Introduction
Early access programs (EAPs) are an opportunity for patients, typically with life-threatening or seriously debilitating conditions, to access medicines prior to marketing authorisation approval. These schemes offer benefits and challenges that mean their implementation across Europe is mixed, with each EAP possessing unique criteria. This article will briefly explore why countries offer EAPs, describe the funded EAPs available across the EU5, and discuss why other countries do not follow suit.
Why offer EAPs?
EAPs have obvious benefits for the stakeholders involved. EAPs offers access to medicines at an earlier stage in the drug development process. In life-threatening differences, this could be the difference between life and death. Even in non-life-threatening diseases, the early treatment of the disease could prevent future manifestations or co-morbidities that makes treatment more challenging for healthcare providers.
Manufacturers can benefit from generating real-world evidence (RWE) in patient populations that can often be extremely difficult to recruit within clinical trials. This evidence can then be used to facilitate pricing and reimbursement (P&R) negotiations with HTA bodies and/or payers. RWE provides more contextual evidence for HTA bodies and payers to consider, especially in understanding how the treatment could be implemented within the healthcare system (e.g. at specialised centres).
Which countries offer EAPs?
A total of seven early access programs exist across the EU4 and the UK: France (Early Access Authorisation – AAP; Compassionate Use Access – CUA) and Italy (Law 648/1996; Compassionate Use) both have two program options and the UK (Early Access to Medicines Scheme – EAMS), Germany (AMHV) and Spain (Royal Decree 1015/2009 – Authorisation for Temporary Use) have one. The common criteria for all schemes is that the therapy is needed to treat a life-threatening, long-lasting or seriously debilitating illnesses, which cannot be treated satisfactorily with any currently authorised medicine. Otherwise, the details of each program varies from market to market regarding application status (e.g. in Germany and Spain an application must have been submitted), expectations for clinical data packages (UK require evidence from phase III trials, unlike Italy that accept phase I (compassionate use) or II (Law 648) trial data)), data collection opportunities and paid access. More detail on the differences between the programs can be accessed via our ISPOR publication here.
Why don’t other countries offer funded early access?
The major concern for the payer is whether the drug should be funded prior to understanding the full level of clinical benefit the product offers. For most EAPs, the perception of the payer is that the manufacturer should be willing to offer the product free of cost while this uncertainty exists, and the evidence is generated to address said uncertainties. It is therefore unsurprising that funded early access is rare. In fact, it is only France’s AAP and Italy’s 648 program offered paid Early Access for manufacturers.
While non-funded access provides a financial drawback to manufacturers, there are still advantages to participating in these schemes as previously described. Manufacturers need to weigh up the cost of providing their products against the valuable opportunity for engagement with stakeholders and collection of additional data that could be involved in future provision, reimbursement, and funding.
Conclusion
There are multiple opportunities for EAPs for manufacturers across the EU4 and the UK. Crucially for manufacturers, only France and Italy provide paid access that allow manufacturers to set a price for their product. The limited number of EAPs that offer paid access could significantly limit the number of manufacturers able to provide early access to potentially lifesaving medicines for patients. However, as discussed above, the opportunity to engage with key stakeholders and collect relevant evidence could outweigh the financial costs in providing the treatment free of charge.
Sources:
Thomas M, Morris L, Foxon G, Craddy P. Comparing and Contrasting Early Access Opportunities Across the EU4 and UK. Poster presented at ISPOR 2022; Vienna.