What processes exist to promote early access to promising medicines?
Early access programmes represent an opportunity to improve health outcomes for patients under exceptional circumstances and can enable accelerated access to promising medicines. However, it can be difficult to determine when early access to an unlicensed medicine is appropriate. This newsletter will discuss the early access processes operating within the UK and discuss the advantages and disadvantages of these systems.
Early access programmes vary across the European Union, but generally promote access to promising medicines prior to marketing authorisation, where there is a clear unmet need. This is usually reserved for patients with life-threatening or debilitating conditions, where there is no effective treatment and where the patient is not eligible to participate in clinical trials, because of age or comorbidities for example.
It is recognised that in exceptional circumstances, the benefits of an unlicensed medicine to a patient may outweigh the risks. After careful consideration of the evidence, should the physician and patient deem that the use of an unlicensed product is appropriate, early access processes should be in place to support improved health outcomes under these exceptional circumstances.
Early access processes in the UK
Within the UK, there is a voluntary scheme in which a scientific opinion from the Medicines and Healthcare products Regulatory Agency (MHRA) can be applied for, which weighs the benefits and risks of a product in order to promote more informed prescriber decision making. This opinion does not replace normal licencing procedures and is only valid for one year, although renewal of the opinion is possible.
The process consists of two steps, the application for promising innovative medicine (PIM) designation and subsequently, the application for an early access to medicines scheme (EAMS) scientific opinion.
Figure 1. The UK early access to medicines scheme is a two-part process, which requires application for promising innovative medicine designation and subsequently the EAMS scientific opinion.
To apply for PIM designation, several criteria must be met:
- The condition must be life-threatening or seriously debilitating
- There must be a high unmet need, with existing treatments either not being available or having serious limitations.
- The new medicine must have a substantial advantage over the current methods utilised in the UK and the benefits of the medicine must outweigh the potential adverse effects.
Once an application for PIM designation is submitted and reviewed, a designation meeting will be arranged to discuss whether the criteria are fulfilled. Assuming the award of a positive designation letter, a further application may then be submitted for EAMS scientific opinion.
A positive scientific opinion requires the medicine to meet the same criteria as for the PIM designation, with the additional requirement that the company can supply the product and manufacture it to a consistent quality standard. Prior to application, the company must attend a pre-submission meeting to confirm that it is likely that the criteria will be met, before the EAMS dossier is submitted. Assuming all criteria and requirements (e.g. pharmacovigilance) are met and the application is successful, a positive EAMS scientific opinion may be published.
Publication of an EAMS scientific opinion carries a range of benefits. It encourages informed decision making by prescribers and early access to promising medicines for patients. Furthermore, it encourages early dialogue between the pharmaceutical company, the NHS and NICE to discuss how the product will be used in practice. Opportunities exist to discuss data collection plans for the generation of real-world data with NICE, with a NICE EAMS meeting offering a potential avenue for companies to prepare for the technology appraisal of their product. These data collection activities could help to better inform decision making. However, due to the short timelines involved it is expected that the real-world data generated may be limited.
Currently, early access supply of medicines is expected to be free, with no funding in place to cover the cost. This can pose a major hurdle for some companies who may have concerns that the benefits do not justify the cost. Despite this, due to the difficulty in assessing cost-effectiveness prior to a health technology assessment, funding is unlikely to be implemented in the foreseeable future. A major advantage to undergoing the EAMS process is that following a positive technology appraisal, accelerated patient access can be expected based on faster implementation. It could be assumed that physicians and patients would have a greater familiarity with the product than if it had not undergone early access.
Overall, the early access to medicines scheme in the UK has a range of advantages and disadvantages. It is hoped that under the correct circumstances, it can promote more informed physician decision making and accelerate patient access to innovative products where appropriate, leading to improved health outcomes for patients.