Early access pathways continue to shape how innovative medicines reach patients across the globe. In this blog, Inbeeo’s Senior Associate Sama Seyedmousavi provides an introduction to early access pathways around the world, highlighting key differences, country examples, and strategic insights to help manufacturers navigate early patient access opportunities for innovative medicines.
Understanding early and alternative access pathways
At Inbeeo, we distinguish between “Early Access” and “Alternative Access” pathways based on their objectives and timing.
Early access pathways allow patients with serious or life-threatening conditions access to investigational products before they receive marketing authorisation (MA) or routine pricing and reimbursement (P&R). They provide an option outside of clinical trials when patients lack comparable or satisfactory treatment alternatives.
By contrast, alternative access pathways refer to routes used instead of the official reimbursement process for treatments that carry significant clinical uncertainty, making them unlikely to succeed in the standard P&R process, or that have been denied reimbursement during the standard P&R assessment for the same reasons.
For ease of reading, we will use the term “early” to refer to both early and alternative access pathways throughout this blog.
Table 1: Examples of early access pathways around the globe
Addressing high unmet needs and uncertainty
Early access pathways play a crucial role in helping patients receive life-changing medicines sooner, especially in areas of high unmet medical need. Specifically, this includes severe conditions where time is critical, rare diseases for which there are no effective options, and therapies with inherent clinical uncertainty that struggle to meet traditional evidence standards:
- Drugs targeting conditions where time is critical: According to EFPIA’s 2024 Patients W.A.I.T. Indicator, the average time between EMA MA and patient access is 578 days across Europe, ranging from 128 days in Germany to 723 days in Poland.
- Orphan drugs: The 2024 W.A.I.T. Indicator report highlights that the average time to availability for orphan drugs extends to 611 days, highlighting the additional hurdles these therapies face.
- Drugs with a conditional marketing authorisation: A study in 2023 analysed health technology assessments (HTAs) across France, England, Scotland, and Canada. It showed that conditionally approved drugs experience moderate delays, averaging six months longer in receiving HTA approval compared to those with standard approvals. Greater clinical uncertainty and limited data linked to conditional approvals complicate the assessment process and contribute to these delays.
Bridging gaps between promising value and reimbursement
By providing early access to therapies, these pathways help bridge the gap between promising results in clinical development and completion of the full regulatory and P&R processes. They also offer the opportunity to gather real-world data, which strengthens the evidence for regulators and supports cost-effectiveness evaluations.
This creates a triple-win: patients gain early access, manufacturers have the chance to refine their value proposition, while healthcare systems gain more robust data, enhancing certainty about the product’s value in the treatment landscape.
The landscape: multiple pathways, multiple models
Programs are far from uniform. Across the globe, many countries have developed multiple access routes, each with its own eligibility rules, funding mechanisms, and duration. Despite differences in terminology, such as expanded access, managed access, or compassionate use, the core goals remain the same: to offer patients early access to promising therapies that address the unmet medical needs, outside of conventional reimbursement channels.
Moreover, these pathways also differ in terms of:
- Time of initiation → Early clinical trials vs post-MA
- Funding mechanism → Manufacturer, healthcare system, patients, or a mix
- Eligible patient population → Individual patients vs cohorts
- Chance of success → Multiple approvals annually vs exceptional use of the pathway
What the numbers tell us: A snapshot of global access pathways
Inbeeo reviewed 47 countries in Europe, North America, APAC, MENAT, and LATAM. In these markets, 91 pathways were identified.
When looking at timing, 38 out of 91 pathways are initiated as early as Phase I or II, whereas 31 can be initiated as of Phase III, and 21 after MA.
Note: One pathway is excluded as it’s dedicated to products that did not follow the formal clinical development process.
Time of initiation
In terms of how pathways are financed, 59 out of 91 pathways have the possibility of funding by the health system, hospital, private insurance, out-of-pocket or charitable organisation. The remaining 32 pathways are fully funded by manufacturers.
Funding mechanism
Looking at who qualifies, among the 59 funded pathways, 34 support access for cohorts of patients, while 25 are designed for individual patient use.
Eligible patient population
for the funded pathways
Finally, in terms of real-world utilisation, out of the 59 funded pathways, 10 pathways are considered highly active, with more than 100 product-indication combinations a year. Meanwhile, 14 pathways showed low activity, highlighting a gap between pathway availability and real-world utilisation.
Chance of success
for the funded pathways
So, what does this mean for manufacturers?
Early access pathways offer a strategic opportunity to accelerate patient access, generate early revenue and/or collect real-world evidence. However, to fully realise their potential, manufacturers must go beyond simply identifying available pathways.
- Country-level variation: Each pathway operates within a unique national context. Due to national differences in funding, eligibility, and regulation, manufacturers must use country-specific strategies, not a one-size-fits-all approach
- Product fit: Not every early access option will suit every product Manufacturers need a clear understanding of what makes a pathway both feasible and impactful for their specific product and objective, considering product-specific factors like development stage, clinical maturity, unmet need, and evidence strength.
- Impact fit: Depending on their configuration, early access opportunities can be significant:
- Faster time to patient access
- Early funding options
- Early evidence generation and refinement of value proposition
- Opportunity to get to know the local landscape in the context of geographic expansion
- Feasibility fit: Early access opportunities also come with trade-offs:
- Operational workload
- Responsibility for treatment continuation after early access ends
- Potential impact on future routine P&R negotiations
Navigating these trade-offs requires a well-informed access strategy and cross-functional alignment early in the development process. Early access should not be seen as a shortcut, but as a bridge, one that, when used strategically, can connect innovation with patient impact more effectively.
Ready to explore opportunities for your asset?
Have an asset you believe could benefit from early or alternative access, but are not sure where to start? At Inbeeo, we help pharma companies navigate this complex landscape, matching client ambitions with the most appropriate pathways across Europe.
With our internal database of early access pathways, and deep expertise in funding models, eligibility, and usage dynamics, we tailor access strategies to your asset and market goals.