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Enabling access for patients with rare diseases

When it comes to meeting patient needs, the well-known counsel to ‘think globally, act locally’ rings true

Rare diseases orphan drugsThe statistics associated with rare diseases are well-known. In Europe, a disease or disorder is defined as rare when it affects less than 1 in 2,000 individuals. In the US, the definition is fewer than 200,000 Americans. Collectively, an estimated 7,000 rare diseases affect 250 million people worldwide, including 30 million Europeans. 

In recent years, the topic of rare diseases has secured a rightful place in the spotlight. Social media channels have allowed individual patients to connect and amplify their need for new treatments, while thousands of patient organisations offer support, education and advocacy. Large pharmaceutical companies such as GlaxoSmithKline and Pfizer have established rare disease divisions, and small and mid-sized drug developers continue to advance their pipelines. 

Despite the groundswell of attention, addressing the medical needs of patients with rare diseases remains a challenge. In response, new models are emerging for accelerating drug development and enabling greater, more timely access to those therapeutics. Industry is working more closely with patient advocacy groups and is adding to existing capabilities that had traditionally focused on very large patient populations. 

Creating global access 

While millions worldwide are affected by rare diseases, gaining access to a new therapeutic can be an individual endeavor for these patients. With a small patient population, thinly spread across potentially dozens of countries, geography, timing and logistics can become the enemy of access. 

There may be no clinical trials in close proximity to the patient or the eligibility requirements for the trial are too limiting. The patient might live in a country where the orphan drug will never become available or the wait for approval could simply be too long for those with rapidly progressing or life-threatening conditions. 

Companies recognising this need and the urgency of it leverage global access programmes to provide access to medicines that would otherwise be unavailable. These programmes follow ethical and regulatory compliant mechanisms, enabling patients to gain access to medicines that are in clinical development, are unlicensed in a specific market but licensed elsewhere, or are in the marketing authorisation application (MAA) process. 

These programmes are referred to using a number of terms including compassionate use, named patient programmes, expanded access and early access, to name but a few. Despite the varied nomenclature, regulation is typically established by local competent authorities, and although the processes vary considerably around the globe, there is almost always a nationally defined route to enable access to unlicensed medicines. 

Critical success factors

Global access programmes can offer significant benefits for patients in need, and in many countries the regulations defining them are well-established. In other countries, however, the situation may not be so clear. The regulations that are in place differ significantly from country to country, can evolve over time, and may leave room for interpretation. Further complicating the process is a large number of stakeholders - both internal and external to the company offering the access programme.

Given this framework (or lack thereof), a number of factors are critical for the success of a global access programme and, when addressed properly, will help deliver the greatest impact.

Define the Aim: At the outset of a global access programme, it is important to clearly define, agree upon, and communicate the aim of the programme within the organisation. As the programme progresses, more internal groups (eg, affiliates) will become aware of it and may want to influence its direction or change the scope. As patients and patient organisations learn about the program, demand is likely to grow and may come from wide range of geographies. In parallel with a growing demand, patients who may not fit the programme's eligibility criteria might seek access. All of these situations must be considered and planned for when structuring the programme. 

While programmes can evolve and expand over time to better meet patient needs, it is important to remain mindful of the agreed aim, to ensure consistency and fairness in delivery of the programme and control costs. 

Engage Internal Teams: During set-up of the programme, internal resources are needed to put in place safety, education, data collection and reporting requirements, define patient eligibility and screening criteria, and determine geographic reach. Typical groups include regulatory, clinical, medical affairs, quality, procurement, legal and finance. Commercial teams are also an important part of the discussion. 

The availability of product to support the programme should be an early consideration, in particular, for those products where commercial supply is not yet established, such as those which are still in development and do not have a marketing authorisation in any territory. In such a situation, a product may be in short supply and lead times can be lengthy, so early involvement of supply chain/CMC groups is essential. 

With a wide range of internal stakeholder needs and input to balance, it is important to consider how each of these groups will be managed as part of the Global Access Programme. Seamless coordination of key functions, along with timely reporting of programme metrics back to stakeholders will help ensure success.

Communicate Effectively: Establishment of an access programme is likely to create interest among internal groups beyond those directly involved with its set-up, implementation, and how the programme will ultimately conclude. As such, it is important to consider how, when, and what is communicated to a broader range of internal stakeholders. Consider local affiliates and medical directors. They will be interested in understanding what the impact of the programme will be in their countries, if and how they will be involved, and how the process will operate. Physicians, patients and patient organisations will want to know if, how, and when access may be available. A robust communications strategy that is delivered proactively will properly set expectations and minimise confusion. 

When it comes to meeting the needs of patients with rare diseases, the well-known counsel to 'think globally, act locally' rings true. While there are thousands of rare diseases and millions of patients around the world who struggle with them, strategies to define access should balance the global and the local. Access programmes must consider the big picture, the geographic breadth of patients, as well as individual needs and the unique barriers to access that each patient faces. A well-defined, well-communicated global access programme that addresses both will deliver important benefits to all participants. 

Mark Corbett is Clinigen's Vice President, Global Access Programmes (GAP). He can be reached at

Author's note: This article is the first in a series on global access programmes. Subsequent articles will discuss the medical, clinical and regulatory aspects of such programs as well as logistical requirements



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