Regulatory Policy and HTA

Our work in Regulatory Policy and Health Technology Assessment looks at the different standards for regulatory approval and reimbursement of cancer drugs in U.S, Canada, and other high-income countries as well as that in low-and-middle-income countries.  We have extensively studied the use of surrogate endpoints and accelerated approval pathway for fast-track approval of cancer drugs in the U.S, and its subsequent impact on cancer drug approval, access, and pricing both in the U.S and globally. In recent years, we have also examined cancer drug reimbursement decisions in Canada.

Relevant Publications:

  1. Gyawali B, Bowman M, Sharpe I, Jalink M, Srivastava S, Wijeratne DT. A systematic review of eHealth technologies for breast cancer supportive care. Cancer Treat Rev. 2023 Jan. PMID: 36736125.
  2. Istasy P, Lee WS, Iansavichene A, Upshur R, Gyawali B, Burkell J, Sadikovic B, Lazo-Langner A, Chin-Yee B. The impact of artificial intelligence on health equity in oncology: A scoping review. J Med Internet Res. 2022. PMID: 36005841.
  3. Lythgoe MP, Desai A, Gyawali B, Savage P, Krell J, Warner JL, Khaki AR. Cancer Therapy Approval Timings, Review Speed, and Publication of Pivotal Registration Trials in the US and Europe, 2010-2019. JAMA Netw Open. 2022 Jun. PMID: 35687337.
  4. Akhade A, Sirohi B and Gyawali B*. Global consequences of the US FDA’s accelerated approval of cancer drugs. The Lancet Oncology. 2022 January. PMID: 35114117
  5. Gyawali B*, Rome BN, Kesselheim AS. Regulatory and clinical consequences of negative confirmatory trials of accelerated approval cancer drugs: retrospective observational study. The BMJ. 2021 PMID: 34497044. Altmetric Score: 305.
    • Covered by STAT News, Medpage Today and several others.
  6. Fundytus A, Sengar M, Lombe D, Hopman W, Jalink M, Gyawali B, Trapani D, Roitberg F, De Vries EGE, Moja L, Ilbawi A, Sullivan R, Booth CM. Access to cancer medicines deemed essential by oncologists in 82 countries: an international, cross-sectional survey. Lancet Oncol. 2021. PMID: 34560006. Altmetric score: 121.
  7. Gyawali B*, Ross JS, Kesselheim AS. Fulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway: The Need for Reforms. JAMA Intern Med. 2021 Jul 13. PMID: 34254981. Altmetric score:
  8. Van Wambeke S and Gyawali B. Atezolizumab in Metastatic Triple-Negative Breast Cancer-No Contradiction in the Eyes of a Dispassionate Observer.JAMA Oncol. 2021 Jun 24. PMID: 34165499.
  9. Gyawali B*, Darrow JJ, Kesselheim AS. Integrating New Effectiveness Data Into US Food and Drug Administration-Approved Drug Labeling. JAMA Intern Med. 2021 May 17. PMID: 33999146.
  10. Thomson S, Witzke N, Gyawali B, Delos Santos S, Udayakumar S, Cardone C, Cheung MC, Chan KKW. Assessing the benefit of cancer drugs approved by the European Medicines Agency using the European Society for Medical Oncology Magnitude of Clinical Benefit Scale over time. Eur J Cancer. 2021 Apr 28;150:203-210. PMID: 33932727.
  11. Gyawali B* and Kesselheim A.S. FDA approval standards for anticancer agents — lessons from two recent approvals in breast cancer. Nat Rev Clin Oncol 2021. PMID: 33767454. Altmetric score: 115
  12. Meyers DE, Jenei K, Chisamore TM, Gyawali B*. Evaluation of the Clinical Benefit of Cancer Drugs Submitted for Reimbursement Recommendation Decisions in Canada. JAMA Internal Medicine. 2021. PMID: 33616606. Altmetric score: 132.
  13. Cherla A, Naci H, Kesselheim AS, Gyawali B, Mossialos E. Assessment of Coverage in England of Cancer Drugs Qualifying for US Food and Drug Administration Accelerated Approval. JAMA Internal Medicine. 2021. PMID: 33616607. Altmetric score: 67
  14. Gyawali B*, Hey SP and Kesselheim AS. Evaluating the evidence behind the  surrogate measures included in the FDA’s table of surrogate endpoints as supporting approval of cancer drugs. Eclinicalmedicine, Published by the Lancet. February, 2020. PMID: 32025656. Altmetric score: 63
  15. Raphael MB, Gyawali B and Booth CM. Real world evidence and regulatory drug approval. Nature Reviews Clinical Oncology 2020. PMID: 32112057.
  16. Gyawali B*, D’Andrea E, Franklin JM and Kesselheim AS. Response Rates and Durations of Response for Biomarker-Based Cancer Drugs in Nonrandomized Versus Randomized Trials. Journal of NCCN. 2020 January. PMID: 31910385. Altmetric score: 59
  17. Gyawali B*, Sharma S# and Booth CM. Is the number of cancer drug approvals a surrogate for regulatory success? Journal of Cancer Policy. 2019. Altmetric score: 51
  18. Hey SP, Gyawali B, D’Andrea E, Kanagaraj M, Franklin JM, Kesselheim AS.  A Systematic Review and Meta-analysis of Bevacizumab in First-line Metastatic Breast Cancer: Lessons for the Research and Regulatory Enterprises. J Natl Cancer Inst. 2019 Oct. PMID: 31651981
  19. Naci H, Davis C, Savović J, Higgins JPT, Sterne JAC, Gyawali B, Romo-Sandoval X, Handley N, Booth CM. Design characteristics, risk of bias, and reporting of randomised controlled trials supporting approvals of cancer drugs by European Medicines Agency, 2014-16: cross sectional analysis. BMJ. 2019 Sep. PMID: 31533922. Altmetric Score: 396
  20. Gyawali B*, Hey SP, Kesselheim AS. Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval. JAMA Internal Medicine. 2019. PMID: 31135808. Altmetric Score: 72
  21. Gyawali B* and Kesselheim AS. US Food and Drug Administration Approval of New Drugs Based on Noninferiority Trials in Oncology: A Dangerous Precedent? JAMA Oncology. 2019. PMID: 30920591 Altmetric score: 127
  22. Gyawali B and Kesselheim AS. Reinforcing the social compromise of accelerated approval. Nature Reviews Clinical Oncology 2018. PMID: 29970919 Altmetric score: 64
  23. Gyawali B* and Addeo A. Negative Phase 3 Randomized Controlled Trials: Why cancer drugs fail the last barrier? International Journal of Cancer. 2018 May. PMID: 29744864. Altmetric score: 63
  24. Gyawali B* and Goldstein D. The US Food and Drug Administration’s Approval of Adjuvant Sunitinib for Renal Cell Cancer: A Case of Regulatory Capture? JAMA Oncology. May 2018. PMID: 29522146. Altmetric score: 105
  25. Hwang TJ, Franklin JM, Chen CT, Lauffenburger JC, Gyawali B, Kesselheim AS and Darrow JJ. Efficacy, Safety, and Regulatory Approval of Food and Drug Administration–Designated Breakthrough and Nonbreakthrough Cancer Medicines. Journal of Clinical Oncology. 2018 Apr 24. PMID: 29688832
    • With editorial by Nicole Kuderer and Gary Lyman.
  26. Gyawali B*. The OlympiAD trial: Who Won the Gold?ecancer 2017, 11:ed75. Published December 6, 2017. PMID : 29290761
  27. Gyawali B, Parsad S, Feinberg B and Nabhan C. Real-World Evidence and Randomized Studies in the Precision Oncology Era: The Right Balance. JCO Precision Oncology 2017:1, 1-5. Altmetric Score: 100
  28. Gyawali B and Prasad V. Health policy: Me-too drugs with limited benefits – the tale of regorafenib for HCC. Nature Reviews Clinical Oncology. (PMID: 28719584) . Altmetric score:83.
  29. Gyawali B and Prasad V. Combining drugs and extending treatment-a PFS endpoint is not sufficient. Nature Reviews Clinical Oncology. (PMID: 28534528) . Altmetric score:75.
  30. Gyawali B and Prasad V. Same data; different interpretations. Journal of Clinical Oncology. 34, 31: 3729-3732., 2016, (PMID: 27573659). Altmetric score: 112
    • 19th most read article in ASCO Journals (JCO, JOP, JGO) (as of Dec.21, 2016)
  31. Gyawali B*. Me, too. Comments and Controversies. Journal of Global Oncology 2, no. 3 (June 2016) 99-104. PMID: 28717688