Market access and patient access schemes
Leela Barham explores the potential for patient access schemes (PAS) to help companies achieve market access.
By on Aug 10, 2010Leela Barham explores the potential for patient access schemes (PAS) to help companies achieve market access.
There has been a rise in the number of patient access schemes (PAS), or risk-sharing schemes, in the UK and other countries.
They are simply a response to uncertainty, says Jim Furniss, director, market access solutions at Bridgehead International Ltd.
That uncertainty is two-foldabout the benefits of a product in real-life use and about budget impact.
The rise of PAS has coincided with the increasing use of Health Technology Assessment (HTA); as payers consider value-for-money, they may find that some products just fail to meet this hurdle.
And if HTA agencies say a product isnt worth it, companies need to decide how to respond.
Understanding uncertainty
It is crucial to know, however, why an HTA agency/payer might think a product isnt worth it.
Is it because the evidence is not good enough? The product is just too expensive compared to alternatives? It might be used in patients who are unlikely to benefit?
Understanding the sources of uncertainty, and designing a scheme that addresses them, are keys to achieving a win-win solution.
Saying no to a product is not likely to be popular even among payers.
Many recognize the intrinsic value of having a range of treatment options, the political downsides of restricting access, and the nature of decision-making when the full value of a product cant be known until some years after its launch.
PAS can help when there are uncertainties in the benefits, which will never be fully known until a product is used in the real world and over a period of time, says Michael W. Thomas, a principal at A.T. Kearney.
Company and payer perspectives
According to Furniss, the real question for companies is, Am I going to need to offer a deal to achieve access? while the payers question is, Can I afford not to reimburse this product?
The decision, he says, is not just about the clinical and economic pros and cons, but a political decision too.
In many countries, such as Australia, there has been a tendency to focus on schemes that deal with financial uncertainties.
Australia is very clear about its approach, Furniss explains. Deals are effectively budget caps, based on the expected patient population who should benefit, with companies paying back if the cap is exceeded.
Both sides therefore know exactly what uncertainty they are trying to address.
Furniss cites the successful approach taken to a primary care COPD product, in which the PAS dealt with the risk that the product might be inappropriately prescribed to asthma sufferers in cases where cheaper and just as effective products were already available.
The scheme set an expected budget cap based on the number of COPD patients who would benefit.
And the approach worked; the company did not need to pay back and had strong incentives to ensure it promoted appropriate prescribing in line with the objectives of the payer.
Outcome-based approaches
Outcome-based approaches like this are sadly few and far between, Furniss notes.
The classic example is the MS scheme in the UK. But the lessons from that scheme are not clear.
It has enabled access, despite NICE saying that the products were not cost effective, says Furniss.
It also solved a political problem, the potential to have to take patients off products. And it did lower prices, between 8 and 15 percent reductions across the products.
But, in Furnisss view, this is not the ideal model, in part because the outcomes were much harder to monitor than anyone thought at the time.
So far, data is only available on the first two years of treatment and theres sufficient noise in the system to prevent conclusions from being drawn.
Even after eight years, Furniss says, we still dont know if the products are cost effective.
The lesson: Keep it simple.
The other UK outcome-based scheme, this one for Velcade, offers some insights for success.
That scheme has an available objective marker for response, and one that is relevant to usual clinical practice, according to Furniss.
Missed opportunities
The UK experience is a missed opportunity, in Thomass view.
The transparency required under the formalized approach to PAS is a hurdle to PAS being a viable route to market. Its an option only some companies will want to accept for some products, given the global impacts, ranging from parallel trade to external reference pricing.
Furniss also points out that administering such schemes can be difficult.
Italy has taken a patient registry approach, bringing data together centrally on oncology products and whether patients respond.
Most of these schemes see companies either offering a discount for the initial period of treatment, often around three months, or offering the product free, Furniss explains.
For those who respond, treatment continues at the price the company has set; for those who dont, a payback is made.
But those who have to collect the data may be not be firm supporters of having to administer such a process.
Its often pharmacists who bear the brunt of such duties, rather than the medicines agencies who negotiate the deals.
And sometimes the healthcare system just doesnt have readily available mechanisms to ensure that those who should receive any payback actually do.
Adapting market access plans
Furniss believes companies need to work harder and adapt their market access plans by identifying earlier whether they need to offer a deal.
Companies find it difficult to be objective about their cost effectiveness, he says.
Naturally, they are close to the product and look to the most favorable assumptions to underpin their modelling, even when its done out of house.
The difficulty is, HTA agencies dont work that way.
There is always a margin of error in modelling, and Furniss believes firms need to look at the worst-case scenario, simply because that is what the HTA is going to do.
They will be rigorously testing the robustness of modelling, he notes.
In asking themselves whether they are likely to need a scheme, pharma firms should realize that the answer will be country-dependent.
In places like the UK, answers are primarily based on whether the product meets the cost-effectiveness threshold.
If it doesnt, then a deal will need to struck or the company may have to forgo that market.
Beyond regulatory needs
Companies can do much more planning much earlier to make sure they build payers perspectives into their clinical development plans, Furniss says.
That means going beyond regulatory needs and looking to what is needed for commercial success.
Thomas, too, thinks approaches to commercial development are changing.
A.T. Kearneys recent work with pharmaceutical companies demonstrates that health economics needs to be done early, and provisional incremental cost effectiveness ratios (ICERs) are being used in key decision points in the commercial development cycle, he says.
They inform decisions at phase II, harden throughout the phase II work, and are absolutely informing decisions at phase III. The ICERs will then determine the discussion on a PAS or approaches to developing evidence on responsiveness in particular patient subgroups.
The need for schemes, according to Thomas, will not go away, especially as reforms in the US gather pace on comparative effectiveness.
Companies should respond, Thomas suggests, by considering cost effectiveness earlier, and conducting a rigorous risk assessment across their portfolios.
Companies also need to consider the execution of a scheme, adds Sol Magaz Marques, also with A.T. Kearney. That means thinking about the design early, and making sure that sales and marketing strategies are aligned.
For more on health economics, see Health economics data and market access.
For more on comparative effectiveness, see Why comparative effectiveness research is a market opportunity.