How medicines are funded in Australia and why it takes so long 

For many people living with cystic fibrosis, medications are not optional. They are what keep lungs functioning, infections controlled, nutrition stable and people alive. 

But have you ever wondered why some medicines are available quickly overseas, yet take years to reach Australians? Or why a medicine can be approved by doctors but still not funded? Or what people mean when they talk about “PBS listings”, “Health Technology Assessment” or “Most Favoured Nation pricing”? 

The process of getting medicines from the laboratory to your local pharmacy is long, complex and often frustrating. For rare diseases like cystic fibrosis, delays can have lifelong consequences. 

Here’s a simple breakdown of how the system works in Australia, and where things can go wrong. 

 

Step 1: Research and Development 

Before a medicine ever reaches a patient, it can take more than 10 years of scientific research, clinical trials and regulatory testing. 

For cystic fibrosis, this has included: 

  • antibiotics  
  • pancreatic enzymes  
  • insulin therapies for CFRD  
  • nebulised medications  
  • and CFTR modulators like Trikafta and Alyftrek.  

Developing medicines is expensive and risky. Pharmaceutical companies may spend billions of dollars before a treatment is ever approved. For rare diseases, the challenge is even greater because patient populations are smaller, making it harder to recover costs. 

Step 2: TGA approval: Is the medicine safe? 

In Australia, medicines first go through the Therapeutic Goods Administration (TGA). 

The TGA looks at: 

  • safety  
  • quality  
  • effectiveness  
  • side effects  
  • manufacturing standards.  

If the medicine passes this process, it can legally be prescribed in Australia. But this does not mean it is affordable. A medicine may cost: 

  • tens of thousands,  
  • hundreds of thousands,  
  • or even more than a million dollars per year.  

Without government subsidy, many Australians simply could not access these treatments. That is where the PBS comes in. 

Step 3: PBAC: Should taxpayers fund It? 

The next step is assessment by the Pharmaceutical Benefits Advisory Committee (PBAC). 

PBAC advises the Federal Government whether a medicine should be listed on the Pharmaceutical Benefits Scheme (PBS). This process is known as Health Technology Assessment (HTA). PBAC doesn’t just ask: “Does this medicine work?” It also asks: 

  • Is it better than existing treatments?  
  • How much extra benefit does it provide?  
  • Is it cost effective?  
  • How many people need it?  
  • What will it cost taxpayers?  

This is where things often become difficult for rare diseases. 

The challenge for rare diseases like cystic fibrosis 

Traditional HTA systems were largely designed around common conditions affecting large populations. But rare diseases don’t fit neatly into those models. 

In cystic fibrosis: 

  • patient numbers are smaller,  
  • evidence can be harder to collect,  
  • long-term benefits may take years to fully understand,  
  • and quality-of-life improvements are sometimes difficult to measure in spreadsheets.  

How do you place a dollar value on: 

  • fewer hospitalisations?  
  • being able to work?  
  • parents sleeping through the night?  
  • improved mental health?  
  • or simply living longer?  

For many in the cystic fibrosis community, these benefits are obvious. But proving them within government funding frameworks can take years. 

The role of advocacy 

This is where advocacy becomes critical. Patient organisations like Cystic Fibrosis Queensland, clinicians, researchers and people with lived experience often work together to: 

  • write submissions,  
  • provide consumer testimony,  
  • meet with government,  
  • speak to the media,  
  • collect data,  
  • and explain the real-world impact of delays.  

Advocacy helps decision-makers understand that medicines are not abstract numbers on a spreadsheet, they are life-changing treatments affecting real people and families. 

Many PBS listings for cystic fibrosis therapies have only happened because the community fought hard for them. 

Step 4: Government negotiation 

Even after PBAC recommends a medicine, the process may still not be over. The Australian Government then negotiates pricing with the pharmaceutical company. 

This can take months or longer. The average time in Australia falls between 15 months to well over two years.  

Governments understandably want value for taxpayers. Pharmaceutical companies want sustainable pricing that supports future research and development. Sometimes negotiations stall. And during these delays, patients wait. For progressive diseases like cystic fibrosis, time matters. Every delayed month can mean: 

  • declining lung function,  
  • more infections,  
  • hospital admissions,  
  • irreversible organ damage,  
  • and worsening mental health.  

 

Step 5: PBS Listing and the Pharmacy 

Once an agreement is reached, the medicine is listed on the PBS. This means Australians can access it at a heavily subsidised cost through their local pharmacy or hospital. Instead of paying tens or hundreds of thousands of dollars each year, patients may only pay: 

  • a general co-payment,  
  • or a concessional co-payment.  

For many families, PBS listing is the difference between access and no access. Australia’s PBS is internationally respected because it protects people from catastrophic healthcare costs. But the system is under growing pressure globally. 

What is “Most Favoured Nation” pricing? 

You may have heard discussion internationally about “Most Favoured Nation” (MFN) pricing policies. In simple terms, MFN policies attempt to ensure a country pays the lowest medicine price available anywhere in the world. At first glance, this sounds reasonable: Why should one country pay more than another? But the reality is far more complicated. 

Why could MFN be a problem for Australia? 

Australia already negotiates aggressively on medicine prices through the PBS. If countries like the United States move toward MFN-style pricing linked to lower-priced countries like Australia, pharmaceutical companies may respond by: 

  • delaying launches in smaller countries,  
  • avoiding lower-price agreements,  
  • or limiting supply.  

Why? 

Because if Australia negotiates a very low price, that price could potentially influence pricing in much larger and more profitable markets overseas. This creates concern that Australia could become: 

  • a lower priority market,  
  • or experience longer delays in accessing new medicines.  

For rare diseases like cystic fibrosis, where treatment populations are already small, this risk becomes even more significant. 

Why delays matter in cystic fibrosis 

Cystic fibrosis is a progressive disease. While modulators have transformed outcomes for many people, they are not cures. Not everyone can access modulators. Not everyone responds equally. And many people still live with: 

  • lung damage,  
  • CFRD,  
  • gastrointestinal complications,  
  • infections,  
  • liver disease,  
  • transplant challenges,  
  • and the daily burden of treatment adherence.  

Delays in access can permanently impact health outcomes. A child who misses years of therapy may never regain lost lung function later. That is why timely, equitable access matters. 

What needs to improve? 

Australia has one of the best medicine subsidy systems in the world. But there is room for improvement. Many patient organisations are calling for: 

  • faster HTA pathways for rare diseases,  
  • greater transparency in decision-making,  
  • improved use of patient experience data,  
  • more flexible assessment models,  
  • earlier access pathways,  
  • and better recognition of long-term societal benefits.  

There is also growing recognition that healthcare should not only measure survival, but quality of life, participation and wellbeing. From advocacy to approval to the pharmacy shelf, medicine funding in Australia is a long and highly complex process. Behind every PBS listing are: 

  • researchers,  
  • clinicians,  
  • policymakers,  
  • pharmaceutical companies,  
  • patient advocates,  
  • and people living with disease who share their stories to drive change.  

For the cystic fibrosis community, access to medicines is not just a policy issue. It is deeply personal. Because while systems debate timelines and budgets, people with cystic fibrosis are living with the reality of a disease that does not wait.