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P.M. Barrios

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    ORAL 27 - Care (ID 123)

    • Event: WCLC 2015
    • Type: Oral Session
    • Track: Advocacy
    • Presentations: 1
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      ORAL27.07 - Impact of Regulatory Delays in Approving Oncology Drugs in a Developing Country: Mortality Associated with Lack of Access to Crizotinib in Brazil (ID 2295)

      10:45 - 12:15  |  Author(s): P.M. Barrios

      • Abstract
      • Presentation
      • Slides

      Strict legislation and regulatory standards for the approval of drugs represent a safety guarantee for the population of any country. However, inappropriate delays in the process of evaluation of new medications have potentially serious consequences that can be measured. The objective of this analysis is to estimate the impact of the delay in the registration of medicines by the Brazilian regulatory agency, ANVISA, on the life span and life with symptoms related to the disease of patients who might potentially benefit from treatment. We use the example of crizotinib (Xalcori® Pfizer, NY, USA), which had its registration refused by the agency in 2014.

      We arbitrarily selected the 3-year period from August 2011 (FDA approval) to June 2014 (refusal by ANVISA) for this analysis. The number and prevalence of NSCLC cases eligible for treatment were estimated according to data from the Brazilian National Cancer Institue (INCA). The percentage of patients with ALK-positive tumors was inferred from the literature. We assumed that every ALK-positive NSCLC patient in Brazil would have access to the drug regardless if seeking treatment through the private or public health systems. The benefits from treatment with crizotinib were considered according to the published literature available at the time of regulatory assessment.

      We estimated 24.460 new cases of NSCLC/year in Brazil (INCA), 70,6% (17.269) of which are diagnosed as advanced disease. Approximately 4,3% (743) would qualify as ALK positive. In a phase III crossover trial, crizotinib treatment ensued an improved PFS (3.0 vs. 7.7 mo; HR 0.49, p<0.001) and a significant extension in the median time to deterioration of symptoms (1.4 vs. 5.6 mo; p<0.001) when compared to standard second line chemotherapy. Survival estimates were obtained from a retrospective analysis (Shaw et al. Lancet 2011) as follows: chemotherapy 6.0 vs. crizotinib 20.3 months. We estimated 707 prevalent cases of ALK+ NSCLC in Brazil at the start of our analysis and 62 new cases per month during the 3-year analysis period were projected. Applying the premises above we calculated 1.367 years of life lost, and 772 additional patients who would remain alive after the selected period between August 2011 and July 2014. Furthermore, a total of 846 years of life free of symptoms’ deterioration are lost with the associated human suffering during the same period of time.

      The delay in the analysis, approval and registration process of new medications in Brazil and other developing or developed countries has an important impact in terms of human lives that can be potentially measured or estimated. While this kind of scrutiny has clear methodological limitations to consider, our main objective in this analysis is to raise the issue of the urgent need for a detailed and transparent evaluation of all the steps involved in the evaluation and registration process to stall this unnecessary suffering and loss of human life.

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