Precision Medicine - Fairness and the Big Pharma Perspective (2017)
The days of the Blockbuster drug are over in oncology. The requirement to demonstrate convincing benefit for patients for a new cancer drug in order to license it is driving increasingly rigorous patient stratification as a means to unambiguously demonstrate precise patient cohorts in whom treatment is justified.
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Big pharma understand that聽these cohorts are increasingly precisely聽molecularly stratified, and in general聽there is an accompanying push to use聽new drugs earlier in the disease journey聽as this trends to an increase in seeing聽benefit. Thus, big science in big pharma,聽richer nations鈥� health budgets and聽insurance systems that can cope with聽this complexity and therefore spiraling聽costs, together with the ability to present聽cogent cases for novel treatments聽in increasingly sophisticated ways聽result in the more affluent microcosmos聽of our societies benefiting first from聽iconoclastic approaches, in contrast to聽far more restrictive approaches, if any聽access at all, from poorer public health聽care systems earlier in the life cycle of聽new drugs. The recent examples of the聽eye-watering cost associated with FDA聽approval of CD19 targeted Chimaeric聽Antigen Receptor T Cell therapy for聽relapsed paediatric acute lymphoblastic聽leukemia is no doubt based on the hope聽of future quality of life and productivity聽for that child together with the relative聽rarity of the condition.
The typical patient utilised en-route to聽registration of new anti cancer drugs is聽not typical of the general population of聽cancer patients and clearly demographic聽and socioeconomic issues contribute聽to that difference (awareness through聽education, nutrition, earlier presentation聽etc). Nevertheless, big pharma has聽a responsibility to follow the science and聽turn that science into good medicine,聽inevitably creating an environment聽where some patients will benefit disproportionately聽through serendipity聽born out of personalisation in addition聽to better access through socioeconomics.聽The successes of precision medicine are聽creating a complex mosaic of trade-offs聽where fairness and equity of access to聽life lengthening or curative but expensive聽treatments have to be scrutinised.聽This of course has a 鈥渞everse translational鈥澛爀ffect on the way drug development聽decisions are made in pharma,聽and the symbiosis of big pharma and聽society continuously evolves around聽these issues.聽
Despite these caveats I remain optimistic聽that good science will give us聽solutions to disease both preventative聽and curative, and that big pharma has聽an essential role in creating the landscape聽of clinically available options that聽can create the debate around fairness聽through an embarrassment of riches聽rather than desert of hopelessness.