Headlines often misrepresent the risks and benefits of novel therapies to the general public and clinical researchers as well. It is not a surprise--or it shouldn't be--that there is confusion at the point of care.
Kite’s treatment, a form of immunotherapy called CAR-T, was initially developed by a team of researchers at the National Cancer Institute, led by a longtime friend and mentor of Dr. Belldegrun. Now Kite pays several million a year to the government to support continuing research dedicated to the company’s efforts. The relationship puts American taxpayers squarely in the middle of one of the hottest new drug markets. It also raises a question: Are taxpayers getting a good deal? “Enthusiasm for cancer immunotherapy is soaring, and so is Arie Belldegrun’s fortune.”
What about transparency? If we are going to continue the "market" debate for pricing then we need to bring more aspects of a free market to bear. Overlooking the obvious violation of free markets (patients don't voluntarily become sick) there is a lot we don't know about the cost of producing clinical grade cellular therapies. Estimates are pretty low and vary around $14000 to $16000 per patient. But production appears to be the smallest influencer. There are supplies, QC testing, services of trained technicians, facility costs and more granular line items not described for public consumption or understanding.
But a quick google shows a bit of toe in the water of what the market may or may not support. The public has questions but there is little in the way of patient centric answers. We might be slowly boiling the frog.
We know that the National Cancer Institute developed CAR-T therapy. But commercialization is an entirely different process...World Congress on Vaccines last year discussed chimeric antigen receptor (CAR) T cell therapy briefly discussed here --> Complex Molecular Diversity of Predictive Precision
If US taxpayers funded the research that allowed development of a new drug how do we reconcile this at the CVS counter? Do we have the Bayh-Dole Act to encourage the invisible hand to lift the perceived brakes applied to innovation?
“The problem was that whenever federal dollars went into research—as almost all of our universities were getting federal grants to do research—and any ideas that were developed from those dollars, those grants, the patents that were secured were owned by the government, and no private individual or company could get access to them,”--Senator Bayh
Reading the headlines the conflicting views in reporting and industry reports are evident. Who do we believe?
There has been discussion of citing the "march in" provision of the act. You may be familiar with a petition backed by Senator Bernie Sanders to allow the government to control escalating drug costs by citing the march in provision of the Bayh-Dole Act. March-in rights give the federal government the right to grant other entities licenses or even to themselves if they gained funding for the patent. The wording is being debated. Should march-in rights consider high drug costs as a threat to public safety?
There are critics of a less restrictive patenting and technology transfer. The outcome--as some believe--promoted excess granting of patents and created restrictive licensing. I suggest reading the opinion rendered in Association for Molecular Pathology v. Myriad Genetics about the claim of ownership of BRCA1 and BRCA2 genes--Patents, Profits, and the American People — The Bayh–Dole Act of 1980
When the Bayh–Dole Act was written, its aim was primarily to stimulate economic growth by more efficiently mining the untapped scientific riches of hospitals, laboratories, and universities. Much has changed since then.
Moreover, some of the most vexing quandaries weren't fully addressed in the original legislation. In Myriad, the Supreme Court has taken on one such question: Who should benefit from discoveries pertaining to nature or the human body? But others remain — for example, what conflicts of interest must be identified and contained in order to protect patients? How can scientific discovery proceed if all innovations and research tools are patented and the discoverers control access to them?--Howard Markel, MD, PhD, NEJM 2013
The Bayh-Dole Act of 1980 mandates that universities take on the responsibility to transfer the novel technologies that arise from federally sponsored research. The objectives are to introduce new products in the market for public benefit and to enhance entrepreneurship--Patent and License Pearls and Pitfalls for Taking an Idea to the Marketplace
Profits and patents can be powerful incentives for scientists, businesspeople, and universities, but new and ongoing risks — including high prices that limit access to lifesaving technologies, reduced sharing of scientific data, marked shifts of focus from basic to applied research, and conflicts of interests for doctors and academic medical centers — should be mitigated or averted through revisions of the law. All Americans should be able to share in the bounties of federally funded biomedical research.--Howard Markel, MD, PhD, NEJM 2013