Streamline 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。また、希少疾患の多くは遺伝性です。加えて、希少疾患の患者は多くの場合に複雑な表現型を有し、提案される治療計画に対する応答が患者によって大きく異なります。疾患が十分に解明されておらず、臨床評価項目・バイオマーカー・アウトカムなどの評価基準の設定が困難となる場合もありえます。患者集団には、新生児から小児、併存疾患を抱える患者など、慎重を要する部分集団が含まれるため、臨床試験を実施すること自体が倫理的に問題となる場合もあります。

M&S 手法は、オーファンドラッグ開発において理想的なアプローチであるだけでなく、規制当局にも活用が奨励されています。M&S は小規模な集団を対象とした薬物 - 病態 - 試験モデルや曝露 - 応答モデルに基づく定量化により、バイオマーカーおよび評価項目に関する知見の獲得を可能にします。また、用量選択・薬物間相互作用 (DDI)・薬物 - 食物間相互作用の評価を促進するのに加えて、成人データのみが入手可能な場合に小児治療の推奨用量の探索や、薬物が他の病態に及ぼす影響のモデル化も可能にします。

Early and careful planning is critical for rare disease development, and Certara’s hundreds of PhD quantitative and regulatory scientists provide expert guidance and active support from pre-IND meetings through clinical development and post-market approval.

We have supported the approval of scores of 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.

Unparalleled experience in orphan drug development

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 orphan patient populations providing insights into biomarkers and endpoints.

Using quantitative approaches, Certara informs trial design, dose selection and dosing regimen, identifying drug-drug and drug-food interactions along with other safety factors to optimize conditions for orphan drug use. We can also model the impact of an orphan drug in development on other disease states or untested populations.

These proven quantitative methods are actively encouraged by global regulators who have demonstrated flexibility in approving orphan drugs either through an accelerated approval pathway, the elimination of a second pivotal clinical trial, or applying modeling and simulation in lieu of certain trials.

サターラに相談する
Demonstrating value and expanding market access

Increased pricing pressure and competition for orphan drugs have impacted how payers and HTAs evaluate these new therapies. The sponsor’s burden to demonstrate value not only for competitive products but also for products with no therapeutic alternatives has been impacted by the doubling of new rare disease approvals, high prices, restricted access and overall budgetary impact.

Certara develops a quantitative, multi-criteria, decision analysis framework adapted to the specific issues in rare disease development and access, including:

  • Quality of evidence
  • Disease severity
  • Ethical considerations
  • Population-level factors
  • Economic impact of the disease
  • 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.

カタログダウンロード
Supporting regulatory interactions and process

医薬品開発の豊富な実績と経験を有する専門家集団として、サイエンティスト・臨床薬理学者・レギュラトリーサイエンスのスペシャリスト・承認申請文書ライターで構成される当社のチームは、お客様のオーファンドラッグ開発担当チームと共に、グローバルな医薬品承認申請案件に取り組みます。Our regulatory team 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.

Certara has supported the approval of scores of orphan drugs using our pharmacometric portfolio of solutions, including physiologically-based pharmacokinetics (PBPK) and population PK (popPK), including:

  • Genetic and hereditary
  • Gastrointestinal
  • 悪性腫瘍領域
  • Central nervous system (CNS)
  • Bone and muscle disease
  • Anti-infectives and antivirals
  • Blood disorders
お問合せ

トップに戻る
Powered by Translations.com GlobalLink OneLink Software