Market access in the pharmaceutical industry is a nuanced and complex process, involving the coordination of treatment delivery at prices that balance the needs of patients, manufacturers, insurance companies, and other stakeholders. 

As the landscape becomes increasingly intricate, ensuring that patients have access to life-changing therapies is more challenging—and crucial—than ever for pharmaceutical and biotech companies.

According to Deloitte, market access needs to be rethought because pharmaceutical assets typically cost about $2B to bring to market, yet about 36% of launches in the US fail to meet launch expectations.

The purpose of this guide is to inform leaders at pharmaceutical and biotechnology companies (along with other stakeholders) about the following:

• What market access is, and who/what it involves
• How to execute an effective market access strategy
• The role regulatory institutions play in market access
• Challenges to market access and how to overcome them

Understanding Market Access in Pharma

As the pharmaceutical industry and commercial models evolve, understanding market access has become increasingly crucial for companies looking to introduce new, innovative products or to expand market share. In the next section, we’ll explore the complexities of market access in the pharmaceutical sector, including the various factors at play and their impact on the industry. 

What Is Market Access?

A team of researchers recently published the first comprehensive meta-analysis on the conceptualization and role of market access in pharma. In many other industries, market access deals primarily with supply, demand, and macroeconomic conditions that control commodity availability. In healthcare and life sciences, market access is about finding the “right” distribution model.

Getting it right, and maintaining access for qualified patients, requires intentionality from pre-commercial market shaping and development through distribution models and network selection and ongoing relationship management.

Ultimately, pharma and biotech companies are optimizing for availability and affordability: they want to make treatments available at prices patients can afford.

In practice, this amounts to optimizing the patient and prescriber experience with a specific product. Is it efficacious, is it covered by insurance companies, and is it readily available for qualified patients? These are the kinds of questions biopharma market access professionals are contending with, and the most forward-thinking firms are investing heavily in internal Access and Patient Services organizations to help streamline the patient journey and optimize access, affordability, and adherence. 

Market Access and the Pharmaceutical Lifecycle

Market access impacts all parts of the overall patient experience, and the most innovative pharmaceutical and biotech companies consider market access at all stages of a product’s lifespan.

Here’s how market access integrates into different phases of the pharmaceutical product lifecycle:

• Clinical phase (Phase 2/Phase 3 assets): During this phase, market access research plays a critical role in narrowing down target populations and informing the design of clinical trials, ensuring that potential therapies can demonstrate value to both patients and payers.
• Pre-commercial (pre-FDA approval): As a drug approaches approval, market access efforts shift towards ensuring patient access. This includes navigating the complexities of authorization, compliance, and distribution strategies to streamline the product's availability.
• Commercialization (launch mode): Post-approval, market access planning focuses on the financial implications, particularly the affordability of the treatment for patients. 

Key Stakeholders in Market Access

Market access teams are primarily concerned with end users—patients—and their ability to procure and utilize approved treatments. However, many other parties are involved in getting treatments to market. Here are the main players:

Pharmaceutical Companies

In simplest terms, pharma companies develop drugs and other treatment options that patients need. These treatments are diverse today, ranging from vaccines and general medicine to high-volume specialty products, oncology treatments, and rare/ultra-rare disease therapies, including innovative cell and gene therapies. 

Put another way, biotech and pharmaceutical companies shape the “goods on offer” in the market. This is inseparable from considerations of availability and access, since even the most promising, cutting-edge treatment options or research discoveries need to be coupled with a way to get them into patients’ hands.

Our recent white paper, 3 Trends Raising the Bar for Patient Services, took a deep look at ways pharma manufacturers are reshaping market access, including:

• Shifting to internal patient access and support services: With so much at stake in a product launch or overall market performance, pharma companies of all sizes, therapeutic areas and product types are investing in internal resources to help manage access, affordability, and adherence, rather than outsourcing entirely.
• Upgrading internal systems with purpose-built software: Pharma companies are upgrading outdated software and tech stacks with integrated, purpose-built solutions that efficiently and effectively support the needs of their personnel.
• Measuring and managing by impact: Related to the above, with greater internal  visibility into and control over the end-to-end patient journey, the most successful pharma operations are powered by data and analytics, continuously measuring and refining market access and patient support strategies. 

Moving forward, the momentum of these trends should only expand as more specialty medicines hit the market, access becomes even more complex, and the commitment to patient-centricity continues to increase.

Healthcare Providers 

Healthcare Providers, or HCPs, are one key contact point through which patients learn about approved products and receive treatment guidance. While the landscape has certainly gotten more complex with patients, particularly rare and ultra-rare disease patients, taking more of a hands-on role in their diagnosis and treatment journey, HCPs are inextricable from market access strategies and tactics. While the mix may vary based on product type and disease state, providers remain key intermediaries between pharma companies and patient populations.

Understanding, engaging, and supporting HCPs is key to pharma market access. HCPs provide guidance to patients that impact utilization and adoption, not to mention that HCPs and office staff play a critical role in access and reimbursement. 

At this point, hopefully you are beginning to see the fundamental dependencies between different stakeholders in the healthcare ecosystem and how each step is interrelated. If healthcare providers believe in a new treatment but face barriers like poor coverage or a complex prior authorization process, it can undermine their confidence in prescribing it, ultimately affecting patient access and health outcomes.

Payers and Insurers

As the name suggests, payers are entities like health plan providers, Medicare, and Medicaid that cover the cost of treatment at the point of service. However, their role goes beyond just payment; they are crucial stakeholders in determining the success of a pharmaceutical product in the market. Payers conduct rigorous cost-benefit analyses, assessing the therapeutic value of treatments against their financial impact on the healthcare system. 

To secure market access, pharmaceutical companies must demonstrate that their products are safe, effective, and economically valuable, often providing data to support these claims. In some cases, patients might initially pay out of pocket but later receive reimbursement, adding another layer of complexity to payer evaluations.

According to a recent study in Frontiers in Pharmacology, the criteria payers and insurers value most in treatment are tangible benefits (in particular, the rarity of the condition treated first in their valuation), followed by the severity of the disease, the novelty of the treatment option, its simplicity, and its capacity to reduce other health risks.

Regulatory Agencies

Another major player in market access is the group of regulatory authorities that define whether a given product can be on the market.  What’s more, regulators don’t just control what launches in the market—they control what stays.

Regulatory bodies monitor performance, safety, and other concerns across pharmaceutical products and can revoke or limit market access based on their findings. (Proof point: between 2012 and 2024, post-market surveillance led to 3,992 drug recall events and 16,397 individual drug recalls, accounting for about 18% of all recalls in that span.)

Further below, we’ll provide a more granular breakdown of the most important regulations impacting market access in pharma. But for now, it suffices to say that regulations define the market through drug/treatment approval, and regulators play a key role in both immediate and long-term market access strategy and success.

Patients and Advocacy Groups

Last but not least, patients are at the center of all market access considerations. Forward-thinking pharmaceutical companies appreciate that what’s best for patients is best for business and they work tirelessly to improve access, affordability, and adherence to help drive better patient health outcomes.  This includes fostering relationships with patient populations and advocacy groups to understand their unique challenges and needs and to shape access strategies that eliminate barriers to accessing treatments.

Focusing on patient needs (and better patient outcomes) as the north star is key whether you’re the pharmaceutical company, healthcare provider, payer, or regulatory agency. Enabling greater collaboration and trust ensures patients aren’t just prescribed treatments or able to have them covered but actually start and stay on them as needed. 

Key Strategies for Successful Market Access

As biopharma companies navigate the complex market access landscape, there are key strategies that can pave the way for success in the short- and long-term, starting with the following. 

Early Engagement With Stakeholders

All stakeholders detailed above should be engaged as early during pre-launch planning as possible. (As mentioned above, this applies especially to patients and advocacy groups. Engaging patient populations, listening to their concerns, and gathering initial impressions and feedback on program design is critical market research.)

Depending on the desired outcome and stage of the planning process, some examples of stakeholder engagement to consider include:

• Town halls and open-access meetings where patients can express desires and concerns and foster discussions about the needs pharma can fulfill (and how).
• Check-ins with doctors and HCP staff to ensure personnel are fully informed about clinical effectiveness, access, and reimbursement policies, and to answer questions they may have.

Pricing and Reimbursement Strategies

Another way to maximize market access success is to take a proactive and dynamic approach to pricing. This starts with considering and preparing for the various pricing models that can be applied to a given treatment. 

For example, value-based pricing emphasizes quality rather than quantity, whereas risk-sharing agreements spread liabilities across payers and manufacturers.

A Health Economics Review study found that reimbursement negotiations dramatically impact drug availability and cost. Researchers found that societal conditions such as the severity of patient needs did not always correspond to greater willingness to pay (WTP) from the payer side. In practice, pharma companies need to build higher-order metrics like quality-adjusted life year (QALY) into internal pricing and negotiation strategies before payer engagement.

Strategies should target specific markets or market segments to secure access early, where possible—a win in its own right and a boon to bargaining power elsewhere.

Leverage Innovative Analytical Models

In general, a successful market access strategy makes the best use of all available tools, including industry-leading data analysis.

Two such lenses used by many top pharmaceutical companies include:

• Health economics and outcomes research (HEOR): These assessments triangulate the costs of healthcare treatments and the outcomes they produce, arriving at dynamic quantitative and qualitative evaluations.
• Health technology assessments (HTAs): These interdisciplinary tests synthesize evidence from various clinical, safety, efficacy, and other trials to determine and inform value, use cases, and best practices for use.

Market access analysts should consider these throughout the planning process to assess how particular partners will value a product.

Understanding Regulatory Considerations

With a solid understanding of what market access is, the key stakeholders involved, and strategies to maximize success, it’s time to explore the diverse facets of regulatory considerations and their implications.

The Global Regulatory Landscape

Some of the most important regulatory bodies that oversee biotechnology and pharmaceutical companies in particular are (inter)national government authorities:

• The United States Food and Drug Administration (FDA)
• The European Union’s European Medicines Agency (EMA)
• Japan’s Pharmaceuticals and Medical Devices Agency (PMDA)

Global market access often requires simultaneously meeting different countries’ or regulatory body’s requirements, including cases where reporting standards and protocols differ.

Approval Processes

Aside from company-wide compliance with basic requirements (e.g., HIPAA) and standards that agencies uphold, pharmaceutical companies also need to obtain approval for each product.

For example, in the United States, the FDA’s drug approval process comprises three essential analyses:

• Analysis of the target condition and available treatments, including the severity of the condition and the quality/cost/etc. of other treatments.
• Analysis of the benefits and risks from clinical trials, including uncertainties and inconclusive evidence about potential positive outcomes and threats.
• Analysis of strategies for mitigating identified risks, including detection and treatment options and/or a Risk Management and Mitigation Strategy (REMS).

The FDA aims to take an objective, scientific approach to testing and maintains ultimate discretion over whether, when, and under what conditions a treatment is approved.

There are also accelerated approval pathways, such as orphan drug status and breakthrough therapy designation. Per a longitudinal Health Affairs study, approval rates are 65% for single rare disease orphan drugs, 15% for those treating multiple rare diseases, and 20% for those treating rare and common diseases.

Post-Market Surveillance

In addition, regulatory agencies such as the FDA can pull a product off the market if it fails to deliver on safety or performance metrics that were promised upon initially entering it. 

One of the most impactful factors in FDA post-market surveillance is real-world data (RWD) or real-world evidence (RWE). RWD is an input that comprises raw data on patients’ health status and outcomes, as triangulated between multiple sources the FDA trusts. Conversely, RWE is an output or the result of rigorous RWD and trial data analysis.

Generating copious amounts of data and carefully analyzing it is one of the best ways to prove a product’s safety to the FDA, patients, and all stakeholders.

Another best practice for staying ahead of potential issues in post-market surveillance is fostering direct relationships with HCPs, prescribers, patients, and advocacy groups. Whether it’s investing in internal Patient Services Managers who check in with assigned clients and document insights around dosing and side effects, or creating a dedicated provider portal for doctors to report adverse effects or to schedule in-person or online meetings, these data points help pharmaceutical companies to stay on top of clinical results and to execute against their mission to improve patient outcomes. 

Challenges in Pharmaceutical Market Access

The pharmaceutical industry is facing many challenges in ensuring market access. These range from regulatory hurdles and the rise of new cell and gene therapies to pricing and reimbursement issues. Economic and competitive pressures are particularly present as market access leaders strive to secure access to innovative drugs and treatments for as many qualified patients as possible, naturally leading to pricing pressures.

These challenges often result in delays in market access, with prolonged approval processes or failed launches potentially preventing access to life-altering treatments. Lengthy negotiations or failure to reach coverage agreements can also stall a product’s commercial launch. Additionally, logistical issues such as manufacturing and supply chain requirements have become more prominent, as evidenced during the COVID-19 pandemic. For example, PwC’s study on market access for gene therapies highlights the necessity for flexible supply chains. Specialty products often demand robust “cold lines” for acute temperature needs and well-honed fulfillment processes to meet faster turnaround times required by specific patients or groups.

Optimize Your Market Access 

Effective market access in pharmaceuticals requires early planning and engagement, cross-functional and stakeholder collaboration, careful cost calculations and negotiations, and a healthy dose of creative thinking and willingness to innovate in support of patient access and better outcomes. It’s a complex, ever-evolving area that blends art and science and is only getting more complex as specialty medicines hit the market and regulations change. 

For more information on emerging trends in market access and what industry leaders should be thinking about, read our Redefining Market Access white paper. It sheds light on how top patient experience innovators are thinking about access strategies today. 

Download our white paper today to learn how patient-focused digital solutions can streamline the overall patient journey.