The Orphan Drug Act Implementation and Impact (2001) https://oig.hhs.gov/oei/reports/oei-09-00-00380.pdf. Accessed on 21-August-2023.

National Cancer Institute. https://www.cancer.gov/pediatric-adult-rare-tumor/rare-tumors/about-rare-cancers#:~:text=Rare%20cancers%20are%20those%20that,children’s%20cancers%20are%20considered%20rare. Accessed on 21-August-2023.

National Cancer Institute. Surveillance, Epidemiology, and End Results Program. https://seer.cancer.gov/seerstat/variables/seer/raresiterecode/. Accessed on 21-August-2023

American Cancer Society. Rare Cancers, Cancer Subtypes, and Pre-Cancers. https://www.cancer.org/cancer/types/rare-cancers.html. Accessed on 21-August-2023

Casali PG, Bruzzi P, Bogaerts J J, and, Blay JY (2015) Rare cancers Europe (RCE) methodological recommendations for clinical studies in rare cancers: a European consensus position paper. Ann Oncol 26:300–306

Article  CAS  PubMed  Google Scholar 

DeSantis CE, Kramer JL, Jemal A (2017) The Burden of Rare cancers in the United States. CA: Cancer J Clin 67:261–272

PubMed  Google Scholar 

Qosa H, Hassan HE, Younis IR (2022) Overview of clinical Pharmacology packages of New Drug Applications approved for the treatment of Rare Diseases. J Clin Pharmacol 62:S72–S78

Article  CAS  PubMed  Google Scholar 

Abuasal B, Ahmed MA, Patel P, Albusaysi S, Sabarinath S, Uppoor R, Mehta M (2022) Clinical pharmacology in Drug Development for Rare Diseases in Neurology: contributions and opportunities. Clin Pharmacol Ther 111:786–798

Article  PubMed  Google Scholar 

New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products. https://www.fda.gov/drugs/development-approval-process-drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products. Accessed on 21-August-2023.

US FDA. Search Orphan Drug Designations and Approvals. https://www.accessdata.fda.gov/scripts/opdlisting/oopd/. Accessed on 21-August-2023

Drugs@FDA. https://www.accessdata.fda.gov/scripts/cder/daf/. Accessed on 21-August-2023.

ICH-E4 Guideline for Industry. Dose-Response Information to Support Drug Registration. https://www.fda.gov/media/71279/download. Accessed on 21-August-2023.

Zirkelbach JF, Shah M, Vallejo J, Cheng J, Ayyoub A, Liu J, Hudson R, Sridhara R, Ison G, Amiri-Kordestani L, Tang S, Gwise T, Rahman A, Pazdur R, Theoret MR (2022) Improving dose-optimization processes used in Oncology Drug Development to minimize toxicity and maximize benefit to patients. J Clin Oncol 40:3489–3500

Article  Google Scholar 

US FDA. Project Optimus. https://www.fda.gov/about-fda/oncology-center-excellence/project-optimus. Accessed on 21-August-2023

US FDA Guidance for Industry. Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases. https://www.fda.gov/media/164555/download. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Adagrasib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/216340Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Belantamab mafodotin. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/761158Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Futibatinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2022/214801Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Infigratinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214622Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Selinexor. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212306Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Sotorasib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214665Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Melphalan flufenamide. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214383Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Mirvetuximab. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2022/761310Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Monsunetuzumab. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/761263Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Tebentafusp. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2022/761228Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Teclistamab. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2022/761291Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Integrated Review. Pacritinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2022/208712Orig1s000IntegratedR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Lurbinectedin. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213702Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Fedratinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212327Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Asciminib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215358Orig1s000,Orig2s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Multi-Discipline Review. Olutasidenib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2022/215814Orig1s000MultidisciplineR.pdf. Accessed on 21-August-2023.

US FDA Clinical Pharmacology Review. Ropeginterferon. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761166Orig1s000ClinPharmR.pdf. Accessed on 21-August-2023.

Poon V, Lu D (2022) Performance of Cox Proportional Hazard models on recovering the Ground Truth of Confounded exposure–response relationships for large-molecule oncology Drugs. CPT Pharmacometrics Syst Pharmacol 11:1511–1526

Article  CAS  PubMed  PubMed Central  Google Scholar 

Moreau P, Garfall AL, van de Donk NWCJ, Nahi H, San-Miguel JF, Oriol A, Nooka AK, Martin T, Rosinol L, Chari A, Karlin L, Benboubker L, Mateos MV, Bahlis N, Popat R, Besemer B, Martinez-Lopez J, Sidana S, Delforge M, Pei L, Trancucci D, Verona R, Girgis S, Lin SXW, Olyslager Y, Jaffe M, Uhlar C, Stephenson T, Rampelbergh RV, Banerjee A, Goldberg JD, Kobos R, Krishnan A, Usmani SZ (2022) Teclistamab in relapsed or refractory Multiple Myeloma. NEJM 387:495–505

Article  CAS  PubMed  Google Scholar 

Girgis S, Lin SXW, Pillarisetti K, Verona R, Vieyra D, Casneuf T, Fink D, Miao X, Chen Y, Stephenson T, Banerjee A, Hilder BW, Russell J, Infante J, Elsayed Y, Smit J, Goldberg JD (2023) Effects of Teclistamab and Talquetamab on soluble BCMA levels in patients with Relapsed/Refractory Multiple Myeloma. Blood Adv 7:644–648

Article  CAS  PubMed  Google Scholar 

US FDA Multi-Discipline Review, Nivolumab, Relatlimab https://www.accessdata.fda.gov/drugsatfda_docs/nda/2022/761234Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Capmatinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213591Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Tepotinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214096Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Tazemetostat. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/211723Orig1s000MultidisciplineR.pdf Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Pexidartinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/211810Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Umbralisib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/213176Orig1Orig2s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Zanubrutinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213217Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Pirtobrutinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/216059Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Pemigatinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213736Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Mosunetuzumab. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/761263Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Selumetinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213756Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

Kesisoglou F, Basu S, Belubbi T, Bransford P, Chung J, Dodd S, Dolton M, Heinbach T, Kulkarni P, Lin W, Moir A, Parrott N, Pepin X, Ren X, Sharma P, Stanatopoulos K, Tistaert C, Vaidhyanathan S, Wagner C, Riedmaier AE (2023) Streamlining Food Effect Assessment — Are repeated Food Effect studies needed? An IQ analysis. AAPS J 25:60

Article  CAS  PubMed  Google Scholar 

Segal EM, Flood MR, Mancini RS, Whiteman RT, Friedt GA, Kramer AR, Hofstetter MA (2014) Oral chemotherapy food and drug interactions: a Comprehensive Review of the literature. J Oncol Pract 10:e255–e268

Article  PubMed  Google Scholar 

Ji Y, Tan E, Hengelage T, Quinlan M, Hendriks BS (2023) Exploratory Food Effect Assessment in patients in early clinical development of Oncology Drugs. Clin Pharmacol Ther 114:288–302

Article  CAS  PubMed  Google Scholar 

Riechelmann R, Girardi D (2016) Drug interactions in cancer patients: a hidden risk? J Res Pharm Pract 5(2):77–78

Article  PubMed  PubMed Central  Google Scholar 

Gatti M, Pea F (2022) The Cytokine Release Syndrome and/or the Proinflammatory cytokines as underlying mechanisms of downregulation of drug metabolism and Drug Transport: a systematic review of the clinical pharmacokinetics of victim Drugs of this drug–Disease Interaction under different clinical conditions. Clin Pharmacokinet 61(11):1519–1544

Article  CAS  PubMed  PubMed Central  Google Scholar 

Sotorasib Prescribing Information. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214665Orig1s000Lbl.pdf

ClinicalTrials.gov. Identifier NCT03028103. https://classic.clinicaltrials.gov/ct2/show/NCT03028103

ICH-E14 Guideline for Industry. Clinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential for Non-Antiarrhythmic Drugs. https://www.fda.gov/media/71372/download Accessed on 22-August-2023.

ICH-E14 and S7B Guideline for Industry. Clinical and Nonclinical Evaluation of QT/QTc Interval Prolongation and Proarrhythmic Potential — Questions and Answers. https://www.fda.gov/media/161198/download Accessed on 22-August-2023

ICH-S9 Guideline for Industry. Nonclinical Evaluation for Anticancer Pharmaceuticals. https://www.fda.gov/media/73161/download. Accessed on 22-August-2023

Pacritinib Prescribing Information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/208712s000lbl.pdf

ClinicalTrials.gov, Identifier NCT 03037385. https://classic.clinicaltrials.gov/ct2/show/NCT03037385

US FDA Multi-Discipline Review. Pralsetinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213721Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Mobocertinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215310Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

Mobocertinib Prescribing Information. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215310Orig1s000lbl.pdf

Melphalan Flufenamide Prescribing Information. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214383Orig1s000lbl.pdf

Umbralisib Prescribing Information. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/213176Orig1Orig2s000Lbl.pdf

US FDA Multi-Discipline Review. Belzutifan. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/215383Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Avapritinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/212608Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Naxitamab. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/761171Orig1s000MultidisciplineR.pdf. Accessed on 22-August-2023.

US FDA Multi-Discipline Review. Entrectinib. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/212725Orig1s000,%20212726Orig1s000MultidisciplineR.Pdf. Accessed on 22-August-2023.

US FDA Guidance for Industry. Immunigencity Assessment for Therapeutic Protein Products. https://www.fda.gov/media/85017/download. Accessed on 22-August-2023.

Fathallah AM, Oldfield P, Fiedler-Kelly J, Ramadam A (2022) Immunogenicity considerations for therapeutic modalities used in Rare Diseases. J Clin Pharmacol 62:S110–S118

Article  CAS  PubMed  Google Scholar 

Nathan P et al (2021) Overall Survival Benefit with Tebentafusp in Metastatic Uveal Melanoma. N Engl J Med 385:1196–1206

Article  CAS  PubMed  Google Scholar 

Tourneau CL, Lee JJ, Sui LL (2009) Dose escalation methods in Phase I Cancer trials. JNCI 101:708–720

Article  PubMed  PubMed Central  Google Scholar 

Kurzrock R, Lin CC, Wu TC, Hobbs BP, Pestana RC, Hong DS (2021) Moving beyond 3 + 3. ASCO Educational Book 41:e133–e144

PubMed  Google Scholar 

Tourneau CL, Gan HK, Razak ARA, Paoletti X (2012) Efficiency of New Dose escalation designs in dose-finding phase I trials of molecularly targeted agents. PLoS ONE 0051039

Yuan Y, Hess KR, Hilsenbeck SG, Gilbert MR (2016) Bayesian optimal interval design: a simple and well-performing design for phase I oncology trials. Clin Cancer Res 22(17):4291–4301

Article