Potential Future Arrangements for Imported Plasma and Recombinant Products - Summary of Consultation Outcomes
March 2018 update: limited Interim Arrangements for Extended Half-Life Clotting Factor Products
The NBA has finalised arrangements with two companies, Bioverativ and Shire, to provide temporary access to extended half-life (EHL) clotting factor products under NBA supply arrangements for a limited number of haemophilia A and B patients with high priority needs.
Further information can be found at https://www.blood.gov.au/plasma-and-recombinant-product-procurement.
Under the national blood arrangements administered by the National Blood Authority (NBA), Australia imports a range of plasma and recombinant products. The NBA last tendered for imported plasma and recombinant products in 2014. Existing contracts do not expire until 2019.
It is clear to the NBA that there is significant interest and demand for Australian patients to have access to new products at the earliest opportunity. This arises from a number of influences, including participation by some patients in clinical trials, an awareness of new variants and alternative therapies for current products, special access arrangements established in some other countries comparable to Australia and perspectives about the potential benefits of new products for patients.
Against this background, the NBA has undertaken extensive consultations with potential suppliers and clinical and patient stakeholders to inform consideration of future procurement arrangements for clotting factor products, specifically recombinant factors VIIa, VIII and IX products. These consultations took the form of an invitation to make submissions in response to a formal Request for Information (RFI), a Stakeholder Consultation paper and additional less formal engagement directly with stakeholders and industry.
The RFI and Stakeholder Consultation papers were issued on 13 January 2017. Final formal submissions were received on 12 April 2017. A period of further engagement followed with industry and stakeholders to clarify information around some of the issues raised and more generally to assist with understanding those issues.
The consultation process elicited a substantial amount of information in relation to new generation Extended Half Life (EHL) clotting factor products as well as other new monoclonal products that offer substitute therapies for haemophilia treatment. Following careful consideration by the NBA of the submissions and information received, the Jurisdictional Blood Committee (JBC) considered the outcome of the consultations at its meetings on 7 July 2017 and 8 September 2017.
The JBC has now determined on behalf of governments what the most appropriate next steps should be. These steps involve JBC authorising the commencement of a health technology assessment for the potential supply of EHL clotting factor products through established processes (the Medical Services Advisory Committee - MSAC), and the implementation of interim and limited managed access arrangements for rFVIII and rFIX products for some patients while that assessment is being completed.
Responses to the RFI were received from eight companies. A total of 15 stakeholder responses were also received to the Stakeholder Consultation paper. Respondents included professional and representative groups, individual health care professionals and individual patients and carers.
A summary of issues identified in stakeholder responses is included below at Appendix 1. The response from the Australian Haemophilia Centre Directors Organisation (AHCDO) also included an independent clinical assessment by a research team at the University of Adelaide in relation to existing data on the use of EHL products for the treatment of Haemophilia A and B. An Executive Summary of this assessment is at Appendix 2.
Apart from the information included in the Appendices referred to, some key information identified through the consultation processes and through other market intelligence is that:
- in general, current and potential suppliers consider that the NBA’s approach to tendering and contracting for imported plasma and recombinant products is appropriate and sustainable;
- no potential availability of additional rFVIIa products was identified that should change current arrangements;
- Australian patients, their representatives and specialist clinicians are increasingly seeking access to EHL products that are funded under the national blood arrangements;
- in the absence of a firm advance assurance of a contract for the supply of a particular product, there is a reluctance by individual potential suppliers to submit a proposal under Schedule 4 of the National Blood Agreement to have registered new EHL products included for supply within the national blood arrangements;
- there are a range of therapies in development or soon to be marketed, including:
- new variants of ‘short half-life’ recombinant clotting factor concentrates;
- new ‘extended half-life’ clotting factor concentrates;
- new bypassing therapies to treat patients with inhibitory antibodies to clotting factor concentrates;
- other products, including monoclonal antibody products, which may prove an alternative to clotting factor concentrates - these products are currently in Phase I, II or III clinical trials; and
- a small number of gene therapy products are in early development.
- EHL products have demonstrated improved pharmacokinetic characteristics and clinical outcomes, which has been reinforced by the experience of local clinicians and patients with the use of these products in the clinical trials setting; and
- there continue to be points of uncertainty concerning EHL recombinant factor FVIII and FIX products, particularly around the availability of a competitive market, clinically relevant product characteristics, likely clinical utility and choice, and price.
After considering the outcomes of the consultation process, the JBC has asked the NBA to progress work in two key areas in relation to EHL products:
1. Initiate a coordinated assessment of EHL products incorporating a national health technology assessment process through MSAC. While the assessment will require the cooperation of potential suppliers, it will be facilitated by the NBA and enable the NBA to engage directly with potential suppliers to accelerate the formal assessment of EHL products.
2. As the health technology assessment will take some time, the NBA should enable initial and time limited supply arrangements to provide access to EHL products for some patients within the national supply arrangements by implementing:
a) a program offered through an existing contracted supplier that will substitute EHL rFVIII product for a current Standard Half-Life (SHL) rFVIII product for suitable patients to be identified by clinicians in four Haemophilia Treatment Centres (HTCs) to be determined, comprising two paediatric HTCs and two adult HTCs. This is expected to involve about 100 patients; and
b) an EHL Access Program through a new supplier utilising EHL rFVIII and rFIX products in a number of HTCs to be determined. This is expected to involve some 75 patients, including those currently participating in extension trials.
These initial and limited supply arrangements are not to involve additional cost to governments and will only be available for a limited period while the MSAC assessment process is being completed.
The NBA will establish a Reference Group to advise the NBA on the detail of implementation arrangements for the access programs outlined in 1 and 2 above and related issues. The Reference Group will comprise:
- the NBA (as Convenor);
- one nominee from the Commonwealth Department of Health;
- two Jurisdictional government nominees;
- two nominees from AHCDO, comprising one paediatric treating clinician and one adult treating clinician; and
- a nominee of the Haemophilia Foundation of Australia.
Other members may be co-opted to the Reference Group as required, including from the Australian Haemophilia Nurses Group (for both paediatric and adult services) as appropriate.
The limited access programs that have been outlined are expected to provide some immediate benefit to some (but not all) haemophilia patients while the evidence-based detailed assessment of EHLs is undertaken. The programs will also enable the collection of real world supply management information in the Australian context. This will assist the NBA with the design and planning of future procurement and evaluation processes and generally support sound and well informed policy and administrative decision making.
To support these objectives, it will be a requirement that data on product supply, usage and relevant outcomes are recorded within the Australian Bleeding Disorder Registry (ABDR) via HTC data recording and the MyABDR application.
In addition to the action determined by the JBC as outlined above, the JBC also agreed that the NBA should accept the opportunity to obtain improved price outcomes in relation to a current contract with an existing supplier and extend the existing contract with that supplier. Accordingly, the NBA has exercised available contract options to extend that contract for the supply of haemophilia products rFVIII and rFIX until 30 June 2019. This contract extension does not preclude the NBA from decisions about future tendering or consideration of the availability of new variant or alternative therapies during the extension period.
The NBA would like to acknowledge and thank all of those who have contributed to the provision of extensive information through the RFI and the consultation processes. It has required significant effort by individuals, representative groups, suppliers and others, both in Australia and overseas. The NBA also appreciates the support and cooperation of key people in getting to where we now are with consideration of these very important issues.
The limited access arrangements are expected to be implemented by the end of 2017. Arrangements to progress the MSAC assessment are also already well advanced. The NBA recognises that these arrangements will not immediately benefit all affected patients and further detail still needs to be developed. However, the action identified does represent a small but significant step forward and, importantly, identifies a pathway that accelerates full and proper consideration of the issues involved as well as interim action.
- Appendix 1 - Potential Future Arrangements for Imported Plasma and Recombinant Products: Summary of Stakeholder Responses (docx) (52.12 KB)
- Appendix 1 - Potential Future Arrangements for Imported Plasma and Recombinant Products: Summary of Stakeholder Responses (pdf) (89.16 KB)
- Appendix 2 - Assessment of Extended Half-Life Clotting Factor Concentrates - Executive Summary March 2017 (docx) (387.3 KB)
- Appendix 2 - Assessment of Extended Half-Life Clotting Factor Concentrates - Executive Summary March 2017 (pdf) (529.29 KB)
Public consultation on the proposed changes to the Criteria for the clinical use of intravenous immunoglobulin in Australia
Public consultation on the proposed changes to the Criteria for the clinical use of intravenous immunoglobulin in Australia closed at 5pm on Friday 9 June 2017. Feedback will be considered by the National Blood Authority and the National Immunoglobulin Governance Advisory Committee as appropriate before being presented to the Jurisdictional Blood Committee for consideration on behalf of funding governments.
BloodSTAR will be updated when the revised Criteria have been approved. The NBA will communicate the changes to all key stakeholders at that time.
The proposed medical condition pro forma documents have been published on the NBA website and are available here for your information.
If you have any questions please contact the Immunoglobulin Governance Program Team at IgGovernance@blood.gov.au.