Regulatory approval and health technology assessment (HTA) are two distinct processes that serve different purposes on the path to market. Regulatory approval, granted by bodies like the European Medicines Agency (EMA), confirms that a medicine or device is safe, effective, and of acceptable quality. HTA evaluates whether that product delivers enough added value to justify its cost within a healthcare system, which determines reimbursement and pricing. Understanding both processes is essential for any company planning a successful market access strategy in Europe. If you would like to learn more about how we work, visit our company page for an overview of our background and approach.
What does each process actually evaluate?
Regulatory approval evaluates the absolute benefit-risk profile of a medicine or medical device. Regulators ask whether the product is safe and effective compared to a placebo or no treatment at all. HTA, by contrast, evaluates relative effectiveness: how much benefit does this product add compared to existing treatments already available to patients?
These two frameworks require different types of evidence. Regulatory submissions are built around clinical trial data that demonstrate a product works. HTA bodies want to know how that product performs against the current standard of care, what the impact is on quality of life, and whether the clinical benefit justifies the price. A product can clear the regulatory bar with strong placebo-controlled trial data and still struggle at HTA if no head-to-head comparator data exist against the relevant comparator used in clinical practice.
Who carries out regulatory approval and HTA in Europe?
In Europe, regulatory approval for medicines is primarily handled by the European Medicines Agency (EMA) through the centralized procedure, which results in a single marketing authorization valid across all EU member states. For medical devices, national competent authorities and notified bodies carry out conformity assessments under the EU MDR and EU IVDR frameworks.
HTA, historically, has been conducted at the national level. Each member state ran its own assessment through its own body, such as the HAS in France, IQWiG in Germany, or NICE in England (which operates under UK rules post-Brexit). This meant companies had to prepare separate HTA dossiers tailored to each country’s methodology, comparators, and evidence requirements. The EU HTA Regulation is changing this structure significantly, as explained below.
Does regulatory approval guarantee reimbursement?
No. Regulatory approval does not guarantee reimbursement, and the two decisions are made independently by different bodies. A product can receive full EMA approval and still be denied reimbursement in one or more member states if national HTA bodies conclude that the added therapeutic value is insufficient or that the price is not justified by the clinical benefit.
This gap between approval and access is one of the most consequential challenges in pharmaceutical market access. In some countries, the reimbursement process can take months or even years after the marketing authorization is granted, leaving patients without access to approved treatments. Companies that treat regulatory approval as the finish line often encounter unexpected delays and costly evidence gaps at the HTA stage. Exploring our full range of services can help you understand how integrated support across both regulatory and HTA tracks reduces this risk.
How does the EU HTA Regulation change the landscape?
The EU HTA Regulation, which began its phased rollout in January 2025 with oncology medicines and advanced therapy medicinal products (ATMPs), introduces a joint clinical assessment (JCA) at the EU level. This replaces the need for separate clinical assessments in participating member states, though national authorities retain the authority to make final reimbursement and pricing decisions.
For companies, this means a single joint clinical assessment dossier must be submitted to the HTA Coordination Group, following a standardized format and addressing a set of pre-agreed comparators and outcomes. The scope of the JCA expands to additional therapeutic areas over the following years. This creates both an opportunity and a challenge: one centralized process reduces duplication, but the stakes of getting the evidence package right are significantly higher when the outcome applies across multiple markets simultaneously.
When should companies start preparing for HTA?
Companies should begin preparing for HTA at the same time as, or even before, they finalize their clinical development program. Ideally, HTA considerations should be integrated into Phase II trial design at the latest. Waiting until after regulatory submission to think about HTA is one of the most common and costly mistakes in drug development.
Early preparation involves identifying the relevant comparators for each target market, designing trials that generate the outcomes HTA bodies value (such as patient-reported outcomes and quality-of-life measures), and engaging in early scientific advice with HTA bodies where possible. Under the EU HTA Regulation, companies can request a joint scientific consultation with the HTA Coordination Group during development, which provides guidance on the evidence requirements for the future joint clinical assessment. Aligning regulatory and HTA strategies from the start significantly reduces the risk of evidence gaps at the time of submission.
What are the key differences between HTA and regulatory approval at a glance?
The core distinction is this: regulatory approval is about safety and efficacy in absolute terms, while HTA is about comparative value in a real-world healthcare context. Every other difference flows from that fundamental distinction.
- Purpose: Regulatory approval establishes that a product is safe and effective. HTA determines whether it provides sufficient added value to warrant public funding.
- Decision maker: EMA or national competent authorities for regulatory; national or now partially joint EU bodies for HTA.
- Evidence focus: Regulatory relies on placebo-controlled trials; HTA requires comparator-based evidence against the current standard of care.
- Outcome: Regulatory approval grants a marketing authorization. HTA informs reimbursement and pricing negotiations.
- Timing: Regulatory approval typically precedes HTA, but HTA preparation must begin during clinical development.
- Scope: A single EMA approval covers the entire EU. HTA decisions, even under the new regulation, remain nationally binding for reimbursement.
Understanding these differences is not just an academic exercise. The evidence a company generates, the comparators it selects, and the endpoints it measures in clinical trials all have direct consequences for both regulatory and HTA outcomes. Treating these as parallel tracks from early development is increasingly the standard for companies that reach patients efficiently.
How Starodub supports your regulatory and market access strategy
Navigating the intersection of regulatory approval and HTA requires expertise that spans both disciplines. At Starodub, we help biopharmaceutical and medical device companies build regulatory strategies that account for HTA requirements from the start, reducing the risk of evidence gaps that delay patient access.
Our support in this area includes:
- Regulatory pathway analysis that incorporates EU HTA Regulation requirements and joint clinical assessment timelines
- Guidance on clinical trial design to ensure the evidence generated satisfies both EMA and HTA body expectations
- Preparation and review of regulatory submissions for the EMA centralized procedure and national procedures
- Support for ATMPs, biologics, combination products, and medical devices across all complexity levels
- Strategic advice on early scientific consultations with both regulatory and HTA bodies
Whether you are in early development or approaching submission, we are ready to help you build a strategy that connects regulatory approval to real market access. Contact us today to discuss how we can support your path to market in Europe.
