Early Access Programs (EAPs) help to give people with life threatening or seriously debilitating conditions early access to new medicines that do not yet have a marketing authorisation. They have the potential to benefit all stakeholders, from patients through to payers and even pharmaceutical companies themselves. Patients for example, are able to access potentially life-saving medicines when there are no other treatments available, whilst pharmaceutical companies can benefit from generating additional data, which in turn facilitates their pricing and reimbursement (P&R) negotiations with payers.
However, because of heterogeneity amongst HTA bodies in Europe, there is often confusion surrounding the EAP process and how it feeds into reimbursement decision making and historically there have been delays in getting reimbursement for EAP products. This article will focus on some of the key changes HTA bodies are making to help support patient access for products previously enlisted under an EAP, and what pharmaceutical companies can do to support.
Why do EAPs cause delays in reimbursement timelines?
Whilst regulatory authorities have become comfortable with granting early access to certain medicines using an immature dataset, reimbursement authorities have been slower to catch up. One might assume that additional data generated by early use of the product would be beneficial to the reimbursement process, however, because patients who use a product via its EAP do not have to follow strict inclusion criteria required for the trial population, the evidence base at the time of launch can often be contradictory and confusing for HTA agencies.
An example of this is demonstrated by Bosulif® (bosutinib) which was granted early access via conditional marketing approval in Europe in March 2013 for the treatment of chronic myeloid leukaemia1. However, once it achieved its marketing authorisation, delays from reimbursement decision makers spanned from 6-18 months2 as HTA agencies failed to adapt to the new and varied evidence base.
For Pharma this can have detrimental effects since innovative, high-cost therapies often lose commercial viability if launch timelines are considerably delayed. The result : less investment in these types of medicines, ultimately leading to the exact opposite of what early access originally set out to do.
How have reimbursement authorities adapted to mitigate access delays?
Across Europe, reimbursement authorities have recognised the importance of granting timely access to medicines which have been available to patients as part of an EAP. A great example of this is the UK’s Early Access to Medicines Scheme (EAMS) which ‘provides an opportunity for important drugs to be used in clinical practice in parallel with the later stages of the regulatory process’ 3. In essence, this means that the scheme allows patients access to products which meet specific criteria, before they have received marketing authorisation. Since its launch, the scheme has also helped to speed up reimbursement timelines once products end the EAMS and are subject to the standard reimbursement process through NICE (the National Institute of Health and Care Excellence). This may be because the scheme allows for considerable engagement with UK stakeholders to discuss the planned evidence collection and current clinical data package, thus allowing pharmaceutical companies to build longstanding relationships with stakeholders as well as gain advice into clinical trial design. In turn, this creates a stronger evidence package for reimbursement decision making and reduces the degree of uncertainty in the clinical data package ultimately speeding up the reimbursement decision making process.
However it’s not just the UK who are making big strides in helping to reduce reimbursement timelines for EAP products. In France, for example, the temporary use authorisation (ATU) scheme was overhauled last year (2021). The reforms allow France’s national reimbursement body, the HAS (the High Authority for Health – Haute Autorité de Santé), access to clinical trial data during the EAP, potentially shortening reimbursement timelines by allowing the Transparency Committee (TC) to utilise such data in their review (to read more on this, see our article titled ‘France’s ATU reforms. What does this mean for manufacturers?’).
What can Pharma do to support EAPs?
To support patient early access there are several opportunities available to pharmaceutical companies. For example, through the supply of products under an EAP free of charge which in turn helps to alleviate the budget impact to the respective health care system and increases the number of products accepted for early access. In addition, stakeholder engagement and early preparation for an EAP can help to reduce the downstream reimbursement process, leading to a smoother review process and ultimately ensuring that patients access the marketed treatment as early as possible and without unnecessary delay. It is therefore imperative that pharmaceutical companies engage with reimbursement authorities as early as possible, to bring awareness to the proposed EAP, answer any concerns or queries and eventually to help reduce reimbursement delays once marketing authorisation is complete.
To conclude, EAPs hold an opportunity to benefit all stakeholders involved, from the patients who receive the treatment to the pharmaceutical company who provides it. Despite this, EAPs have sometimes led to reimbursement decision delays because of heterogeneity across HTA bodies and a lack of understanding surrounding the EAP itself. However, we are seeing a shift in the industry and the UK market particularly is setting a strong precedent as to how regulators and reimbursement agencies can work together to help ensure that patients are able to access life changing medicines as soon as it is safe for them to do so.
- European Medicines Agency. Bosulif product overview. Accessed [11/04/2022]. https://www.ema.europa.eu/en/medicines/human/EPAR/bosulif
- Leyens L, Brand A: Early Patient Access to Medicines: Health Technology Assessment Bodies Need to Catch Up with New Marketing Authorization Methods. Public Health Genomics 2016;19:187-191. doi: 10.1159/000446537
- National Institute for Health and Care Excellence. Early Access to Medicines Scheme (EAMS). Accessed [14/04/2022]. https://www.nice.org.uk/about/what-we-do/life-sciences/office-for-market-access/early-access-to-medicines-scheme