Small patients, big expectations
How to ensure PIPs are feasible
When embarking on paediatric projects, clients often find themselves confronted with a scarcity of available data. Since 2013, CDDI has been filling this gap by providing real-world data on children who are seen by expert clinicians at a representative sample of hospitals around the world.
Clients are using this data to inform their Paediatric Investigational Plans and negotiations with PDCO.
There are two things that usually happen as a result of this process.
The client gains confidence with its planning and PIPs agreed with PDCO, based on realistic data. The participating clinicians say: “This data should be published!”.
Why are PIPs so challenging ?
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PIPs are required to determine safe and effective doses of drugs in children…
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…but the indication in children may be different from that in adults.
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Age-appropriate formulations should be available.
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Paediatric populations with specific indications are often smaller in comparison to adults.
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Protocols in paediatric studies usually progress down age-specific cohorts, further limiting the population size.
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Pharmacokinetic sampling involves timed blood samples, which can be problematic and unpopular and blood draw samples are limited.
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Obtaining parental informed consent (and child assent where appropriate) is stressful for all parties.
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Accrual is usually slow, meaning many sites may be required.
Where do we come in ?
We provide data to inform the design of the PIP to support negotiations with Regulators and to ensure the agreed plan is feasible in the agreed time.
Our proprietary process, called Evidence-based Feasibility (EbF), uses retrospective, de-identified, real-world data to assess the feasibility of a study concept or draft protocol.
EbFs help de-risk PIPs, preventing the need for amendments and ensuring timely completion which can have significant commercial implications.
If you would like to discuss how we can help you with your PIP, please contact us here.