市場調査レポート

小児用医薬品の開発:規制面での課題と商業面での機会

Pediatric drug development - Regulatory challenges and commercial opportunities

発行 CBR Pharma Insights 商品コード 312351
出版日 ページ情報 英文 64 Pages
納期: 即日から翌営業日
価格
本日の銀行送金レート: 1USD=101.32円で換算しております。
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小児用医薬品の開発:規制面での課題と商業面での機会 Pediatric drug development - Regulatory challenges and commercial opportunities
出版日: 2014年07月31日 ページ情報: 英文 64 Pages
概要

小児人口は世界の総人口の2/3を占めていますが、小児用医薬品の市場規模は全体の10%以下と相対的に小さな状態に留まっています。米国(1997年)やEU(2007年)に小児科関連法案が成立するまで、製薬企業には小児用医薬品を開発するインセンティブがほとんど働いていませんでした。しかし、法案成立後は巨額の資金が小児用医薬品の分野にも投じられ、数多くの製品が開発され、現在治験にかけられています。一方で、複雑な法規制が治験の進行を妨げているという側面も存在し、市場改革の余地がまだ大きく残されています。その規制が見直され、また新興国で処方・投薬・剤形の面での改善や、難病や「顧みられない病気」(neglected diseases)への対応が進めば、巨大な市場機会が今後もたらされるものと思われます。

当レポートでは、全世界の小児用医薬品の開発の現状と将来展望について分析し、現在の規制状況(米国・EU等)や今後の改革の方向性、製品開発・市場拡大に関する今後の主な課題、将来的な市場機会、各企業がとるべき戦略について調査・考察しております。

エグゼクティブ・サマリー

  • 小児用医薬品の開発の商業的誘因性について
  • 製薬企業が小児用医薬品へ投資する理由

分析手法

小児用医薬品:概況

  • 世界の小児人口
  • 小児用医薬品の開発の基礎知識と主な課題
  • 臨床上のアンメットニーズ

市場の現状

  • 規制・法令
  • 世界レベルでの規制の調整

老年用医薬品開発のための戦略的考察

  • 治験に関する考察
  • 小児用医薬品の処方に関する問題
  • 小児科のネットワーク
  • 患者側のアクセス
  • 商業的機会
  • ジェネリック医薬品の処方と剤形
  • 難病や「顧みられない病気」に関する問題
  • 小児用ワクチン

付録

目次
Product Code: GBI050CBR

Although pediatrics represent around two-fifths of the global population the market for pediatric medicines remains relatively small (accounting for <10% of global pharmaceutical sales). Until pediatric legislation was introduced in the US (1997) and EU (2007) there has been little incentive for the pharma industry to evaluate drugs in children due to the low medical need of chronic illnesses and the high off-label use of generic drugs (Milne & Bruss, 2008).

Since the introduction of pediatric legislation there has been a substantial investment in pediatric research and the number of clinical trials performed in children has increased significantly. In the US more than 350 product labels include new pediatric information and more than 130 products have undergone a pediatric focused post-labelling safety review. In Europe there has been 221 changes regarding the safety and efficacy of medicines from the submission of old or new studies in children and 89 additions of dosing information for children as a direct consequence of Paediatric Investigation Plans (PIPs).

Despite the apparent success of the pediatric regulation, the submission of pediatric information is complex and the incentives are often insufficient to encourage industry innovation (Rose & Della Pasqua, 2011). No new drugs can be registered in the EU without a detailed PIP being approved by the EMA's Pediatric Committee (PDCO) (Rose, 2014). Without a PIP, the registration process for a new drug can be blocked. In the US, the FDA has adopted a more pragmatic approach to pediatric drug development, providing voluntary and mandatory routes for pediatric evaluation.

Many experts agree that there is significant room to improve the EU pediatric regulations and a revision of the legislation is scheduled to take place in 2018. In the meantime, there is considerable potential for the industry to develop pediatric formulations and doses of approved and generic medicines, particularly for use in transition countries where access to high quality pediatric formulation is improving. In addition, new commercial opportunities exist to develop medicines that target pediatric specific conditions in neonates as well as rare and neglected diseases and pediatric vaccines.

Scope

  • Pediatric medicines: an overview
  • The pediatric population
  • Underlying/key issues for pediatric drug development
  • Unmet clinical needs
  • Current landscape
  • Regulation and legislation
  • Global harmonization
  • Strategic considerations for developing pediatric medicines
  • Clinical trial considerations
  • Pediatric formulation issues
  • Pediatric networks
  • Patient access
  • Commercial opportunities
  • Generic formulations and dosage forms
  • Rare and neglected diseases
  • Pediatric vaccines

Key Reasons to Purchase

  • Reviews the current regulatory landscape and helps sponsors to understand the potential impact of FDA SIA regulations and the variations between EU regulation on global pediatric plans;
  • Identifies the key challenges associated with pediatric drug development and the ways to overcome these hurdles;
  • Assesses what issues need to be addressed in order to improve access to pediatric medicines and commercial opportunities in transition countries based on KOL insights;
  • Analyses different strategies to develop age-appropriate medicines in niche therapeutic fields in neonates, rare and neglected pediatric diseases and pediatric vaccines.

Table of Contents

  • Executive summary
  • How commercially attractive is pediatric drug development
  • Why should pharma invest in pediatric medicines
  • Methodology
  • Pediatric medicines: an overview
  • The pediatric population
  • Underlying/key issues for pediatric drug development
  • Unmet clinical needs
  • Current landscape
  • Regulation and legislation
  • Global harmonization
  • Strategic considerations for developing geriatric medicines
  • Clinical trial considerations
  • Pediatric formulation issues
  • Pediatric networks
  • Patient access
  • Commercial opportunities
  • Generic formulations and dosage forms
  • Rare and neglected diseases
  • Pediatric vaccines
  • Appendix
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