To establish market access, start with unmet need: Why patient insights are central to reimbursement strategy

Biopharma organizations share a single purpose: delivering critical treatments to the patients who need them. And market access is essential to that mission. No matter how innovative the science, no matter how well designed the study — if payers won’t agree to reimburse the cost of a therapy, patients will never benefit from it. 

This is why market access planning is essential to any product’s success. Too often seen as a final hurdle, market access is integral to the entire development pathway. Access experts can help guide the course of a development program through patient-focused reimbursement strategy: a comprehensive approach built on deep understanding of the population a therapy seeks to serve. 

I recently presented a plenary session at the World EPA Congress that explored this topic. The themes I touched on are hardly new; they have been a consistent part of my conversations with clients for the last 12 years. As pressures on the industry grow, however, the following principles are worth revisiting, and resonated with the session’s audience.  

The payer perspective 

Development teams sometimes see regulatory approval as their primary goal, assuming that a marketing authorization will put all other pieces into place. But products regularly meet regulatory requirements only to be declined for reimbursement.  

Health technology assessment (HTA) bodies and payers often have a higher evidentiary threshold than that of regulators. For example, one study found that among 105 FDA-approved drugs assessed by HTA bodies in Australia, Canada, and the U.K., approximately 40 percent were not recommended for reimbursement. Reasons for negative reimbursement decisions included uncertain clinical benefit, small comparative clinical benefit, comparative safety concerns, and lack of cost-effectiveness at the proposed price.¹   

Private payers in the U.S. are also increasing restrictions on drugs. Another study reviewed coverage policies for 187 specialty drugs among 17 large commercial health plans and found that from 2017 to 2021, the proportion of coverage guidelines consistent with FDA labeling declined from about 57 percent to about 45 percent. In the same timeframe, the proportion of policies that were more restrictive than FDA labeling increased from 39.5 percent to nearly 52 percent.²  

Unlike regulators, HTA bodies and payers consider a therapy’s economic value. Clinical success is only one factor in coverage decisions — and failure to demonstrate economic value can negatively impact a promising therapy. When bluebird bio, for example, couldn’t reach reimbursement agreements within Europe for its gene therapy Zynteglo, the company closed its operations in the region.³ Similarly, Pfizer’s Vyndaquel and Vyndamax failed to receive reimbursement recommendations in Australia⁴ and the U.K.⁵ due to unacceptably high pricing and uncertain clinical evidence. Canada’s CADTH recommended reimbursement for the product only if specific clinical and economic criteria were met, including a price cut of greater than 92 percent.⁶ In the U.S., an effectiveness analysis of these drugs recommended the same price reduction to meet cost-effectiveness thresholds.⁷ 

Addressing unmet need 

Too often, people think of market access as pricing and commercialization — efforts to be undertaken only after drug study and approval. And while access experts can lead this important late-phase work, they should also be involved in study planning and execution, ideally starting from phase II proof of concept studies in patients but no later than phase III design, and for any pivotal study. Market access considerations will help direct — and support — a drug’s development journey.  

Every market access strategy begins by asking and answering this question: How can we best address unmet patient need? Whether providing a treatment where no options exist or by improving upon the current standard of care, sponsors must demonstrate that a treatment will likely result in better outcomes, better quality of life (QoL), fewer side effects, fewer hospitalizations, or greater patient convenience — and that it will do so in an economically feasible way. 

When included in the full development process, from phase II proof of concept studies in patients, through phase III design and for any pivotal study, access professionals can help teams: 

1. Define the patient unmet need: A team’s first task is to understand the patient population for which they are developing a product. Innovative science is exciting but ultimately fruitless if it won’t be welcomed by patients, who want treatments that appreciably improve on their current options. And researchers can’t make assumptions about whether patients will welcome a new therapy. For any program, patient insight is essential. Only by studying patients’ real-world challenges can researchers determine whether their product will be useful in addressing the situations patients face. 
2. Determine how effectively the product addresses unmet need: Once a team understands a population’s needs, they must consider whether the benefits of the treatment will be compelling to HTA bodies and payers. A therapy, for example, that costs significantly more than the standard of care and results in only modest QoL improvements will likely not earn favorable coverage decisions. Both patients and payers want therapies that improve significantly — and cost-effectively — on existing options. Simply put: If we are not addressing an unmet need, we are not providing any value. 
3. Generate evidence that will demonstrate clinical and economic value: When planning phase III studies, teams must understand evidentiary thresholds for both regulatory and reimbursement decisions. It’s critical, then, that access experts help shape study design and determine endpoints, some of which will be used to make a case for the therapy’s economic value. In generating evidence, teams must remember that regulators and clinicians may use different measures of value than HTA bodies and payers do. When treating glaucoma, for example, health care providers often rely on intraocular pressure measures. Payers, however, will likely want to measure improved vision and may not find intraocular pressure endpoints compelling. When access experts participate in all phases of research, they can help adjust output measures to make them more relevant to payers. They may also suggest adjustments to the patient population that will make it more representative of real-world populations. 
4. Create a pricing approach based on improved outcomes: Payers want products that provide clinical benefit while, if possible, reducing resource pressures on the health care economy. For some products, those dual benefits may be easier to quantify, like a treatment that results in fewer hospitalizations — creating both cost savings and a better patient experience. But the value of some improvements will be more difficult to assess and agree on. For example, convenience is important to patients, so a therapy that is simpler to use than the standard will meet an unmet patient need, even if that therapy doesn’t improve clinical outcomes. Payers, however, are often unwilling to reimburse for convenience-based improvements. We believe that the aspects of treatment that matter to patients have inherent value and that we should advocate for the value of those qualities with HTA bodies and payers. Developing evidence to support this is critical if patients are to benefit from a product’s full value. 

Access planning: Never too early

Every drug developer can benefit from bringing access experts into the process by phase II to understand patient – as well as health care professional and payer unmet needs. These perspectives are critical to developing the target product profile, and product value proposition which can be refined through phase III. Equitable reimbursement is essential to product success. Building payer requirements into the drug pathway will make reimbursement — and ultimately, patient benefit — easier to secure.

For access professionals, it’s critically important to engage with your development teams and to advocate for patient-relevant evidence collection. Data that demonstrate both clinical and economic value will position your product for favorable coverage decisions so the appropriate patient population can benefit from this new therapy. 

Ready to harness patient insight to generate compelling evidence? Our Access Consulting team is always available for a conversation.

References

¹ Assessment of FDA-approved drugs not recommended for use or reimbursement in other countries, 2017-2020, JAMA Internal Medicine, February 2023.

² Commercial coverage of specialty drugs, 2017–2021, Health Affairs Scholar, August 2023. 

³ With the pricing situation “untenable” in Europe, bluebird will wind down its operations in the “broken” market, Fierce Pharma, August 2021. 

⁴ Public summary document, Pharmaceutical Benefits Scheme (Australia), March 2021.

⁵ Tafamidis for treating transthyretin amyloidosis with cardiomyopathy, NICE technology appraisal guidance, May 2021. 

⁶ Canadian Drug Expert Committee Recommendation, CADTH, May 2022.

⁷ Cost-effectiveness of tafamidis therapy for transthyretin amyloid cardiomyopathy, Circulation, 2020. 

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