UTILISING HUMAN CHALLENGE TRIALS

As A Tool In Expedited Review Programs For Infectious Diseases

Expedited review programs in Europe

In 2016, PRIME (PRIority Medicines) was launched by the EMA, with the objective to strengthen support for medicines that target an unmet medical need.

The scheme focuses on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options.

The PRIME scheme offers the below advantages:

• Appointed rapporteur from the Committee for Medicinal Products for Human Use (CHMP) / Committee for Advanced Therapies (CAT)

• Organise a kick-off meeting with the rapporteur and a multidisciplinary group of experts, so that they provide guidance on the overall development plan and regulatory strategy

• Assign a dedicated contact point

• Provide scientific advice at key development milestones, involving additional stakeholders such as health-technologyassessment bodies, to facilitate quicker access for patients to the new medicine

• Confirm potential for accelerated assessment

What is the added value of Human Challenge Trials?

In each of the expedited pathways listed above, the key element is the availability of early clinical efficacy data.

HCTs are an ideal tool to collect these data on infectious diseases. They allow the collection of efficacy data to support the evidence that the medicine shows promise in serious conditions and, where applicable, show superiority over existing treatments.

The efficacy data is collected in a well-defined model, without the ‘noise’ of a field trial. This allows the collection of ‘crisp’ data while being able to focus on the required clinical outcomes.

A number of RSV vaccines currently under development, including the J&J, Pfizer and Bavarian Nordic vaccines, received breakthrough and/or PRIME designation based on preliminary clinical evidence collected in a Human Challenge Trial.

An additional advantage is that the data is collected quicker than if they would have been collected in a field trial, allowing drug developers and regulators to make faster decisions on candidates and progress those that have a greater chance of improving public health.

Conclusion

A designation in an expedited review program gives a strong advantage in your development pathway. For infectious disease medicines, a Human Challenge Trial can provide the needed early efficacy data which grants the required designation, both in the United States and Europe.

BRUNO SPEDER, VP of regulatory affairs & consultancy services, hVIVO, discusses how Human Challenge Trials can be used in the framework of expedited review programs as part of drug and vaccine development.

Kimberly Remillard, RAC, senior regulatory affairs manager at Thermo Fisher Scientific, examines how leveraging the power of Pharma 4.0 can ensure data integrity within biopharma.