Medicines for the 21st Century: Safe, Better, Cheaper
On May 8th, 2019, my company, PharmaFlow (just me) hosted a Conference at Techniquest, Cardiff Bay, titled Medicines for the 21st Century: Safe, Better, Cheaper (see 3 minute video above).
Its aim was to curate the views of stakeholders in the pharmaceutical industry in relation to safety, efficacy, and affordability of medicines. It comprised three panel sessions to discuss the various issues, and come up with suggestions on how things could be radically improved.
From all the inputs, I compiled a white paper, which was sent to the then Chair of the UK House of Commons Health and Social Care Committee, the Rt. Hon. Jeremy Hunt, MP.
Receipt of the paper was acknowledged by both the Chair and the Secretariat, albeit I’ve not heard anything since.
Little did I know at the time what was about to break the following year!
Wise words from Ray Perkins PhD
It was fortunate that Dr Perkins was able to travel from Kentucky to join us on the day of the conference, to share with us his deep understanding of the history and science of medicines discovery and development. Dr Perkins is an acknowledged world expert and kindly provided an account of how and where the system is in need of repair at the Conference.
I knew Dr Perkins to be a brilliant scientist from our discussions on LinkedIn over many years previous to COVID. What I had not realised was the scale and scope of his insights in relation to the world of medicine. Dr Perkins joined our panel, contributed from the floor, and best of all, he spoke to our headline video recording (see 3 minute video above).
He summed up his thoughts with the words “I’m just here pointing out the problems, put forward as a backdrop for any solutions that may come forward.”
These are extracts from the Appendix Dr Perkins wrote for the white paper:
“Here at the end of the second decade of the 21st century, the word “broken” best describes our ability to diagnose and treat disease. Our ability to select therapies that work is broken. Our ability to diagnose, even define, disease is broken.
Our ability to perform research that is reproducible is broken. Our fundamental hypothesis of biology is broken. The list goes on. This is certainly a depressing and, to many, surprising state of affairs. Commercial and non-commercial media outlets are crowded with excited faces assuring us that something akin to immortality is just around the corner. Even old and once-respected science publications now are filled with only “positive” findings, with headlines adjusted to secure maximum attention.
Institutions and universities employ public relations offices whose influence on scientists’ careers equals that of their peers.
The application of medicine is broken. Specifically, the ability to select medicines for patients presenting with symptoms is worse than hit-or-miss. In 2018 in the United Kingdom, some one billion prescriptions were written. Of that number, the best that can be said is that 900 million of those did nothing, providing neither benefit nor harm. The reality is much worse. Nine of ten patients are exposed to side effects, some worse than the disease itself. Misuse of antibiotics leads directly to drug resistant strains of bacteria.
The Genes-are-Destiny model is broken. Readily refuted by simple arguments, genes-are-destiny persists as the century-long basis of biology. Gene mutations, for example, have long defined “treatable targets” in drug and diagnostic discovery.
As such, it must be seen as a primary contributor to their failures. Even the recent “breakthroughs” in cancer immunotherapy reinforce this conclusion, all exhibiting NNTs (Number Needed to Treat) comparable to chemotherapy.
The picture is understandably bleak. Enormous amounts of money are wasted on therapies that don’t work, and development of new therapies and diagnostics are hamstrung. However, there are findings that point the way forward. The impact of microbiome findings has only scarcely been felt.
Humans are composite creatures and must be approached as such. Further, the failure of genes-are-destiny must be seen in contrast to its inverse: biology must be approached at the functional level of complex, interacting networks of molecules, cells, tissues, organs, organisms and the environment. Within this new and bracing context, the activity of proteins becomes paramount, as well as the relationship between that activity and biological function.
“Functional Proteomics” must occupy centre stage in the eyes of researcher and decision maker alike.
Else a pig is simply a ham sandwich.”
More from Dr Perkins can be found here: Making the Case for Functional Proteomics
Recommended Reading from Dr Perkins:
Dance to the Tune of Life: Biological Relativity, Denis Noble, Cambridge University Press, 2016
The Black Swan: The Impact of the Highly Improbable, Nassim Nicholas Taleb, Random House, 2007
What Should we Conclude?
I’ll leave the last words to Dr Perkins:
The Genes-are-Destiny model is broken. It is recognised now humans are composite creatures and must be approached as such.
One of the first scientists to sequence the human genome, recognised the limitations of using genes to predict and prevent disease: “We simply don’t have enough genes for this idea of biological determinism to work.” Craig Venter, Chairman and CEO of J. Craig Venter Institute.
The failure of genes-are-destiny must be seen in contrast to its inverse: biology must be approached at the functional level of complex, interacting networks of molecules, cells, tissues, organs, organisms and the environment.
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