Medicine made free for extremely rare disease

The Government has made a new medicine available for free for an extremely rare and life-threatening disease, potentially saving patients hundreds of thousands of dollars a year.

The new medicine Galafold® (migalastat) for the treatment of Fabry disease is now listed on the Life Saving Drugs Program, which provides free access to highly specialised medicines to treat patients with rare and life-threatening diseases.

Patients with Fabry disease have a rare enzyme deficiency, which means their bodies have trouble breaking down a fatty substance called globotriaosylceramide.

The condition usually presents in childhood with episodes of severe pain. Other symptoms include skins rashes, headaches, fatigue, vertigo, fever and vomiting and diarrhea.

It can result in potentially life-threatening complications including kidney failure, heart attack and stroke. It can have a major impact on patients and their families.

Currently, there are about 100 Fabry patients receiving enzyme replacement therapy through the LSDP.

Galafold® is a new oral medicine alternative for the treatment of Fabry disease patients aged 16 years and older.

It provides greater treatment choice for Fabry patients, reduces disease symptoms and dramatically improves quality of life, whilst also allowing patients to manage their own treatment at home without the need to have painful injections or infusions.

Without subsidy, Australian Fabry patients would pay hundreds of thousands of dollars for this treatment, putting them beyond the reach of most families who have to fight this extremely rare condition.

Health Minister Greg Hunt said it is the first medicine included on the Life Saving Drugs Program following the implementation of reforms to make the process more timely, transparent and improve patient access through the program.

“Our Government currently funds 14 different life-saving medicines for nine very rare diseases through the program, providing physical, emotional and financial relief for 400 Australian patients,” he said.

“These medicines are very expensive and would be too high of a financial burden on patients.”

Medicines funded through this program include high cost medicines that do not meet the criteria to be funded on the Pharmaceutical Benefits Scheme (PBS).

The new the Life Saving Drugs Program medical expert panel was announced in August. The panel, chaired by Australia’s former Deputy Chief Medical Officer Dr Tony Hobbs, supports the evaluation of medicines for funding on the program and provides advice to the Chief Medical Officer.

Medical research building the economy

Australia’s investment in medical research has significantly boosted the country’s welfare, economy and future potential, according to a new study launched at Parliament House, Canberra.

The Association of Australian Medical Research Institutes (AAMRI) President Professor Tony Cunningham AO released a report of the study in October and said for every dollar invested in medical research, Australia gains a $3.90 return to the economy.

“A near $4-to-$1 is an extraordinary return on investment. This return is far higher than the level needed to secure government funding for just about any other investment in infrastructure,” Professor Cunningham said.

The new study, by KPMG Economics, has identified that the medical research sector – including the downstream medical technology and pharmaceutical sector – employs more than 110,000 people.

It also found that the health gains that flow from medical research result in a larger and more productive national workforce.

“Investing in medical research must remain a top priority for Australia – not only for the health and wellbeing of all Australians, but also to help build a strong economy through employment, knowledge creation and through our burgeoning medical technology and pharmaceutical export industry,” Professor Cunningham said.

The findings demonstrate the national workforce is estimated to be significantly larger than it would have been in the absence of medical research – 23,000 full-time employees more.

The report also shows today’s economy, as measured by GDP, is $2.6 billion larger as a result of historical medical research. Significantly, welfare, a measure of how well off we are as a population, is $1.5 billion higher that it would have been in the absence of medical research.

Professor Cunningham said the in-depth study of the impact of medical research in Australia was a timely reminder that we can only expect the positive health and economic outcomes if we are willing to put in the investment.

“I’d like to thank the Australian community and our politicians for making that investment,” he said.

To view the full report, visit https://aamri.org.au/KPMGReport.

MBS Review process leaves much to be desired

BY CHRIS JOHNSON AND LUKE TOY

The AMA is growing increasingly frustrated at the lack of transparency in the MBS Review process.

AMA President Dr Tony Bartone has written to the MBS Taskforce Chair, Professor Bruce Robinson, warning him that the peak medical body is fast losing confidence in the Review (see separate article in this edition).

The Taskforce has recently dumped numerous Clinical Committee reports out for consultation at the same time, demanding responses within short timeframes in a “targeted consultation” – meaning that the reports are not widely available.

Dr Bartone said the Taskforce had worked on recommendations over the past few years, but now wants the medical profession to respond in just a few weeks or months.

He has asked Professor Robinson to ensure these “unreasonable timeframes” are extended and that his Taskforce be more flexible in its consultation.

Dr Bartone also asked that the reports be made publicly available on the internet.

“This will ensure transparency of the review process,” he said.

The MBS Review Taskforce is assessing upwards of 5000 items on the Medicare schedule with regards to best practice and value for money.

Glaring examples of the irregularity of the Taskforce’s work include the oncology review failing to consult with the Royal Australasian College of Surgeons; and the colonoscopy review inadvertently excluding the Australian Private Hospitals Association.

These are just a few examples.

“But it’s not just the Review – if we don’t change how the implementation is carried out then we will have major financial issues as well,” Dr Bartone said.

The AMA has raised with the Health Department, and the Health Minister on behalf of the AMA and the membership, the fact that making significant changes, without adequate lead time before commencement, means that neither the health funds, members, the AMA, or patients are able to be part of an informed financial consent process.

This is because while the Department may be ready to implement items on a set date, unless they give the AMA, the health insurers and the profession time to know what the changes are, how they relate to the previous items, and then the ability to adapt their own schedules using the same methodology, a level of confusion will be created.

“You’ll either end up with insurers not ready with their benefit schedules, or insurers pricing the same service under the new items at a different price due to not having the information they need, or both,” Dr Bartone said.

“How do I know this? Well that’s what is happening right now with the 1 November changes.”

AMA members are upset because they don’t know what to charge under particular insurance arrangements or insurers gap schemes, and therefore can’t do an informed financial consent.

One insurer has already lowered one fee for the same service, while other insurers are not yet ready with their benefit schedules. Indeed, most of the major funds have not yet been able to revise their schedules, despite it being two weeks after the items have taken effect.

Without insurance schedules many patients will suffer from increased out of pocket costs, since insurers haven’t been able to help doctors set fees at the no or known gap levels.

Worse, without insurance schedules, insurers may have to revert to default payments which are only 25 per cent of the MBS rate. This could deliver even larger out of pockets and significant short time bill shock, even when patients have high levels of coverage and have undertaken their due diligence.

In the long-term we could see potentially even more variation across benefit schedules and inconsistencies with the intent of both the MBS Review and the Private Health Insurance reforms.

In reviewing the new items, it is clear the MBS Review team has given considered thought to amalgamating, deleting, streamlining and creating new items.

It has also employed a detailed process for generating new fees across the new items, with consideration to fee relativity compared to the old item structure.

“Yet these changes and their intent will not be realised if the methodology for achieving the new structure is not released, and if sufficient time isn’t allowed for the sector to adapt,” Dr Bartone said.

“And it wouldn’t be the insurers fault, nor the profession, nor the patients – we’ll have managed to create yet another problem simply from the implementation process.”

To that end, the AMA has called Government to:

Increase the lead time it provides before new items take effect
Provide the methodology and logic behind the changes, to give the funds and the chance to consider this
Convene a roundtable or consultation with the AMA and the funds about what else is required to protect patients from the potential of out of pocket expenses during the interim period.

“Of course, this becomes all the more important when you consider the Gold Silver Bronze system has clinical definitions that are underpinned by stipulated MBS items,” Dr Bartone said.

[Correspondence] China—USA trade dispute could affect health care

The US Government has already started to impose a 25% tariff on approximately US$50 billion of goods imported from China. The Chinese Government decided to take equivalent retaliatory measures,1 which could have different effects on health care, and thus requires careful consideration.

AMA success on My Health Record

AMA lobbying regarding the My Health Record system has paid off, with the Senate Committee conducting an inquiry into it accepting many of the AMA’s suggestions and the Government moving to legislate some of them.

Health Minister Greg Hunt has announced measures to strengthen safety and privacy measures, and to protect against domestic violence and misuse of the system.

“We have examined the recommendations from the Senate Inquiry, we have listened to concerns raised by a range of groups and My Health Record users,” he said.

The Government is moving amendments to Labor’s original legislation to further strengthen the My Health Record Act.

These include:

Increasing penalties for improper use of a My Health Record.
Strengthening provisions to safeguard against domestic violence. The proposed provisions will ensure that a person cannot be the authorised representative of a minor if they have restricted access to the child, or may pose a risk to the child, or a person associated with the child.
Prohibiting an employer from requesting and using health information in an individual’s My Health Record and protecting employees and potential employees from discriminatory use of their My Health Record. Importantly, employers or insurers cannot simply avoid the prohibition by asking the individuals to share their My Health Record information with them.
No health information or de-identified data to be released to private health insurers, and other types of insurers for research or public health purposes.
The proposed amendments also reinforce that the My Health Record system is a critical piece of national health infrastructure operating for the benefit of all Australians, by removing the ability of the System Operator to delegate functions to organisations other than the Department of Health and the Chief Executive of Medicare.

“Furthermore, the Government will conduct a review looking into whether it is appropriate that parents have default access to the records of 14-17 year-olds,” the Minister said.

The proposed amendments are in addition to those announced in July, which have already passed the Lower House. They include that law enforcement agencies can only access a person’s My Health Record with a warrant or court order and anyone who chooses to cancel a record at any time will have that record permanently deleted.

AMA President Dr Tony Bartone supported the Government’s proposed amendments.

“We initially worked with the Government on a first draft of the Bill to fix the concerns about warrant access, and to allow people to delete their record, which gives them the practical ability to opt-out at any time should they choose,” Dr Bartone said.

“These amendments are now in the Bill.

“We also called for a significant national communications effort to ensure that people know more about the My Health Record.

“In a positive move, the Senate Committee agrees that the legislation should now be passed.

“The AMA also supports the Labor amendments to the Bill. We consulted Labor about their suggestions and agree that they further improve the Bill, and provide stronger protections for our patients.

“We have had successful Committee review of the legislation, improvements made with the input of the Opposition, and consultation to hear and respond to major stakeholder concerns.

“We also welcome the commitment to review the issue of parental access to the records of 14-17 year-olds.

“This and other concerns that arise can be addressed through policy change once the My Health Record Act is passed.”

Shadow Health Minister Catherine King said more needed to be done.

“The Liberals are finally moving to clean up their My Health Record mess – by adopting Labor’s proposed changes – but they still need to act and extend the opt-out period,” she said.

In its final report, the Senate Standing Committee on Community Affairs has acknowledged the AMA’s input to the inquiry and the AMA agrees with many of the Committee recommendations.

Senior executives and doctors from the AMA appeared before Senate hearings on the matter, as well as submitting written recommendations for the way forward with My Health Record.

Of particular concern for the AMA were privacy issues and the sharing of information to third parties from a patient’s My Health Record.

The AMA called for warrant-only access to My Health Record data for law enforcement and other Government purposes; permanent deletion of all data in a patient’s My Health Record if the patient opts out; and stronger provisions to prohibit health insurer and employer access to My Health Record data – this includes a prohibition on health insurers access under the secondary use framework.

CHRIS JOHNSON

[Correspondence] From silos to sustainability: transition through a UHC lens

The transition to higher-income status is a positive step forward for countries, but this transition brings with it the prospect of declining external assistance, both in general and in particular for health. Most health donor agencies rely at least in part on an income threshold to establish eligibility for support. Such a donor transition implies that government is increasingly responsible for the financing of a health programme and its supported interventions.1

New mental health program tells kids to just be you

Former Prime Minister Julia Gillard has launched a new school-based mental health initiative that aims to give teachers the tools to help students manage their mental health.

The Federal Government has invested $98.6 million.

The program Be You will be delivered by Beyond Blue – of which Ms Gillard is the chairwoman – in partnership with Headspace and Early Childhood Australia.

The program will be rolled-out by Beyond Blue in 6,000 schools and 2,000 early learning services in 2019.

Teachers and educators, including those still in training, will have access to free online courses and materials on mental health and suicide prevention.

The program will also be supported by more than 70 frontline staff from Early Childhood Australia and Headspace who will help schools and early learning services around the country implement the program, through online, telephone, and face to face consultations.

“Half of all lifelong mental health issues emerge before the age of 14,” Ms Gillard said.
“We have the opportunity to grow Australia’s most mentally healthy generation. It’s a big ambition and to achieve it we are asking everybody to get involved.”
Health Minister Greg Hunt said the program will provide Australian teachers with the skills and resources to be able to teach students how to manage their mental health and wellbeing, build resilience, and support the mental wellbeing of other students.
“It will ensure that students have all the support required for healthy social and emotional development,” Mr Hunt said.
“Be You will teach educators to identify any students who may be experiencing mental health difficulties, and to work with the families and local services to get the right help early on. It will also help educators look after their own mental health.”
Education Minister Dan Tehan added that Be You builds on the strengths of current school-based mental health programs, and complements the recently launched Australian Student Wellbeing Framework.
“I encourage all Australian schools and early learning providers to engage with beyondblue and Be You to support the mental health and wellbeing of our students,” Mr Tehan said.

“As half of all mental health disorders in Australia emerge before the age of 14, schools and early learning services in Australia represent one of the best opportunities for mental health issues to be detected early and managed.

“Schools also play a vital role in prevention by helping our children and young people learn the skills they need to look after their own mental health and wellbeing.”

The Government is also providing $2.36 million over four years to the University of Queensland to evaluate the program. This will assess the effectiveness and cost-effectiveness of the program, and identify opportunities to strengthen or improve it.

[Perspectives] Shekhar Saxena: making mental health a development priority

Softly spoken Shekhar Saxena is a giant in the world of mental health. As Director of the Department of Mental Health and Substance Abuse at WHO for the past 8 years—a role from which he stepped down in June, 2018—he has seen a shift in attitudes to mental health. “The seriousness with which mental health is taken at a political and societal level has certainly increased”, says Saxena. However, there is a chasm between commitments made by governments and what happens on the front line, he says: “When it comes to mental health, all countries are developing countries.

Unconscionable to leave children on Nauru

The AMA’s paediatric representative Dr Paul Bauert has delivered a blunt message to the Federal Government – get the kids off Nauru.

While addressing protest rallies, speaking to the media and handing over a petition to parliamentarians that has been signed by thousands of doctors, Dr Bauert repeated his insistence that it is unconscionable to leave children suffering on the Pacific island.

Dr Bauert has treated asylum seeker patients on Nauru.

“This is the only situation I’ve come across where it is deliberate government policy which is causing the pain and suffering of these children,” he told reporters in Canberra in October.

“Many are damaged already, but we don’t want this damage to be permanent. They need to be assessed and treated as a matter of urgency.

“It’s a miracle we haven’t had a death already.

“I have reviewed many cases of these children myself. It is simply unconscionable that we are keeping these children and their families in a situation which we know is a critical threat to their health and wellbeing.

“The situation for children on Nauru is a humanitarian emergency requiring urgent intervention and removal of all these children and their families to medical treatment in Australia.”

On October 15, Dr Bauert and Sydney-based GP Dr Sara Townend delivered to Prime Minister Scott Morrison an open letter signed by almost 6,000 Australian doctors, urging children in detention on Nauru be transferred to Australia for medical and psychological treatment.

The number of signatories amounts to about five per cent of all registered doctors in Australia.

CHRIS JOHNSON

Father of Medicare dies

Professor John Deeble AO, the man universally known as the “Father of Medicare” has died at the age of 87.

Most recently, Emeritus Fellow of the Australian National University, Sax Medallist, and Patron of the Deeble Institute for Health Policy Research – the research arm of the Australian Healthcare and Hospitals Association, of which Professor Deeble was a life member – it was his much earlier work that gave him nickname.

In 1968, together with Dr Dick Scotton, Professor Deeble co-authored the original proposals for universal health insurance in Australia.

He subsequently became the architect of the reintroduction of universal healthcare in Australia – Medicare – in 1984.

His other appointments included First Assistant Secretary in the Commonwealth Department of Health, Founding Director of the Australian Institute of Health and Welfare, and from 1989 to 2005, Senior Fellow in Epidemiology and Adjunct Professor in Economics at the National Centre for Epidemiology and Population Health at the ANU.

He was Special Adviser to the ministers for health in the Whitlam and Hawke governments, chairman of the planning committees for both Medibank and Medicare, and a commissioner of the Health Insurance Commission for 16 years.

In addition, Professor Deeble was a World Bank Consultant on healthcare financing in Hungary, Turkey and Indonesia, and for more than10 years to 2005, an adviser to the government of South Africa.