Why drug reimbursement is about science, politics and art
Written by Athena Kolivos

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.