currently DHBs would approve funding for applications received from general practice.

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As the DHB is responsible for the funding of medicines under HPC, we consider it is appropriate for the application to be made by a clinician working on behalf of the DHB.

SUBMISSION COMMENT

PHARMAC RESPONSE

We have amended to Policy to state that only cheaper funded alternatives need to have been tried prior to application under HPC. The new wording (included in Section 4(a) of the Policy) is: The patient has reasonably tried and failed all alternative cheaper funded treatments (or these alternative treatments have been contraindicated) or has experienced such serious side effects with all other cheaper relevant funded treatments that treatment has been ceased or cannot reasonably be continued.

A number of submitters considered the requirement to have trialled and failed all other treatments appeared to apply to HPC in the flow chart, but was inconsistent with the intent of HPC.

A number of submitters supported the trial of a DHB discretion for treatments less than $500, and a number of DHBs expressed an interest in being involved in the trial. Some asked for further clarification of the information requirements. One submitter considered the limit for discretion could be $1,000. One submitter suggested that the information collected from DHBs could contain indication and specialty of prescribing clinician to assist with Schedule listing assessment.

PHARMAC welcomes this support and will be working with DHBs in the coming months to set this up. We will be in touch with all DHBs to work though some of the issues associated with information collection raised by some DHBs.

PHARMAC considers that $500 is an appropriate level at this time however we will review the level over time. We will discuss with DHBs what information should be provided in order to participate in the trial. We agree this level of information would be useful in Schedule assessment, however we are cognisant of ensuring the information can be provided without significant overhead to DHBs. We have addressed this with the inclusion of the following in Section 7 of the NPPA Policy:

A few submitters queried what would happen to existing patients if PHARMAC decided a particular medicine should not be used under HPC.

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SUBMISSION COMMENT

Some submitters sought clarification on what would happen to Discretionary Community Supply under the proposed Scheme.

PHARMAC RESPONSE

We do not intend to make any changes to DCS as a result of this review. The role of DCS will be reviewed following the development of the National Preferred Medicines List for DHB Hospitals.

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Metadata

Title

PHARMAC's responses to submitter's views

Abstract

Pharmaceutical Management Agency Review of Exceptional Circumstances Responses to Submitters’ Views June 2011 A433632 - qA17559 Introduction This Responses to Submitters’ views document provides PHARMAC’s responses to key points raised in submissions on proposed changes to the way PHARMAC assesses…

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