• Contents
• Letter of transmittal
• User guide
• part 1: overview of the nba and the blood sector
• part 2: highlights of 2011–12
• NBA's Performance
  (PARTS 3,4, AND 5)
• part 3: performance: securing the supply of blood and blood products
• part 4: performance: improving the sector and its management of risk
  • Introduction
  • Sector Improvement
  • Risk Management
• part 5: performance: supporting appropriate and safe use of blood and blood products
• part 6: Corporate management
• part 7: financial management and accountability
• part 8: appendices

Part 4: Performance: Improving the Sector and its Management of Risk

Sector Improvement

In the 2012-13 Federal Budget the NBA received strong endorsement from governments for its drive to improve data capture and analysis across all aspects of the supply chain. This area of activity, together with our focus on knowledge acquisition and management, and other management and accountability initiatives, are key strategies to improve the overall efficiency and sustainability of the sector.

The Portfolio Budget Statements particularly promoted the appropriate and efficient use of blood and blood products through better supply management and information capability. In response to the outcomes of the Budget, the NBA started developing a new corporate plan in consultation with relevant government departments and chief medical officers.

Sector improvement through data collection and analysis

The availability of comprehensive, consistent, relevant, timely and robust data, and the capacity to analyse that data, are powerful tools to identify areas where improvements can be made in supply management and clinical demand. In 2011-12 considerable progress was made in the capture and analysis of data, building on the achievements of the previous year. In addition to the projects described below, see also page 93 for a description of the red blood cell usage project.

Integrated data management system

The Integrated Data Management System (IDMS) is used by the NBA to manage the budgeting and forecasting of supply and demand for blood and blood products, inventory management and contract administration.

In 2010-11 the NBA's internal auditor had conducted a review of the system including an assessment of risks, evaluation of controls, assessment of security, interfaces with other business systems and a general review of the performance of the system. The review confirmed the system's functionality and its appropriateness as a platform for further development of NBA information and data strategies and made a number of recommendations for enhancements.

During 2011-12 the NBA addressed several issues such as developing technical specifications for the interface between BloodNet (see below) and IDMS for goods ordering and receipt verification. Build 7 of IDMS enabled the automated production of key parts of the NSP&B documentation which will reduce the potential for errors in future.

Implementation of BloodNet

Initially known as ORBS, the web-based ordering and inventory management system for blood and blood products was developed by Queensland Health. The first site went live in January 2008 and was Queensland-wide by December 2008. Last year the NBA and Queensland Health conducted a national proof of concept trial of the system. Following the successful completion of the trial, the JBC provided interest monies to implement the system throughout Australia and to undertake further development to provide additional capabilities. Reports generated from BloodNet will assist approved health providers to comply with the NSQHS standard for blood and blood products (see page 91).

During 2011-12 the roll out of the ordering and receipting modules of BloodNet throughout Australia gathered pace, and by 30 June 2012 BloodNet was processing 75 per cent of total volume issued by the Blood Service (see Table 4.1 below).

Throughout the year the equivalent of two full-time trainers visited hospitals and pathology laboratories to implement BloodNet, in addition to the NBA providing training, user support groups and help desk support. The information being generated from the BloodNet system is significantly assisting the work on a national inventory management framework (see page 42).

At the same time, we started to roll out a module of BloodNet focusing on the fate of products; we expect to complete this by late 2012. This function enables users to record details of product discards, transfers of products between approved health providers and actual transfusions-essential information which has not been readily available before. Approved health providers can generate reports on demand containing reliable data to help them in managing discards and reduce wastage of blood products. They can also upload data from the Blood Service's legacy Electronic Returns Information Capture (ERIC) system.

The reliability and accessibility of BloodNet were continuously improved during the year. In addition, in collaboration with those states, a proof of concept trial to interface BloodNet to approved health providers' laboratory information systems commenced in New South Wales (with eBlood) and Queensland (with AUSLAB).

The development of a central user management and authentication system for all blood sector-wide ICT systems was largely completed during 2011-12, with implementation to occur between August and December 2012. This new system, Guard, will enable users to access the relevant systems with ease, without the need for multiple usernames and passwords. When the National EHealth Transition Authority (NEHTA) completes the implementation of the National Authentication Service for Health (NASH), our intention is to integrate Guard into NASH to further enhance the user experience and provide enhanced security.

Connecting Australia's hospitals to BloodNet

BloodNet, the NBA's national online blood ordering and inventory management system, took a major leap forward in 2011-12 when a trial was launched in Newcastle to connect BloodNet with the John Hunter Hospital's own computer system. This diagram illustrates how the data flows between hospitals, the NBA and the Blood Service:

The proof of concept trial is ongoing, but progress is so promising that the NBA is hopeful of connecting more hospital information systems to BloodNet during 2013.

The trial is testing the feasibility of connecting a web service between the two systems (known as an interface), so that whenever John Hunter Hospital pathology staff receipt blood orders into BloodNet, it automatically talks' to their hospital's own information system passing on unit data (which BloodNet receives from the Blood Service's ICT system). This eliminates the need to re-key the information. This will not only save valuable time for laboratory staff, it will also remove the possibility of any data entry errors by abolishing manual data entry processes.

The ultimate goal is to build an interface between BloodNet and the laboratory information systems used in all major Australian hospitals. Not only will this dramatically reduce the time spent on data entry for hospital staff, it will also provide a real-time view of blood inventory levels across the country for the first time.

The new features offered by the interface will benefit hospitals in several ways:

• hospital inventory levels will automatically be passed every 15 minutes from the hospital to the NBA to enable the NBA, the Blood Service and jurisdictions to more actively manage the national inventory
• the fate of the blood unit (whether it has been transferred to another hospital, discarded or transfused) is recorded in both systems
• component details (such as component type, blood group, collection and expiry dates, modifiers and phenotypes) upon being receipted into BloodNet will be passed to the hospital's laboratory information systems.

Early in 2012 the JBC approved expenditure on new infrastructure to host ABDR and BloodNet. Implementation of the new hardware was largely completed by the end of June 2012 in readiness for the launch of the redeveloped ABDR in mid-August 2012. We believe that there is scope to use our new infrastructure to support other sector-wide systems, thus achieving further economies for governments- as well as ensuring standardisation and consistency in quality- as part of the national health reforms.

Australian Bleeding Disorders Registry (ABDR)

The ABDR is used on a daily basis by clinicians in all Australian haemophilia treatment centres (HTCs) and clinics as a clinical tool to assist in managing the treatment of patients. The NBA also uses the registry to collect data relating to the ordering, supply and use of clotting factor products, in order to create demand models which assist in negotiations with suppliers on the provision of these products. Hence the system is designed to produce data that ful?ls the needs of clinicians, patient representative groups and governments within a highly controlled governance framework. Oversight of the registry is provided by a steering committee consisting of representatives of AHCDO, the HFA, jurisdictions and the NBA. The AHCDO supports the use of the registry.

Major progress on increased data collection and quality was again achieved during the year, with a noticeable increase in the quality of data; the data capture rate exceeded eighty per cent, enabling the publishing of the second ABDR Annual Report (see www.nba.gov.au/abdr). The data in the report is useful to clinicians in a variety of settings; as at 30 June 2012 the report was downloaded 1,074 times.

The ABDR report is enabling us to compare Australian performance with other countries' data. This will assist the NBA in planning appropriate long-term supply arrangements. An example of comparative data is shown in Table 4.2 below.

How the Australian Bleeding Disorders Registry helps children

FIGURE 4.1 ABDR haemophilia treatment centre dashboard report*.

Children with bleeding disorders often face a life of uncertainty due to their regular bleeds' preventing them from activities that involve the usual bumps and scrapes of childhood.

To give them more certainty in their medical treatment, the NBA developed the ABDR which collects clinical data on patients at all haemophilia treatment centres (HTCs) across Australia, including at Melbourne's Royal Children's Hospital.

Data and Product Manager at the hospital's HTC, Julia Ekert, said the ABDR was used to keep a record of each child's medical data, height, weight and dosage. From this, she can accurately order products for patients. The clinical staff also use the database on a day-to-day basis as it provides a current patient record and is used to offer treatment advice over the phone to concerned parents. In order to ensure that the ABDR is a good clinical tool, Julia works closely with the clinical team to make sure that data is accurate and up-to-date.

If a parent says a child has had a bleed, we can deal with that because we have a record of the child's height and weight on the registry including their treatment plan and what doses they are on,' she said. We have an accurate history of the patient in order to give them the best advice.'

The Melbourne HTC looks after 250 children with bleeding disorders across Victoria, including about 80 who are regular visitors due to the severity of their condition. The registry is also used to monitor patients who have their products delivered at home. This provides continuity of care to patients by allowing other staff involved in ordering products access to the information required.

If I'm not there, all the data is in one spot for the nurse who covers product ordering when I'm away,' Ms Ekert said. Prior to the registry, the hospital had paper records listing every haemophilia patient's name and diagnosis. If we wanted to get a medical record from the basement, it could take hours, if not the next day before you had it. The new ABDR was a significant advance in that it allowed me to use one system for many tasks, which streamlined my workload.'

The ABDR's capacity to search the data, for example by age group, has made it easier to contact patient groups when running education programs. We've done various education days with kids but we find the informal method works better, so we organise a Boys Day Out where patients aged eight to 14 go out for a fun day with clinicians and nurses,' she said. They go to arcade games and ten pin bowling where they get to talk to other kids about having haemophilia and they end up feeling like -it's not just me. At the same time, the staff can get in some education messages.'

The ABDR is the only database of its kind in Australia, collecting clinical information related to the treatment of people with bleeding disorders. With the support of the NBA, the ABDR allows accurate information regarding national clotting factor usage which can then be used in product procurement. The NBA and the ABDR are pivotal in ensuring there is an adequate supply of clotting factor concentrate which is essential to optimise the quality of life of people with bleeding disorders in Australia.

* This screenshot is an example of a HTC dashboard report from the redeveloped ABDR and includes a summary of HTC details for patients, interactions, bleeding disorder severity measures, products ordered and usage comparisons. This has been provided for illustrative purposes only to demonstrate one of the newly available reports in ABDR however, at the time of provision, this report was still in production and may have since undergone further refinement. The de-identified data provided relates to a HTC for a specific time period and statistical calculations and representations within this report are based on publicly available information. The data used has been migrated from the previous version of ABDR for the retrospective period of July 2012, which at the time of provision, may have included incomplete data. The NBA and ABDR Steering Committee have approved the use of this image for the NBA Annual Report only.

In March 2011 the JBC approved expenditure on the redevelopment of the ABDR using interest monies to employ experts to undertake the project in house. This fourth generation redevelopment will improve the efficiency, performance and stability of the system, increase security of sensitive data, provide a sufficiently flexible platform to support the development of new and future enhancements, and enable the integration of the ABDR into other sector systems such as BloodNet in a cost effective way. The redevelopment is being guided by extensive consultation with key stakeholders such as nurses' groups, HTCs, data managers, jurisdictions, the HFA and representatives of haemophilia physiotherapy, social worker and counsellor groups.

As the project neared completion a workshop was held in June 2012, involving all haemophilia treatment centre staff, for final user testing, training and review of the data migrated from the earlier system. Support from the haemophilia community and users has remained high throughout the redevelopment project and the go-live date of mid-August 2012 is keenly anticipated.

In May 2012 the JBC approved additional expenditure to develop a patient interface for the ABDR. This will enable us to capture data in real time on patient infusions, problems, and home inventory management. This initiative is strongly supported by patients and the clinical community.

NBA's business intelligence system

Launched in January 2010, the NBA's business intelligence system, known as Big Red, provides a single electronic repository for the secure storage, manipulation, integration and analysis of data drawn from other NBA systems.

During the year, efforts on Big Red have focused on data cleansing and integrating data from disparate systems into a combined report. Another major effort was to complete technical work to enable secure remote access by jurisdictions to Big Red, a key feature of the system when it was originally designed. The function will enable jurisdictional users to link data on demand and supply to compare ordering practices among approved health providers. Work on the arrangements governing access to data is continuing.

National IVIg management system

The National IVIg Management System (NIMS) was to capture information on the use of IVIg in order to support and align with the Criteria for the clinical use of IVIg in Australia. Further development work was placed on hold following the decision of the CTEPC to undertake an independent review of the authorisation and clinical governance arrangements for IVIg (see pages 76-77).

Our annual Report on the use of IVIg 2010-11 may be found on the NBA's website.

Barcoding

The 2007 JBC decision to mandate speci?c standards for barcoding of blood and blood products was subject to a further decision of the JBC in 2010 to align the implementation of the policy with the implementation of blood ordering and receipting systems.

In 2011-12 the NBA commenced work to identify the data set to be captured across the supply chain and the barcode functions needed. Extensive consultations were undertaken with NEHTA, GS1 Australia and all major laboratory information system vendors to enable the NBA to develop an implementation plan that is robust and achievable. A formal discussion paper will be issued for comment later in 2012. This project is now being undertaken in parallel with the NBA's development of the interface between BloodNet and the laboratory information systems of approved health providers (see pages 62-63).

Sector improvement through knowledge management

In depth knowledge of global trends, both in medical and pharmaceutical developments, in supply markets, and in how blood and blood products are managed elsewhere in the world, enable the NBA to provide high quality advice to governments and stakeholders, and to negotiate value for money contracts with its suppliers.

Monitoring international trends

The NBA monitors international developments that may influence the management of blood and blood products in Australia. The horizon scanning program, which forms an integral part of our knowledge network, provides up to date intelligence on emerging or potential issues, processes, techniques and technologies relevant to the sector. It enables us to provide current, proactive and informed analysis to governments and is essential in negotiating contracts for the supply of blood and blood products. We monitor advances in processes, techniques and technologies that enable the NBA to ful?l its functions under the National Blood Agreement (clause 2 refers).

Our focus is on:

• information that may have an impact on global supply, demand and pricing
• potential new product developments and applications
• trends in clinical practice, government regulation and legal decisions
• emerging risks that could put financial or other pressures on the Australian sector

Matters of interest are posted on the NBA website www.nba.gov.au/supply/sector-monitoring and a summary of highlights from the year under review is also posted. In the highly abridged printed extract below, we describe the types of information we seek, with limited and brief examples. More details on these and other matters can be found in the summary on the website.

Products

The NBA follows product development and clinical trials that may, within a reasonable time frame, introduce new products, new uses or new treatment schedules to the market. Areas of interest in 2011-12 included:

Clotting factors

or patients with the bleeding disorder haemophilia, convenience of treatment increases with the length of half-life of clotting factors. For both Factor VIII (haemophilia A) and Factor IX (haemophilia B) there are longer-acting products in the pipeline.

Competitive pricing may develop in the markets for rFVIIa and Factor IX. There is currently only one product commercially available for each of these, but in both cases new products are in clinical trials.

Immunoglobulin

Immunoglobulin is used by patients with primary immunodeficiencies. While this has usually been given by infusion, in some countries self-administered subcutaneous injection is now possible. Intravenous immunoglobulin (IVIg) is also variously used round the world in treating certain disorders of the nerve and muscles, including Guillain-Barr- syndrome, chronic inflammatory demyelinating polyneuropathy, and multifocal motor neuropathy.

Of particular significance for the NBA is the current interest in immunoglobulin as a possible therapy or even prophylactic for Alzheimer's disease including:

• Baxter initiated a second Phase III trial evaluating the use of its Gammagard Liquid in patients with mild to moderate Alzheimer's. This complements the company's first Phase III trial, which is fully enrolled and will conclude by the end of 2012. The company also announced an extensive study for some patients who completed 18 months of treatment in the first Alzheimer's Phase III trial. This will provide additional data related to the longer-term effects.
• Grifols launched a second trial of treating Alzheimer's with plasma derivatives, combining apheresis with the administration of albumin and IVIg.

If immunoglobulin were to become a recognised therapy for Alzheimer's the increased demand would have a major impact on price, particularly in the short-term. There are a number of potential drugs in the pipeline, not based on plasma products, which are therefore of interest.

Regulatory matters

The NBA follows overseas regulatory decisions on products, processes or procedures which are or may be of relevance to its responsibilities. In the current year the frontier of stem cell research has been a focus of attention:

• The US Food and Drug Administration has collaborated with the US National Institutes of Health (NIH) on a series of workshops on moving pluripotent stem cell therapies into clinical practice
• In Canada, Osiris Therapeutics won approval for its stem cell drug, Prochymal, for a disease that can attack patients who received bone marrow transplants. Prochymal was approved for the treatment of acute graft versus host disease in children for whom steroids have not worked.

Market structure and company news

The NBA follows company profitability, business forecasts, capital raisings or returns, mergers and takeovers, arrangements for joint research and/or development, contracts for supply of manufacturing inputs, and marketing agreements. Companies of interest include suppliers, potential suppliers and developers of products which may be of interest. In general terms, the industry is currently in growth mode. Developments of interest included:

• The US State of Georgia announced a $US78 million incentive package to Baxter to build an integrated plasma processing facility in Georgia, with greater capacity than its Los Angeles plant. Construction in Georgia will begin in 2012 and commercial production in 2018.
• In February 2012 CSL announced that first-half net profit fell 3.4 per cent as the high Australian dollar put pressure on its earnings, but that without the impact of exchange-rate movements, net profit would have grown 16 per cent. The company also upgraded its full-year profit guidance, citing vigorous demand for its products. North America was CSL's biggest market, accounting for 42 per cent of revenue, followed by Europe with 32 per cent and Australia with 10 per cent. CSL will begin reporting in US dollars in the year starting 1 July 2012, in line with industry practice and to reflect the predominance of the company's global sales.
• Grifols acquired 51 per cent of Araclon Biotech, which specialises in diagnosis and therapies for neurodegenerative diseases, especially Alzheimer's
• Amongst recent agreements, Shire and Sangamo BioSciences will develop therapeutics for haemophilia based on Sangamo's zinc finger DNA-binding protein technology transfusions, while Axerion Therapeutics and MedImmune will develop and commercialise a biologic approach to treat Alzheimer's.

Overseas events

The NBA follows both safety concerns and instances of good practice. It monitors health issues in countries from which its visitors and immigrants come.

United Kingdom: Since 2002, in order to minimise the risk of potential variant Creutzfeldt-Jacob Disease (vCJD) transmission through blood transfusion, imported FFP has been used to treat those born on or after 1 January 1996, and therefore unlikely to have been exposed to bovine spongiform encephalopathy (BSE) through their diet. The Advisory Committee on the Safety of Blood, Tissue and Organs (SaBTO) reviewed this measure at its March 2012 meeting and concluded that it should continue, even when those recipients reach 16 years of age

Canada: The Canadian Blood Services urged Canadians of African and Caribbean heritage to celebrate their unique culture through blood and stem cell donation. Individuals with African heritage possess certain minor blood groups that may make it difficult to find compatible blood when repeated transfusions are needed as in sickle cell disease treatment', said Dr Isaac Odame, Medical Director of the Global Sickle Cell Disease Network at Sick Kids Hospital in Toronto.

Israel: Pluristem Therapeutics announced in May that a seven year-old girl suffering from aplastic bone marrow, who was rapidly deteriorating, had seen a reversal of her condition following intramuscular injection of Pluristem's PLacental eXpanded (PLX) cells (in aplastic bone marrow the patient has no blood-forming haematopoietic stem cells in bone marrow).

United States: The US Department of Health and Human Services said it would re-examine the ban on blood donations from men who have sex with men, with four studies planned.

Safety issues

The impact of transfusion on outcomes in cardiac surgery remains an interest, as does the relationship between age of red cells and surgical outcome. Examples include:

• Risk of infection: Patients given packed red blood cells had an increased risk for major infection after cardiac surgery, according to study results presented at The Society of Thoracic Surgeons 48th Annual Meeting.6
• Volume transfused: At a professional update on paediatric and congenital cardiovascular disease in February 2012, researchers reported that in paediatric heart transplantation, increasing amounts of blood transfused appeared to be associated with worsening outcomes.7
• Age of red cells: The duration of red blood cell storage did not adversely affect outcomes in ventilated patients receiving transfusions, according to a small randomised trial reported by Daryl Kor, from the Mayo Clinic in Rochester, Minnesota8. The investigators noted that their results differed from earlier study results that found storage duration led to adverse clinical outcomes. One possible reason is that patients in this study received a similar red blood cell dose, while earlier researchers did not adjust for dose, they suggested. Also, pre-storage leukocyte reduction was used for all red blood cell units transfused.
• Transfusion threshold: The American Association of Blood Banks issued new guidelines on the level at which a patient’s red blood cell count can be viewed as so low as to require a transfusion. The Association concluded most patients would do just as well if the threshold was at seven or eight grams per decilitre in hospitalised, stable patients.
• Appropriate use: Johns Hopkins researchers said a new study confirmed there is still wide variation in the use of transfusions and frequent use of transfused blood in patients who do not need it. Some recent studies have shown that surgical patients do no better, and may do worse, if given transfusions prematurely or unnecessarily. Steven M. Frank, leader of the study described in the journal Anesthesiology said ‘Blood conservation is one of the few areas in medicine where outcomes can be improved, risk reduced and costs saved all at the same time. Nothing says it is better to give a patient more blood than is needed. The exceptions’, Frank says, ‘are cases of trauma, haemorrhage or both, where infusing blood quickly can be lifesaving.’

Research

A wide range of scientific research has some potential to affect the use of blood and blood products, but with variable time horizons. Even research which achieves its desired scientific outcomes may not lead to scaled-up production, clinical trials, regulatory approval and market development. A selection of brief reports follows:

Stem cells

• Scientists for the first time isolated single stem cells that give rise to many different types of blood cell: white cells, platelets and red cells. It may be possible one day to use stem cells to regenerate the entire blood system, or to isolate stem cells from individual patients in order to grow their own personal supplies of blood cells or clotting factors. John Dick of the University Health Network in Toronto led the study9, which used genetically modified mice without their own immune systems. Scientists grew human bone marrow cells within the animals to target those that are the stem cells of the blood system.

Genetics

• University of Iowa researchers are using 14 sets of twins (identical and non-identical) to determine if the rate at which red blood cells decay is inherited.
• Two recent USA studies examined two specific health issues faced by African-Americans:
  • Research10 is shedding light on why kidney disease is far more common among African-Americans than other ethnic groups. Finding the gene believed responsible (APOL1) is the first step in developing treatments. It is a variant that wards off sleeping sickness, a disease mainly borne by tsetse flies that kills tens of thousands of people in Africa each year. The gene is carried by as many as 12 per cent of African-Americans.
  • Researchers have found five previously unknown gene mutations believed to be associated with elevated blood platelet counts in African-Americans. ‘The findings’, they say, ‘could lead to the development of new drugs to help prevent coronary artery disease’. The study11 is believed to be the first of its size to focus on platelet genetics in African-Americans, who have a higher risk of stroke than other racial groups. They also have relatively higher platelet counts and average platelet volume, and worse outcomes after a heart attack.
• In a small study, researchers compared plasma proteins in male and female blood. Of the 231 proteins identified, there were differences in abundance between genders. If these are confirmed in further work, male or female plasma could be selectively transfused to patients, depending on their condition12.
• Scientists have developed a gene therapy strategy they say could feasibly treat both ß-thalassaemia and sickle cell disease. The Weill Cornell Medical College-led team that reported on the development13 devised in parallel a simple assay to predict how well individual patients are likely to respond to the treatment.

Patient blood management

• In a recent presentation14 Dr Mark Pagnano of the Mayo Clinic said that administering fluids first rather than transfusing patients, and using an antifibrinolytic agent such as tranexamic acid (TXA), can reduce blood loss and the need for transfusion in patients undergoing hip or knee arthroplasty.
• A study15 from the London School of Hygiene and Tropical Medicine has concluded that ‘the use of TXA in the treatment of traumatic bleeding has the potential to prevent many premature deaths every year’.

Alzheimer's Disease

International research activity is broad, focusing on why the disease develops, its diagnosis, prevention and treatment options. In 2011-12, research included investigating the genetic basis of susceptibility and defining the molecular pathways responsible for neuronal degeneration. A number of drugs were in clinical trials:

• A preliminary study16 of 46 patients found that bapineuzumab may reduce the development of tau tangles in the brain, thought by some to be a hallmark of Alzheimer’s. The results of a more conclusive study are expected later in 2012.
• Science reported in April 2012 that researchers using nuclear resonance imaging and computer modeling had glimpsed proteins turning into the distinctive clumps of Alzheimer’s, which may be a crucial target for preventive medicine. Researchers found compounds that disrupt formation of these amyloid clumps17.
• A drug designed by Genentech to stop Alzheimer’s before it can take hold will be tested on a family in Colombia with a genetic mutation that leads to the disease in its members, usually in their 40s.
• Intellect Neurosciences announced two new programs in its Alzheimer’s pipeline. Their monoclonal antibodies target early neurotoxic forms of tau protein, and could yield therapeutic and diagnostic uses.
• Four articles in the journal Alzheimer’s and Dementia in 2011 described new criteria for Alzheimer’s, dementia and mild cognitive impairment. They emphasise that the Alzheimer’s pathophysiological process starts years and perhaps decades before clinical symptoms, and that biomarkers can detect amyloid ß deposition and the effects of neurodegeneration in the brain18.

Haemophilia

Studies have variously:

• demonstrated the potential of gene therapy to convert severe bleeding in haemophilia B into a mild form of the disease, or reverse the disease19
• found the overall success rate to immune tolerance induction is independent of FVIII dosing regimen although a high dose of FVIII compared to a low dose achieved tolerance more rapidly20
• reported the in vivo therapeutic efficacy of an approach to bypass FVIII with FIX variants engineered to directly activate Factor X (FX) and propagate the intrinsic coagulation pathway in the absence of FVIII21.

Synthetic blood substitutes

Scientists say worm blood could hold the key. The haemoglobin found in earth and sea worms carries about 50 times more oxygen than human blood, prompting University of California researchers in San Diego to examine the potential to replicate the same oxygen-carrying capacity in artificial blood22.

Infectious diseases

Disease burden within a community (e.g. dengue in North Queensland in summer) may limit blood collection for a time. Disease in individual donors (e.g. influenza), or potential disease resulting from travel (e.g. malaria) means a donor must be deferred. Some people may not be permitted to donate at all (e.g. people who lived in the UK for a period critical in the history of vCJD). Blood donations are tested for a number of diseases (e.g. HIV and Hepatitis B), and there are others globally for which it may become necessary to test in Australia in the future (e.g. Chagas disease, the tick-borne babesiosis23 and Lyme disease).

Vaccines: Progress was made during the year on developing universal flu vaccines, pre-pandemic avian flu vaccines protecting against a range of strains, four-strain seasonal flu vaccines, intranasal (inhaled) vaccines, and plant-expressed virus-like particle vaccines. Testing continues on tetravalent dengue vaccine.

Prion diseases

• French researchers found that prions are more easily able to jump between species than has been previously thought24.
• In January 2012, neurologists across Britain were told by the Nationlal Health Service’s National Prion Clinic, part of the University College London Hospitals Trust, and the Medical Research Council’s Prion Unit that a new blood test is now available for vCJD. Until then the only way of confirming the diagnosis had been through tonsil biopsies or after the patient has died when brain samples can be taken.

Overseas intelligence gathering

The collaboration of national plasma product supply planners (NPPSpa)

The fourth meeting of the collaboration of national plasma product supply planners was held in Madrid in March 2012 and attended by the NBA's Deputy General Manager, Commercial Contracts. The group consists of a number of national agencies that have responsibility for plasma products. The aim of the group is to support participants' needs for a secure, cost effective supply of plasma for fractionation, plasma derivatives and their clinically substitutable recombinant products. It continues to be the only international forum that shares data and experiences in the management of plasma products and is especially valuable in the context of procurement mechanisms and outcomes. Meetings of the group are timed to coincide with the annual International Plasma Protein Congress. Issues discussed included:

• comparative per capita product usage trends
• product utilisation and authorisation processes
• overviews of recent and upcoming tender processes and contract terms, including product portfolios, prices and price disclosure policies, inventory holding and the role of each agency
• impacts of non-reversible anti-coagulants
• availability and implications of product home delivery/treatment
• the emergence of long-acting haemophilia products, and country strategies around licensing and contracting
• product management strategies for FVIII and immunoglobulin products
• mechanisms for feedback from clinicians and patients on products and the performance of suppliers
• relationships between registered indications, actual usage, costs and supply
• market reaction to the global recall and subsequent re-introduction of Octagam.

As in previous years, the information obtained at the meeting place the prices for plasma-derived and recombinant products achieved in recent Australian tenders at a very competitive level.

The meeting was chaired by Canada. Members agreed to continue meeting annually, and to explore the possibility of inviting other countries to join the group.

International Plasma Protein Congress

The International Plasma Protein Therapeutics Association is an industry organisation for the commercial plasma products industry. Members include all of the major global plasma fractionation companies, as well as commercial plasma collectors, suppliers of equipment and consumables, and other associated companies. The congress is attended by companies, industry suppliers, analysts, regulators, purchasing bodies and patient group representatives. It presents opportunities to gather intelligence about company strategies and hot topics'. For example, market analysts were very interested in the range of new recombinant clotting factors which will become available in the next few years.

Items of relevance to the Australian blood sector included: impacts of the global financial crisis, regulatory developments, including: collaboration and information sharing in the USA and Europe-including that relating to tests for prothrombotic activity in products; health technology assessment methodologies and value; plasma demand and donation; developments in tolerisation for severe haemophilia; and updates on progress on clinical trials on the use of IVIg to treat Alzheimer's disease.

Sector research framework

One of the DoHA-funded projects completed by the NBA in 2011 identified a number of significant gaps in critical information needed by the blood sector in order to make key decisions for improvement.

Under the National Blood Agreement, one of the roles of the NBA is to ‘facilitate and fund appropriate research, policy development or other action in relation to new developments by relevant governments, or non-government persons or bodies’25. In December 2011 JBC agreed that there would be value in establishing a research framework for the blood sector which would identify priority areas for research, governance and funding arrangements and administrative processes. The national blood arrangements place the NBA in a unique position to coordinate resources for research across the blood sector.

During 2012 we commenced work to develop this framework. The NBA believes that through relevant research, evidence could be generated to improve clinical and laboratory practice, ensure product adequacy and safety into the future, and ensure patients are receiving the most appropriate products to meet their clinical needs. Work will continue through 2012-13 to refine and finalise the research strategy and develop an implementation plan for the NBA research program.

Dissemination of information

The 2010 amendments to the Freedom of Information Act 1982 require agencies to release data under an open license unless there is a cogent reason not to do so. We see this as an opportunity to publish key documents in order to increase knowledge of blood and blood products, and awareness and understanding of governments' expenditure on them.

In May 2012 the JBC members agreed to the publication on the NBA website of the annual NSP&B and monthly supply reports, reflecting their general support for the publication of data at a national and jurisdictional level in relation to product volumes and costs. In addition, the NBA's series of product monographs, initially developed in 2010, will be published on the NBA's website in future. The monographs are updated annually and contain factual information on trends on issues and costs for each supply group.

Sector improvement through management and accountability initiatives

An important focus for the NBA in 2011-12 was the review and assessment of policies and procedures across aspects of the blood supply chain, in order to increase efficiency and accountability within the blood sector, and internationally. See also pages 40-41 for descriptions of selected performance measures included in contracts with suppliers. Implementation of the new National Safety and Quality Health Service (NSQHS) standard for blood and blood product safety (page 91) and our work on inventory management (page 42) will also lead to improved efficiencies and accountability within the sector.

Review of the authorisation and clinical governance framework for intravenous immunoglobulin (IVIg) and normal immunoglobulin (NIg)

In 2010-11 the NBA and key stakeholders undertook a preliminary analysis of the existing management arrangements for IVIg and alternatives that may be used. This was in response to an identified need for a higher level of cost-effectiveness to be applied to the use of IVIg. Growth has averaged approximately 11 per cent per annum since 2003, although there were reductions in the rate of growth after the release of the initial Criteria for the clinical use of intravenous immunoglobulin in Australia (the Criteria). The range of indications for which IVIg therapy demonstrates some clinical benefit is expanding. This factor, and the potential for a signifcant and ongoing growth in demand, poses challenges for both supply security and affordability.

In addition, the analysis found that current authorisation arrangements are not consistent across jurisdictions. While arrangements need to be flexible enough to accommodate the capacities of different facilities, some fundamental authorisation requirements should be in place across all jurisdictions and all facilities. The review found that current authorisation arrangements differ in their focus on the public and private sectors and the extent to which there is a dedicated review of individual patients on IVIg treatment to determine efficacy. The analysis also noted that there is no national peak body for reporting and analysing trends on how IVIg is used. In May 2011, the CTEPC commissioned an independent review of the authorisation and clinical governance framework for IVIg to identify improvements. The NBA is coordinating the review on behalf of governments. The review has the following goals to:

• ensure that funded immunoglobulin use reflects best clinical practice and is cost effective
• ensure that the outcomes of decision-making regarding access to IVIg funded under the national blood arrangements are consistent with the Criteria (see page 94)
• improve the capture of information on the need for, use and outcomes of treatment with intravenous or normal immunoglobulin (IVIg or NIg) and improve the evidence base that will inform future changes as to what is regarded as best practice in use and prescribing
• improve governments’ understanding of the issues, benefits and risks of including NIg and subcutaneous immunoglobulin (SCIg)26 in any new management framework.

The CTEPC appointed Dr Stephen Christley to chair a review advisory group which contains representatives from health consumer and patients groups, clinicians, large and small jurisdictions, DoHA, the Blood Service and the NBA, as well as an IVIg nurse and a health economist.

In March 2012 the NBA engaged Ernst and Young to manage aspects of the review. During the initial phase the consultant reviewed and analysed current authorisation procedures and systems to access IVIg and NIg (see page 36-37). The objective of this was to determine the extent of compliance, efficiency and satisfaction of all stakeholders with the current authorisation and clinical governance arrangements for IVIg. This included identifying what is known at each step in the process to support decision-making. The consultant made a detailed study of practices in both small and large jurisdictions to determine the benefits and weaknesses in how IVIg is currently accessed and managed in different facilities.

To ensure that the NBA and the consultants had a comprehensive understanding of how IVIg is accessed, used and managed across Australia, a survey asking about prescribing and use of IVIg was developed and distributed widely in the medical community. Several follow-up focus groups with leading medical disciplines using IVIg were also undertaken. How IVIg is managed and accessed in other countries will also be investigated and research will be conducted into management frameworks for other high cost medications within Australia. Following these activities, the consultant will develop a number of models for possible future arrangements for IVIg, and subject the key models to risk and cost-benefit analysis. We anticipate that the review will be completed early in 2012-13.

Implementing the Statement on National Stewardship Expectations for the Supply of Blood and Blood Products

The Statement on National Stewardship Expectations for the Supply of Blood and Blood Products was approved by health ministers in November 2010. The statement is a core foundation document that defines the commitment and cooperation expected of approved health providers in how they manage these products and contribute to the objectives of the National Blood Agreement. The Agreement does not address the roles and responsibilities of health providers such as laboratories in hospitals and clinics and other institutions that receive blood and blood products for dispensing to patients.

We believe that to ensure success, it is essential to integrate our strategies to implement the statement with those of the NSQHS to implement the new blood and blood product safety Standard 7 (see page 91). During 2011-12 the NBA collaborated with the ACSQHC (Australian Commission of Safety and Quality in Health Care) on plans to implement the statement and Standard 7 nationally. We will use three approaches to ensure adoption:

• promote the statement at national, jurisdictional and local levels: with a suite of supporting publications targeted at specific groups within health providers; engaging with the private sector; and holding forums to maintain a high level of sector engagement for governments' shared objectives for, and showcase best practice of, stewardship in the management and use of blood and blood products
• integrate and prioritise the statement, for example in clinical education, quality improvement systems and processes and in national health reforms. The NBA is collaborating directly with the ACSQHC to detail the activities that are expected at hospital level to ensure compliance with Standard 7. Consideration will also be given to incorporating the principles of the statement into national agreements and relevant NBA contracts
• establish effective data linkages within and across the health sector to support accreditation, quality improvement, performance measurement and reporting. Good progress is already being made on standardising a methodology to support red blood cell data linkage (see page 93), and integrating the NBA's BloodNet system with the laboratory information systems of health providers (see pages 62-63). We have also started work on improving national inventory management (see page 42).

Some actions can be taken at a national level, while others would be more effective at a jurisdictional level. The NBA will give clear explanations on how performance will be measured and provide information on the tools available to health providers to meet these obligations.

In 2012 the NBA started work on a promotional brochure for approved health providers and consumers to outline the expectations of the statement in the context of the broader national blood arrangements and the requirements under the standard. The SA government developed a framework of key activities for implementing the statement, and in May 2012 all JBC members agreed to implement this approach at both policy and practical levels.

We also undertook research and preliminary consultations to increase our understanding of the relationships between the private health sector and the blood sector and to identify opportunities for the NBA to engage with appropriate sections of this important group. The first major step in this activity will be to invite key representatives from the private health sector to attend a forum at which we can discuss how the NBA could assist private organisations to implement reforms in the clinical governance and management of blood and blood products.

Performance scorecard for the sector

Key performance indicators are essential tools for both monitoring and improving the quality of health services. Significant advances have been made over the past decade in the development of indicators for the Australian health sector. Performance indicators originally focused mainly in the field of acute hospital care, but now extend into services such as community health, general practice and public health. The language and culture of performance measurement is now well established in the day-to-day life of acute public hospitals, and in the health care sector.

The vision of the National Health Performance Committee is for a health system that searches for, compares, learns from the best and improves performance through the adoption of benchmarking practices across all levels of the system. (National Health Performance Framework Report, 2001)

This vision has underpinned the development of performance indicators for national health services and the blood sector. The emphasis has been to have a set of indicators for blood related services that serve as tools for improving service quality through collaborative benchmarking.

The stated primary policy objectives of the National Blood Agreement27 are to:

• provide an adequate, safe, secure and affordable supply of blood products, blood related products and blood related services in Australia
• promote safe, high quality management and use of blood products, blood related products and blood related services in Australia.

In order to fulfill these objectives, the Agreement requires the NBA to:

• undertake or facilitate national information management, benchmarking and cost and performance evaluation for the national blood supply
• facilitate the development of national information systems for safety and quality issues in relation to the Australian blood sector.

The objectives have been summarised as three perspectives:

• supply security-to provide an adequate, safe, secure and affordable supply of blood products, blood related products and blood related services in Australia
• clinical safety and quality-to promote safe, high quality management and use of blood products, blood related products and blood related services in Australia
• sector management-to undertake or facilitate national information management, benchmarking and cost and performance evaluation for the national blood supply; and facilitate the development of national information systems for safety and quality issues in relation to the Australian blood sector.

During 2011-12 the NBA has been developing and identifying a set of key performance indicators using these perspectives, aligned with the national health performance framework, for use in benchmarking and monitoring the blood sector.

The preliminary development of performance indicators for the blood sector is occurring in collaboration with key sector stakeholders. As an example, the NBA discussed the draft indicators with suppliers at the annual suppliers' forum held in April 2012.

However, while agreement on performance indicators is a necessary first step, it will not in itself lead to their introduction for blood related services. A number of issues need to be resolved for this to occur, including how the indicators are produced, how frequently they are reported, and who is responsible for reporting them. We will need to continue to collaborate with blood sector stakeholders to outline the steps required to embed performance indicators within the sector.

6 Horvath KA. ‘Do blood transfusions affect the risk of infections after cardiac surgery? Experience of the NIH/CIHR Cardiothoracic Surgical Trials Network’. Presented at: The Society of Thoracic Surgeons 48th Annual Meeting; Jan. 28-Feb. 1, 2012; Fort Lauderdale, Fla. Howard-Quijano K, et al, ‘The effect of red blood cell transfusions on pediatric heart transplant patients’ PCCD 2012; Abstract 43. : Annual Update on Pediatric and Congenital Cardiovascular Disease, Orlando, February 2012.

7 Howard-Quijano K, et al, ‘The effect of red blood cell transfusions on pediatric heart transplant patients’ PCCD 2012; Abstract 43. : Annual Update on Pediatric and Congenital Cardiovascular Disease, Orlando, February 2012.

8 Kor DJ, et al ‘Fresh red blood cell transfusion and short-term pulmonary, immunologic, and coagulation status:
A randomized clinical trial’ Am J Respir Crit Care Med 2012.

9 Faiyaz Notta*, Sergei Doulatov, Elisa Laurenti, Armando Poeppl,Igor Jurisica, John E. Dick, ‘Isolation of Single Human Hematopoietic Stem Cells Capable of Long-Term Multilineage Engraftment’ Science 8 July 2011: Vol. 333 no. 6039 pp. 218-221 DOI: 10.1126/science.1201219.

10 at the Beth Israel Deaconess Medical Center, led by Dr Martin Pollak.

11 Published in the online journal PLoS Genetics and reported 5 March, 2012.

12 ‘Proteomic analyses of human plasma: Venus versus Mars’. Transfusion 2012, 52, 417-424.

13 Stefano Rivella, and colleagues report in PLoS One: ‘Therapeutic Hemoglobin Levels after Gene Transfer in ß-Thalassemia Mice and in Hematopoietic Cells of ß-Thalassemia and Sickle Cells Disease Patients’.

14 Pagnano M. Minimizing blood loss: An acid trip in 2011. Paper #38. Presented at the Current Concepts in Joint Replacement 2011 Winter Meeting. Dec. 7–10. Orlando, Florida.

15 Katharine Ker, Junko Kiriya, Pablo Perel, Phil Edwards, Haleema Shakur and Ian Roberts: ‘Avoidable mortality from giving tranexamic acid to bleeding trauma patients: an estimation based on WHO mortality data, a systematic literature review and data from the CRASH-2 trial’, BMC Emergency Medicine 2012, 12:3 doi:10.1186/1471-227X-12-3 Published:
1 March 2012.

16 Published April 2, 2012 in Archives of Neurology.

17 The study, published April 30 in the online journal PLoS ONE, is entitled ‘The influence of spin-labeled fluorene compounds on the assembly and toxicity of the Aß peptide’.

18 Andrew E Budson; Paul R Solomon. ‘New Diagnostic Criteria for Alzheimer’s Disease and Mild Cognitive Impairment for the Practical Neurologist’, Practical Neuology Pract Neurol. 2012;12(2):88-96.

19 Nathwani AC, Tuddenham EGD, Rangarajan S et al. ‘Transfer in Hemophilia B’ N Engl J Med. 2011 Dec 22;365(25):2357-65. Epub 2011 Dec 10.PMID: 22149959.

20 Hay RM and DiMichele DM. ‘The Principal Results of the International Immune Tolerance Study: A Randomized Dose Comparison’ Blood 2012;119(6):1335–1344.

21 Milanov P, Ivanciu L, Abriss D et al. ‘Engineered Factor IX Variants Bypass FVIII and Correct Hemophilia A Phenotype in Mice’ Blood 2012;119(2):602–611.

22 At the Australian and New Zealand College of Anaesthetists ANZCA) conference in Perth, May 2012.

23 The first reported Australian human case of the potentially lethal tick- borne infection babesiosis has been reported in the Medical Journal of Australia.

24 Béringue, V., Herzog, L., Jaumain, E. et al. Facilitated Cross-Species Transmission of Prions in Extraneural Tissue. (2012). Science. 335: pp. 472-475. Accessed 23 February 2012.

25 National Blood Agreement, Part 3, section 25 (n) refers.

26 However, SCIg is not yet funded under the national blood arrangements.

27 National Blood Agreement paragraphs 1(a) and (b), 25 (o) and 35(f)