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 ?
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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.
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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.
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EbFs help de-risk PIPs, preventing the need for amendments and ensuring timely completion which can have significant commercial implications.
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If you would like to discuss how we can help you with your PIP, please contact us here.