Evidence matters to payers

Since 2000, the payer has emerged as the critical audience for pharmaceutical and medical device companies to educate and communicate to about their proposition.

But who are payers? What evidence do they need to see to assess a product? And, critically, what is the best way to communicate with them effectively?

A little understanding
In simple terms, a payer is any person or body who is involved in making decisions to pay for a healthcare intervention. This can be a national body conducting health technology assessments (HTAs) and issuing legally-binding advice to healthcare providers.

It can also be a regional health authority which, as a budget holder, must decide which drugs to fund to optimise health outcomes. At hospital level, a pharmacist may be the gatekeeper to which medicines are used by local physicians.

So the payer can have many faces and therefore many evidence requirements. Understanding which evidence influences payer decision-making should be an important step for those involved in planning the research programme, or even making 'go/no go' decisions for early-phase products.

One way to understand what evidence payers need to see is simply to collect and examine the decisions published by payer bodies worldwide. From this, it is often possible to understand how decisions have been made and what evidence was required.

For instance, was cost-effectiveness data requested and what thresholds were set? How many randomised studies were needed to influence positive endorsement across different disease areas? Are competitors using initiatives such as patient support programmes to convince payers to endorse their products?

Positive influence
Answers to questions such as these can allow strategic planners to positively influence the continued clinical development programme at an early stage and optimise chances of successful market access later.

Clinical data remains pivotal, of course, whether it is the payer or prescriber who decides.

While regulatory authorities need to ensure a new medicine is safe and efficacious, the payer thinks comparatively: will this new intervention work as well as, if not better than, existing therapies?

To prepare for this, companies need to ensure they are entering the market with a comprehensive picture of their evidence. The best-practice approach involves conducting a systematic literature review to collate and summarise all the relevant data. At an early stage, development of this evidence platform should allow medical teams to plan the design of phase III trials adequately to capture the 'right' outcomes and patient populations, as well as feed into return-on-investment analyses.

Meta-analysis
Homogeneity permitting, meta-analysis allows for indirect comparison to competitor interventions, and in the case of network meta-analysis (NMA), ranking of interventions against each other. With positive results, this analytical approach can demonstrate added value for the product.

More importantly, this evidence is becoming a commonplace requirement for payers; recent reimbursement reforms in France and Germany have now placed fresh emphasis on the need for comparative effectiveness data, and even linking this to price setting.

A question of costs
Payers seek evidence of value for money and the financial case needs to be made in an evidence-based way. For many markets, such as the UK and Canada, national HTA bodies require formal cost-effectiveness  analyses as part of manufacturer submissions. These modelling exercises distil health benefits into a single outcome, often a generic measure of health status (known as utility). In parallel, the cost of delivering the interventions in question is considered and calculated. Combining these as an incremental cost-effectiveness ratio, decision makers can compare new interventions across disease areas and prioritise funding accordingly.

As they play such a pivotal role in many health economies, these cost models are rigorously interrogated by academic assessment groups (at the behest of HTA bodies). To stand up to this scrutiny, models need to be evidence-based. This means systematically assessing the existing health economic literature to inform a best-practice model design. Moreover, data inputs, whether they are clinical or economic endpoints, need to be collected in an unbiased manner. All of this serves to minimise the sources of uncertainty within the model and increase transparency.

For many companies, the growing need for these types of analyses has prompted global teams to commission one core model to be used across their target markets. Compared with each market developing its own model, this appears an efficient strategy. However, a careful, considered approach is needed to ensure that a core model meets the needs of the majority, if not all, of the key markets.

Feasibility of core economic modelling should be conducted at an early stage in the product's development. This can feed into trial design and endpoint planning, but will also identify the requirements of each individual market and how a core model can best address these. To attain this knowledge, global teams need to gain expert health economic advice on model design and payer evidence requirements from each of the target markets. This international work needs strategic co-ordination and implementation in a time frame synchronous with product launch and relevant reimbursement applications.

Adaptable core model
One key attribute of any core model itself is adaptability. In most scenarios, some adaptation of the core model will be required for a national setting. This may be simply updating local cost inputs, or it could mean restructuring the modelled treatment pathway based on local practices. For this reason, models need to be easy to use and transparent. For instance, centralising all inputs into one location will allow users to input their own costs and run a revised model easily.

With technical assessment by HTA and reimbursement authorities, models need to be clearly laid out and navigable. Complicated, impenetrable spreadsheets risk raising uncertainties in a model's robustness and contributing to negative decision-making.

So far, the need for market access teams to develop three types of evidence have been described:

• Evidence of how payers make decisions
• Comparative clinical data
• Health economic impact of the new intervention.

But once all of this data is collected, how can it be communicated to payers? And what are the challenges?

Less conversation
Unlike the traditional 'physician-rep' relationships, payers have limited time for face-to-face meetings. In addition, payers often have varying appetites and capabilities in assessing complex data. As a consequence, presentations, whether in paper or electronic format, need to be layered – allowing the reader to 'drill down' to the level of detail each requires to understand the evidence.

Complicated, impenetrable spreadsheets risk raising uncertainties in a model's robustness and contributing to negative decision-making

When describing the disease, for instance, complex presentations of pathological mechanisms are unlikely to interest payers greatly. They will, instead, focus on understanding what the standard of care is and what the current unmet needs are. This can hinge on the sub-optimal health status of patients, but can also be a deficit in health system efficiency, such as over-long hospital stays. Either way, a payer must be convinced that patients with a specific disease need an additional, improved intervention.

Once need is proven, value must be shown. Presentations of health benefits need to focus on real-life implementation. If an antihypertensive reduces blood pressure, what subsequent healthcare event is offset by this investment? If risk of heart attack is reduced, how many events will now be avoided? Payers need this spelt out, clearly and concisely, while maintaining an evidence-based approach and appropriate level of detail.

Once a payer is convinced by the positive health impact of a new intervention, the economic case has to be made. For some markets, formal cost-effectiveness analyses are relied upon, as described above. For other markets, however, demonstrating how a new intervention can offset additional treatment costs due to, for example, reduced doctor visits, can be enough. These calculators must be transparent and evidence-based so that the payer can easily see how inputs have been derived.

Modern technology
Using modern communication platforms, such as the iPad, budget impact scenarios can be quickly customised in collaboration with the decision maker and the resultant business case emailed instantly to his inbox for use in his local decision-making processes.

Indeed, a step further is to combine this active approach with a passive solution. Once the active presentation is complete, a payer can be provided with a link to a web-based version of the budget impact calculator. This would allow the payer to customise the scenario further, and download iterative business cases as necessary.

As healthcare budgets get slashed year-on-year, so justification of incremental healthcare spend becomes increasingly challenging. Market access teams must begin planning their evidence platform at an early stage, assessing the payer market carefully and understanding what analyses and tools they will need. Time is required, as analyses such as cost-effectiveness models and systematic review need to be developed to best-practice standards to be fit for purpose. Once the evidence platform is established, innovative communication methods must be employed to facilitate payer decision-making.

The Author
Ruairi O'Donnell is commercial director, Abacus International