The Price of Neglect: Why Startups Must Care About Market Access

Startups face an uphill battle from securing funding to navigating the labyrinth of clinical trials and regulatory approvals, the journey is as grueling as it is rewarding. Yet amid the focus on scientific discovery and regulatory hurdles, one critical aspect is often neglected: market access.

Drawing on over a decade of experience in this field, I’ve witnessed countless startups excel in early development, only to falter when their breakthrough therapies fail to reach patients due to inadequate pricing, reimbursement, or payer engagement strategies. In today’s environment—shaped by stringent health technology assessments (HTAs) in Europe, the Inflation Reduction Act (IRA) in the US, and ever-rising payer thresholds—market access is no longer optional. It is an integral part of drug development that must be addressed as early as Phase Ib or II.

What Is Market Access?

Market access refers to the process of ensuring that a new therapy is not only approved by regulatory agencies but also made available to patients through pricing agreements, reimbursement, and distribution. This multifaceted process involves:

• Pricing Strategy: Determining a price that reflects the therapy’s value while meeting payer expectations and patient affordability.
• Reimbursement Negotiations: Engaging with public and private payers to secure coverage and formulary inclusion.
• Health Technology Assessments (HTAs): Demonstrating cost-effectiveness and clinical value to justify payer investment.

For startups, market access ensures that a therapy not only reaches the market but thrives within it. Without it, even the most innovative treatments risk becoming commercial failures.

The Historical Oversight

Startups often view market access as a final step—something to address after securing regulatory approval. This approach stems from several misconceptions and constraints:

1. Resource Limitations: Early-stage companies focus their limited resources on clinical development and funding, leaving market access as a lower priority.
2. Sequential Thinking: Many believe that regulatory approval automatically leads to reimbursement and patient access, overlooking the complexities of payer negotiations.
3. Complexity of Systems: Market access requires engaging with diverse healthcare systems, each with its own priorities and methods, which can feel overwhelming for lean organizations.

The Changing Landscape: Europe and the US

Europe: JCA and National HTAs

The introduction of the Joint Clinical Assessment (JCA) under the European Union Health Technology Assessment Regulation marks a significant shift. As of 2025, all new oncology drugs and advanced therapies will undergo a centralized clinical assessment at the EU level, streamlining evaluations but also introducing additional scrutiny.

• Advantages: JCA harmonizes assessments across member states, reducing redundancies.
• Challenges: National bodies like IQWiG (Germany) will still conduct independent evaluations for pricing and reimbursement. This dual process means startups must prepare for both EU-level and country-specific requirements.

United Kingdom: NICE

The UK’s National Institute for Health and Care Excellence (NICE) employs rigorous cost-effectiveness thresholds. Drugs must demonstrate a cost per quality-adjusted life year (QALY) below £30,000 to secure reimbursement in most cases. Startups that fail to design trials with NICE’s criteria in mind often face delays or outright rejection.

Germany: IQWiG

Germany’s Institute for Quality and Efficiency in Health Care (IQWiG) evaluates new therapies based on added benefit compared to the standard of care. Pricing negotiations are tied to these evaluations, and therapies that fail to demonstrate significant improvement face substantial price cuts.

United States: The Inflation Reduction Act (IRA)

In the US, the Inflation Reduction Act introduces Medicare price negotiations for high-cost drugs, a landmark change in a historically market-driven pricing system.

• Impact: Startups developing therapies likely to be included in the IRA’s negotiation list must rethink their pricing strategies early.
• Payer Fragmentation: Private payers and pharmacy benefit managers (PBMs) continue to dominate the US system, requiring tailored strategies for each payer type.

Real-World Consequences of Neglect

Case Study: Bluebird Bio’s Zynteglo

Zynteglo, a gene therapy for beta-thalassemia, represented a scientific breakthrough with its potential to cure a debilitating disease. Priced at $1.8 million, it struggled to gain traction in Europe, where payers balked at the high cost despite the therapy’s value. The lack of an outcomes-based pricing model or payer engagement strategy led to Bluebird Bio withdrawing the therapy from the European market entirely.

Case Study: Novartis’ Kymriah

Kymriah, a CAR-T therapy for leukemia, faced reimbursement challenges in both the US and Europe. Despite strong clinical results, delays in payer negotiations slowed patient access, costing Novartis valuable time and revenue in the early post-launch period.

The Statistics Are Clear

The proliferation of late-stage assets underscores the need for better market access planning:

• Phase III Trials: Over 300 unpartnered assets are currently in Phase III globally, representing a record high. Many of these come from startups with limited experience in commercialization.
• Launch Failures: A report by Deloitte found that 36% of new drug launches in the US fail to meet revenue expectations, primarily due to pricing and market access issues.
• Late Planning: Nearly one-third of biotech companies do not begin market access planning until after Phase III, despite evidence that early integration significantly improves outcomes.

Why Startups Must Act Early

To navigate these challenges, startups must integrate market access strategies early—ideally by Phase Ib or II. This allows them to:

1. Design Payer-Friendly Trials: Align trial endpoints with payer expectations, such as real-world effectiveness and health economic data.
2. Engage Stakeholders Early: Build relationships with payers, HTAs, and patient advocacy groups to understand their priorities and address concerns proactively.
3. Anticipate Regional Differences: Tailor strategies for markets like the US, UK, and Germany, considering unique payer thresholds and pricing frameworks.
4. Mitigate Financial Risks: Develop pricing and reimbursement models that reflect both the therapy’s value and market realities, reducing the risk of post-launch revenue shortfalls.

Conclusion: Market Access as a Strategic Imperative

Market access is no longer a postscript to regulatory approval—it is a determinant of success or failure. Startups that integrate market access considerations into their development strategies position themselves not only to secure regulatory approval but also to achieve commercial success.

The stakes are too high to ignore this reality. Therapies that fail to address payer expectations risk becoming scientific footnotes, while those that do can transform lives and redefine markets. For startups, the message is clear: market access is not a hurdle to clear; it is a cornerstone to build upon.