NICE and PBAC – TACS Healthcare

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.


Established	NICE 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 for	Technology 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 Fees	New 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 Scope	Evaluation 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.
Stakeholders	Substantial 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 evaluation	16.0 months (2009 – 2016)	4.5-month cycle from submission to PBAC consideration
Average time registration to reimburse (2012-2017)*	4.2 months	13.8 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%
Pricing	Voluntary 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 review	Introduction 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 reviews	An 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 deals	UK-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 Entry	Review 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 Interference	Health 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 groups	The 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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