April 29, 2016
Today, The Cancer Letter published a guest editorial written by iPRI President, Professor Peter Boyle.
The Moonshot: A View from Europe
By Peter Boyle
“And I believe we need a moonshot in this country to cure cancer.”
With these words, Vice-President Joe Biden gave the first public hint of a new specific, major program to be launched and funded by the U.S. government.
President Barack Obama reiterated this development in his State of the Union address announcing a new national effort to get it done and placing Vice President Biden as leader of this initiative.
The appointment of Vice President Biden to head the initiative is an inspired choice.
Of course, let’s not forget that in 1971 President Richard Nixon launched a not dissimilar initiative and yet 45 years later, there still remains an on-going war against this feared group of diseases, despite progress in many aspects. Times change, knowledge advances, and there are many signs that this new initiative holds out a better chance of success.
To emphasize the seriousness of this Moonshot, one of the initial moves was to convene a meeting of the White House Moonshot Task Force involving the heads of relevant executive branch departments, agencies, and offices. Vice President Biden emphasized that it would take a whole-of-government approach to help achieve the goal of the moonshot–to make a decade of advancements in the next five years.
The entire federal government is engaged.
Detailed plans have emerged slowly, but the address delivered by Vice President Biden at the recent meeting of the American Association for Cancer Research, captured in a recent edition of The Cancer Letter, was quite informative.
Last year, the 2016 budget funding for the National Institutes of Health was increased by $2 billion, the largest increase in a decade, and he added that the White House is requesting another $800 million to support research activities across multiple federal departments as part of the anticancer effort.
Biden provided another focus—other than ‘cure’—during this speech emphasizing the initiative’s goal to advance research more efficiently and more rapidly. He noted that President Obama had signed essentially what is an executive order giving him control over all the federal agencies and departments, from Veterans Affairs to the Department of Energy, to engage whatever resources necessary to achieve the Moonshot goals.
The coordination across a wide variety of government departments is a quite remarkable development in the history of research. It says very clearly that this is a very serious endeavor with whatever federal resources considered necessary to be engaged on the Moonshot.
In addition, the convening power of the Office of the Vice President in bringing together what are often politically and culturally entrenched research interests to put aside their differences and focus on what matters, the patient, is remarkable.
Biden launched a consultation for recommendations and has announced that the Task Force has received several including increasing research budgets across the Federal government; making it easier to share data; removing paywalls around published research; and incentivizing verification of study results.
Viewing the evolution of this Moonshot from Europe, there is much to be admired and much that we can learn.
Among other issues, Biden emphasized the need to close the time gap between submission of a research grant and the announcement of the outcome. This would be a valuable lesson that should be learned in Europe.
The amount of time scientists spend writing grants, often with copious detail as a requirement, is dispiriting and the long gap between submission and outcome is a major impediment to progress in science.
Three cheers for Biden changing this and there will be the same when the European Commission and many national research funding organisations follows suit.
Sharing research data is an issue which has been much debated. In principle, it is a great idea, but there are pragmatic hurdles to overcome. It is clear that data sharing could become a condition when receiving federal funding. How can recipients of grants from other non-federal sources be forced to make data available for sharing?
Perhaps the most important data to share are between the public and private sector.
Much progress could be made more rapidly if the public sector (e.g. academia) could share data with the private sector (e.g. Big Pharma), and vice-versa in the pre-competitive space.
Such cooperation could be a vital cog of making rapid progress towards improving prospects for a cure for some cancers. It cannot be legislated for, but would provide a major stimulus to progress.
Too many in academia take a snooty view of the private sector, frequently until they require financing for specific research. This approach needs to change and progress towards sharing information and data is a major challenge for this Moonshot and will require significant negotiation.
Biden makes a great point about paywalls surrounding published research.
The current journal model is difficult to justify.
A group of scientists find funding, complete their research and send the results to a Journal for publication. Following peer-review, the manuscript may be accepted for publication.
Too frequently, the authors are obliged to pay hundreds (or in the low thousands) for the privilege of their work being published in a journal and frequently have to transfer copyright of their original work to the publisher as a condition of publication.
Not only that, but anyone wanting an electronic copy of the research report is frequently obliged to pay several tens of dollars to have a copy.
Alternatively, they or their institution should pay thousands of dollars for journal subscriptions. This is a strange business model, which requires change: frequently the best medical research is published in journals which report millions of dollars of advertising annually.
All research should be published for free and it should be freely available to anyone who needs a copy to examine in detail. The current model is broke and needs fixing.
It’s also difficult to argue with the recommendation to involve patients earlier in clinical trial design and focus. Clinical trial recruitment is a huge problem in the U.S.
Patients either don’t know about the trials, or they’re not consulted about how the trials are designed and targeted. Only 4 percent of all the patients with cancer in the U.S. are involved with a trial.
There is something to learn from the United Kingdom where many times that percentage are participating in clinical trials.
Apart from formulating specific areas requiring modification to improve the research situation, this Moonshot should also open the door for a re-think of a wide variety of potential impediments to making progress.
For example, within the Moonshot, it would be timely to re-evaluate the clinical trial paradigm of phase I, phase II and phase III.
Modern medicine has made some remarkable strides forward, particularly in the past decade, and it would appear timely to re-evaluate this paradigm so as to shorten the time it takes to do trials required for approval of a new drug without losing any important information about efficacy and safety.
In addition, much more consideration should be given to effectiveness as well as efficacy: trials are performed currently on highly selected groups of patients who are eligible to enter a trial because of strong restrictions laid down by entry criteria. Healthcare costs are a significant issue today, and there could be a profitable discussion about basing reimbursement and pricing on effectiveness rather than efficacy.
Value sits at the heart of this. The door is slightly open: let’s take the opportunity to push it.
One pitfall to avoid is the Moonshot attempting to fix with injections of finance important issues that are not really related to a scarcity of money, but more to do issues such as vanity, commercial or academic conflict of interest, legal hurdles, poorly designed research (reductionism), and poor peer review (compounded by the enormous increase in the number of journals being published).
Failure to address such malfunctions of the scientific machinery could interfere with reaching the goals of the Moonshot.
Vice President Biden continued: “It’s personal. But I know we can do this.”
There is so much to be admired about the scope and organization of the White House Moonshot Initiative.
Apart from the specific goals, which will be defined before the end of 2016, it provides a unique opportunity to allow broader and strategic thinking to take place around current barriers to progress, such as clinical trial design and FDA requirements for efficacy and safety before approval for general use not only of medicines but of other treatments such as devices where power morcellation has been a focus of many articles recently.
The final goals established are currently being discussed. The idea to measure progress by improving patient outcomes rather than counting publications is essential to its success. The focus of all cancer research should ultimately be to do something for the patient with cancer or someone who is at high-risk of developing the disease. It was timely to see the recent letter in The Cancer Letter from deans of schools of public health emphasizing the importance of prevention.
I have always struggled as to why there has not been such an effort to improve cancer outcomes on such a scale internationally: cancer is a global problem. Let’s hope that the Moonshot is a success and that it can be a model to be taken up by top-level groups internationally.
Global society should not have to depend on the United States alone to solve one of the great problems of our times: the European Union at least should volunteer to share the financial burden (not to dilute federal government contributions by cost-sharing, but to add additional matching funds).
In such a way, more rapid progress should expected.
From a personal perspective, the Moonshot should have its main focus on the patient with cancer, but should not ignore other elements of cancer control.
Initial reports highlighted the search for a cure, but there are four pillars of oncology: Prevent those cancers that can be prevented; Treat those cancers that can be treated; Cure those cancers that can be cured: and, provide Palliation whenever required.
The continually aging demographic worldwide will result in a continually increasing number of new cases of cancer being diagnosed every year. The U.S. will not be spared these increases, and the changing healthcare system, coupled with progress in drug delivery, will have many consequences including seeing more and more patients receiving chemotherapy being treated as hospital out-patients.
I have a special plea. I have experienced the deaths of my mother, father and younger sister from this vile disease.
Please Vice President Biden, pay more attention to the protection of care givers and the environment. Care giving is emotionally draining as well as having financial considerations. It can also be hazardous.
Certain types of chemotherapy excrete identified human carcinogens in the hours and days following treatment (e.g. adriamycin), in sweat, faeces, urine or vomit exposing care-givers to these dangerous chemicals.
In hospital, there are special procedures for dealing with such hazardous waste but these do not exist in the home environment.
General advice such as “restrict use of one toilet to the cancer patient” or “flush twice” do not solve the problem.
Apart from posing a threat to care givers and family members, these hazardous chemicals enter into our sewage systems and general environment and have been doing so on a daily basis for several decades.
I enthusiastically endorse the appointment of Vice President Biden to head up this important initiative. He has seen cancer at close up and has experience of what it is like to be a parent of a cancer victim and he has seen the situation of care givers at first hand. This group has been largely ignored until now.
The federal government, the cancer research community, the entire medical profession, the insurance industry, the pharmaceutical companies and politicians must join forces to fight this cause of human suffering on all fronts. America has taken the initiative: the rest of the world must follow their example and join in.
Godspeed, Mr. Biden.