Is that drug listed on the Pharmaceutical Benefits Scheme? NSW Cancer Conference 2023 Poster #186

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https://tacshealthcare.com.au/wp-content/uploads/2023/11/NSW-CC-Sep-2023-Poster-186.pdf

Objective

As one of the three pillars of Australia’s universal public health system, the Pharmaceutical Benefits Scheme (PBS) provides equitable access to medicines for all Australians. Cancer is a leading cause of death of Australians (18% in 2020).1 Hence, timely availability of new treatments on the PBS has become more critical than ever.

This retrospective review of the listing and use of cancer pharmacotherapies on the PBS considers whether the current scheme is meeting the needs of Australian medical oncologists, haematologists and their patients.

Methods

PBS and Repatriation PBS (RPBS) claims data for all funded programs, including chemotherapy, were sourced from the Services Australia 2 and PBS3 websites. Services (prescription dispensed for a PBS item) for and benefit statistics were graphed by Anatomical Therapeutic Chemical (ATC) first level code over time. Proportions of Government expenditure and average cost per service were calculated. 

Outcomes of Pharmaceutical Benefits Advisory Committee (PBAC) Meetings4 were reviewed to identify considerations for the first indication of new products for non-small cell lung cancer (NSCLC) and Multiple Myeloma (MM), as representative of pharmacological treatments for solid tumour and haematology indications. The duration between marketing approval by the Therapeutic Goods Administration (TGA) and time points to PBS listing were determined.5 

Figure 1. PBS/RPBS services provided by ATC Code 1992/93 to 2022/23
Figure 2. Australian Govt. PBS/RPBS expenditure by ATC Code 1992/93 to 2022/23

Results

PBS/RPBS claims data was available by ATC for the 31-year period 1992/93 to 2022/23. Changes in the number of services provided and benefit paid by the Federal Government (excluding patient co-payments) for ATC groups contributing over 5% are shown in Figures 1 and 2. Table 1 provides cost breakdowns by ATC, including patient out of pocket contributions.

All ATC groups grew over time mirroring societal demographic changes. The higher growth of Cardiovascular, Nervous System and Alimentary groups reflect treatment innovations with statins, anti-depressants and proton-pump inhibitors, respectively.

Table 1. PBS/RPBS costs by ATC codes in 2021/22

The introduction of Section 100 High-Cost Drugs to reimburse States for in-hospital usage of certain therapies, and agreements between the Commonwealth and all States/Territories, except NSW/ACT, to permit PBS out-patient dispensing have also added service volume to the scheme. Increased costs have been offset by pricing policy changes, mostly directed at manufacturers, such as splitting into Formularies by molecule patent status, Price Disclosure and Efficient Funding of Chemotherapies (EFC).

While the proportion of total services for cancer treatments grew over time, the ATC group represented only 2.3% of PBS/RPBS activity in 2021/22. However, in the same year, the group represented 41.5% of total Government expenditure on the PBS/RPBS program and continuing to grow.

Table 2. New medicines for NSCLC and first PBS listing (1999-2023)

Table 2 lists new medicines requesting PBS listing for a NSCLC indication, and Table 3 for Multiple Myeloma from PBAC Meeting outcome documents available from December 1999. Public Summary Documents were introduced  from July 2005. The number of PBAC considerations and date of TGA approval and PBS listing are noted.

Table 3. New medicines for MM and first PBS listing (1999-2023)

Discussion

Innovative medicines are now considered by the PBAC at similar times to TGA approval due to policies such as Parallel Processing and TGA Provisional pathways. Streamlining of PBS processes in the past 5 years see clinically effective drugs navigating the process usually within two PBAC considerations. Beyond this, intractable issues can introduce a significant lag between local treatment practices and internationally accepted standards of care.

The finding that there was an average 13-month difference in time to access new treatments for MM and NSCLC on the PBS over the period supports ongoing policy reform to accelerate access.

Table 4. Time to PBS listing for NSCLC and MM drugs (1999-2023). †Excludes afatinib as an outlier at 58 months due to global pricing policy

Conclusions

•Although variable time to access, the PBS/RPBS is serving patients with cancer, and their clinicians by subsidising innovative treatments.

•Treatments for cancer account for an increasing proportion of total Government expenditure on the scheme. Currently, over 40% and growing.

•Although the proportion of patient out-of-pocket costs are comparatively low, the actual amount can be significant and inequitable relative to other therapeutic areas.

Streamlining PBS processes – initial submission categories

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While the “Streamlining PBS Processes” work has completed, and the Strategic Agreement 2017-2022 of which it was a part, has passed into history, what is the ongoing impact?

The new classification of submission types became effective from the PBAC July 2021 Meeting (March 2021 cut-off). This meant the loss of the ‘major’ and ‘minor’ labels traditionally used to distinguish between those submissions requiring full evaluation and those managed at Committee Secretarial level. Definitions applying to both new submissions and re-submissions have been changed.

For new submissions, five categories apply based upon the complexity of the submission and evaluation required. The new processes also apply to vaccine listings on the National Immunisation Program (NIP).

Categories 1 and 2, formerly major submissions, require the PBAC to assess the magnitude of clinical improvement or toxicity reduction, the incremental cost and the comparative costs and outcomes where an economic evaluation is required to support a claim of cost-effectiveness, cost-utility or cost‑minimisation.

Categories 3, 4 and Committee Secretariat, formerly minor submissions do not require a full evaluation (clinical, economic and financial evaluation).

The chart shown includes submissions by category for formal and out-of-session PBAC meetings held in the 12 months from July 2021. This encompasses the three formal meetings of July 2021, November 2021 and March 2022; and three out-of-session, September 2021, December 2021 and May 2022. Unfortunately, despite 14 considerations at the May 2022 meeting, these were not classified during the new terms. For the purpose of analyses, these are assumed to be resubmissions.

Source: PBS website – PBAC Outcomes

Of the 113 initial submissions categorised over the 12-month period, 74 (66%) were allocated as Category 1 or Category 2. Of these, approximately one third (23) qualified as Category 1 with an associated complexity or innovation. Given that overall, Category 1 submissions accounted for 21% of all those lodged, it does not reflect a decrease in novel pharmacological treatments being brought to Australia.

Cost-recovery

Interestingly, it is The National Health (Pharmaceuticals and Vaccines – Cost Recovery) Regulations 2022 that “prescribes the requirement for categorising submissions”. However, in relation to cost recovery matters, nothing takes precedence over the Cost Recovery Regulations.

Using the recently announced revised Cost Recovery Implementation Statement (CRIS), effective 1 August 2022, fees of approximately AU$ 15 million would have been received by the Commonwealth Department of Health and Aged Care over the 12 month period for new submissions. Fees for subsequent submissions and other services are not included in this total.

Category definitions

Category 1

  • A first in class medicine and/or a medicine for a new population OR
  • A drug with a codependent technology that requires an integrated codependent submission to the PBAC and MSAC OR
  • A drug with a TGA Provisional determination related to the proposed population.

Category 2

Category 2 is the default for submissions requesting a PBS listing that does not meet the criteria for Category 1.

  • A new medicine, a new indication of a currently listed medicine, or to make one or more changes to an existing listing that requires a full evaluation (clinical, economic and financial evaluation).
  • A request for the PBAC to reconsider an existing recommendation where there is a change to the clinical, economic and/or financial information most recently relied on by the PBAC.
  • A new form or strength of an already-listed medicine that is not bioequivalent to an existing listed form of the medicine. A Category 2 submission may be necessary to demonstrate that the new form delivers similar clinical outcomes to the existing form.

Category 3

Category 3 submissions relate to requests to change existing listings that do not change the population or cost-effectiveness of the medicine AND that do not meet the criteria for a Category 4 submission.

Although the PBAC will assess the clinical need for and clinical effectiveness of the requested listing, an economic evaluation is not necessary to support the claims made in the submission. Additionally, the financial estimates do not require the PBAC to assess any substantial financial implications for the supply of a listed medicine or designated vaccine.

They may also relate to a request for the PBAC to reconsider an existing recommendation where there is no change to the clinical, economic or financial information most recently relied on by the PBAC.

As PBAC advice is required on a case-by-case basis regarding the potential for schedule equivalence for biosimilar listings, Category 3 submissions are also appropriate for a new biosimilar brand of an existing pharmaceutical item with no indication changes.

Category 4

Category 4 submissions involve a request for one or more of the following:

  • Listing of a new pharmaceutical item of a listed medicine.
  • Consideration as an exempt item (Exempt item as per subsection 84AH of the National Health Act 1953).
  • Varying or including a listed medicine on the prescriber bag.
  • A change to the existing, or the addition of a new form or manner of administration of a listed medicine.
  • A change to the maximum quantity and/or number of repeats of a listed medicine.
  • A change or addition to the prescriber type(s) of a listed medicine.

Committee Secretariat

Committee secretariat submissions relate to applications where the requested listing changes do not require the PBAC to consider comparative effectiveness, cost-effectiveness or clinical need:

  • there is no difference in patient safety or population for the new pharmaceutical item in the submission compared to an already-listed pharmaceutical item; AND
  • there is no financial effect associated with the proposed change to the PBS.

Note: Applications that do not require PBAC consideration for listing an additional brand (a generic medicine) or new oral form of an existing TGA-approved and PBS-listed pharmaceutical item should be lodged directly to the Department of Health &Aged Care.

References

Procedure guidance for listing medicines on the Pharmaceutical Benefits Scheme version 2.4

2022-2023 Cost Recovery Implementation Statement (CRIS)

The transparency conundrum

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True transparency requires more than making information available.

According to Rio Tinto, it was a ‘misunderstanding’ with the local Indigenous community that resulted in the destruction of 46,000 year-old sacred sites in May this year. The company had Government approval and stakeholders had been informed. All boxes checked. Obviously, that was not enough.

Since 2015, the Australian Government has been a member of the Open Government Partnership (OGP), committing to support the goals of increasing the transparency and accountability of government. The first OGP Global Report (May 2019, page 20), identifies these as generally lacking in health procurement decision-making in the 78 nations involved. In this article, I assume procurement includes the registration and reimbursement of therapeutic interventions by the Government on behalf of Australian taxpayers. My thesis being that openness may not necessarily be transparent, using two examples to illustrate.

Example 1. The presentation of options by the Therapeutic Goods Administration (TGA) for which type of medicines would have evaluation status made public.

Option 1: maintain TGA’s current publication arrangements [to not make public]

Option 2: list all applications accepted for evaluation

Option 3: list all applications at two different time points

Option 4: list applications of innovator medicines of highest public interest, but not generic or biosimilar medicines

The explanations given for providing Options 3 and 4 include: ‘Generally, there is less public interest in whether a generic or biosimilar medicine is under evaluation by TGA in Australia’, and ‘Earlier publication of generic or biosimilar approvals prior to ARTG entry allows more transparency of forthcoming competition to sponsors of originator medicines and potentially, purchasers of biosimilar and generic products.’

The TGA has been in the unenviable situation of neither being able to confirm nor deny whether a particular medicine is currently in the evaluation process. Other similar jurisdictions, such as Europe and Canada publish this information. It is a good initiative. The particular drivers of this 2019 consultation on transparency were: from the TGA side, inconsistencies with other agencies, especially evident during joint evaluations of new molecules; from the innovator sponsor side, more time to initiate legal proceedings against potential patent infringing generics/biosimilars and avoidance of liability for damages by the Commonwealth; and from generic/biosimilar applicants, maintain the status quo.

The inclusion of Options 3 and 4 show that there is an awareness of the potential negative commercial/financial implications to applicants and the Government of originator sponsors knowing the timing of registration of a generic or biosimilar. Is this made clear? Does ‘less public interest’ justify being selectively transparent?

Example 2. Measures associated with the ongoing Medicines Australia-Commonwealth Strategic Agreement PBS process improvements include the PBAC preference for greater transparency to be introduced in Public Summary Documents (PSDs) through a standardised approach to redactions.

Objectives of proposed PSD Changes – PBAC (Medicines Australia, Feb 2020)
•Increase transparency of PBAC’s decision-making processes.
•Publish PSDs in an efficient and consistent manner through establishment of a standardised approach to redacting information.
•Provide consumers with access to information to assist with making decisions about their individual health needs.
•Increase the public’s understanding about PBAC decisions.
•Align Australia’s practices with leading international jurisdiction approaches*.

*This refers specifically to the additional, and unexpected, push for PSD inclusion of all clinical data provided in PBAC submissions, irrespective of the publication status nor commercial impact. While no other jurisdiction currently requires it, other countries are considering in response to calls for increased pricing transparency (WHO, WHA, 2019).

While appropriate that the PBAC want to clearly indicate how each recommendation was reached, what is the point of providing even more information in PSDs when the existing format is, realistically, inaccessible to anyone without disease and technical knowledge and an appreciation of the meaning of uncertainty in this context? Inclusion of a summary in lay-man’s language in PSDs would go much further towards openness. It is like providing a patient with a copy of his/her CT scan and the report without an explanation as to what it means.

Pew Research Center, August 2019,
“Trust and Mistrust in Americans’ Views of Scientific Experts, Page 24

Do we want people to be able to understand the information OR is the fact that it is openly available more important?

According to a 2019 Pew Research survey, it is the later. Americans confidence in scientific findings are most influenced by open public access to data and independent committee reviews. There appears to be no similar survey providing Australian opinions.

Scientific journals are strongly encouraging authors to make datasets open source at the time of study publication with the intent of it becoming a mandatory requirement. Should sponsors of reimbursement submissions expect anything different? It is notable that journals target technical audiences, while PSDs are ostensibly for consumers (patients, healthcare professionals, competitors).

Before public release of information, consideration should be given as to who will use it, for what purpose(s), and the most appropriate release format, which is what the TGA and PBAC have undertaken via consultation in the examples provided. Is it enough?

Note: There is an opportunity to voice your opinion on ‘What does open government mean to you’ as input to the Third National OGP Australian Action Plan 2020-2022.

Image source: Jim Pavlidis

NICE and PBAC

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The current review of NICE is a useful foil upon which to consider the announced refresh of the Australian National Medicines Policy. Both are being driven by concern that access processes are not keeping pace with biomedical innovation.

The table below provides a side by side comparison of the two appraisal systems and HTA reimbursement environments. Of interest is the relatively recent increase in NHS responsibility and focus on overall budget impact of reimbursing a new technology.

The need to effectively manage uncertainty whilst still making decisions is a key challenge of providing timely access, especially as patient populations are becoming smaller and more targeted.

 
EstablishedNICE became a legal entity in April 1999.
First guidance published was an assessment of zanamivir for flu.		The PBAC evolved from the Formulary Committee and became an independent statutory body under s 101 of the National Health Act (NHA) in 1953.
Cost-effectiveness was introduced into the NHA in 1998, with the first PBAC Guidelines published in 1992.
Responsible forTechnology appraisals (TAs) that assess the clinical and cost effectiveness of health technologies (pharmaceuticals, biopharmaceuticals, procedures, devices, diagnostic agents) and highly specialised technologies (HST). The primary role of the PBAC is to recommend new medicines for listing on the Pharmaceutical Benefits Scheme (PBS), taking into account the medicine registration, its clinical effectiveness, safety and cost-effectiveness compared with other treatments.
Cost Recovery FeesNew regulations came into effect 1 April 2019 allowing NICE to charge for appraisals. A single TA costs approximately US$ 156 K for large, and US $40 K for small companies.Significant increases from 1 July 2019, with the full process to listing, including one major submission, costing approximately US$ 300 K. A second stage is to be implemented from July 2020 but may be delayed.
Current review Review of Methods and Processes
Announced July 2019		Federal Government review of the National Medicines Policy. Formerly announced October 2019
Review ScopeEvaluation methods of technology appraisals and highly specialised technologies (HST).   The QALY as a decision tool is not included with NICE indicating that changes to methods will only occur if there is a compelling case.Consider whether the policy in its current form continues to meet the needs of Australians.  
Task force established, Terms of Reference pending. On-hold as resources directed to COVID-19.
StakeholdersSubstantial interest from external stakeholders and interest groups.    Wide-spread interest and involvement of health stakeholders, patient and consumer groups.
Review Concerns – access and impact on patients
-affordability in the NHS
-clearer criteria for HST
-improved representation of the patient view
-inclusion of non-health benefits
-success of the life sciences sector		-timely and equitable access to new, innovative therapies
-affordability (Govt, community, patient)
-supply chain (shortages, rebates)
-integration of patient voice
-viable medicines sector
Average evaluation16.0 months (2009 – 2016)4.5-month cycle from submission to PBAC consideration
Average time registration to reimburse (2012-2017)*4.2 months13.8 months
% NCEs registered, subsequently reimbursed (2012-2017)* 84.3%
(29% not funded for full licence, and 5% only recommended for the Cancer Drugs Fund).		46%
PricingVoluntary Pricing and Access Scheme (VPAS) came into effect in January 2019, replacing the Pharmaceutical Price Regulation Scheme (PPRS). VPAS promises more and faster NICE appraisals for NCEs and speed up of appraisals for non-cancer medicines to be in line with cancer medicine timelines. Following loss of the Pharmaceutical Benefits Pricing Authority (PBPA) in 2014, the Department of Health has become the sole arbitrator of pricing. Reforms in 2015 introduced statutory price cuts at 5, 10 and 15-year anniversaries from PBS listing targeting patented medicines.
Last major reviewIntroduction of end-of-life criteria and formation of the Cancer Drugs Fund (CDF) that relaunched under NICE in 2016.Strategic Agreement 2017-2022 – Streamlining PBS Processes in progress.
PBAC Guidelines Version 5.0 published September 2016.
Parliamentary level reviewsAn inquiry by the All-Party Parliamentary Group (APPG) on Access to Medicines highlighted demand for wider value thresholds and modifiers, better management of uncertainty and improvements in the use of data and real-world evidence be brought into the methods. Senate Inquiries into the Supply of Chemotherapy Drugs (2013); Availability of New, Innovative and Specialist Cancer Drugs in Australia (2015) and Funding for Research into Cancers with Low Survival Rates (2017). Recommendations accepted and implemented by Goverment to varying degrees.
US trade dealsUK-US post-Brexit trade agreement under negotiation.
While US propose to redesign the NICE process, NHS England has been taking steps to increase its role in affordability and to effectively bypass NICE when appraising innovative medicines.
NHS will play a key role in applying pressure on drug prices irrespective of any trade deal.		2005 AUSFTA was instrumental in introducing major reforms to PBS listing process.
Many, such as Independent Reviews and Public Summary Documents have been diluted, or configured to suit DoH purposes. For example, recent call for 100% transparency of clinical data. Others, such as PBAC hearings, continue to be invaluable.
Budget impact
(Ghabri and Mauskopf 2018).		NHS Commercial Medicines Unit responsible for Patient Access Schemes and negotiation of outcomes-based pricing agreements. Introduction of the budget impact test makes a high budgetary impact the reason for manufacturers to reduce their price, either directly or indirectly, by lowering the cost-effectiveness threshold.Since 2010–2011, any recommendation by PBAC that has a financial impact for the Federal Government is considered by the cabinet. The estimated financial impact of a drug on the Australian drug budget is a significant predictor of the PBAC recommendation for reimbursement
Managed EntryReview will consider how to better managing uncertainty around early data and surrogate endpoints, such as collecting RWE while trials pending.Process available since February 2011. Infrequently utilised following outcomes of initial agreements, .
Political InterferenceHealth Secretary Matt Hancock’s recent intervention in funding decisions around cystic fibrosis drugs, Orkambi and Trikafta, shows that NICE’s independence can be compromised.Parliamentarians exert pressure on the Minister of Health (MoH) to enact listings once a positive PBAC recommendation has been made. However, MoH can deflect back onto Sponsor for not meeting the advice (conditions) of the PBAC recommendation. Strong commitment to this ‘independence’ of the PBAC.
Role of consumer and patient advocacy groupsThe Review proposes new responsibilities for external stakeholders, such as delivering quantitative evidence of disease modifiers to be used in assessments and decision making. Disparity of resource and HTA skills within the patient group community to address these issues could risk furthering the inequality and lead to a misrepresentation of some therapy areas.Increasing call, and opportunities for patient voice to be heard during evaluation of new medicines.
Pilot initiative whereby patient groups meet with PBAC members prior to consideration of a relevant submission, is to be formalised. Industry increasingly engage with patient groups, especially around relevance of clinical endpoints and capturing real impact of disease. Government focus on transparency has lead to development and launch of a publicly accessible Medicines Status Website.

References and Notes:

Lanning R. Does NICE hold the cards to drug pricing and reimbursement? PharmaField 30 March 2020.

Boyd N. A NICE Transformation? PharmaTimes Magazine, April 2020, p. 25-26.

*Medicines Australia 2018 COMPARE 4 Report

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Globalisation and the pricing of biopharmaceuticals

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As the Sponsor of a new medicine seeking listing on the national Pharmaceutical Benefits Scheme (PBS), pricing used to be relatively straightforward once a positive Pharmaceutical Benefits Advisory Committee (PBAC) recommendation had been received.

The Pharmaceutical Benefits Pricing Authority (PBPA), defunct as of 1 April 2014, would determine the price based on: (1) PBAC advice accompanying the recommendation and, (2) the Sponsor’s pricing submission. Calculation methods were explained in the PBPA Manual (last edition 2009) and, sometimes further discussion with the Pricing Section of the Department of Health (DoH) was required to finalise, for example, exact amount of cost offsets etc. From a company perspective, as long as you stayed above the specified ‘global floor’, the local price was a matter for the local affiliate.

Prior to August 2007 reforms, when the PBS was split into two formularies (F1, F2) based on the availability of a generic brand following lost of exclusivity, the ‘reference pricing method’, was most commonly used. This is where drugs are priced based on their relative safety and efficacy, as determined by the PBAC and documented in Therapeutic Relativity Sheets. Where drugs are considered to be of similar efficacy and safety, the lowest priced brand or drug sets the benchmark price (‘cost-minimised’).

The ‘cost plus method’, where a gross margin of 30% is considered reasonable based on costs declared by the Sponsor in the PB11b form, became applicable to a larger range of drugs (F2, multiple brands) after the reforms, with reference pricing only applying to F1 (single brand, on patent) drugs.

A Price Disclosure policy was introduced for F2 medicines. This was necessary for the Government to realise the savings when generic brands cost-minimised to originator brand prices. Generic companies were using the excess margin above cost, due to lower development investment, to incentivise prescribers and pharmacists. Various iterations to price disclosure processes have been extremely effective in finding the lowest viable price. This policy is almost wholly responsible for over 30% of PBS services currently being below the general patient co-payment ($40) and in no need of Government subsidy.

Source: PBS Expenditure and Prescriptions Report 1.7.18 to 30.6.19

Continuing adjustments to policies and Health Technology Assessment (HTA) evaluation guidelines maintained Government pressure on prices and risk mitigation. This resulted in the need for Special Pricing Arrangements (SPAs) and Risk Share Agreements (RSAs), respectively. Rebates of 100 % are now the norm. When ‘effective’ (i.e. real) prices being paid for new medicines are lower than the global floor these must be confidential otherwise long delays to PBS listing are likely. This is because of the increasing number of countries who base their local drug prices on those paid by a basket of countries (international reference pricing). This approach is proposed in the USA as part of the Pelosi bill.

Where a comparator is listed under a SPA, preparing an economic evaluation has become a guessing game as to the effective price. Needing to use the published list price disadvantages a new medicine in the evaluation process.

Things became more complicated when the PBS Access and Sustainability Package, introduced in mid-2015, included a 5% price cut to all F1 brands listed for five or more years. As the Table shows, F2 is a smaller source of potential savings for the Government. Anniversary cuts were extended to include 10 and 15 year listing marks as part of the 2017 Commonwealth-Medicines Australia Strategic Agreement.

Affiliate pricing of a new medicine is increasingly centralised by companies as Government payers push to use taxpayers’ money wisely. In both situations, prices expected, based on perceived and relative value, may be very different to those modelled on the available clinical evidence.

Collaboration between payers and pressure for reform (2), including transparency of real prices, may result in pricing becoming very simple if companies choose to set only one global price.

(1) Impact of introduction of value-based pricing in Germany (2) See articles by Neil Gubert on cross-border collaboration and the Commonwealth Fund on drug pricing reform.

Photo source: BIG Maze, National Building Museum, Washington DC, 2014

It’s almost MMXX!

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Yes, 2020; a Leap year; summer Olympics; US Presidential race; and UN International Year of Plant Health!

Whatever is forecast with respect to Australia’s weather and economy in the coming year, here are a few predictions that haven’t quite come to fruition (Courtesy of BestLife):

Source: Cincinnati Refined
  • In 1937, Nikola Tesla predicted that “within a century, coffee, tea, and tobacco will be no longer in vogue.” His idea was that “it will simply be no longer fashionable to poison the system with harmful ingredients.”  Tobacco, yes; but coffee, definitely not!
  • A 1951 edition of the magazine Popular Mechanics was confident that every family in the 21st century would have at least one helicopter in their garage. This guy in Cincinnati has achieved it!
  • In 1900,  John Watkins Jr., a curator at the Smithsonian Institution, predicted that by the 2000s, “there will be no C, X, or Q in our everyday alphabet. They will be abandoned because unnecessary.” This was before Alfred Butts invented Scrabble in 1938!
  • However, for those familiar with the habits of teenagers, in one facet, Watkins was accurate when he noted that people would only communicate with “condensed words expressing condensed ideas.”

On a serious note, the past decade has seen the Australian Pharmaceutical Benefits Scheme effectively flat-line at AU $11 Billion (US $7.45 B) per year in real dollars. This is despite the upward pressures of the listing of multiple innovative products, especially in the oncology and infectious disease areas; increases in service numbers associated with an ageing demographic; and 50% of the population managing a chronic condition. Forces pushing expenditure down have included highly effective pricing policies, in particular following loss of patent exclusivity; approximately 30% of prescriptions being self-funded as the cost is below the indexed co-payment threshold; and down-scheduling of products to over the counter and off the PBS.

While this stagnation is of concern to those whose livelihood depends on the PBS, i.e. manufacturers, other suppliers, wholesalers, community and hospital pharmacies; in political terms, the majority of the sector has been neutralised. Only retail pharmacies, and a successful negotiation of the 7th Community Pharmacy Agreement (7CPA) stand between the Commonwealth Government and budget control of the program. New and amended listings are carefully added for maximum political capital, with only those in the background appreciating the full extent of the delays to patient access.

Further policy initiatives should be expected in 2020 and beyond to make up the short fall in savings to Government committed to by Medicines Australia in the 2017-2022 Strategic Agreement (SA). Savings of AU $1.8 Billion (US $1.22 B) are to be delivered for reinvestment into listing of new medicines, however the delay in entry of Humira biosimilar competition has left a significant gap to date. The Government may make trade-offs between the Industry and the Pharmacy Guild, representing owners of Australia’s 5,700 community pharmacies, and the Pharmaceutical Society of Australia (PSA), particularly around services focused on ‘Medicines Safety’ as the 10th National Health Priority.

Other activities to expect locally next year:

—-My blogging companions—-
  • Review of the National Medicines Policy;
  • Roll-out of Tranche 2 of PBS Streamlined Process improvements;
  • Impact of full Cost-Recovery to take effect from 1 July;
  • Introduction of electronic and active ingredient prescribing requiring clinicians to take extra steps to prescribe a specific brand;
  • Further biosimilar uptake initiatives;
  • Continuing formalisation of consumer and patient input to steps in product R&D and Market Access;
  • Activity around proposed changes to the Supply Chain, which have not progressed in 2019. This has the potential for global impact in terms of price transparency and product access.
  • Increasing patient activism in New Zealand.

Thank you for your patronage during 2019.

Surviving Community Pharmacy

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As the purchasing of medicines moves towards a commodity model in Australia, with off-patent (F2) molecules a marketplace, and ‘me too’ R&D programs and associated payer behaviour creating as much in the patent (F1) space, providers all along the supply chain are being impacted.

What are Community Pharmacists, as one of the key groups affected by stagnation of the PBS, doing to remain viable?

Community Pharmacy Programs and Services

The first Community Pharmacy Agreement (CPA) in 1990 between the Commonwealth and the Pharmacy Guild, representing pharmacy owners, introduced a new remuneration framework for pharmacies supplying PBS medicines and created incentives to optimise the distribution of pharmacy services around the country.

Specific programs and services appeared in the third CPA from 2000, and subsequent CPAs have included an ever-increasing number of remunerated clinical activities. These still represent a small proportion of the overall value of the agreement, but offer more revenue with no increase in costs. Hence, in addition to retaining existing components, such as 30-day dispensing, expansion of service programs are a key feature of negotiations of future agreements. Figure 1 shows the average annual dollar amount a pharmacy can expect to generate from the components of each CPA.

Figure 1. Actual Expenditure minus CSO from 3CPA onwards, divided by number of approved pharmacies. Note: Pharmacies purchase PBS items as stock and only claim this cost back from the Government following dispensing. # not including additional $600 million committed as part of Pharmacy Compact 2017. Sources: AIHW, DoH, ANAO, Pharmacy Guild.

This is despite continuation of Government payment for programs being contingent on demonstrating clinical and cost-effectiveness. A 2017 evaluation by HealthConsult was unable to make conclusive assessments on the benefit of Dose Administration Aids, Staged Supply, MedsCheck, Diabetes MedsCheck and Home Medicines Reviews conducted by pharmacists due to a lack of robust data. Further research is required in this, currently, evidence free zone.

The Pharmacy Guild and Instigo initiated the Health Advice Plus program in 2016 to assist pharmacies to maximise the opportunities and revenue afforded by the CPA programs. Based on 1,000 pharmacies, they recently reported that, of those pharmacies : 87 % have a private consultation room; 44 % are maximising their clinical Intervention potential; only 28 % are maximising MedsChecks with an average of 12 a month (up to 20 permitted); 56 % have not reached their DAA cap level (individually calculated based on previous 12 months); and 72 % are currently providing vaccination services.

The Aged Care Royal Commission Interim Report prompted this month’s decision by the COAG Health Council to name “Medicine Safety” as Australia’s 10th National Health Priority Area. The report also notes the need for the Federal Government, Pharmacy Guild and Pharmaceutical Society of Australia (PSA) to consider the effectiveness of the Residential Medication Management Review program and make it stronger and more accessible. A timely opportunity for pharmacy as the 7CPA negotiations continue.

From supply function to pre-primary healthcare

The Guild and PSA have recognised the need to broaden the role of community pharmacy and are actively working to expand the sectors offerings for treatment of minor aliments and preventative health (vaccinations, health checks, risk assessments, self-care, lifestyle issues).

Source: https://www.tga.gov.au/scheduling-news (accessed 301119)

From a policy and revenue perspective, this is also a sweet spot that the Government is prepared to support in the face of a healthcare system showing the strains of an ageing population, increasing prevalence of chronic diseases, higher public expectations and rising costs of new technologies. According to a RACGP report, General Practitioner workloads, inadequate Medicare rebates and decreasing numbers of graduates choosing the speciality, with numbers in training dropping by 20% year on year since 2016, will not see an improvement in the foreseeable future.

This is a gap that community pharmacy is driving to step into. One means is via down scheduling of Prescription Only medicines to Pharmacist Only (a process managed by the TGA) which has the added value to Government of reducing visits to the GP (capacity and expenditure benefit) but also the cost of these medicines moves to the patient’s pocket from the PBS (if listed). Table 1 shows the products most likely to be re-scheduled in the near future.

Meanwhile the debate about extending pharmacists’ scope of practice to include prescribing of Prescription Only medicines continues.

Figure 2. Pharmacy Mark-up: the increase in the pharmacy mark up between 2014-15 and 2015-16 is a result of the introduction of the Administration Handling and Infrastructure fee, replacing the previous percentage based mark-up. Source: https://www.pbs.gov.au/statistics/expenditure-prescriptions/2015-2016/cpa-exp-rpt-2015-16.xlsx (accessed 301119)

De-linking dispensing fee from medicine price

As successive Price Disclosure policies dropped the average price of many frequently dispensed former block buster molecules for chronic conditions, community pharmacies watched their revenue similarly plummet. Coupling payment for completing the same dispensing process to the price of a product was no longer such a good idea. The 6CPA moved compensation for dispensing from a percentage of product value to a flat fee. The Administration, Handling and Infrastructure (AHI) fee has stabilised the impact of price disclosure. Figure 2 shows the impact of this changeover comparing Government payment in the last year of the 5CPA with that in the first of the 6CPA when the AHI was introduced. Unlike PBS listed medicine prices, the AHI is indexed annually.

The 7CPA is due to commence in July 2020. Negotiations continue between the Australian Government, Pharmacy Guild and PSA.

Image source

3 Reasons why Medicines Shortages will continue

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Mandatory reporting of medicine shortages† from 1 January this year has seen new notifications to the Australian Therapeutic Goods Administration (TGA) increase by over 400% (n=1,455 for 2018-19) compared to the previous period (n=274).  Currently, over 10% of the drugs on the TGA ‘list’ are classified as critical with the potential to have a life-threatening or serious impact on patients. 

Miljkovic N et al. Results of EAHP's 2018 Survey on Medicines Shortages. Eur J Hosp Pharm 2019;26:60-65.
Miljkovic N et al. Results of EAHP’s 2018 Survey on Medicines Shortages. Eur J Hosp Pharm 2019;26:60-65.

The FDA Drug Shortages Task Force Report, released today, compared drugs that went into shortage from 2013 to 2017 to similar drugs that did not go into shortage. Drugs in shortage were more likely to be relatively low-price, in particular genericised sterile injectables, including anaesthetics, chemotherapy and pain treatments. Australian hospital (2017) and European Association of Hospital Pharmacists (EAHP) 2018 Medicines Shortages surveys reported similar groups of drugs most often impacted (Figure 2 from the report shown).

Shortages can and do have a significant impact on patient care, especially when there is little or no notice. Required reporting provides authorities, health care professionals and patients time to prepare. Unfortunately, this measure, like those taken elsewhere in the world will not reduce the problem because:

(1) Commodity pricing policies

Treating drugs as commodities exposes them to the rigours of supply and demand.

Although demand is increasing globally, due to ageing populations and availability of more effective medicines, the FDA Task Force found ‘prices rarely rose after shortages began, and during shortages, production typically did not increase enough to restore supply to pre-shortage levels.’ This points to a ‘broken marketplace‘, where scarcity does not result in the price increases predicted by basic economic principles.

RELATION BETWEEN PRICING POLICY AND MEDICINE SHORTAGES. From https://publicaties.vereniginginnovatievegeneesmiddelen.nl/magazine/mm2019uk/medicines-for-tomorrow/
RELATION BETWEEN PRICING POLICY AND MEDICINE SHORTAGES. From https://publicaties.vereniginginnovatievegeneesmiddelen.nl/magazine/mm2019uk/medicines-for-tomorrow/

As the graph shows the relationship between introduction of a preference policy in the Netherlands, where only the cheapest medicine for a specific disorder is reimbursed, has resulted in a greater proportion of these medicines being in shortage.

Companies seeking to enter the generic marketplace may not have the manufacturing history and quality safeguards in place to ensure sustainable supply. This is despite providing guarantees to Governments who preference suppliers based on price.

The production and supply of pharmaceuticals is regulated by Good Manufacturing Practice (GMP), as prices decrease, companies may consolidate manufacturing facilities to maintain profitability. Hence, those medicines with the most competition will be the most vulnerable to shortages.

(2) Expansion of reference pricing

As more Governments, including the US (see Pelosi Lower Drug Costs Act 2019) introduce reference pricing into their drug procurement policy mix, others are losing their appetite for the quid pro quo of access to new products at ‘hidden’ prices. The Dutch Health Minister has recently called to ignore the confidentiality of pricing agreements, while the Australian pharmaceutical industry was rocked last year by the presentation of a poster listing rebates by ATC code at an international conference. In addition, changes to supply chain rebate arrangements continue to be progressed by the Australian Government despite concerns around the impact on availability of new drugs.

There is a real possibility that companies will set a price for a product and that will be the price, irrespective of country. This will restore the marketplace but patients in countries that have come to expect, and demand, substantial discounts on new medicines will be left waiting for access.

(3) Solutions to date have been ineffective

TGA Annual Performance Statistics. Table 80. https://www.tga.gov.au/book-page/15-reporting-medicine-shortages
TGA Annual Performance Statistics, 2018-19, Table 80.
https://www.tga.gov.au/book-page/15-reporting-medicine-shortages

The EAHP 2018 survey found that medicines shortages have become more troublesome since the last survey in 2014, with 91.8% respondents reporting shortages impacting patient care. The FDA Task force found that the number of ongoing drug shortages has been rising, and that their impact is likely underappreciated. Note: the FDA infographic shows shortages averted, the how likely includes requested intervention by other suppliers.

Manufacturing issues continue to be the most common reason for supply shortages. As reported in 47% of cases to the TGA in 2018-19; and 37%, plus 27% other quality issues, to FDA in 2012. Medicines are not commodities.

The FDA conclude that: ‘The root causes of shortages involve economic factors that are driven by both private- and public-sector decision-making.’

Private sector decisions serve business interests. While public sector decisions aim to benefit societal, and political, interests. The Task Force suggests quality ratings of manufacturing facilities and new contracting approaches with incentives as possible levers.

In the meantime, there will always be someone seizing the day, in this case, a plethora of global wholesalers!

Notes: † Defined as when supply of a medicine in Australia will not, or will not be likely to, meet the demand for that product in Australia any time within the next 6 months; *Reportable medicines are Registered Schedule 4 (Prescription Medicine) or Schedule 8 (Controlled Drug) products, and certain non-prescription medicines considered critical and listed in the relevant legislative instrument. Chain photo from Google Images

The PBS goes to Canada

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Taxpayer funded national programs providing universal access to prescription drugs are longstanding policy in Australia, NZ and UK.

Considering the establishment of a similar program in Canada, where largely private province-based schemes* currently operate, presents an opportunity to consider what is lost when these programs are the sole focus of pharmaceutical policy.

Advocates of such schemes point to cost-effective product selection and savings generated by lower and reducing generic and brand name prices. The principles of universality, equity of access, safe & appropriate prescribing, and value for money are paramount.

The Minister of Health, the Hon Greg Hunt MP, regularly commends the foundation role that the PBS plays in the Australian health system. While such programs have reduced both overall expenditures and the average price paid per drug, like all policies, they often have unforeseen repercussions.

In her recent essay, Kristina Acri examined the potential unintended consequences of a publicly-funded national Pharmacare program in Canada. She reviewed the experience of New Zealand, Australia and United Kingdom to identify those aspects where reality has not matched the promise:

  • Drug supply is put at risk by sole tendering & reference-based pricing policies, as well as use of cost-effectiveness analysis. For off-patent medicines, seeking the lowest price within a guaranteed supply framework should protect against shortages. However, whether appropriate or not, global factors and profit seeking influence where available drug supplies are sent. Dealing with drug shortages are an increasing occurrence for pharmacists, clinicians and patients;
  • The scheme may not be universal. In Australia, 50 % of total drug expenditure is directly borne by patients, one of the highest cost-sharing rates in OECD countries after the far Northern European countries (ranging from Sweden 48 % to Iceland 58%) (OECD, 2015). Before introducing the scheme, how medications will be selected and what the costs will be to patients and taxpayers must be fully understood; and
  • The likely impacts on access, health outcomes, costs, and innovation for patients, physicians, the market and the economy should be examined.
Infographic, Globerman & Barua (2019)
The Patented Medicine Prices Review Board (PMPRB) is an independent quasi-judicial Federal agency established in 1987 under the Patent Act. The PMPRB ensures prices of patented medicines sold in Canada are not excessive.

In a similar review, Crosby et al. (2016) discuss that to control spending on universal schemes, access to new drugs is rationed, downward pressure is imposed on prices, and costs are pushed to individuals. They conclude that introducing a universal scheme to Canada would increase public spending, reduce patient choice to only subsidised medicines, delay access to new drugs and lead to inequities in coverage compared to the existing situation.

Globerman and Barua (2019) discuss the impact of the Health Canada proposed amendments to the Patented Medicine Prices Review Board (PMPRB) procedures which will include the evaluation of the cost effectiveness of drugs by CADTH, into the determination of maximum allowable prices for patented drugs. They note that that these changes ‘seem to be focusing more on controlling expenditures on pharmaceuticals than on ensuring that Canadians have access to new therapies’. They advocate for an efficient level of expenditures on pharmaceutical drugs, not simply containing expenditures on those drugs.

‘In principle, cost-efficiency analysis is a technique for comparing the social benefits of a drug relative to its cost.

In practice, the conventional application of the technique arguably leads to an underestimation of the social benefits of new drugs.’

Despite these concerns, last month legislation introduced by Health Canada was passed and paves the way towards a PBS Canadian-style! ‘Government of Canada Announces Changes to Lower Drug Prices and Lay the Foundation for National Pharmacare.’ News Release, August 2019.

*A third of Canadians are covered by public drug plans which vary from province to province. Most are covered through their workplace by private insurance plans. Another 10% of Canadians have no coverage at all (Canadian Health Coalition, 2017).

References: (1) Kristina M. L. Acri (née Lybecker) The Unintended Consequences of National Pharmacare Programs. The Experiences of Australia, New Zealand, and the UK. The Fraser Institute, December 2018. Vancouver BC, Canada. (2) Steven Globerman and Bacchus Barua. Pharmaceutical Regulation, Innovation, and Access to New Drugs. An International Perspective. The Fraser Institute, January 2019. Vancouver BC, Canada. (3) Crosby L, Lefebvre C, Kovacs-Litman A. Is pharmacare the prescription Canada needs?. UWOMJ Drugs 2016;85(1):23-5.

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Who is subsidising who?

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Over 30% of all prescriptions written in Australia for PBS-listed medicines are self-funded as they cost less than the relevant Co-payment (under co-pay).

General patients (co-pay $40.30, 1 Jan 2019) are the sole recipients of this cost-shifting as the dispensing fee ($7.29) is greater than the Concessional co-pay amount ($6.50).

So why do the 8% of Services to General patients account for over 30% of Government PBS/RPBS expenditure?

The bar graph shows Benefit paid and differences in proportion of Services attributable to patient categories with and without inclusion of under co-payment numbers. Another way to present this, and as reported in the annual PBS expenditure & prescription tables, is as Average Government Benefit paid per Service.

The line graph shows the evolution of Average Benefit per Service by patient category over time. This graph is based on figures downloaded from Medicare Australia website (includes both Section 85 & Section 100). The total services (approximately 205 K) match the prescription numbers presented in the PBS Expenditure and Prescription Report for the same periods, which exclude under-co payment prescriptions (since reporting started in 2012-13).

Why is the Average Benefit per Service currently over five times higher for the General patient category compared to Concessional?

 

Is there a difference in the demographics of General and Concessional patients such that higher cost (F1) medicines are prescribed more frequently to General category patients?

Benefit paid per service ($) by ATC CV CNS Anti-Infectives GI Respiratory Oncology-Immuno Other
2017/2018 $ 15.24 $ 28.65 $ 149.68 $ 31.64 $ 46.16 $ 966.48 $ 62.98

In recent years, the peak and drop for average benefit paid per service for the General ordinary category mimics that of usage of new Hepatitis C treatments. Is there disproportionate prescribing of these and other higher cost drugs, such as oncologics and immunomodulators, between categories? To test this, benefit figures were downloaded from the Medicare Australia website for Hepatitis C medicines and reveal a split between General and Concessional patient categories of 39% to 61% in $1.37 billion paid by Government during 2017-18. Total service numbers (72,266) show a similar ratio with General 37% and Concessional 63%. For the ImmunOncology medicine, Nivolumab (Opdivo, BMS) total services (40,560) were split General 32% to Concessional 65% in 2017-18.

Perhaps the cause is an artefact of the huge volume, relative to General patients, of low cost (F2) items prescribed, and paid by Government for Concessional patients? Rosuvastatin and Atorvastatin had the highest prescription volumes in 2017-18, of these 37% and 30%, respectively were under co-payment prescriptions.

As a proportion of over co-payment prescriptions in 2017-18, Safety Net (SN) accounted for by 1.3% of the General patient category and 18.2% Concessional. The addition of approximately $40 and $6.50, respectively per script to Government expenditure is unlikely to be responsible for the large difference in average benefit per service paid, given the General SN category is so small.

Suggestions on what may be driving this difference are welcome. Hope you can provide me with a D’oh moment!

 

Sources: Simpsons; Medicare Australia Statistics; PBS expenditure prescriptions report tables