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  1. 1. Submission from the Australian and New Zealand Association of Physicians in Nuclear Medicine to the Health Technology Assessment Review PO Box 73 Balmain NSW 2041 Australia Phone: +61.2.9818.4824 Fax: +61.2.9818.4806 e-mail: secretariat@anzapnm.org.au web: www.anzapnm.org.au ABN 99 665 425 983
  2. 2. May 2009 2
  3. 3. Summary The ANZAPNM supports the current Review of Health Technology Assessment as it provides an opportunity to improve the current processes and deliver more effective outcomes. Health technology assessment should provide a means of evaluating new technologies and medicines in a reasonable and timely manner. However at the same time, it should not be used as a means of rationing access to medical procedures or medicines that can improve the delivery of health care and treatment outcomes. Currently the processes under the Medical Services Advisory Committee (MSAC) and the Therapeutic Goods Administration (TGA) as defined by their respective legislation place significant requirements on those seeking approval of new procedures and products. In some cases, as discussed in this submission, the requirements are overly burdensome and do not appear to deliver the intended outcome. For nuclear medicine, there are number of issues, as outlined below. The inflexibility of the current processes 3
  4. 4. Under the MSAC arrangements, the current process that evaluates against patient outcome disadvantages diagnostic technologies because, while diagnostic technologies provide information about the presence or absence of diseases which are used by clinicians to determine initial and ongoing management of the condition, clinical outcome(s) can only rarely be linked directly to the result of a diagnostic test. Furthermore, the levels of evidence applicable to therapeutic procedures do not necessarily have the same relevance to diagnostic procedures. The current MSAC arrangements do not adequately address situations such as the evaluation of the reports from the PET Data Collection Project. There appears to be no mechanism to assess these reports, even though they were based on MSAC-approved protocols, in a more timely manner. Further, the lack of “corporate memory” in the area has contributed to difficulties with respect to why the project was structured in particular ways. There is concern about the objectivity of the external evaluation process and the application of inappropriate evidence requirements to diagnostic technologies, as already noted. Given that a positive MSAC recommendation remains the first step in having a procedure included on the Medicare Benefits Schedule, there is no process that allows the adoption of a technology already in wide use in the public hospital sector (that is, with public 4
  5. 5. funding) to be funded under the Medicare Scheme with a Medicare item and available to patients in the private sector. In the case of the TGA, there are particular issues concerning the availability of radiopharmaceuticals and the loss of licensing when there is a change of ownership of the product. The current mechanisms offer some flexibility but it is inadequate to maintain ready availability of essential radiopharmaceuticals for patient diagnosis and treatment. The time and cost of the MSAC process Under the present arrangements preparation of an application requires significant work and cost. Once the MSAC evaluation is completed, there are multiple steps involving preparation of briefings, interdepartmental consultation concerning cost, regulatory drafting, external consultation through the MBCC process – all of which add significantly to the timing of introduction of a new item. The timeframe also has the potential to adversely affect the funding allocation for the introduction of a new procedure that has taken an extended time to be implemented, in that costs increase between the time of the application and the time the decision is made about funding. This extended evaluation and implementation process takes years, not months. From the perspective of both applicants and the government, there is also significant cost involved in the process. 5
  6. 6. Issues around outcome From the perspective of the medical profession seeking funding for new diagnostic and therapeutic procedures for which they consider there is robust evidence that the procedures will improve medical treatment, there remains potential for significant delays in the implementation of recommendations that have been approved for public funding. While it is not clear whether MSAC’s processes represent international best practice, there would appear to be value in developing cooperative approaches internationally to facilitate the use of international assessments, particularly in specialty areas where Australian patient numbers will be small, in order to facilitate the availability of new diagnostic and therapeutic procedures. Conclusion The ANZAPNM believes this review provides the opportunity to develop a health technology assessment process that provides more flexibility to deal with different procedures and products and which facilitates involvement in the process. Health technology assessment must not be used as a means of restricting access or slowing down the diffusion of diagnostic and therapeutic procedures that are able to improve the health of the Australian community. 6
  7. 7. Recommendations The ANZAPNM recommends that: There be a simplified framework and increased flexibility in the health technology assessment process, in order to facilitate assessment of both new applications and matters referred for further consideration and to reduce the need for full external evaluations. Consideration be given to developing evidence requirements more suited to diagnostic procedures. The public interest be considered in all regulation and related documentation particularly with respect to the availability of radiopharmaceuticals. Suitable regulatory solutions be identified to facilitate the use of safe, long established radiopharmaceuticals, particularly where the originally licensed manufacturer has relinquished ownership of the product. International cooperative approaches and standardisation and alignment of processes be developed in order to facilitate the use of international health technology assessments to augment or substitute for MSAC evaluations. 7
  8. 8. The PET Data Collection Project be the model for future data collection arrangements for the evaluation of new technologies. Ongoing financial support be provided to facilitate such data collection exercises. As part of this development consideration be given to funding the establishment of a diagnostic imaging economics and statistics advisory body to improve the quality of applications and reduce the need for external evaluation. In finalising Medicare item descriptors and fees for Medicare benefit following an MSAC assessment, consideration be given to replacing the current MBCC process with a mechanism by which the relevant medical specialty group has direct involvement and that the recommended fee for Medicare benefit be adjusted to take account of costs current at the time of introduction of the item. Any new provisions for post marketing surveillance take account of and not duplicate existing State and Federal reporting requirements relating to the medical use of radiation. In any realignment of health technology assessment processes the primacy of the assessment and the importance of ensuring appropriate new technologies are available for the treatment of Australian patients should outweigh application fee considerations and 8
  9. 9. accordingly the introduction of application fees for MSAC applications should not be considered. 9
  10. 10. Contents Page Background 8 2004 review of MSAC 9 The Australian and New Zealand Association of Physicians in Nuclear Medicine 10 The Process of Health Technology Assessment in Australia 12 Term of Reference 1 13 Term of Reference 2 20 Term of Reference 3 26 Term of Reference 4 27 Term of Reference 5 30 Attachment Health Technology Assessment Review – Terms of Reference 32 10
  11. 11. Background In December 2008, the Minister for Health and Ageing and the Minister for Finance announced there would be a review of health technology assessment (HTA) as a “Better Regulation Ministerial Partnership” with a stated commitment to “deregulation, to reduce costs to business and consumers and contribute to the Government’s productivity agenda”. The stated objective of the HTA Review is to “recommend options for improving process efficiency and reducing regulatory burden for Commonwealth HTA processes to facilitate medical innovation without compromising timely and affordable patient access to medical services and devices that: are demonstrated to be safe, effective and cost effective, and deliver improved health outcomes and value for money.” The Australian and New Zealand Association of Physicians in Nuclear Medicine (ANZAPNM) acknowledges the need for ensuring that new technologies are safe and contribute to improved health outcomes and, at the same time, are cost effective, as noted in the Discussion Paper. It also recognises that health technology is one of the drivers of both public and private health expenditure as new medicines, devices, procedures and tests continue to be developed. 11
  12. 12. It is further noted that the HTA Review is to present options that are: sustainable within existing funding levels, and consistent with Government policy objectives, including: policy on regulation of therapeutic products policy on access to and financing of medical procedures and therapeutic products the “interpretation of the term ‘public funding’ to mean direct and indirect funding of health technologies whether that funding fully or partially covers the cost” ensuring the regulatory processes are effective and efficient. Previous review of MSAC A review of MSAC was undertaken in 2004. ANZAPNM made a submission to that review, at a time when there was discussion of the role of MSAC in peer reviewed medical journals, and associated coverage in the media. Two particular points were raised by the ANZAPNM in response to that review: Technology assessment should be conducted on the basis of the best available evidence, whether it is Australian or from elsewhere. 12
  13. 13. The evaluation process for diagnostic technologies is necessarily different from that used for pharmaceuticals or treatment paradigms in general. It is extremely difficult and in many cases impossible to either use randomised-controlled trials or provide proof of survival advantage as a result of using new diagnostic modalities. It is the ANZAPNM’s view, supported in the international literature, that new diagnostic technologies should simply demonstrate statistically and clinically significant improvement in precision, sensitivity and specificity and cost-effectiveness. These views are reiterated. 13
  14. 14. The Australian and New Zealand Association of Physicians in Nuclear Medicine The Australian and New Zealand Association of Physicians in Nuclear Medicine (ANZAPNM) is the peak organisation representing nuclear medicine specialists and the practice of nuclear medicine in Australia. The membership of the ANZAPNM includes both physicians with advanced training in nuclear medicine, and dual-trained radiologists (i.e. radiologists who have undertaken advanced training in nuclear medicine). Nuclear medicine is the medical specialty that utilises a range of high technology imaging equipment, most commonly SPECT and Positron Emission Tomography (PET) together with medical isotopes to deliver both diagnostic and therapeutic procedures. Nuclear medicine specialists are highly trained in the medical application of radiation. Nuclear medicine is a mature medical specialty in that it is well-established and its use in a wide range of applications is accepted as both efficient and effective. Both pathology and diagnostic imaging, including nuclear medicine, can assist in more accurately identifying those patient subsets that are more likely to gain from a therapy, and 14
  15. 15. can enable monitoring of therapy response. Accordingly, both pathology and diagnostic imaging, including nuclear medicine, are fundamental to the provision of health care. The Impact of MSAC and the TGA on Nuclear Medicine As a high technology medical specialty, diagnostic and therapeutic nuclear medicine procedures may be subject to assessment under both the MSAC – for new procedures, or the application of existing technologies for new or amended indications, and the TGA – in relation to radiopharmaceutical production and supply. Radioisotopes require registration under the TGA. Increasingly, as radiopharmaceutical manufacturers rationalise their manufacturing operations and their “radiopharmaceutical portfolios”, well-established, effective radiopharmaceuticals are losing their registration – not because they have lost their efficacy, but for the lack of a sponsor in a country where the economics of serving a relatively small population does not justify a new company spending significant amounts of money to seek registration. The TGA guidelines provide for the registration to be transferred between sponsors without the product having to be re- evaluated at the full fee, with only the annual registration charge being applicable, but this transfer has to happen simultaneously. The problem arises when there is no sponsor willing to accept transference of registration at the same time, resulting in a cancellation of registration. 15
  16. 16. Therefore, nuclear medicine is one of a number of medical specialties that is significantly affected by regulatory and non-regulatory health technology assessment requirements in Australia. 16
  17. 17. The Process of Health Technology Assessment in Australia The following issues will be discussed by the ANZAPNM in this submission under the relevant term of reference. The majority of the ANZAPNM’s comments relate to the operation of MSAC, although a number of issues concerning TGA’s processes are also raised. the opportunity provided by the Review of Health Technology Assessment to improve the current processes and deliver more effective outcomes; the inflexibilities built into the MSAC assessment processes that need to be addressed, including the application of different levels of evidence, the use of a range of data to support applications including international data and evaluations, enhanced processes for “references” in place of full evaluations; the cumbersome nature and significant work and cost involved in the application and assessment requirements for all involved in both MSAC and TGA applications; the extended evaluation and implementation timelines; the need for transparency in all the processes; the need for flexibility in TGA’s processes and mechanisms to ensure that access to clinically valuable radiopharmaceuticals is facilitated, particularly where there has been a loss of licensing through a change in owners. 17
  18. 18. 18
  19. 19. Term of Reference 1 1 Simplification and better coordination between the Commonwealth HTA processes (as identified in the Review scope), which includes: consideration of a single entry point and tracking system for applications for market regulation and funding; making time to affordable access as short as possible for new technologies while maintaining or improving the rigour of evaluation processes; and examination of the feasibility of conducting concurrent assessments for market registration and funding and coverage purposes, noting current work in this area. The Discussion Paper comments that the “lack of high quality evidence for new medical procedures and associated devices is a shared problem that affects the medical industry, health professionals, consumers and governments”. This is true in relation to Level 1 evidence (RCTs), and governments need to recognise that for many reasons this standard is unlikely to be reached. In the absence of level 1 evidence, there is significant clinical investigation underway and published in the scientific literature, on most, if not all new medical procedures and associated devices. The ANZAPNM supports the continuing use of available data, both Australian and international, in health technology assessments. In Australia, applicants are requested to submit a significant set of data to support the safety, clinical effectiveness and cost effectiveness of the diagnostic or therapeutic service for which they seek public funding under Medicare. In reality, an externally contracted 19
  20. 20. evaluating agency (or agencies) undertakes what is essentially a parallel process. The Department of Health and Ageing has indicated that the data provided in applications are poor and cannot be used – this is not unique to Australia. Other than rejecting all such applications, the department’s contracted evaluating agency searches, documents and evaluates all available relevant information as part of conducting a review of the evidence for MSAC’s consideration. This accounts for much of the cost and time associated with assessments. 20
  21. 21. In 2007-08 MSAC received 14 requests for assessment: four from the medical industry one from a professional college two from individuals seven references from government including the states and the Department of Health and Ageing. From this data, government is the single largest user of MSAC and this is understandable given its responsibilities. All parties recognise the factors that inhibit the ability of applicants to prepare submissions comparable to the pharmaceutical and devices industries. At the same time, the parallel work undertaken by the external evaluators is a time consuming, inefficient and costly process. There is a reluctance to rely on health technology assessment undertaken in other countries, and in Australia a full assessment of current Australian and international evidence is undertaken, irrespective of the results of overseas HTAs. The ANZAPNM undertook an extensive review of health technology assessment processes in a number of countries comparable to Australia. These processes did not offer any one perfect model, and health technology assessment was undertaken for a range of reasons. Nevertheless, there would appear to be value in a formal review of these approaches being undertaken, particularly with respect to increasing Australia’s 21
  22. 22. ability to utilise the results of international health technology assessment in order to avoid duplication. The limitations, risks and opportunities associated with using overseas health technology assessments relate to whether the actual processes are comparable and whether the ultimate purpose of the assessment is the same or different. The ANZAPNM appreciates the need for assessments to consider diagnostic or therapeutic procedures in the Australian context. However, this element could supplement the findings of overseas health technology assessment agencies, particularly those undertaken in the recent past and which align with Australian imperatives, rather than duplicate them. As noted above, the acceptability or otherwise of overseas based health technology assessments could be based on a comprehensive evaluation of the processes of other agencies undertaking health technology assessment internationally, against a set of agreed requirements for Australia’s purposes. Where one or more international agencies whose approaches to assessment were found to align with those of Australia, formal agreements could be made to access their assessments. This could pave the way for applicants and MSAC to focus on the Australian aspects of the diagnostic and therapeutic service, such as epidemiological considerations, limitations of current interventions, and cost effectiveness, where appropriate. In the continuing challenge posed by data quality issues, MSAC could take on a stronger role in defining interim funding arrangements where a pre-requisite to funding is local data collection. 22
  23. 23. The benefits of using relevant aspects of overseas health technology assessments would be shorter assessment timeframes overall, earlier introduction of services found to be of value, and data collection for services where the evidence base needs to be strengthened. It would also result in cost savings from process efficiencies. The other consideration is that Australia’s population does not necessarily support the recruitment of large numbers of patients to studies where the condition may be relatively rare. In these cases, international data will be important. The ANZAPNM would be willing to work with the department on exploring collaborative approaches to data collection, particularly under interim funding arrangements to build the evidence base. One of the issues frequently raised is the time taken for an application to go through the MSAC process. As an example, the submission dates of the reports of the PET Data Collection Project and the date of implementation of the resultant Medicare items indicates the extended time taken in completing evaluations: Date Report Submitted Indication Date Medicare Item Available Elapsed time in months from 23
  24. 24. to MSAC submission to available item 30 May 2006 Colorectal Cancer 1 Dec 2008 30 months 14 June 2006 Ovarian Cancer 1 Dec 2008 29 month 10 July 2006 Melanoma 1 Dec 2008 28 months 13 Oct 2006 Oesophageal cancer Gastro-oesophageal junction cancer 1 July 2009 (anticipated) 33 months 9 May 2007 Head and Neck cancer 1 July 2009 (anticipated) 26 months 19 July 2007 Low Grade Non-Hodgkin’s Lymphoma Not completed > 22 months for MSAC evaluation phase only 13 Nov 2007 Glioma Not completed > 18 months for MSAC evaluation phase only 23 April 2008 Sarcoma Not completed > 13 months for MSAC evaluation phase only Given the clinical protocols for each of the indications were approved by MSAC prior to the commencement of the study, the duration of these evaluations could not be considered timely. As indicated above, it is difficult to demonstrate cost effectiveness for diagnostic technologies. What is needed is an evaluation mechanism that recognises that it is not possible to demonstrate an outcome based on the use of a diagnostic technology; it is possible to demonstrate changed management that affects outcome. The PET Data Collection Project is a case in point. Any amended MSAC or similar process needs to have a distinct evaluation process for diagnostic technologies or a 24
  25. 25. mechanism to recognise the differences between evaluating diagnostic and therapeutic procedures. The development of economic data is also frequently the stumbling block for MSAC assessments; and the availability of health economic advice presents a significant problem. Assessing diagnostic technology, Fryback and Thornbury 1 proposed a hierarchy of diagnostic efficacy as follows. 1. Technical Technical imaging quality 2. Diagnostic accuracy Sensitivity, specificity, positive predictive value, negative predictive value 3. Diagnostic thinking Likelihood ratio (Bayesian approach using pre-test and post- test probabilities) 4. Therapeutic Changes in therapeutic choices (patient management) 5. Patient outcome Improvement in morbidity/mortality 6. Societal Cost–benefit analysis This hierarchy continues to be used as a framework for assessing diagnostic technologies. 2 The link between diagnostic accuracy and patient outcome is not direct. Diagnostic test accuracy influences diagnosis which in turn influences treatment decisions; outcome depends on treatment effectiveness. Thus, there is only an indirect link between diagnostic accuracy and patient outcome, particularly medium to long-term outcome. The 1 Fryback, D.G. & Thornbury, J.R. The efficacy of diagnostic imaging. Medical Decision Making, 1991, 11:88-94. 2 Facey, K., Bradbury, I., Laking, G. & Payne, E. Overview of the clinical effectiveness of positron emission tomography imaging in selected cancers. Health Technology Assessment, 2007; Vol. 11: No. 44. 25
  26. 26. Canadian Institute of Clinical Evaluative Sciences, in an assessment of PET, shared this view, concluding that it was difficult to conclusively assess the impact of PET on morbidity and mortality but intermediate outcomes such as the avoidance of inappropriate surgery could be assessed. 3 A further issue raised with respect to the consideration of the PET data has been the decision that for every indication for which PET may be used a separate MSAC application must be developed and assessed. It is difficult to find a comparable decision in relation to other diagnostic technologies. From the demographic data collected during the PET project, additional data was provided to the clinician that resulted in a change in clinical management in most cases. From this, it is reasonable to assume that PET’s impact on patient management is significant in the majority of cases. Therefore, the decision that PET will not be accepted as useful across all cancer indications is disappointing. The ANZAPNM believes there is sufficient evidence for this aspect of the MSAC process to be re-assessed, particularly where there is data generated in consultation with the department and MSAC that supports wider application of this technology. Ideally the health technology assessment process aims to deliver information that is accurate in terms of the ultimate cost. However the problem with the current process is 3 Institute for Clinical Evaluative Sciences. Health Technology Assessment of Positron Emission Tomography (PET) – A Systematic Review, 2001. 26
  27. 27. that the costings included in the application may be directed more towards eliciting a successful outcome in a comparison with any existing funded technology, than the longer- term objective of ensuring that the final funding model reflects the true costs of the procedure. As a result, because this same set of costings is used in the ministerial briefing (assuming a positive recommendation) the actual projected outlays are under-estimated and the resulting fee for Medicare benefit has not reflected all relevant costs. The nature of the evaluation process encourages the minimisation of costs and is consistent with the government’s position that it may choose to fully or partially fund or subsidise a medical service. However, the consequence of underfunding new and existing services, particularly those that may be considered expensive, is that the difference has to be met through out of pocket costs by patients. One interpretation of the implications of government policy on minimising costs may be that applicants have to submit cost data that underestimates actual costs in order to enhance the chances of the submission being successful. A further issue that influences the overall time taken from an assessment - from submission, through to final recommendation by MSAC to a new item on the MBS - is the number of steps that need to be undertaken. These include: preparation of a briefing by DoHA for the Minister, 27
  28. 28. consideration of the financial impact on Medicare outlays by the Department of Finance and Regulation the Minister’s consideration the DoHA’s preparation of items including liaison with legislative drafting and with clinicians about technical issues the involvement of the AMA through the convening of an MBCC meeting subsequent work by DoHA and Executive Committee approval. All of these steps contribute to the time taken to make a diagnostic or therapeutic service available to patients via the MBS. The value of the involvement of the MBCC has been questioned on a number of occasions, particularly with respect to health technology assessment in areas where the AMA has no particular expertise other than as a convenor of a meeting and has, on occasions, not ensured the relevant specialty groups have been involved in the meetings. While the AMA through the MBCC has had an important role in negotiations about Medicare fees in the past, the ANZAPM believes that this might not be the most effective arrangement into the future. In summary, the ANZAPNM makes the following points: 28
  29. 29. it is possible to conduct national studies to collect and collate clinical data in order to gather data for the evaluation of health technologies; there is value in having a single coordinating body / organisation; there is value in the government providing support for these exercises; there would be value in there being additional support, such as economic and statistical advice; and consideration should be given as to whether the MBCC process is the most effective mechanism for review and input at the final stages of the process. There may be value in establishing a broader evaluation framework in order to facilitate the use of international health technology assessment reports and data to speed up the evaluation process. There may also be value in further review of the practicalities of using multi-centre studies to gather evidence and the introduction of mechanisms to facilitate this process, such as enhancing the capacity for ethics approvals across a number of sites and providing support for protocol development. While there are strengths in having individual site assessment and protocol development, there is also a need for a mechanism to facilitate approvals when the site is involved in a national study. Term of Reference 2 Improving role clarity and addressing duplication between processes, where it exists, including consideration of consolidating functions with the Australian HTA system. 29
  30. 30. The ANZAPNM has no particular experience of difficulties associated with the duplication between the different health technology assessment processes for drugs, medical services and devices. However, recent experiences in relation to the availability of certain radiopharmaceuticals has highlighted the need for a more responsive capability to allow for the use of radiopharmaceuticals different from those specifically identified in Medicare item descriptors. While this issue does not necessarily fall under the focus of this review it does however highlight the need for greater adaptability across a range of processes not just those associated with health technology assessment. Standardisation and alignment of processes, consolidation of application information, greater cooperation between agencies and committees are appropriate objectives for health technology assessment. However, at the same time, consideration needs to be given to the size and coverage of a single agency and potential inefficiencies if all the above processes were carried out within the one agency. Therapeutic Goods Administration (TGA) The ANZAPNM has had no involvement in submitting applications involving approval or recognition across two or more assessment schemes. However, the ANZAPNM has a number of problems with the current process of registration of radiopharmaceuticals by the TGA. 30
  31. 31. These problems relate to the situation where a manufacturer who holds registration for a particular product sells the licence for that product to another manufacturer who is disinclined to retain the registration. The product then become unregistered – for no other reason than the want of a sponsor – and then may only be available through arrangements such as the Special Access Scheme (SAS) or by individuals obtaining endorsement as Authorised Prescribers under Section 19(5) of the Therapeutic Goods Act. Such endorsements relate to the use of the product for which application is made. The most prominent and ongoing recent example of this problem is cholecystokinin (CCK) for nuclear medicine hepatobiliary scans. CCK is used in patients with suspected chronic acalculous cholecystitis, to make the gallbladder contract. to clear sludge (caused by prolonged fasting) from the gallbladder, prior to imaging. (iii) to diagnose sphincter of Oddi dysfunction. The clinical justification for the use of CCK is that, although a number of agents can make the gallbladder contract, CCK is the only agent to mimic the evacuation of bile that occurs in normal physiology, i.e. a prompt contraction that is limited in duration. Simulation of the normal physiological response of the gallbladder is highly desirable in patients with complex gallbladder pathology. The gallbladder ejection obtained from infusion of CCK is used by surgeons to select patients who are likely to benefit from cholecystectomy or sphincterotomy. 31
  32. 32. There is no alternative supplier of the drug locally or internationally. There are no alternative drugs that do not require an authorised prescriber licence. While there are provisions for products registered on ARTG to be transferred to another sponsor without cancelling the registration and thereby having to go through the process of submitting a new full application with full application and/or evaluation fees payable to ensure continuing supply of products, this requires action by both the relinquishing company and by the accepting company. This process simply requires completion of documentation to transfer the therapeutic product to another sponsor. However, the reality is that, as noted above, increasingly the new company is disinclined to take over the product – probably due to the relatively low number of doses that would be sold in Australia, compared with overseas countries where the sale of the product has occurred. The question arises as to whether, in the case of products such as radiopharmaceuticals that have proven efficacy and very few adverse reactions, and where the only reason they become unregistered is because of the sale of a suite of products from one manufacturer to another, there should be a different process applicable from the one currently existing. The impact of the current registration problem for products such as CCK is that: 32
  33. 33. there is significant additional workload for nuclear medicine specialists to individually seek authorised prescriber status, or to obtain approval for single use of a product under the Special Access Scheme; there is added workload for the TGA that should be able to be eliminated; and patients who will benefit from the use of products, such as CCK, do not undergo those procedures because of the difficulties associated with using the product – an outcome that disadvantages the patients. These are proven therapeutic and diagnostic products that should be available under normal circumstances. The SAS refers to arrangements that provide for the import and/or supply of an unapproved therapeutic good for a single patient, on a case-by-case basis. Patients are defined as either Category A patients or Category B patients. For Category A patients, medical practitioners can supply unapproved goods to very seriously ill patients without the approval of the TGA as long as the medical practitioner notifies the TGA within 28 days. These patients are defined in the legislation as "persons who are seriously ill with a condition from which death is reasonably likely to occur within a matter of months, or from which premature death is reasonably likely to occur in the absence of early treatment". For Category B patients, approval of an application by the TGA to supply an unapproved product is required on a patient-by-patient basis to reflect the needs of different patients. Applications should be made in writing. 33
  34. 34. The document Access to Unapproved Therapeutic Goods, TGA 2004 explains the rationale for TGA’s approach to monitoring access to unregistered products, as: In keeping with its charter, the TGA also has a responsibility to encourage at all times the availability of approved (evaluated) products. Thus the various mechanisms for supply of unapproved products are intended to be temporary measures pending general market approval of the product. TGA requires that applications to use unapproved products justify adequately why available approved unapproved (fully evaluated products) are not suitable for use. Unfettered access to unapproved products amounts to de-facto marketing and would remove any incentive for a sponsor to seek registration of the unapproved product or for other sponsors to seek of alternative similar products. The supplier has to provide the TGA with six monthly reports on access and supply of the unregistered product. Clearly, where long-term use is anticipated, it is expected that the product becomes registered. However, this again raises the problem of manufacturers supplying products for a small population and balancing the relatively low number of sales against the significant cost of developing and maintaining registration. It may be reasonably asked why a new owner would not simply take the step of transferring registration, as currently allowed – ANZAPNM is unable to speak on behalf of the manufacturers in this regard, but assumes it relates to ongoing cost. 34
  35. 35. Radiopharmaceuticals are classed as chemical entities and are considered under Category 1 which is provided for under the Therapeutic Goods Regulations sub-regulation 16(C)(3)(b) and 16 (D)(3)(b). The evaluation fee for Category 1, New chemical entity (including radiopharmaceuticals) where they have not been included previously on the ARTG, is $176, 000. However, if a sponsor can show that the new isomer, mixture of isomers, complex or derivative of or salt of a registered substance does not change the pharmacokinetics, pharmacodynamics and/or toxicity of the moiety in a way which could change the safety/efficacy profile, the new product may be considered to be essentially similar and the fee level would be for a generic product rather than a new chemical entity. If not, then the application would be considered as a new chemical entity. There are other provisions for “extension of indication” ($104,800) and new generic product (essentially the same as a registered medicine chemically, same form, bioequivalent and same safety and efficacy properties) ($67,300). There are annual charges of $5,250 for biologics and $3,140 for non-biologics. Change in ownership of the sponsor/supplier does not in itself incur a charge – it is the lapsing or cancellation of registration that results in the need to re-register the product that incurs the evaluation fee. Transfer provisions allow for continuity of registration with payment of the annual fee as advised by the TGA. 4 4 It is noted that amendments to the Therapeutic Goods Amendment (2009 Measures No. 1) Bill 2009 will be considered by Parliament in the 2009 winter sittings. These include clarifying the arrangements for setting conditions on medicines so these are more transparent, and also to enable sponsors who have asked for the registration or listing of a medicine to be cancelled to apply for this to be revoked (within 90 days) to keep the 35
  36. 36. On that basis, it is difficult to see why a manufacturer/supplier would not – in the case of a radiopharmaceutical – simply organise to transfer the registration but, as noted above, the ANZAPNM is not in a position to comment on why this does not occur. The answer may be for a provision to be introduced that deals specifically with this issue in the case of radiopharmaceuticals – i.e. where there is no sponsor available at the time the registration lapses and a new sponsor comes forward within a year or so, re-registration of the product is allowed under the same provisions, without incurring the evaluation fee. This would reduce the significant financial disincentive for another company to seek re- registration of a product that is used for a relatively small population. Similarly, the possibility of recognition of certain radiopharmaceuticals as “orphan drugs” has been raised. However, given an orphan drug is defined in the legislation as a medicine, vaccine or in vivo diagnostic agent which is intended to treat, prevent or diagnose a rare disease or must not be commercially viable to supply to treat, prevent or diagnose another disease or condition, this provision does not assist with the radiopharmaceutical problem. It is noted that the TGA also has provisions for considering products for use of medicines in special population groups, e.g. geriatrics, paediatrics, hepatic or renal impairment which medicine on the register. However, these amendments if passed, will do little to address the current problems. 36
  37. 37. may involve less than 2,000 users a year. However, for products such as CCK, the condition they are treating is not rare. The use of the special TGA arrangements under which a number of radiopharmaceuticals are supplied generates significant work which, in the case of radiopharmaceuticals, is mostly related to products that have previously been registered by the TGA. The ANZAPNM wishes to acknowledge efforts by the TGA to facilitate the requirements for authorisation and special access. It is recognised that, in the long term, utilising such arrangements for very safe and effective radiopharmaceuticals is not desirable. However, given there has been no inclination to transfer registration it is highly unlikely there will be moves to re-register these radiopharmaceuticals. This means that to maintain access to currently unregistered radiopharmaceuticals other mechanisms need to be developed to ensure that patients continue to have access to the diagnostic and therapeutic procedures for which they are used. Therefore, the ANZPANM firmly believes that, in the same way that government makes very high cost medicines available to prolong life for some cancer sufferers, equally there are public interest arguments to identify a suitable regulatory solution that enables low use, safe radiopharmaceuticals to retain registration independent of the originally licensed manufacturer when that manufacturer has relinquished ownership of the product. 37
  38. 38. Term of Reference 3 Enhancing post marketing surveillance mechanisms to ensure the ongoing safety and efficacy of medical devices. The ANZAPNM notes that data on adverse events (that equates to post marketing surveillance) is collected in relation to both radiopharmaceutical use and on nuclear medicine procedures. In both cases, the number of adverse events is negligible. However, it is relevant to note that nuclear medicine can be identified as having a high number of incidents reported to state radiation advisory councils, for example. The reason is that nuclear medicine has a strong culture of reporting any accidents and has generally adopted a systemic risk approach like that of the aviation industry rather than a “name and blame” approach common in the rest of the medical profession. This approach has encouraged good practice and minimisation of serious accidents. At the same time, other diagnostic modalities using radiation have not yet developed this same approach and do not automatically categorise incidents such as repeating a scan or Xray to expand the area scanned, or to ensure the image is satisfactory, as a reportable incident – this is merely classified as “taking another view”. In this regard, there would be value in developing some uniformity of reporting, although we question whether this would be a role for an enhanced post-marketing surveillance capacity. 38
  39. 39. In initiating any additional post-marketing surveillance it is important to recognise that, in the case of nuclear medicine (and presumably many other specialties), there are already multiple regulatory requirements relating to the submission of data (at both federal and state levels) and these must be coordinated, rather than duplicated by any post marketing surveillance exercise. 39
  40. 40. Term of Reference 4 Strengthening transparency and procedural fairness in the assessment, decision making and fee negotiation arrangements for processes (as outlined in the Review scope) through improved communication with stakeholders about processes, methodologies, outcomes and performance against key indicators. The Review has asked what aspects of Australia’s health technology assessment system work well in relation to transparency and procedural fairness, what improvements could be made, and what performance indicators could be instituted. Concerns about procedural fairness and transparency have been examined in detail elsewhere in relation to the assessment of two applications concerning PET. With respect to issues raised in those examinations, concerns are expressed to the ANZAPNM in relation to: the objectivity of the technical evaluation process; the need for an evaluation methodology that is specifically designed to assess diagnostic technologies and which is different from that applicable to therapeutic technologies; the potential for politicisation of elements of the process; the inter-relationship of the roles of the main MSAC and the supporting committees. 40
  41. 41. The ANZAPNM appreciates that changes have been made to some MSAC processes in response to the issues raised in relation to the early PET assessments. The ANZAPNM understands the government's rationale for considering any further scope for aligning processes for pharmaceuticals, devices, diagnostic technology and therapeutic procedures/interventions and there is sense in considering this. It is also noted that one area that would be under consideration is the introduction of fees for lodging and assessing applications for funding under Medicare in a similar fashion to the way that fees currently apply to assessments by the TGA and PBAC. On the surface this would seem a logical step. However PBAC and TGA applicants are manufacturers, suppliers or distributors of products. In the majority of cases their request for MBS or PBS listing is the culmination of multi-year and multi-million dollar research and development investment projects. MSAC applicants tend to be less homogeneous than the TGA and PBAC applicants, comprising individuals, professional organisations, governments and some from medical industry who have been through the TGA and/or PBAC process. For the medical industry group, MSAC is the end of the line where the product’s therapeutic value has to be established before medical and other health professionals can 41
  42. 42. perform a service (using that product) that attracts a patient benefit. In many cases the final amount of the determined schedule fee for the procedure using the new product includes a professional component (taking into account the training of the professional), a technical component (allowing for the complexity of the service), and a capital component (the costs of the equipment to provide the service and its running costs etc). Consequently the MSAC process focuses on the value of the service (involving the use of an approved product) to health care - be it diagnostic and/or therapeutic. Particular procedures provided by medical practitioners not involving new products require assessment by MSAC before the procedure can attract a patient benefit. The ANZAPNM supports the need for assessment of new medical technologies to establish their diagnostic and therapeutic value and assess the acceptability of their risk to the community. In 2007, of 14 applications received by MSAC seven were from government agencies including the Department of Health and Ageing, applications from governments likely to continue to increase as they seek to contain costs through various demand and supply measures. Cost recovery of all costs relating to processing and assessing applications is one such measure. However, if the average cost of an MSAC assessment is $250,000 full cost 42
  43. 43. recovery to this amount for professional services would be prohibitive for individual professionals and for professional organizations. The effect of this would be a stifling of the introduction of new and potentially safer and more cost effective diagnostic and therapeutic services. With the opening up of the MBS to health professionals other than the medical profession, and the pressure to expand roles on the basis of equivalent safety and effectiveness but greater cost-effectiveness, the burden of cost on those health professionals or professional associations to put forward their case will be even more onerous than it is now. This is an area that needs to be explored carefully, including the possibility of introducing a financial contribution, to ensure that the original objective of establishing MSAC as a public interest facility is not lost in the face of budgetary pressures. 43
  44. 44. Term of Reference 5 Enhanced arrangements for assessment of co-dependent 5 and hybrid technologies 6 . The process whereby the ANZAPNM was able to coordinate the national PET Data Collection Project provides a valuable model for future assessment. While this project was very large, involving multiple sites and multiple indications – with a cost that reflected the extensive nature of the project – it nonetheless represents a useful approach in developing a dataset on particular technologies. In this case, there was a need to assess the role of PET in relation to a number of cancer indications; Commonwealth funding provided the opportunity to conduct several protocols in parallel. The terms of reference for this Review seek examples to illustrate points. One such example relates to the usefulness of having an extensive data set. The PET Data Collection Project had an additional benefit in that from the extensive data collected and analysed it was possible for ANZAPNM to provide additional information to further inform the MSAC process in relation to the lymphoma evaluation. Concerns were expressed by MSAC when it was reviewing the report from the Project about the selection of low grade rather than high-grade lymphoma for review. The ANZAPNM was also in a position to 5 Where therapy involving the use of one health technology to directly improve health (eg a medicine or a medical device or a procedure) is improved by the use of another health technology (eg a pathology or imaging diagnostic technology), which might more accurately identify patient subsets most likely to gain from the therapy or monitors therapy response. 6 Where the characteristics of different health technologies (eg a medicine or a medical device or a biologic) are combined in one intervention (eg laser activated medicines such as photodynamic therapy, or drug eluting stents. 44
  45. 45. provide additional data on the stage change information for low grade, high grade and Hodgkins disease patients, both for initial staging and for restaging, from the PET Data Collection Project database. It is understood these additional data enabled the MSAC evaluation to progress. The ability to respond to such enquiries from MSAC significantly enhances the evaluation of new technologies and reinforces the value of government support for similar clinical research activities. While the PET Data Collection Project process presented a number of difficulties over the life of the project, not the least of which was the time taken to finalise protocols, it still provided valuable lessons in how to undertake such a multi-centre research program to inform government policy. 45
  46. 46. Attachment Terms of Reference The terms of reference for the Review, issued in April 2009, seek a report on: 1 Simplification and better coordination between the Commonwealth HTA processes (as identified in the Review scope), which includes: consideration of a single entry point and tracking system for applications for market regulation and funding; making time to affordable access as short as possible for new technologies while maintaining or improving the rigour of evaluation processes; and examination of the feasibility of conducting concurrent assessments for market registration and funding and coverage purposes, noting current work in this area. 2 Improving role clarity and addressing duplication between processes, where it exists, including consideration of consolidating functions within the Australian HTA system. 3 Reviewing post marketing surveillance mechanisms to ensure the ongoing safety and efficacy of medical devices. 4 Strengthening transparency and procedural fairness in the assessment, decision making and fee negotiation arrangements for processes (as outlined in the Review scope) through improved communication with stakeholders about processes, methodologies, outcomes and performance against key indicators. 46
  47. 47. 5 Enhanced arrangements for assessment of co-dependent 7 and hybrid technologies 8 . Scope of the Review The Scope of the Review includes: regulation of therapeutic goods approval of funding under the Medicare Benefits Scheme currently informed by the MSAC and relevant implementation consultative committees listing of prostheses for private health insurance coverage, as currently informed by the PDC; and listing of hybrid and co-dependent technologies as currently informed by the MSAC, Pharmaceutical Benefits Advisory Committees (PBAC) and PDC. MSAC’s current role is defined by its Terms of Reference, namely: Advise the Minister for Health and Ageing on the strength of evidence pertaining to new and emerging medical technologies and procedures in relation to their safety, effectiveness and cost-effectiveness and under what circumstances public funding should be supported. Advise the Minister for Health and Ageing on which new medical technologies and procedures should be funded on an interim basis to allow data to be assembled to determine either safety, effectiveness and cost-effectiveness. 7 Where therapy involving the use of one health technology to directly improve health (eg a medicine or a medical device or a procedure) is improved by the use of another health technology (eg a pathology or imaging diagnostic technology) which might more accurately identify patient subsets most likely to gain from the therapy or monitors therapy response. 8 Where the characteristics of different health technologies (eg a medicine or a medical device or a biologic) are combined in one intervention (eg laser activated medicines such as photodynamic therapy, or drug eluting stents. 47
  48. 48. Advise the Minister for Health and Ageing on references related either to new and/or existing medical technologies and procedures. Undertake health technology assessment work referred by the Australian Health Ministers’ Advisory Council (AHMAC) and report its finding to the AHMAC. Under these Terms of Reference, MSAC has the responsibility of accepting applications in relation to new medical technologies and procedures, or where there are significant changes in the use of already publicly-funded medical technologies. However it may also accept “references” from both the Minister and from the Australian Health Ministers’ Advisory Council. 48

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