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Ready for takeoff?

A new pathway promises expedited approvals for innovative medicines
Ready for takeoff?

A new expedited approval pathway in Europe expects to allow innovative medicines to reach the market at a pace on par with US accelerated assessment pathways.

 Priority Medicines, or PRIME, was launched by the European Medicines Agency (EMA) earlier this year in an effort to accelerate the medicine approval process in the European Union. It focuses on priority medicines, which are those that may be significantly better than existing treatments or treat diseases where no options currently exist. EU regulators modelled PRIME on the FDA's Breakthrough Therapy programme in the US.

 Early data is a key requirement for gaining PRIME eligibility. Based on early clinical data, PRIME applicants must demonstrate how their medicine will improve a patient's quality of life. Once selected, PRIME applicants will receive support from the EMA earlier in development with the goal of improving clinical trial design for marketing authorisation application (MAA), evaluation and long-term commercial prospects of PRIME medicines.

EU accelerated pathways before PRIME
To understand how PRIME came into existence, it's helpful to know about PRIME's predecessors and their limitations. These regulatory pathways include Accelerated Assessment, Conditional and Exceptional Marketing Authorisation, Compassionate Use Programmes and the Adaptive Pathway.

Accelerated Assessment created an accelerated approval process of medicines that were of major public interest or therapeutically innovative. Eligible companies submit an Accelerated Assessment application two to three months prior to submitting an MAA, and it is highly recommended that they request a pre-submission meeting at least six months before the MAA submission. Although the Accelerated Assessment pathway reduces the marketing authorisation evaluation process, the complexity of the approval process has limited the number of accepted medications to date.

Conditional and Exceptional Marketing Authorisation is available for expedited review of products that prevent, diagnose or treat seriously debilitating or life-threatening diseases, as well as those that treat orphan diseases or are used to provide emergency care. To qualify, applicants must demonstrate a favourable benefit-risk ratio. Under the Exceptional Marketing Authorisation route, data is reviewed annually and a full dossier of data is not required. For a Conditional Marketing Authorisation, scientific advice or protocol assistance is available early in a product's development. Unlike an Exceptional Circumstances MAA, Conditional Marketing Authorisations are expected to eventually provide a full dossier of data post approval. Although Conditional Marketing Authorisation may benefit public health, few companies have chosen this approach.

A more efficient pathway will allow innovative medicines to reach the market much faster

Compassionate Use Programmes (CUPs) are designed for patients with no satisfactory treatment options or who cannot enter clinical trials. CUPs allow sponsors to make products available without an authorisation. Although the EMA provides suggestions through the Committee for Medicinal Products for Human Use, CUPs are governed by different member states in Europe. Currently, there are five ongoing CUPs throughout Europe that have included the Committee for Medicinal Products for Human Use's recommendations, as well as more nationally. However, many eligible patients are unaware of CUPs due to a lack of publicity.

The most popular of the early access options is the Adaptive Pathway Pilot Programme, a precursor to PRIME. This programme promotes innovation and early access to medicine by allowing companies to gain conditional approval early on in the development process. Data is gathered on a rolling basis, beginning with smaller patient populations and then expanding to more patient groups. Data is gathered from real-life use and added to clinical trial data, which is reviewed by patient groups and Health Technology Assessment bodies. Although only 10 applications have been accepted for the Adaptive Pathway, 34 applications have been received since the programme's inception in 2014.

A new track for innovation
By aiding companies with EMA expertise and knowledge early in the developmental process, the PRIME pathway aims to create an expedited medicine approval process. A more efficient pathway will lower regulatory hurdles and development costs, as well as allow innovative medicines to reach the market much faster. All sectors within the pharmaceutical industry will benefit from greater access to innovative new medicines across the EU.

Article by
Anusha Bala, BSc, LLB

is global head of regulatory affairs at Novella Clinical

10th November 2016

From: Regulatory



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