Shefali Shah, principal of market access consulting, Evidera, and John Rafa, senior director of market intelligence, Accelerated Enrollment Solutions (AES), write about payer/health technology perspectives and trial design, optimal approaches for incorporating feedback and more.
Imagine this: After years of painstaking research and clinical trials, you successfully secure FDA approval for your new drug. The launch date and sales projections are set. Commercial plans go into motion. Then the worst-case scenario happens: Payers determine that your data do not sufficiently justify your product’s price relative to the perceived benefits to patients.
Payers restrict access to your product, prompting difficult decisions about the trade-offs between price discounts versus conducting additional trials to reinforce your product’s value proposition. You wish you could turn back the clock since such foresight could have driven a different set of decisions—ones that could have helped speed patient access to your compound.
How could this happen?
As public and private payers grow more stringent in their data reviews and selective about the products they will cover, manufacturers are increasingly being denied reimbursement for approved products. In 2019, roughly 400 products were excluded from Express Scripts and CVS formularies, a trend that has been ticking upwards.
- Express Scripts did not publish exclusion lists for 2012 and 2013.
- Express Scripts data for 2017 have been restated. Its 2018 list initially had 159 products, then grew to 196 products with midyear additions.
Source: Drug Channels Institute analysis of company reports.
Published on Drug Channels on August 16, 2018.
Market access is the process to ensure that all appropriate patients who would benefit from a product get rapid and sustained availability to it at the right price. A multitude of decisions along a drug’s lifecyle can significantly hamper its commercial potential, if not considered early in the process. Market access to stakeholders is one of the most essential considerations.
PCSK9 inhibitors are a prime example. The launch of these cholesterol-lowering drugs was expected to “revolutionize” care for as many as 10 million American cardiac patients. But when insurers and other payers balked at the economic rationale, prescribers were required to navigate a “maze of prior authorization,” thus compromising patient access. In one study, 80% of patients who tried to get PCSK9 inhibitors were met with initial rejections by insurers, and only 50% received approval after appealing.
How to help avoid market access delays, denials or misses
If market access considerations are only a distant thought in your clinical research planning, you’re not alone. Historically, clinical development and market access teams have operated separately, with the clinical mindset focused on what’s required for regulatory approval and the commercial mindset focused on what’s required for commercial success. In some cases, an assessment of payer needs may not happen until months prior to a product’s launch.
Today, however, this paradigm is changing. Payers are under increased pressure to control costs, and they are demanding stronger evidence of a drug’s economic value before they will cover a marketed product. As a result, the industry is slowly waking up to the need to integrate market access strategies earlier in the clinical development process.
It is critical to seek feedback early in the process directly from payers, government agencies and other market access stakeholders about protocol design, primary and secondary endpoints and related evidence necessary to differentiate a new drug from its competitors, asking questions like:
- What are the most critical endpoints?
- What do early modeling results tell us about the value of the product?
- What value messages will deliver broad access to patients?
- How should I communicate my evidence?
Gaining payer insights helps optimize the trial design to include payer-relevant endpoints for a more accurate value story that leads to broader unencumbered access. Those who consider market access early are reaping the benefits. Integrating the payer’s perspective early in trial planning can shift the decision-making process to 1) improving the odds of gaining favorable market access, 2) achieving faster adoption once launched and 3) speeding access to patients.
The growing influence of health technology assessments in securing market access
As the industry moves toward pay-for-performance and value-based frameworks, sponsors are under increased pressure to demonstrate economic value. A health technology accessment (HTA) is a formal research process that evaluates existing evidence, including clinical, economic, ethical, legal and societal perspectives.
HTAs ask different questions than traditional regulatory reviews, with the key differentiating question boiling down to economic value.
HTAs now play a formal, post-approval role in Europe. In the U.S., a formal HTA is not required for coverage or widespread use, but payer decisions are increasingly influenced by HTA recommendations, a trend that’s expected to continue.
What you should know today
Navigating the increasingly complex landscape of payer and HTA perspectives requires a depth of knowledge and experience that only comes from having faced and overcome market access challenges across many different scenarios.
Access strategy considerations may not fall within the scope of your day-to-day responsibilities, but it’s worth investing a small amount of time now to reap significant benefits in the future, when your drug’s market success is at stake. Our experience tells us that a little foresight goes a long way.
To hear more from our experts on payer perspectives and trial design, register and listen to our recent webinar.