Gideon M.Blumenthal and Richard Pazdur, Nature Reviews Clinical Oncology.2019

1. Drilon, A. et al. Efficacy of larotrectinib in TRK fusion-positive

Cancers in adults and children. N. Engl. J. Med. 378, 731–739

(2018).

2. US Food and Drug Administration. Expansion cohorts: use in first-inhuman clinical trials to expedite development of oncology drugs and

biologics. FDA https://www.fda.gov/downloads/Drugs/

GuidanceComplianceRegulatoryInformation/Guidances/

UCM616325.pdf (2018).

3. US Food and Drug Administration. Master protocols: efficient

clinical trial design strategies to expedite the development of oncology

drugs and biologics. FDA https://www.fda.gov/downloads/Drugs/

GuidanceComplianceRegulatoryInformation/Guidances/

UCM621817.pdf (2018).

4. Blumenthal, G. M. & Pazdur, R. Approvals in 2017: gene therapies

and site-agnostic indications. Nat. Rev. Clin. Oncol. 15, 127–128

(2018).

5. Beaver, J. A., Kluetz, P. F. & Pazdur, R. Metastasis-free survival —

a new end point in prostate cancer trials. N. Engl. J. Med. 378,

2458–2460 (2018).

6. US Food & Drug Administration. Nonmetastatic, castration-resistant

Prostate cancer: considerations for metastasis-free survival endpoint

in clinical trials. FDA https://www.fda.gov/downloads/Drugs/

GuidanceComplianceRegulatoryInformation/Guidances/

UCM625703.pdf (2018).

7. US Food & Drug Administration. Clinical trial endpoints

for the approval of cancer drugs and biologics. FDA

8. US Food & Drug Administration. Hematologic malignancies:

Regulatory considerations for the use of minimal residual disease in

the development of drug and biological products for treatment. FDA

https://www.fda.gov/downloads/Drugs/GuidanceCompliance

RegulatoryInformation/Guidances/UCM623333.pdf (2018).

9. US Food & Drug Administration. FDA alerts health care

professionals and oncology clinical investigators about an efficacy

issue identified in clinical trials for some patients taking Keytruda

(pembrolizumab) or Tecentriq (atezolizumab) as monotherapy to

treat urothelial cancer with low expression of PD-L1. FDA https://

www.fda.gov/Drugs/DrugSafety/ucm608075.htm (2018).

10. Gormley, N. J. & Pazdur, R. Immunotherapy combinations in

multiple myeloma — known unknowns. N. Engl. J. Med. 379,

1791–1795 (2018).

11. Blumenhal, G. M. et al. Real-time oncology review and the

assessment aid: increasing review efficiency through standardization

and earlier data access. Friends of Cancer Research https://www.focr.

org/sites/default/files/ROTR%20White%20Paper%201.pdf (2018).

12. Sutter, S. Project renewal: US FDA’s plan to update generic cancer

drug labels depends on NDA holders. Pink Sheet Pharma

intelligence https://pink.pharmaintelligence.informa.com/

PS124262/Project-Renewal-US-FDAs-Plan-To-Update-GenericCancer-Drug-Labels-Depends-On-NDA-Holders (2018).

13. Usdin, S. FDA to facilitate access to unapproved drugs. BioCentury

https://www.biocentury.com/biocentury/regulation/2018-12-14/how-fdaplans-help-patients-get-expanded-access-unapproved-drugs (2018).

https://mp.weixin.qq.com/s/PLFB4a-2g02tonEZZ5l4aA