Overcome the challenges in orphan drug development with modeling and simulation

希少疾患治療薬(オーファンドラッグ)の開発には、臨床・規制・商業面において多様な課題を伴います。患者が少数であるため、臨床試験に向けた被験者の同定および組み入れは非常に困難です。

We have supported the approval of over 100 orphan drugs, including over 90% of new novel drug approvals with orphan designation in the past 6 years by the US FDA with our software or services. As the global leader in model-informed drug development, we have worked on more rare and orphan drug development programs than any other consultancy.

 

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Challenge: Small patient pools

Solution: Our expertise in clinical pharmacology strategy and modeling and simulation (M&S) services enable us to quantify drug-disease-trial and exposure-response models from small patient populations providing insights into biomarkers and endpoints.

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Challenge: Small patient pools
Challenge: Sensitive patient populations

Challenge: Sensitive patient populations

Solution: We can model the impact of a drug in development on other disease states or untested populations. These proven quantitative methods are actively encouraged by global regulators who have demonstrated extraordinary flexibility in approving orphan drugs either through an accelerated approval pathway, the elimination of a second pivotal clinical trial, or applying M&S in lieu of certain trials.

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Challenge: Unique regulatory landscape

Solution: Experience in early and careful planning is critical for rare disease development. The Certara regulatory team has more than 200 experts and writers with proficiency in orphan global filings, as well as a deep understanding of how to articulate the results derived from modeling and simulation. We provide quantitative and regulatory expert guidance and active support from pre-IND meetings through clinical development and postmarket approval.

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Challenge: Unique regulatory landscape
Reimbursement challenges

Reimbursement challenges

Solution: Certara develops a quantitative Multicriteria-decision analysis-based framework adapted to the very specific issues in rare disease development and access including quality of evidence, disease severity, ethical considerations, population-level factors, economic impact of the disease, and specific budgetary impact. By combining disease information, patient input, real world data, and innovative pricing and contracting strategies with the wider social and health benefits, we build robust, evidence-based results to justify market access and pricing.

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