October 30 2023

The evolution of Early Access Programmes (EAPs) design for gene therapies and single-administration treatments


Sara Ahmad

Stefan Focsa

Gene therapies hold immense promise in revolutionising healthcare as they aim to correct the underlying genetic origins of diseases rather than merely treating symptoms. These therapies are administered within narrow windows of opportunity to change the course of a disease, heightening the importance of early intervention beyond that of conventional medical treatments. Although the preferred route for patients to early-access investigational therapies is through clinical trials, not all patients are eligible or able to participate in these studies. EAPs are a valuable strategy that facilitate therapy access for indications with high unmet needs, granting patients with life-threatening illnesses access to new treatments, prior to official regulatory approvals. However, EAPs are often less suited to single-administration therapies such as gene therapies due to uncertainties regarding their long-term safety and the complexity in selecting the right type of program to support the subsequent potential product reimbursement.

The unique difficulties surrounding EAPs for gene therapies:

  1. The complexity of gene therapies

With their intricate development and deployment, gene therapies differ from traditional drugs. Since they target the patients’ genetic material, it is necessary to ensure the therapy's safety, efficacy, and long-term impact which requires extensive preclinical and clinical testing. Waiting for these tests may not be an option for some patients with chronic diseases that require rapid access. However, regulators will only recommend treatment authorisation when the benefits outweigh the risks. With EAPs which run parallel to clinical trials, gene therapies may not have the requisite duration to conclusively demonstrate the absence of uncertainties regarding their safety and efficacy profile. An example on that would be Astellas’ AT132 gene therapy product for X-Linked Myotubular Myopathy.

  • The process of EAPs for medicine varies in different international contexts

While these programmes are known as EAPs in the U.S., they are named differently in Europe. Compassionate Use Programmes (CUPs) and Named-Patient Programmes (NPPs) are the main programmes in the EU, while the UK program is named Early Access to Medicines Scheme (EAMS). Europe presents the most attractive option for manufactures seeking gene therapy EAPs, as most countries have formalised pathways for reimbursed therapies prior to market authorisation. Despite EAPs being highly regulated, their guidelines are continually evolving. There are more differences than similarities among these programmes. These differences concern who pays for EAPs, the period of coverage, formal requirements for data collection, the bureaucratic application process, and even patients’ eligibility once the EAP is approved.

  • EAPs may represent a financial risk for gene therapy manufacturers

Despite gene therapies being a prime candidate for EAPs as they target rare diseases, they are considered financially risky, unsustainable, and unfeasible for manufacturers. This is primarily because in those countries where only CUPs are allowed as EAPs, they are required to provide the treatment for free with no certainty for future reimbursement of the therapy. Ultimately, this deters future investment in cell and gene therapies and diminishes their cohort of patients, especially for single-administration therapies.

  • Gene therapies are progressing rapidly, healthcare sectors must adapt adequately 

The landscape of medicine is rapidly evolving with the imminent approval of 32 cell and gene therapies in the U.S. These innovative therapies signify a paradigm shift from traditional treatment approaches. To fully harness their potential, the entire healthcare sector must adequately adapt and innovate ensuring they adopt and utilise these therapies in a sustainable manner.

Finding opportunity amidst the challenges of gene therapy EAPs

EAPs stand as indispensable pillars within the healthcare sector, serving as a vital tool to assess the rising potential of gene therapies. EAPs play a pivotal function in shaping formal reimbursement through the generation of Real-World Evidence (RWE), validating a product's worth to payers. To harness EAPs full potential, they should be strategically established at least 18 months prior to launch, affording ample time for meticulous planning, precise execution, and comprehensive data collection. The latter is essential to gather detailed insights into single-administration therapies. The early incorporation of patient perspectives into the design of EAPs is paramount, ensuring that these programmes harmonize with patients' needs and preferences while addressing other pivotal considerations in a manner agreeable to all stakeholders. In this synergy of foresight, data, and patient-centered design, EAPs emerge as a dynamic force propelling healthcare into a future where gene therapies hold boundless potential.

In the forthcoming years, it is plausible that we may also witness the implementation of outcomes-based or alternative risk-sharing arrangements in the context of EAPs. These arrangements would have the potential to satisfy payers' concerns surrounding the reimbursement of high-cost products associated with clinical uncertainties. Simultaneously, they could expedite the introduction of innovative therapies to reach patients with substantial unmet medical needs at an earlier stage, and allow pharmaceutical manufacturers to establish earlier relationship with healthcare authorities and providers, and support their market penetration plans.

Get in touch to find out how we can support you in assessing the feasibility of EAPs, and the supporting strategy to drive success.


1. https://www.ohe.org/wp-content/uploads/2021/06/Key-considerationsfor-early-access-schemes-for-single-administration-one-timetherapies.pdf

2. https://www.lse.ac.uk/business/consulting/news/gene-therapies

3. https://www.gov.uk/government/consultations/early-access-to-medicines-scheme-eams-consultation

4. https://www.valueinhealthjournal.com/article/S1098-3015(21)00306-5/fulltext

5. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9998493/