How to Navigate the FDA Landscape for an Orphan Drug
Rare diseases are a public health priority. FDA has launched several programs and a final guidance to address the unmet need for orphan drugs.
Tag: 希少疾患
人工知能：希少疾病治療薬の開発を加速させる方法とは？
Recently, artificial intelligence (AI) has been front and center of newspapers, trade journals, and social media. It gleams in the limelight the same way as a host of other past innovations. Like many scientists, I look at innovations as an advancement. However, little did I know what the business outlook on innovation was until I … Continued
A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease
New therapeutics discovery and development for ocular diseases have been traditionally associated with a low probability of technical and regulatory success. The significant unmet medical needs for ocular diseases include but aren’t limited to glaucoma, age-related macular degeneration, and retinopathy. Recently, there has also been considerable focus on thyroid eye disease (TED). This research gained … Continued
World Orphan Drug Congress
The Rare Disease Drug Dilemma: Great Value, High Price, Limited Access?
This webinar will explain how pricing has evolved for the rare disease drug market and best practices for supporting value-based pricing.
Certara 2019年のベストブログ
A selection of short essays from our blog, written to empower our clients with modeling and simulation (M&S), regulatory science, and real-world value assessment solutions to help them solve the toughest drug development problems. In the Best of the Blog, Certara’s scientists and regulatory experts share their learnings, technological advances, and thought leadership.
希少疾患の治療薬を迅速に開発するための革新的なソリューション
This webinar discussed which pharmaceutical payer strategies work and which don’t in our rapidly changing regulatory and reimbursement world.
Found in Translation: 複雑な生物製剤の早期開発戦略の構築
This webinar described some important strategic considerations for successful development of complex biologics including appropriate data collection and timings, analyses requiring cross-functional data integration, and key pharmacology-related critical questions.
Immunogenicity Prediction and Dose Optimization using Clinically-Validated In Silico Modeling and Simulation
Biologic drug development is a rapidly evolving sector in the biopharmaceutical industry. Immunogenicity is an inherent challenge with this complex class of drugs. A quantitative systems pharmacology approach can be used to predict and better manage immunogenicity, and as a tool to guide clinical and regulatory decision-making in biologics drug development.
6MWTにおける変動性を理解する：DMD病態進行モデルの開発
デュシェンヌ型筋ジストロフィー (DMD) は致死性の伴性劣性遺伝性の小児希少疾患であり、通常男児のみ発症します。進行性の筋変性、筋力低下をきたし、最終的には機能損失につながるのが特徴です。DMDは、筋肉の統合性の維持、および筋組織の分化を促すシグナルと成長の促進に必要なタンパク質である、ジストロフィン遺伝子の変異が原因となり発症します。… Continued
