Strategic Market Access Ensures the Right Treatments at the Right Time

Craig Caceci, Executive VP and Managing Director, EU/UK, AscellaHealth

Market access is a process that ensures patients have access to the right treatments at the right time—and for a fair price that aligns with the effectiveness of a drug in treating disease. It is a critical part of drug development and go-to-market strategy that establishes pricing and reimbursement sustainability for the healthcare payer and the pharmaceutical manufacturer.

Rigorous, proactive methodologies support pharmaceutical companies as they endeavor to receive the best reimbursement possible for their product. It guarantees these products remain affordable and attainable for payers and patients.

Navigating market access and reimbursement is essential in achieving this mission. A robust market access strategy ensures that pharmaceutical products, including those with limited distribution, reach patients and are covered by diverse healthcare systems—whether through public or private payers in the US or single-payer systems in the European Union (EU).¹

The critical first steps are to set the right price, demonstrate product value and generate data to document its safety and effectiveness. This information forms the foundation for achieving adequate reimbursement and payer coverage, essential for patient access to a drug, ultimately enhancing patient care.

Global Market Access

Market access strategies vary by country and payer model. In the EU, for instance, obtaining European Medicines Agency (EMA) approval through the centralized process does not guarantee that a drug can be prescribed² until it is included in each country’s reimbursement catalog. Similarly, in the United States, despite FDA approval, there are no guarantees that commercial, private, public, or government-funded insurers will cover the cost.

It is essential to initiate the market access process early in the product lifecycle, ideally during Phase II or III clinical trials. This proactive approach involves engaging with regulatory authorities, payers, and key stakeholders to align on clinical and economic evidence requirements.

Early dialogue with these entities can help identify potential barriers and facilitate the development of robust evidence packages that demonstrate the value of the product. This strategy not only ensures that a product meets regulatory requirements but also aligns with payer expectations, increasing the likelihood of timely reimbursement decisions. By addressing these critical components early, manufacturers can accelerate patient access to treatment, ultimately improving health outcomes and maximizing the product's market potential.

Key Strategies for Market Access

Many pharmaceutical manufacturers, most commonly smaller biotech companies, may struggle to execute a successful market access strategy due to limited resources. Increasingly, companies outsource this function to experienced professionals who can manage and steer comprehensive campaigns.

In the EU, post-EMA approval challenges include negotiating pricing and reimbursement with each country’s distinct payer system. In the US, overcoming payer barriers involves deep market contacts and expertise in negotiating reimbursement terms.

Early and ongoing engagement with payers is crucial for developing effective market access strategies. This approach involves gathering robust real-world evidence, evaluating market conditions and competitive insights, and clearly articulating the product's value proposition.

By proactively addressing potential barriers and issues through cross-functional collaboration, manufacturers can enhance their negotiation tactics with payers. Conducting thorough market access analysis helps uncover reimbursement gaps and coverage limitations, enabling the development of effective pricing strategies to secure coverage and reimbursement.

Drug Formulary Access

Securing favorable positioning on drug formularies and national guidelines is critical to indicating market access success. This positioning involves understanding the intricacies of formulary or guideline management, which includes navigating the policies and procedures that dictate the drugs selected for inclusion. To achieve this, pharmaceutical companies must not only demonstrate a drug’s efficacy through rigorous clinical trials, but also provide robust, real-world evidence of its clinical outcome.

Additionally, it is essential to substantiate the drug’s cost effectiveness, highlighting its value proposition to the patient, payer, and healthcare provider. This comprehensive, collaborative approach ensures that the drug is accessible to patients who need it, driving better health outcomes and maximizing the therapeutic potential of the drug in the marketplace.

Value-Based Contracting

Manufacturers must prepare for value-based contracting,³ including alternative payment models such as risk share and pay-for-performance. These contracts tie reimbursement, coverage, or payment to a treatment’s clinical outcomes and effectiveness, aligning incentives across the healthcare system. The prevalence of value-based contracting is increasing as payers and providers aim to reduce healthcare costs, improve healthcare outcomes, and enhance patient access and care.

Affordability of specialty drugs for plans and members with complex, chronic conditions drives this interest, with growth in specialty drug costs representing an underlying factor in overall price increases: average annual price growth was 13.2%⁴ for specialty drugs compared with 2.6% for non-specialty drugs. According to the most recent government report, specialty drugs represented 50% of total drug spending in 2021⁵ with a projected growth of 5.5%–9.5%⁶ growth rate for specialty drugs from 2023–2024. Looking ahead, the specialty drug pipeline may represent up to two-thirds of newly launched drugs in the next five years, further underscoring the significant impact specialty drugs will continue to have on healthcare spending and patient care.

In this current environment, demonstration of value builds as the demands of product launch and commercialization line up alongside the economic pressures of the Inflation Reduction Act,⁷ which is introducing a new era of government price scrutiny and negotiations. This convergence continues to squeeze the monetary lifespan of drugs in the United States, requiring increased evidence beyond clinical trials that include health economic outcomes research, real world evidence, patient-reported outcomes, and integrated combinations.

Assembling the data and documenting longitudinal evidence can take years to develop—but it is well worth the effort. Industry analysts report that drugs that achieved launch excellence have approximately twice the evidence within the first two years as all other launches.⁸

Financial Support Solutions

Financial support solutions help patients access programs designed to make medicines more affordable and accessible. For patients who do not qualify for insurance or assistance programs, patient assistance programs⁹ (PAPs) provide free or low-cost medications to low-income individuals, with individual companies setting different eligibility criteria for enrollment in their PAPs.

In the United States, customized co-pay assistance programs help reduce the out-of-pocket expenses insured patients must pay, making expensive therapies more affordable. Manufacturers rate high out-of-pocket costs as the top patient access barrier.¹⁰

To address this issue, specialty pharmaceutical solutions companies work with manufacturers in establishing reimbursement programs and identifying ways to help patients access the therapies they need. These financial solutions help patients manage financial burdens, enhance medication access, and increase medication adherence.

Drug affordability is particularly challenging for individuals with one or more complex, chronic diseases who are currently taking multiple prescription medicines. An estimated 129 million people in the US¹¹ have at least one characteristic chronic disease, defined by the CDC¹² as a condition that lasts at least one year and requires ongoing medical attention or limits daily activities. An analysis of the CDC Behavioral Risk Factor Surveillance System estimates that 53.8% of adults aged 18─34 years report at least one chronic condition, and 22.3% have more than one.

Reimbursement Nuances

Reimbursement of medicines differs by geography, often distinguished by whether or not a payer will require a health technology assessment (HTA) on the drug. In the US, each insurer performs internal assessments to decide on payment for new products based on the criteria of safety, efficacy, and cost. In Europe, Australia, and Canada, for example, an additional level of assessment is required in the form of an HTA. As a result, HTAs play a crucial role in global market access strategies.

HTAs help ensure that new therapies are clinically effective and economically viable, influencing reimbursement decisions and patient access worldwide. Increasingly, national HTA bodies are collaborating on their assessments, as evidenced in the Joint Clinical Assessment collaboration, whereby the clinical features of new medicines are assessed centrally at the European level. Individual member states will handle pricing and reimbursement negotiations based on this clinical evidence as part of the new Joint Clinical Assessment Regulations.¹³ Implementation of the Act will begin in January 2025.

Future Outlook

The market access landscape is complex and ever-changing, with payers continually reassessing their business models, priorities, and strategies. Manufacturers face many go-to-market challenges, including emerging prior authorization restrictions—especially for expensive cell and gene therapies, step therapy requirements, formulary restrictions, and exclusions.

To overcome these challenges, processes should ideally focus on a collaborative, patient-centric care approach that enhances the quality of life for patients with complex, chronic conditions by removing barriers to treatment and improving patient access to essential therapies.

Global marketing teams are tasked with providing the expertise and support needed for successful market access and reimbursement, adapting to regulatory changes, and ensuring a worldwide approach that prioritizes patient access and care.

About the Author

Craig Caceci, Executive VP and Managing Director, EU/UK, AscellaHealth, brings over 18 years of experience in the pharmaceutical industry, holding various positions in sales, sales leadership, training, and managed care. At AscellaHealth, Craig oversees the day-to-day operations of the EU/UK division. He manages US-based group purchasing initiatives, generating incremental savings for members in managing their Specialty and Rare/Orphan disease pharmaceutical utilization. His responsibilities encompass working with PBMs, hospitals, long-term care facilities and strategic pharmaceutical manufacturers.

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