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There is a lot of crowd speak about reforming how we develop drugs--especially in the US. Don't get me wrong, I listened carefully during the DIA/FDA Statistics Forum about adaptive frameworks, pragmatic trials, how to analyze real world evidence and integrate findings with clinical trial data generated from well-defined, perhaps ineffectual, clinical endpoints. There also seems to be an inexhaustible variety of innovative strategies being circulated to manage the ginormous data deluge and focus on patient driven outcomes. As a writer coming of age both personally and professionally during the early years of the HIV crisis I remain curious about our relative amnesia or short attention spans for the hard-won lessons learned during a pivotal time in clinical science. The most powerful patient advocacy in the world drove the positive outcomes and clinical successes most of us take for granted. The leading edge of the science existed at the front line--well before long-term outcomes in efficacy and safety were established. I don't want to ruin your morning but dear reader, "safety" is not even a scientific term. Because of the "interesting" evolution of the Food And Drug Administration (FDA)--and you definitely should read up on how we landed where we are today--and how gaps persist where real science can occur (The Cure in The Code: How 20th Century Law is Undermining 21st Century Medicine)--we are forgetting, at our own detriment, how off-label prescribing, critical thinking, and physician/clinical scientist group-think led to Euresist. The development of an international computer-based clinical management of antiretroviral drug resistance informed providers about what to do if a patient's viral load--once suppressed--escalated. What therapy or cocktail has proven effective?
The physicians were prescribing drugs effective in leprosy to patients with aggressive fungal disorders, wasting syndromes, and oral and genital canker sores characteristic of AIDS--not because they were FDA approved--but because they worked. The evolution of EuResist provided a tool for physicians to make evidence-based decisions (even if evidence was still evolving at the clinical level) at the point of care outside the government process that had proven to cumbersome to respond to the scale of morbidity and mortality. EuResist Network is a partnership of eighteen institutions in Europe and beyond, which promotes and coordinates joint research and dissemination efforts of its partners. As my inbox "pings" with late breaking news of ASCO data and invitations to webcasts I think we are witnessing the pivot from compassion and improving patient outcomes to expanding market share and market driven profits. I am not tossing babies out with volumes of bathwater--but seriously--where is the framework for a free open-sourced group think? I know there are pockets of sharing. I have written about them here. The model of for-profit data outpaces the network open-source solutions. Huge databases are amassed with partnerships boasting genetic libraries, claims data, clinical trial data and real world data--for a fee. A whopping fee with no promise of relevance or operational insights. What is the solution? Provide context and conceptual understanding so healthcare providers can group-think knowing they have actionable data. An ever increasing number of immune-oncology (I/O) therapies are being granted break-through designations. I am excited to hear the latest data from larotrectinib, a novel selective TRK tyrosine kinase inhibitor (TKI). The high response rate is intriguing. Those of us lucky enough to be present in science have a responsibility. Not to repeat sensationalized headlines but to help unpack the data in an industry agnostic manner. What do findings mean? Real world data is generated one patient at a time. It is up to us to assess real world evidence and share the narrative. It will be the providers at the point of care evaluating tumor genetics, micro-environments, mutational attributes of exomes, neoantigens, cell-cell signals of kinase pathways, and more as 'n of 1' findings outnumber epidemiological population estimations. Despite durable responses to TRK kinase-directed therapy in patients with NTRK-rearranged A sentence in The Cure in the Code has stayed with me, "A drug can be judged unfit for the market, one arm of government limits what the drug company may say while another invites juries to read lies or broken promises into its silence-Peter Huber
Stay-tuned for findings from the LOXO webcast scheduled June 4th... Comments are closed.
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