So, you have a novel therapeutic progressing well through clinical trials but are already receiving access requests for numerous markets from patients you know could benefit from access to your product.

This is a common occurrence now with many pharma companies facing the dilemma of how to support patients who do not have an alternative treatment, do not qualify for the clinical trial and in many instances suffer from a serious debilitating if not life-threatening condition?

So, when do companies consider the whole question of early access. Many are already engaging at phase III/IIIb while others consider them as early as during phases I and II, it’s the ole adage it’s never too early to start thinking about your early access strategy!

Access programs can be used to widen access to patients who do not meet the eligibility criteria for a clinical trial, or even continue access for patients who have completed a clinical trial. Even while marketing approval has been granted and pricing negotiation and final availability of a commercial product can take several years in some markets, so an access program works there too.

And what is to be gained… what are the benefits to both the pharma company and the patient of running an access program.

Most importantly they allow access to be provided in an ethical and compliant manner allowing clinicians to meet the needs of their patients. For the patient it can be a lifesaving opportunity, as many do not qualify for what can be quite specific criteria for a clinical trial population. The early access patient will tend to be more representative of the real-life patient the drug is eventually going to be used with, so early access provides a better reflection of the realities of its use in the future.  For the pharma company there is an opportunity to build relationships with potential future prescribers, patient advocacy groups and real-world data collection which may even be used in regulatory submissions. In some markets there is the possibility to charge for product, but care should be taken in setting a benchmark price for the future and really the driver should be patient access not commercial return.  So, it can be a very patient centric approach where the pharma company can set inclusion and exclusion criteria and not erode clinical trial potential population, as only no qualifying clinical trial patients should be considered.

There are always two sides to every coin so what are the challenges to be weighed up if you are thinking about early access. The single biggest hurdle is the regulatory environment whilst in the past 10years no single piece of legislation in any market has sought to restrict access, the interpretation of how that increased access plays out in every market is far from unified. Take Europe for example you have essentially 4 different procedures to choose from to gain marketing authorization, National procedure, mutual recognition procedure, centralized and decentralized procedure. Underneath that for early access you have countries in Europe with their own national procedures, those that follow European Medicines Agency guidelines and several including the UK shortly who sit outside of EMA but treated as European market. Once you have picked through the peculiarities of each procedure and digested hurdles to achieve access in each you will have a range of country approaches all working to different timelines to manage. When commercialization does happen, it is unlikely to be in every market at the same time, so a pharma company needs to be prepared to manage both licensed and unlicensed supply over multiple markets.

There is also the risk of an adverse event tripping up a future commercial launch so how access programs are run, and patient eligibility criteria are set is critical. There are requirements for safety and adverse event reporting which also must be managed. Whilst a commercial launch is always in the distance the regulations around early access mean products cannot be promoted so governance on this issue need to be in place. Patient demand can also be challenging, and the supply needs of a potential parallel clinical trial also need to be considered need. There can be perceived inequality if access programs are only available in countries where product can be charged for.

Access programs can be a huge success for both the Pharma company and the patients involved but it may be wise to seek support from a specialist provider such as Sciensus Rare who have experience of running multiple programs across many markets.

Sciensus Rare will support you with a full regulatory assessment of the countries you are interested in running an access program. This will cover advice on type of access program most suited to your needs, whether it can be charged for or not, what data you may be able to collect. We can also advise on your communication strategy. You will be supported by a dedicated project manager, and a multilingual customer service team.

We will ensure health care professionals are properly validated and given access to our online ordering portal Sciensus Axcess. Registered healthcare professionals will be able to view documentation for the access program including product information and any relevant training materials.

Our logistics teams will ensure product is delivered most of the time within 24hrs of order confirmation. We will ensure there is full track and trace capability to allow auditing of all activity. We can provide a full order to cash service. We will deal with any issues in an efficient, ethical and regulatory compliant manner. If you require additional support for your patients, we can advise on a range of support services via our nurse lead patient support programs. We can support your clinicians with Individual funding requests as required. You will be provided with full transparency on the activities we carry out on your behalf with accompanying adherence to a range of mutually agreed KPIs.

When your access program completes as you move to commercialization in that country, we can also handle all your support needs for commercial supply.

If you wish to discuss your requirements in relation to Early Access any do not hesitate to contact Andrew Cummins at Andrew.cummins@sciensus.com