Content produced by Clinical Research Corporation (member of The IQ Group Global) in association with Australian Doctor Group

Drug reimbursement in Australia has become particularly challenging in recent years as an increasing number of high-cost yet innovative drug therapies face both health technology assessment as well as PBS budget hurdles.

Even when marketing approval is expedited for a particular biotechnology advance with high potential for patient outcomes, the road to reimbursement and patient access can be long and complicated with the intense focus on therapy value (improved outcomes relative to cost) and PBS (payer) affordability.

So what can we do to support reimbursement and minimise the time it takes for PBS listing? At the Clinical Research Corporation, which specialises in medical and government affairs for the healthcare industry, here are three crucial factors we consider:

1) The science – have the evidence right first time to support clinical and economic claims

The clinical trial program ideally should include evidence needed to support both regulatory and reimbursement outcomes.

Yet this rarely happens with the focus instead on registration, i.e. approval to sell based on efficacy, safety and quality. Meanwhile, reimbursement (comparative effectiveness and safety, therapy value, budget impact) usually makes do with whatever evidence exists to show therapy value and support the ‘buy’.

However, if the data is insufficient to show therapy value, it will be difficult to support reimbursement. This means a therapy without a buyer and hence out of reach to patients who cannot afford to pay.

The more the evidence is lacking to support reimbursement, the greater the reliance on a more complex economic model built on alternative inputs and assumptions. This, in turn, increases uncertainty for the PBAC and government (payer) about the new drug’s effectiveness and value.

It’s crucial that a submission gets it right first time with studies containing clinical efficacy and other endpoints (e.g. quality of life, healthcare resource utilisation) and an economic model to show cost-effectiveness or therapy value in the intended patient population.

2) The politics – understand the healthcare environment

Reimbursement decisions are not made in a vacuum. While an evidence-based submission is pivotal to the funding decision, it is not all about the data.

The politics of reimbursement is very much driven by clinical demand and the PBAC’s deliberations are only one step in a process involving multiple stakeholders and activities.

Even a technically sound submission showing clinical need, comparative effectiveness, safety and cost effectiveness can be rejected or delayed because of concerns about the target patient population size, how the company ‘positions’ the therapy, price, PBS budget affordability or other factors.

While submissions must be evidence based – ideally with data from head-to-head randomised studies with the appropriate comparator(s) to help reduce uncertainties comparing the new drug and the alternative(s) – reimbursement outcomes are based on a range of factors.

In addition to the PBAC’s consideration of the submission, there is policy, debate, varying expert opinions and points of view within government and the community (e.g. HCP and patient groups) that will affect the decision.

Any market access strategy, ideally planned years in advance of the reimbursement submission and working with the company’s global teams, should include an analysis of the funding environmental and all stakeholders.

3) The art – communicate value to government and community stakeholders

While reimbursement is all about demonstrating the therapy’s comparative effect and value, communicating this and other key information tailored to all relevant stakeholders is paramount. This includes information about the disease and its management, patient population, clinical need, clinical and economic evidence, health outcomes, budget impact and any other considerations.

Translating complex technical information for those within government and the community who will advocate or lobby for PBS listing should be part of the plan.

As new high-cost drug therapy advances continue to present to a healthcare system that demands patient outcomes, value to different stakeholders and PBS budget certainty, market access planning and implementation is more important than ever and should be considered up front alongside the registration strategy and clinical trial program.

Only then can the science, politics and art of market access success be truly realised.




Recently, there has been an increasing focus on patient centric approaches to healthcare by various stakeholders in the quest to improve outcomes and value for patients and their support networks. These parties include regulators, payers, healthcare providers, health technology assessors, as well as device and pharmaceutical companies. They seek to understand patient experiences throughout the course of a disease in addressing the needs and improving the outcomes that matter most to patients.

From regulatory agencies such as the FDA, EMA and TGA, to payers seeking patient reported outcomes evidence, it seems patient centricity is on everyone’s agenda.1,2 Indeed, the TGA in March 2018 held a consumers’ workshop to discuss introducing patient cards and consumer device information for all permanently implantable medical devices and plans ongoing consumer engagement for this initiative.2

However, it is important to balance patient centricity at the ‘macro’ patient population level with personalised care for individual patients.



Patient centricity has been defined as “keeping benefits to patients a primary concern” via “integrated measures for listening to and partnering with patients, and placing patient well-being at the core of all initiatives. In essence, it represents a holistic approach to disease management.”1,3

This definition suggests focus at a population level as opposed to the individual patient. Thus, it has been argued a fundamental shift by medical device and pharmaceutical companies is needed to engage or collaborate with, and achieve value for, patients and their families/carers and so move beyond a brand/product focus.1,3 This means having an innovative outlook whereby companies involve patients throughout the product development lifecycle in deciding the best course of action.1

Some proposed changes include1,3:

  • Shifting the cultural company mindset from a product to patient focus so patient centred outcomes become the core of business strategy. This means capturing patient perspectives and establishing associated performance indicators based on patient input.
  • Restructuring staff roles, responsibilities and how their behaviours are rewarded.
  • Building trust with patients, healthcare providers and the public, e.g. via collaborative initiatives of mutual interest.
  • Gaining insights from other organisations within the healthcare sector experienced with patient interaction or who have established frameworks for patient engagement (e.g. NICE, EMA, TGA, PBAC and other regulatory/health assessment technology bodies).
  • Patient engagement in R & D and commercialisation activities.
  • Standardising patient reported outcomes, i.e. outcomes important and relevant to patients, which should properly reflect patient experience of the disease and its management.

However, there are some challenges to achieving patient centricity, among which is that all healthcare stakeholders need to be aligned and working together as this is not a feat for device or pharma companies alone.1

Does adopting a patient value focus have to be so grandiose? In the age of digital technologies and medical devices that help drive patient centric outcomes, much can be gained by focusing on the individual patient, their ecosystem and needs, rather than a ‘one size fits all’ approach. This does not require seismic shifts in company structures and processes. Instead, it means personalising the management of a patient’s condition such that their individual preferences, experiences and personal ecosystem (healthcare networks, carers, providers) are considered in tailoring a solution or program for them.

Diabetes is one condition gaining momentum in the area of personalised medicine with the emergence of alternative monitoring technologies to blood glucose testing. These newer devices are helping the individual patient to ‘see’ their own glucose patterns and understand and manage the likely causes of fluctuating levels. Future technologies such as saliva based, pain-free non-invasive methods offering patient ease of use and convenience, combined with accurate/reliable results, can only further improve a patient’s health literacy so he/she takes an interest in, and gains control of, their glucose levels. It is important that the patient data from these technologies be used to educate, encourage and empower individuals to make appropriate lifestyle/behavioural changes that will lead to improved outcomes in diabetes. Now such personalised (tailored) disease management is true patient centricity.



  1. Du Plessis D et al. Patient Centricity and Pharmaceutical Companies: Is it Feasible? Therapeutic Innovation & Regulatory Science 2017;51(4): DOI: 10.1177/2168479017696268
  2. https://www.tga.gov.au/tga-presentation-health-consumers-workshop-medical-devices-1-march-2018
  3. Beveridge D. Medical Devices: A Practical Guide to Maintaining Patient Centricity. August 2017 https://www.alexandergroup.com/blog/medical-device/medical-devices-a-practical-guide-to-maintaining-patient-centricity/




In recent years there has been a rise in the availability of precision medicines, many of which require use of companion diagnostic tests to determine which patients will respond to the therapy.1 Innovative precision medicines can potentially cure previously untreatable diseases, however, they are expensive and represent a challenge for payers deciding which therapies to fund. Companion diagnostics may help prove the cost effectiveness of therapies along with providing a number of other benefits.

Tackling healthcare costs

Increasing healthcare costs globally have led to an emphasis on demonstrating both the clinical and economic value of therapies, which requires forward planning from companies seeking reimbursement. Recent European analysis has shown that companion diagnostics represent a small segment of in vitro diagnostics spending and less then 1% of total healthcare expenditure.2 Yet these specialised diagnostic tests represent a way to minimise inefficient use of healthcare resources by identifying sub-populations of patients that are most likely to achieve positive outcomes from a therapy.2

Increasing need for personalised therapies

Oncology is a therapeutic area where treatments have become increasingly personalised therefore creating a growing need for companion diagnostics.3 Traditionally, treatment options (including surgery, radiotherapy and chemotherapy) may have been limited resulting in most patients receiving the same or similar treatment based on the type and stage of their cancer.4 This approach achieved varying degrees of success among patients with some being cured of their disease, while others were exposed to harsh side effects with little or no improvement to their health.

In recent years, advancements in cancer biology research have revealed high levels of variation in the genetic mutations that lead to the development of cancer, which could account for the variation in patient response to a therapy. Recently, many drugs, particularly those to treat cancer, have been developed to target specific aspects of the disease. For example, a drug therapy (such as a monoclonal antibody) may bind to a specific biomarker on cancer cells or turn on an aspect of the patient’s immune response. By developing companion diagnostics to identify these specific biomarkers in a patient’s blood or tissue biopsy, the patients most likely to respond to the targeted therapy can be identified.

Benefits of improved data collection

Companion diagnostics can add value throughout all stages of clinical development of a therapy by providing a mechanism to collect useful data. During the pre-clinical stage, a diagnostic test could be used to assess the likely success of a proposed therapy, thus reducing the chance of incurring substantial costs at the later stages of development.5 As a potential therapy advances through the clinical trial stages, use of a companion diagnostic could facilitate selection of those patients most likely to respond favourably to the drug. By identifying a well-defined patient population, companies could enrol fewer patients and potentially complete clinical studies in a shorter timeframe which could result in reduced costs.3,6

Inclusion of a companion diagnostic has been attributed with taking a therapy from second or third line treatment in the general population to a first line therapy in a sub-population which can lead to increased revenues.3 Personalised medicines may also result in less adverse reactions which may contribute to healthcare savings.4 This precision approach allows companies to collect impactful data that may provide a more attractive package to payers by treating a select patient segment with greater potential to achieve the desired health outcomes.

CRC’s team has a wealth of experience to assist our clients in their Medical Affairs needs throughout the drug development lifecycle. 


  1. Brooks E. A. & Bergstrom F. Companion Diagnostics: Important Health Economics and Reimbursement Considerations. Decision Driver Analytics. Available at: http://www.decision-driver.com/companion-diagnostics-white-paper.aspx
  2. Wurcel V. et al. The value of Companion Diagnostics: Overcoming Access Barriers to Transform Personalised Health Care into an Affordable Reality in Europe. Public Health Genomics Journal.
  3. Agarwal A. et al. The current and future state of companion diagnostics. Pharmacogenomics and Personalised Medicine Journal. Available at: https://www.ncbi.nlm.nih.gov/pubmed/25897259
  4. ADC Review. Companion Diagnostics: Paving the way for personalised medicine. Available at: https://adcreview.com/news/companion-diagnostics-and-personalized-medicine/
  5. Leamon C. 2013. Companion Diagnostics: Improving development success and patient care. Available at: https://www.genengnews.com/gen-articles/companion-diagnostics-improving-development-success-and-patient-care/4957
  6. Danner S. et al. Capitalizing on precision medicine: How pharmaceutical firms can shape the future of healthcare. Available at: https://www.strategyand.pwc.com/reports/capitalizing-precision-medicine





We have previously reported this year will be big for biosimilars with several therapies on the horizon to challenge some of the biggest biologic brands. 1 The Australian government has put mechanisms in place to increase awareness of and drive uptake of biosimilars. 2 However, government backing and a more affordable price for these therapies does not guarantee their commercial success. Here we discuss how MSLs can contribute to their company’s strategy for maximising uptake of biosimilars.

Communicating biosimilar data

The science behind both reference brands and biosimilars is complex and healthcare professionals (HCPs) may be more comfortable prescribing and dispensing established brands which have more evidence to support their use in a given patient population. Biosimilar clinical studies vary from traditional studies because they typically include analytical data showing how the structure of the biosimilar compares to the reference drug and how that structure influences the therapy’s function. HCPs may not be familiar with terminology such as bioequivalence and extrapolation across indications, which are commonly used to discuss biosimilars. 3

Therefore, a key activity for MSLs is to educate HCPs about biosimilars along with sharing product specific information. This should help HCPs to understand and embrace the importance of analytical data over traditional clinical study data when discussing biosimilar development. 4 MSLs can be vigilant for potential gaps in HCPs’ knowledge and seek to address their concerns with the aim of helping them become more comfortable prescribing biosimilars to their patients.

Supporting the switch

Controversy remains around the safety of multiple switches between reference biologics and biosimilars. HCPs may have concerns about changing a successfully established brand for a particular patient and also be more wary of the potential for adverse reactions among patients receiving biosimilars. It is important for MSLs to become aware of these concerns and the potential medico-legal implications from a HCP perspective. This issue is relevant to both clinicians and pharmacists. In Australia, pharmacists have the authority via ‘a-flagging’ to substitute a biosimilar product at the point of dispensing provided the prescriber has not indicated that brand substitution is not permitted.

HCPs may not be convinced of the evidence around new biosimilars, which can result in them waiting to assess their peers’ experience before prescribing. 5 MSLs could address this reluctance in a number of ways for example, by engaging with key opinion leaders (KOLs) from abroad where biosimilars are more established to provide peer to peer education. MSLs could also assist HCPs to become involved with ‘switching studies’ for biosimilars listed on the PBS to provide the opportunity to capture real-world evidence for the use of biosimilars in a specific patient group.

Embracing patient centricity


Patients should also be considered as key stakeholders. When gathering intelligence about the competitor landscape, MSLs should seek information about patient support programs and other benefits that competitor companies offer. They can gain these insights not only from HCPs but by engaging directly with patient support groups and patient advocates. By doing this they can become knowledgeable about patients’ needs and assist in establishing improved, if not similar, services to ensure the biosimilar enters the market with maximum opportunity for commercial success.

CRC’s experienced team can provide strategic and forward-thinking Medical Affairs solutions to provide our clients with biosimilars maximum competitive advantage.


  1. O’Reilly N. 2018. Growth of Biosimilars in Australia. Available at: https://crcaustralia.com/media-releases/growth-of-biosimilars-in-australia/
  2. PBS Website. 2017. Biosimilar awareness initiative and biosimilar uptake drivers. Available at: http://www.pbs.gov.au/info/general/biosimilars
  3. Turner M. and Franzen M. 2017. Biosimilars: An Evolving Commercial Model. Available at: https://www.lifescienceleader.com/doc/biosimilars-an-evolving-commercial-model-0001
  4. Welch A. R. 2016. What Biosimilars Need to Succeed in Clinical Practice. Available at: https://www.biosimilardevelopment.com/doc/what-biosimilars-need-to-succeed-in-clinical-practice-0001
  5. Turner M. and Franzen M. 2017. Biosimilars: Differentiating and Surviving the Cost of Entry. Available at: https://www.lifescienceleader.com/doc/biosimilars-differentiating-and-surviving-the-cost-of-entry-0001




Patient reported outcomes and experiences can be an effective and practical means of capturing necessary real-world evidence of the impact of an intervention not available elsewhere. The following case outlines how Australian patient feedback filled an absence of real world evidence required by decision makers in considering the potential reimbursement of a consumable medical device for a specific subpopulation.


A client of CRC had been seeking reimbursement for several years for a consumable medical device under Australian state governments’ medical aids and equipment schemes. The device is for a well-defined post-surgical subgroup in the community and supported by a longstanding body of clinical evidence, as well as reports of improved quality of life (QoL).

A key benefit known anecdotally for years, yet not assessed in a dedicated study is the reduction of hospital days due to complications from the surgery. In addition, the QoL benefits of the device had never be shown among Australians, which was highly relevant to demonstrating need by patients in the local community setting. The absence of both Australian economic and QoL information was a major barrier to progressing reimbursement.

Exacerbating the situation was a silent patient population without a national advocacy voice nor representative bodies in most Australian states. The issue was also too small for national and state healthcare professional groups to address.


It was clear a compelling economic argument based on local evidence was needed to drive a funding decision in the various states.

A VBHC approach would have been ideal to generate real world ‘pay for performance’ data in showing the value of the medical device for this subpopulation. Yet VBHC was not logistically feasible, given the time, budget, resources and stakeholders required. Our client and the affected subpopulation needed a timely practical solution to gather real world evidence that could be shared with state health departments.

Going directly to the source, i.e. the patients themselves, was the only pragmatic solution. Thus, CRC proposed, prepared and implemented a patient survey conducted online and manually to collect the necessary economic and other data that was ‘fit for purpose’. The short simple survey captured patient experiences with and without use of the medical device to understand, in particular, their consumption of healthcare resources post-surgery and QoL.

The survey generated double the number of responders anticipated. Importantly, it clearly showed fewer hospital days due to post-surgery complications for individuals using the device compared with non-users, as well as better quality of life.


Interim survey results have been shared so far with two state health departments who are interested because for the first time they have a reference point from which to verify key data such as hospital days.

A further (unintentional) benefit of the survey is that it has invigorated a patient group previously silent who had given up hope of the possibility of the device ever being funded.

Survey results continue to be collected for which the plan is to share final results with state governments in progressing a funding decision.

CRC provides Medical Affairs solutions to the Pharmaceutical industry throughout the Drug Development Life Cycle. Our objective is to maximise the value of therapeutic compounds from pre-launch through to commercialisation and beyond.

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