The challenges of waiting for RCTs vs treating patients now — ASN Events

The challenges of waiting for RCTs vs treating patients now (#12)

Josh Mailman 1
  1. NorCal CarciNET Community, Ripon, United States

Background:

2016 marks the 20th anniversary of PRRT and may mark the year that a commercially available PRRT product is approved in the United States for the treatment of Neuroendocrine Tumors (NETs).  During these 20+ years PRRT was available to many in either small institutional studies or via single center compassionate care programs. Many retrospective analysis were done on this data showing the extended progression free survival (PFS) compared to drugs approved for the treatment of NETs. Over the past 20 years thousands of patients traveled the world in search of PRRT at centers that were able to offer PRRT. Thousands more were unable to make this journey due to lack of resources or knowledge about this treatment, or were steered away by members of the medical community that would not recommend a treatment before a randomized control trial was complete.

Observation:

As patients we seek the best chance to live a longer life or find a cure, we tend to overlook the details of why a treatment is offered in one country over another only to assume that it is over regulation in one country vs. another, we mostly fail to look at the underlying issues and challenges regarding drug development in our respective countries. The pathway that PRRT was developed on, while allowing many of us to have access to treatment, hindered the wider NET population of having access to a therapy that may greatly lengthening the quality and quantity of our lives.

Conclusion:

As a community we must work harder to have focused investigational periods where patients can benefit from new treatment while at the same time having some common methodology among institutions so that the time to a randomized control trial and the length of the trail can be considerable shorter than 20 years.