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European Pharmaceutical Contractor

Think Global

Close your eyes and stick a pin randomly into a map of the world. That’s where you might find a patient in urgent need of a new therapeutic option.

But what if there are no clinical trials in close proximity, or the eligibility requirements are too limiting? What if the patient lives in a country where the drug will never become available? Or perhaps the wait for approval will simply be too long.

When patients with unmet medical needs are unable to access a drug via a clinical trial or commercial channels, a global access programme can be an effective way to ensure the medicine reaches the patient. 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 process.

Around the world, these programmes are referred to by a number of terms, including compassionate use, named patient use, expanded access and early access. Regulations governing this type of access are typically established by local competent authorities, which define the parameters by which programmes can be established and how they must operate.

It is clear that these programmes can positively impact patients and provide a critically important solution for their treating physicians. For example, the former French Health Products Safety Agency (now the National Security Agency of Medicines and Health Products, ANSM) reported that by 2007, more than 20,000 patients had been treated with over 200 products under compassionate use legislation introduced in 1994 (1).

Where they exist, regulations governing patient access outside clinical trials and commercial supply provide much needed structure and guidance in an area which is generally not well understood by drug manufacturers, physicians and patients. Combined with an in-depth understanding of nuances and local best practices, a robust global access programme can be effectively established and implemented.

Not only do such programmes afford companies the opportunity to meet ethical obligations and goals, but they can also offer patients a potentially life-saving or life-extending treatment option, ahead of its commercial availability. In addition, they are highly effective in helping foster positive relationships with key opinion leaders and treatment centres, as well as providing opportunities to gather limited, yet valuable, information about the use of drugs in wider populations.

Starting Point

If your organisation is considering a global access programme, a number of topics should be addressed at the outset by a cross-functional team including representatives from regulatory, clinical, medical affairs, commercial, quality, procurement, legal and finance.

When developing a programme, initial discussions with internal stakeholders should focus on how the company wants to allow access and include the following aspects:
  • Geographies in which the programme will operate
  • Patient eligibility requirements
  • Pharmacovigilance and safety reporting requirements and processes
  • Processes by which physicians will request access and the documentation they will need
  • Training that will be needed for local affiliates, medical directors and others
  • Timing and exit strategy for completion of the programme
As a drug progresses through the trial process, unsolicited requests for access are likely to be received – especially if the drug represents a breakthrough therapeutic option. Combining information on these requests with market research will help define the appropriate geographic reach for a programme.

Details such as the time and resources needed to gain regulatory approval, the proposed indications, the number of patients to be treated, the need for a local wholesaler or agent, and the applicable import regulations also need to be considered and planned for ahead of time.

Where the drug resides in the product lifecycle is also an important factor in determining the possible routes for unlicensed supply. How available the product is in the marketplace, if it is licensed elsewhere and if clinical studies are complete can all affect the way in which a drug can be imported, if at all. Regulations can define if a company can charge for the product – this can be an important point of discussion when developing the scope of a programme.

Once a programme launches, it is likely to evolve, especially for drugs that are early in the development cycle and not yet licensed. Thus, the programme needs to have processes in place to ensure regular and timely review of all relevant information and materials. As new safety information becomes available, documentation used as part of the programme will need to be updated. Similarly, as approvals are obtained, patients in those countries will be transitioned from the unlicensed supply to commercial packs.

Regulatory Environment

Regulations governing global access programmes are typically set by local competent authorities and define the parameters by which access can be established and how programmes must operate. Unlike regulations governing the clinical trial process, which are very structured, the regulations related to unlicensed medicines can vary considerably from country to country.

For example, in the European Union (EU), the legal framework for providing unlicensed medicines is outlined by top-line legislation (Article 83 of EC Regulation No. 726/2004). Its objective is to:
  • Facilitate and improve the access of patients in the EU to compassionate use programmes
  • Favour a common approach regarding the conditions for use, distribution and patients’ targeted use of unauthorised medicines
  • Increase transparency between member states in terms of treatment availability
However, this regulation is interpreted in various ways by each of the member states. In 2010, the European Clinical Research Infrastructures Network published results of a survey on the regulatory requirements of compassionate use programmes (2). The results demonstrated the range of different requirements adopted by each of the countries, clearly showing that compassionate use is not interpreted in the same way across Europe. The authors concluded that “there are more differences than similarities in ‘compassionate use’ programmes in Europe.”

Adding to the complexity created by country-specific regulations, there are also inconsistencies in the way in which the process can be governed on a national level. National agency opinions can vary according to the particular assessor involved.

Given the myriad rules and regulations – as well as nuances in their interpretation – it is important for a company to have a thorough understanding of what is permitted in each country where an access programme will operate. For countries that are well-established in this area, such as the US, UK and France, the rules are clear. However, in those geographies where programme parameters are evolving or not firmly defined, setting one up can be challenging. Simply identifying the established legislation is not enough, as exceptions are often possible and the practicalities of local import processes must be well understood.

An additional point to consider is that global access is an evolving landscape. Countries with long-standing regulations may update their rules in an effort to clarify legislation and improve the facilitation of access. Regulations in the US, for example, were clarified by the Food and Drug Administration in 2009, when new types of access for treatment use were added to ensure “broad and equitable access to investigational drugs for treatment.”

Some countries with very basic regulations or no established supply routes are strengthening or establishing access routes. In some cases, this evolution in regulation is being driven in response to pressure from patients themselves, as well as advocacy groups. However, changes are not always quickly implemented, not only due to local bureaucracy and political procedures, but also because care must be taken to ensure that access to unlicensed drugs remains in the best interests of patients and the supply is not open to misuse.

Critical Success Factors

Clearly, global access programmes can offer significant benefits for patients in need and their physicians. Several factors are critical for the success of a programme which, when addressed properly, will help deliver the greatest impact.

Define the Aim
At the outset of a global access programme, it is essential to clearly define, agree upon, and communicate its aim within the organisation. As the programme progresses, more internal groups, such as 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 programme, demand is likely to grow and may come from a wide range of geographies. In parallel with a growing demand, patients who may not fit the 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 need to ensure consistency and fairness in delivery of the programme, and control costs. As it progresses, a regular review of milestones, successes and areas for improvement should be conducted.

Engage Internal Teams
During the set-up of the programme, internal resources are required to put safety, education, data collection and reporting requirements in place, 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 – particularly 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/chemistry and manufacturing controls groups is essential.

With a wide range of internal stakeholder needs and input to balance, it is necessary 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 to ensure success.

Communicate Effectively
The establishment of an access programme is likely to create interest among internal groups, beyond those directly involved with its set-up, implementation and conclusion. 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.

Conclusion

When it comes to meeting the needs of patients around the world, the well-known counsel to ‘think globally, act locally’ rings true. Access programmes should consider the big picture – the geographic spread of patients – as well as their individual needs and the unique barriers to access each patient faces. A well-defined, well-communicated global access programme that addresses both areas will deliver important benefits to all participants.

References

1. Compassionate use in Europe: A patchy framework for early market entry, Jones Day, August 2010. Visit: www.jonesday.com/ compassionate-use-in-europe-a-patchy-frame-work-for-earlymarket- entry-08-20-2010

2. Compassionate use of interventions: results of a European Clinical Research Infrastructures Network survey of ten European countries, Trials 11(104), 2010. Visit: www.trialsjournal.com/ content/11/1/104

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Mark Corbett joined Clinigen in 2010 as Senior Vice President of Clinigen Global Access Programs. He has overall responsibility for Clinigen’s Global Access Programs Division, which specialises in the consultancy, development, set-up and implementation of access programmes on behalf of the biotech and pharmaceutical industries. Mark has gained extensive knowledge and operational expertise in the implementation of more than 120 global early access/named patient programmes for a variety of clients, ranging from niche biotech to global pharmaceutical companies.
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