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Early access to orphan medicines – a glimpse into the future

By Kieron Lewis, Director of Strategic Consulting

Following on from my blog post in early December, where I touched upon some of the key highlights from the World Orphan Drug Congress, I would now like to share some of the insights from Rob Fox, our Head of Business Development – Managed Access, presentation which provided a glimpse into the future of Managed Access Programs (MAPs). Also known as early access programs, MAPs are designed to provide patients with ethical, compliant and controlled access to investigational medicines before commercial launch in the country requesting access. Unsurprisingly, a large proportion of MAPs support the early access to rare and orphan drugs.

With over 30 years of experience in enabling early access to drugs, Clinigen has been a key player in the progression of the sector. To date, we have helped our pharmaceutical and biotech customers implement over 250 MAPs in over 130 countries worldwide. During this time, we have seen a lot of change and especially over the past decade there has been a big shift in how companies manage their MAPs. Herein, we have outlined how the industry is changing.


In 2007, 80% of our pharmaceutical customers had no policy on early access to medicines. Their approach to MAPs was very much reactive, with companies only responding and initiating programs due to pre-launch demand.

Nowadays, our customers have a much more proactive approach and want to discuss Managed Access options early in the clinical development cycle, usually when demand is anticipated ahead of launch. Many companies now see early access as a key part of their market access strategy, supported by the fact that seven of the ten medicines launched in 2017, which are predicted to be billion-dollar drugs by 2022, had an early access program in place before launch.

In 2017, 55% of all MAPs were implemented prelaunch. We predict this to increase to 90% by 2027. The expectation is that given the increased focus on rare disease and personalised therapy, the majority of companies will be initiating early access before the medicine is approved anywhere.

In fact, the 21st Century Cures Act, which was signed into US law in December 2016 states that pharmaceutical and biotech companies developing a phase II or III investigational product must have an expanded or compassionate/early access policy that they make public via their website.


In 2007, when the majority of MAPs were initiated post-first launch, 50% were owned by a member of the commercial team and there was a real need to educate on early access.

As MAPs have moved to be considered earlier within the product development cycle, we have seen a shift away from individual ownership (now only 15% led by commercial team members) to multiple stakeholders (such as medical, clinical, regulatory, supply chain, drug safety, patient advocacy and market access).

By 2027, we expect to see more internal early access experts within companies and separate functions to deal with MAPs which is increasingly common amongst our larger clients.

Patient selection

The most important aspect of MAPs are the patients and how they are selected for being eligible to participate in a MAP. Historically, this has been managed with the eligibility criteria being decided by the Chief Medical Officer or equivalent at the pharma/biotech company. If the same person also takes responsibility for granting exceptions, the eligibility criteria may not always match the needs of healthcare professionals and patients.

Nowadays, rather than only one person being responsible for decision making, we now see internal committees responsible for providing insights and decisions. Some companies are even appointing dedicated Chief Patient Officers, who can work with patient groups to more accurately predict who will be involved in early access and give patients a say in how MAPs are implemented.

With disease specific selection, we expect both internal and external parties to have an even bigger role to play in the future. These dedicated groups are likely to encourage more patient input to collectively formulate their inclusion and exclusion criteria.


Data collection can be extremely beneficial to the progression and direction of MAPs. In 2007, in addition to the required data collection on safety, the only other data collected was on which hospitals were accessing the drug, which physicians were prescribing to patients, and how many patients were being treated. A lot of the time this data wasn’t even used.

Today, Clinigen-initiated MAPs have seen a third of its customers capturing some element of real world evidence (RWE), i.e. additional clinical and efficacy data, to provide insights into how the drug is really being used and how effective it is.

We expect that, in the future, over three quarters of early access programs will be utilised to collect RWE. Also, with an increase in more personalised, high value therapies, RWE is going to be critical for getting drugs to market. We can also expect more technology to capture Quality of Life (QoL) data for patients and patients’ families.

Non-core markets

MAPs didn’t used to specify which countries they would run in. Often companies would open them up globally and just wait to see where they got demand from or respond to requests from outside the specified region on a case-by-case basis.

We are now seeing a more phased approach, with companies initially opening MAPs to a few core markets and then further once they have their first launch. We also see companies going a step further and using dual programs, known as the ‘International Pharmacy’ model.

In the future, companies will develop more defined strategies for these non-core markets and introduce teams that will oversee the entire portfolio of early access functions across the organisation.


We have seen huge changes over the last decade and differences to how companies approach early access, from very reactive to proactive. We expect there to be many more changes as companies further develop and adapt their MAPs so that they form an integral part of the commercial launch, especially to ensure they are underpinned with patient centric strategies.

To find out more about how Clinigen can help you with any aspect of your MAP, from creating a new policy to entering a new market, visit

Kieron Lewis Director of Strategic Consulting

Early Access
01 Dec 2017Insight Blog
Early Access to Orphan Medicines: Highlights from the World Orphan Drug Congress Barcelona, 2017
By Kieron Lewis Director of Strategic Consulting
  • Orphan Medicines
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  • Experimental Medicines