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14 Jan 2019 Insight Blog

Current Trends in Early Access

By Neil Watson, Director - Global Business Development

Current trends in Early Access

Clinigen chaired a roundtable on Early Access at the Early and Managed Access Programmes Europe conference in London late last year.  The conference was an informative event, well attended by pharma and biotechs, as well as healthcare professionals and advisers, with much debate on the current trends and attitudes towards providing access to treatments that are currently unlicensed.

The programme with many names

Known as ‘Named Patient’, ‘Compassionate Use’ or ‘Early Access’ one of the first challenges is navigating the complicated nomenclature that has been attached to this specialised space in the development lifecycle of a prescription medication. 

Wrapped in country-specific regulations designed to protect both patient and drug developer, many companies have established their own lexicon which can further add to the confusion both inside and out. Leaving the regulatory specifics to the professionals and using the term ‘Managed Access’, coined by Clinigen, helped to focus the discussion and establish a common theme!

Key points included:

  • Organisational size and profile has a huge influence on whether pharmas or biotechs will provide access to their highly valuable assets
  • Resource is important - larger corporations can often provide access at no cost to the patients
  • Smaller biotechs with multiple demands on their cash may have limited room for access versus their organisational survival
  • Biotech representatives felt that this often forces them to limit access to solely the clinical trial environment, based on the concept of future treatment for the many rather than early access for the few
  • All agreed that regardless of size, corporate alignment is essential. The decision to provide access to an unlicensed treatment will involve virtually every part of an organisation from reception to medical affairs, clinical development to supply chain
  • Taking the time to collect and collate feedback from all stakeholders and establish a policy which can be disseminated both inside and outside the company is critical. In the US it is a mandatory part of the ‘21st Century Cures’ Act

Access models

Once the access decision has been taken there are many overriding principles which may determine how, where and to whom access is provided. Proof of efficacy is usually at the forefront, but commercial hurdles will always be present with some companies only granting access in countries where the drug will eventually be filed.

Clinical trial authorities now often encourage drug developers to document how they will manage the provision of treatment to chronic patients once their involvement in a trial has ended so broad brush principles may also be applied to the type of patient too.  Patients who have contributed to the clinical development in a trial may be provided ongoing, seamless free of charge access until the treatment is commercially available through a protocol-led approach whereas ad hoc patients not previously involved may get access through a less rigorous regulatory framework.

It is widely recognised that a Managed Access approach for patients comes with significantly less oversight than for instance on open label extension study, meaning access can be provided with lower overall infrastructure costs.

Real World Data

Providing access to its unlicensed drugs can give a developer a whole new world of useful information. Unlike the protocol-restrained environment of a clinical trial, the Managed Access environment can provide privileged access to a potentially huge pool of real-life patients.

However, delegates commented that the confusion surrounding the capture of real-world data in this patient population is holding back developers from taking advantage of this unique opportunity. The regulatory framework has been interpreted differently in different countries, which also applies to taking and using data. Some authorities will demand that a trial is undertaken, whereas others have a more pragmatic approach, more aligned with the workload of the treating physician. As global dissatisfaction with the trial process gains momentum, more agile sources of competently collected and analysed data may be encouraged, both by regulators and developers. This is another reason for trusting experts to set up and run Managed Access activities.

To outsource or not to outsource

The decision to outsource versus in-house is always based on a compromise between available resources creating apparent cost efficiencies and the need to pay for the required expertise that doesn’t reside in the organisation.

Delegates felt that this dilemma is best resolved by looking at the cost of failure and the time taken to learn whilst implementing internally. Outsourcing to experts and maintaining organisational flexibility can reduce fixed costs and speed treatment to patients in need.

Buying in expertise enables a developer to benefit from the combined learning of multiple organisations and multiple programmes. However, some organisations feel that they know their asset better than anyone so they alone should manage its availability. This was also a misconception voiced in the 90’s before the growth in outsourcing clinical development to CROs who now frequently understand the asset better than the developer, but with limited negative consequences.

So what are drug developers looking for in a Managed Access provider?

The messages from the delegates were consistent with the feedback we regularly get from our customers:

  • Managed Access providers should be experts in the provision of up to date regulatory advice interpreted correctly on a country by country basis
  • Developers want help navigating the complex and ever-changing regulatory landscape internationally, so it’s great if a provider can liaise in local language with local affiliates
  • With the advent of new technologies such as cell and gene therapies, the ability to stay close to regulators and even work with them to develop regulatory strategies will be vital
  • Interpretation of legislation, including labelling requirements is essential as well as helping to convince the authorities on what can be done in their country
  • Developers need to be assured that their provider has a real understanding of the distribution pathways in various countries
  • Providers must have good knowledge of the supply chain processes in every country, particularly the relevant customs and import rules

The Clinigen difference

The round table was a useful opportunity to hear directly from the drug developers what they want and need from Managed Access programmes, and the regulatory and resource challenges that go with them.  We look forward to continue to support pharma companies to navigate through some of these difficult and important challenges in providing access to unlicensed medicines.





Neil Watson Director - Global Business Development