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· 분류 : 외국도서 > 의학 > 약리학
· ISBN : 9783030786045
· 쪽수 : 409쪽
· 출판일 : 2021-11-09
목차
1. Introduction to Rare Diseases and Market Overview ? Raymond A. Huml, MS, DVM, RAC
2. The Patient Perspective - Meredith Huml
3. The Caregiver Perspective - Pat Furlong, RN
4. The Critical Role of Patient Advocacy Groups - Keri McDonough, MA
5. A Mental Health Perspective - Michelle Bailey, MD, MPH
6. Investment Decisions Related to Rare Disease Drug Development - Jonathan Tunnicliffe, MS, MBA and Devin Rosenthal, PhD, MBA
7. Optimizing Rare Disease Registries and Natural History Studies ? Sharon Hesterlee, PhD
8. Innovative Clinical Trial Design - Zoran Antonijevic, MS, and Jonathan R. Huml
9. CNS Rare Disease Drug Development ? Jane Williams, MD, MPH
10. Oncologic Rare Disease Drug Development ? Laura Vidal, MD and Jozsef Palatka, MD11. Hematologic Rare Disease Drug Development ? Daniel Mazzolenis, MD and Liat Vidal, MD
12. Ophthalmic Rare Disease Drug Development ? Nick Spittal, MBA
13. Pediatric Challenges and Issues ?Alexander Cvetkovic Muntanola, MD
14. Cell and Gene Therapy - Peter Robinson, MBA
15. The Feasibility Assessment ? Gloria Alley, RN and Marie Emms
16. The Evolving Regulatory Space ? And the Advent of Patient Focused Drug Development - Annie Kennedy and James Valentine, JD, MHS
17. Operational Aspects Related to Rare Disease Drug Development ? Cinzia Dorigo, PharmD
18. Accelerating Rare Disease Drug Development in Light of COVID-19 ? Zizi Imatorbhebhe, MS, MBA, PMP
19. Rare Disease Drug Approvals: Lessons Learned ? Raymond A. Huml, MS, DVM, RAC
20. Commercial and Reimbursement Challenges ? Maryna Kolochavina, PharmD
21. Integrated Life Cycle Management ? Maryna Kolochavina, PharmaD
22. The Case for Real World Evidence in Orphan Drug Development ? David Thompson, PhD and Maryna Kolochavina, PharmD
23. Summary & Integration of Rare Disease Drug Development Concepts ? Raymond A. Huml, MS, DVM, RAC
