Regulatory approval without reimbursement results in unrealized revenue and, most importantly, patients left untreated. Parexel market access strategy experts Sangeeta Budhia and Jep Poirrier have advised hundreds of companies on how to gain reimbursement and attractive pricing based on value and differentiation. They help sponsors navigate a dynamic, global marketplace increasingly dominated by regulators and payers who demand that new products fill unmet medical needs and deliver cost-effective benefits to patients and healthcare systems. Sangeeta and Jep discuss early practical strategies to succeed in a fast-evolving environment.
Reimbursement is the ultimate patient-relevant outcome.
Poirrier: Recent events are validating what we have been advocating at Parexel for the last ten to 15 years: map out an evidence-generation plan early that will satisfy regulators and payers. As per regulation passed by the European Commission in 2021, the European Network for Health Technology Assessment (EUnetHTA) will begin performing joint clinical assessments of new products by 2025.¹ Meanwhile, in the United States, the Inflation Reduction Act of 2022 authorized Medicare to negotiate prices directly with biopharmaceutical companies for the first time in history.² These laws, on separate continents, are changing the evidentiary threshold for drug developers. The new HTA agency regulation was designed to guarantee market access for sponsors who present sufficient data and follow the rules—the goal is for patients to get access to these treatments. Although the United States does not have HTA agencies, Medicare’s new powers mean an increased focus on fair pricing. That, combined with the ascending influence of the Institute for Clinical and Economic Review (ICER), has altered U.S. market dynamics. In September 2023, ICER updated its Value Assessment Framework to include a “societal perspective” and capture productivity data from patients and caregivers.³ We advise clients to adjust their evidence plan nimbly to the new challenges because reimbursement is the ultimate patient-relevant outcome. Patients do not benefit if a sponsor spends $500 million on a drug that is not reimbursable.
We advise clients to adjust their evidence plan nimbly to the new challenges because reimbursement is the ultimate patient-relevant outcome. Patients do not benefit if a sponsor spends $500 million on a drug that is not reimbursable.
Patients and caregivers are vital to quantifying unmet needs.
Budhia: In this changing environment, demonstrating value—specifically, the comparative value of a product amongst those available—is more critical than ever. One way to do that is to substantiate the criticality of the unmet need. We are helping more and more sponsors engage with patients and caregivers to understand the impact of the disease and potential treatments on their quality of life. Traditionally, quality-of-life (QoL) measures in clinical trials try to capture benefits, but—depending on the tool's subtlety—that can be like using a sledgehammer when a fine-tooth comb is required. An unmet need needs to be quantified but also segmented. We advise sponsors to use patient and caregiver surveys to understand nuances in quality of life, disease burden, and resourcing needs. In line with ICER’s recent methodology refresh, more traditional HTA agencies are welcoming quantified patient and caregiver insights. For example, in amyloidosis, a rare disease that can lead to degeneration and dysfunction of the nervous system, a patient survey revealed that patients with Stage 1 or 2 disease required one caregiver, but those with Stage 3 disease required two caregivers. That’s a substantial financial and human impact. Duchenne muscular dystrophy (DMD) is a genetic disorder that causes muscle degeneration and weakness. Clinical trials have historically focused on lower limb mobility, using the six-minute walk test. However, patients said its impact on upper limb function was also meaningful. That insight requires quantifying the effect of a treatment on the upper limbs.
Gather evidence to support HTA agency coverage decisions.
Poirrier: We often work with sponsors who want to capture patient-reported outcome (PRO) data that is fit to appear on a product’s label. Although there are many PRO instruments available, not all of them are validated or accepted by regulators, HTA agencies, and payers. PRO data must be collected in an unbiased, robust manner without confounders. This is not easy. For example, we recently worked with a sponsor seeking a label claim that says its product reduces fatigue. Fatigue can negatively impact a patient’s productivity and engagement with daily living. Lost wages and productivity burden communities, employers, and societies as well. It is a quantifiable endpoint that could justify premium pricing if you can prove cost-effectiveness. However, many standard QoL scales don’t measure fatigue, which varies significantly by disease. Sponsors who try to incorporate a new fatigue endpoint late in development often struggle. HTA agencies routinely scrutinize PRO data and patient surveys in coverage decisions. The new EU joint clinical assessment program will standardize those analyses. Many sponsors are also looking at demonstrating value in terms of health equity. The pandemic highlighted disparities in healthcare access, spending, and co-morbidities. We are testing these arguments and data with specific HTA agencies.
Analyze a product’s impact on the healthcare system.
Budhia: We advise sponsors to generate data relevant to healthcare systems, not just to patients. During development, most sponsors focus on designing and conducting clinical trials that meet their primary endpoints. Then, they focus on regulatory approval. When those hurdles are cleared, they ask: how will payers look at it? But along that journey, sponsors need to assess how patients and caregivers in the target condition interact with the healthcare system. That is the only way to determine whether evidence will be fit for purpose for payers focusing on cost-effectiveness. For example, say a sponsor is developing a product to save an organ, such as preventing a colostomy in Crohn’s Disease (CD). Even if the drug is more expensive than the procedure, an HTA body would likely accept the additional cost due to the immense benefit to patients of keeping their colon intact. Likewise, kidney-saving drugs that prevent a kidney transplant (which requires lifetime treatments to sustain) would pass the cost-effectiveness test.
Sponsors need to assess how patients and caregivers in the target condition interact with the healthcare system. That is the only way to determine whether evidence will be fit for purpose for payers focusing on cost-effectiveness.
Contributing Experts