Lives unaffected by cystic fibrosis

Australian Government Department of Health - Budget 2020-21   >

Last nights’ Budget saw the injection of $2.8 billion into the PBS for the New Medicines Funding Guarantee. This is great news for the CF community and drugs like Trikafta currently under review. The Government has promised to fund all medicines recommended by the PBAC however this Funding Guarantee means that there is more money to go around. We can feel confident that Trikafta will be available soon after ‘recommendation’. Let’s put some pressure on the negotiators to act fast and get Trikafta into the hands of people who need it. 

NDIS ADVOCACY FOR PEOPLE WITH CYSTIC FIBROSIS

CFA's Advocacy National Disability Insurance Scheme - October 2020 >

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HTA PARLIAMENATARY INQUIRY

TIME TO MOVE A MOUNTAIN

Cystic Fibrosis Australia (CFA) welcomes the Parliamentary Inquiry into new drugs and novel technologies. The Inquiry by the House of Representatives Standing Committee on Health, Aged Care and Sport will include a focus on access to treatment for rare diseases and conditions where there is high and unmet need. In other words, trying to eliminate the mountainous obstacles we so often see in front of us.

The Inquiry will be chaired by Trent Zimmermann MP and Dr Mike Freelander MP and CFA has written to both asking to be included in the HTA review. All CF drugs go through the HTA process and we would all like it to be a swifter process.  Let’s have our say and try and move one enormous mountain out of the way because quite simply, it’s time to level the playing field.

We believe that the Cystic Fibrosis (CF) Community has a wealth of knowledge and real-life experiences that should be shared. They will help ensure that the Inquiry’s outcomes benefit the communities in need.

Consumer submissions addressing the Terms of Reference (ToR) must be submitted by Tuesday, 13 October 2020 and CFA has developed key points to assist you in the process.

We have added suggested consumer comments for each ToR point below and we encourage the CF Community to use this information as a guide. Your own words will be so much more powerful.

The Inquiry’s ToR points are designed to ensure Australia is well positioned to access new drugs and technologies in a timely manner in the future.

Your personal stories and heartfelt experience are incredibly important. These will really make the difference and have a considerable impact. With that in mind we encourage you to share this information if you feel comfortable about doing so.

It is also important when looking at the ToR Topics to consider what has not been included. CFA sees this as a vital component of the ToR review, and we need to be sure the Inquiry is robust and inclusive. You do not have to address all the ToR topics. Just focus on those that have impacted your life.

As we live in a digital world the House of Representatives Inquiry will accept submissions online in the form of a letter, a short document, a more substantial proposal or a video. More information is available here Preparing a submission to an inquiry.

ToR Topics

1.   The range of new drugs and emerging novel medical technologies in development in Australia and globally, including areas of innovation where there is an interface between drugs and novel therapies.

Current Issues:
a)  Speed to market in Australia when drugs are available overseas
b)  Lack of transparency by pharmaceutical companies regarding their submissions
c)  The HTA process and timeline relating to a specific drug or treatment should be agreed to by Government departments, Regulators and Sponsor and shared with consumers to manage expectations
d)  Consumer consultation should be held at TGA stage and prior
e)  Consumer co-design should be rewarded.

2.   Incentives to research, develop and commercialise new drugs and novel medical technologies for conditions where there is an unmet need, in particular orphan, personalised drugs and off-patent that could be repurposed and used to treat new conditions.

Current Issues:
a)  Innovative trial should be encouraged and incentivised – N of 1, adaptive, organoids and basket trials are all available in Australia – approval pathways for rare diseases should be established
b)  Double blind placebo clinical trials are no longer the only option or ‘best practice’ method especially in rare diseases
c)  Treatments should be personalised, and precision medicine embraced and encouraged
d)  Research into ‘evidence gaps’ for rare diseases should be funded
e)  Repurposing of existing treatments should be incentivised and acceleration pathways established.

3.  Measures that could make Australia a more attractive location for clinical trials for new drugs and novel medical technologies

Current Issues:
a)  Support for disease specific clinical trial networks
b)  Incentivise international clinical trials to include Australia by providing benefits such as an expedited HTA approval process when Australian data is available
c)  National infrastructure for clinical trials
d)  Consumer co-design
e)  Streamlined national ethics approval process
f)  Every Australian should have access to clinical trials
g)  Not for Profit Consumer bodies should be supported to aid clinical trial participant recruitment
h)  Embrace innovative clinical trials that include rural and remote communities.

4.  Without compromising the assessment of safety, quality, efficacy or cost-effectiveness, whether the approval process for new drugs and novel medical technologies, could be made more efficient, including through greater use of international approval processes, greater alignment of registration and reimbursement processes or post market assessment.

Current Issues:
a)  Open collaboration with FDA and EMA
b)  Clinical registries should be accredited and then part funded by Government for use in the drug evaluation process
c)  International reimbursement contact negotiations to run while consumers benefit from treatments. This would also provide valuable data through post marketing surveillance
e)  Set time limits for commercial (pricing) negotiations
f)  New clinical trial techniques should be valued in the reimbursement process
g)  Add consumers to the HTA process from the beginning
h)  Require consumer comments to Pharma TGA and PBAC submissions
i)  Provide support, educate and update consumers throughout the process
j)  Incentivise compassionate access for a great number of people who are critically ill.

I realise this is a great deal of information to digest so as always CFA is here to help. If you have any questions, please email [email protected] and we will get back to you with a solution.

You can also contact the Government Inquiry Secretariat on 02 6277 4145 or [email protected] . There are three ways to deliver your submission…

1.      create or log in to My Parliament and set up an account
2.      email [email protected]
3.      email Nicki at CFA and we will submit for you – her email is [email protected].

Thank you to everyone who decides to support CFA’s Health Technology Assessment advocacy. It will help eliminate the obstacles and pave the way for faster access to drugs in the future for people with CF. This is a mountain well worth moving because a level playing field is everyone’s right, and in every society’s best interests.

 


About CFA's Advocay plan and strategy

Cystic Fibrosis Australia has an advocacy strategy designed to ensure …

  • cystic fibrosis is kept top of mind with our politicians and decision makers
  • researchers are professionally drawn to the CF field and become involved
  • funds are forthcoming from the corporate and community sectors for a worthwhile cause and
  • the broad Australian community understands the inequity that surrounds CF.

CFA is a robust and authoritative voice for people with cystic fibrosis and their families, carers, partners and support networks. We are often speaking for the powerless to the powerful.

Government, business and the health and community sectors need to understand and appreciate the challenges people with CF face. This includes key issues such as drug affordability and availability, infection control, gene testing and access to the best medical and allied health services.

Over the past 24 months CFA has driven the following advocacy campaigns.

Ongoing campaigns

Pills to the Pollies

Lolly Jar awareness - Send us an email to [email protected] with your name, contact details and location, local newspaper name, favourite radio station announcer, local MP or Senator name and electorate area and we will send them all a ‘lolly jar’ full of sweets.

We will ask these community leaders to guess how many lollies are in the jar. The answer will be ‘a member of your community knows as they take this number of tablets every day’.

Make your voice heard

Community Videos ‘Why I need Orkambi’

CFA are always bringing videos and letters from the community to present to The Government. If you have a message you want to share and are confident to turn it into a video, please send it to [email protected]

Upcoming campaigns

Siblings Events

The impact of living with CF reaches far greater than just the person with CF. To highlight this, Cystic Fibrosis Australia hosted a Siblings afternoon tea in Parliament House, Canberra. 

Outcomes from this special event coming soon.

NFP forum

The process of having drugs approved in Australia is a long and arduous task. With this being a common issue amongst those NFPs working in the treatment and accessibility space.

In October, we invite key representitives from various NFPs around Australia to share their experiences and work togther to devise an easier and more efficient process. 

For more information on this event, please contact [email protected]

Indigenous and Ethnicity Report

Mental Health Roadshow

The Roadshow took place in 2018, for more>

Previous campaigns

PBAC Consumer Hearing

Oh Oh Oh Orkambi

Canberra Protest

Bronchitol and Pulmozyme

Department of Health and Minister’s Office Stakeholder Meeting

Make an ‘O’ for Orkambi

Christmas Card campaign to Minister for Health and Vertex