Our in-house clinical experts have considerable experience in developing therapies for rare diseases and paediatrics, with an emphasis on innovative programmes.
We work with our clients to conceive a drug development programme which utilises all the clinical, regulatory and statistical approaches available and integrates this with Aparito’s tools for collecting patient generated data to support efficacy and safety endpoints. Our aim is to provide a streamlined drug development programme, reducing the number, complexity and duration of the clinical trials.
We endeavour to make use of the EMA’s Adaptive Pathways, which itself utilises Parallel Scientific Advice, Conditional Marketing Authorisation (perhaps with biomarker endpoints), Marketing Authorisation under exceptional circumstances, Risk Management Plans, centralised compassionate use and patient registries.
By integrating Aparito technology into a drug development programme from the very start, we also collect healthcare utility data, and patient preference or disease impact can be integrated seamlessly from early phases of drug development. This provides a rich dataset that can be used as part of a MAA as well as economic analysis for HTA and payer discussions.
At Aparito our experts have over 20 years’ experience of regulatory strategy for rare diseases and paediatric therapies.
We know how important regulatory buy-in is for every drug development and understand the common pitfalls that lead to unnecessary road blocks and delays, such as not having a sound regulatory strategy, not engaging with regulators early on and taking on board their recommendations, poor trial design and poor choice of endpoints
We provide support for high level strategic regulatory advice and buy-in around regulatory requirements and clinical development plans, whilst marrying innovative digital tools with regulatory demands.
We can help with:
Developing strategic plan and gap analysis to reduce risk
Scientific Advice (and Protocol Assistance) for Paediatric & Rare diseases (National and EMA)
Orphan Designation applications, PIPs, PRIME
EMA / HTA parallel scientific advice
EMA / FDA joint scientific advice