Cancer HC Statistics Pharmaceuticals

Which cancer treatment is best?

This seems like a straightforward question, but clearly depends on what you mean by “best”.  Some drugs will be more efficacious and have more adverse events; other drugs may be less efficacious but have fewer adverse events.  What if a one drug shows an 80% improvement in progression free survival (PFS), but a 50% improvement in overall survival (OS); whereas another drug shows only a 50% increase in progression free survival but an 80% increase in overall survival.  Patient preferences over these treatment outcomes can vary.

But even if we just consider gains in OS, how should this be measured?  Average survival?  Median survival?  Share of patients that survive X years?  Number needed to treat (NNT)?  Does this matter?

In fact, a recent article by Karweit et al (2017) found that it does.  The authors looked at the following ways of measuring improvements in overall survival:

  • Median  OS
  • mean OS
  • 1-year survival rate
  • NNT to avoid 1 death at 1 year

The authors compare these outcomes across treatments for breast cancer (BC), colorectal cancer (CRC), melanoma, non–small cell lung cancer (NSCLC), prostate cancer (PC), and renal cell cancer (RCC).

So which drug was best?  It depends on the outcome you use.

  • Breast cancer: ado-trastuzumab emtansine demonstrated the greatest improvement in median OS, pertuzumab in mean OS, eribulin mesylate in 1-year survival rate; trastuzumab demonstrated the lowest NNT. For CRC, bevacizumab demonstrated the greatest improvements in median OS and 1-year survival rate, as well as the lowest NNT, whereas cetuximab had the greatest improvement in mean OS.
  • Colorectal cancer: Capecitabine, the only agent assessed in CRC with a nonplacebo comparator, showed the least improvement across all 3 outcomes. For melanoma, ipilimumab showed the greatest benefit with all 3 outcomes.
  • NSCLC: Pemetrexed exhibited the greatest improvements of median and mean OS, whereas erlotinib showed the greatest improvement in 1-year survival rate and the lowest NNT.
  • Prostate cancer: Enzalutamide demonstrated the greatest improvement in median OS, sipuleucel-T showed the greatest improvement in mean OS, and abiraterone had the greatest improvement in 1-year survival rate and the lowest NNT.
  • RCC: Sunitinib demonstrated the greatest improvements in both median and mean OS, whereas temsirolimus had the greatest improvement in 1-year survival rate and the lowest NNT.

The authors propose a measure portfolio to more comprehsnively measure efficacy.  They conclude as follows:

Our preliminary qualitative and quantitative analysis, which used more and different metrics than may be the standard, suggests that a broad array of survival outcomes are required to fully assess and benchmark the relative clinical value of anticancer agents. This approach becomes progressively more important as drugs transition from clinical development to regulatory approval and widespread application. The portfolio of measures assessing impact needs to be more broadly meaningful in general populations; our concept of a measure portfolio starts to move in that direction.

Source:

  • Jennifer Karweit, MS; Srividya Kotapati, PharmD; Samuel Wagner, PhD; James W. Shaw, PhD, PharmD, MPH; Steffan W. Wolfe, BA; and Amy P. Abernethy, MD, PhD. Jennifer Karweit, MS; Srividya Kotapati, PharmD; Samuel Wagner, PhD; James W. Shaw, PhD, PharmD, MPH; Steffan W. Wolfe, BA; and Amy P. Abernethy, MD, PhD. An Expanded Portfolio of Survival Metrics for Assessing Anticancer Agents. JMCP. January 17, 2017

4 Comments

  1. Breast cancer is one of the leading causes of deaths in India. According to an estimation by the World Health Organisation, roughly 144,937 women in India were detected with breast cancer in 2012 and 70,218 died because of it, making it one death for every two new diagnoses. With the incidence of the disease rising by more than 20 per cent since 2008, India is expected to have a whopping 200,000 new cases of breast cancer per year by 2030.
    Recommendations:

    To go for self-examination every month after 25 years of age.
    To undergo annual clinical breast examination after the age of 35.
    To undergo annual mammography screening after the age of 40.
    Those with a family history of breast cancer must get their genetic testing done for BRCA1/BRCA2 gene mutations.

  2. Dully noted, that many consulting firms do this to leverage the resources and increase income. I am working on establishing similar types of partnerships to leverage my company’s resources. This story is very interesting as it relates to a few of the trends that we are seeing in the healthcare arena; there will be some major changes to the industry over the next few years.

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