Cancer research — especially when it comes to new treatments — is high risk, high reward. Fewer than four out of 100 drugs that enter the first stage of clinical trials ultimately make it to the market, but those that do can command high prices.
The risks are high for patients, too. Treatments like chemotherapy are essentially poisoning the body, hoping the tumors disappear before the whole body wastes away, and new immunotherapies come with a range of a debilitating side effects. And the reward is sometimes questionable: sometimes just a few more months of life. And occasionally the cure becomes the cause: a therapy that results in full remission of one cancer can cause others down the line.
As researchers get more ambitious about treating rare cancers and designing bespoke treatments based on an individuals genes and cells, the picture only gets more complicated. Regulators and scientists are grappling with how to prove that a medicine works when its only meant to treat a tiny fraction of cases.
The POLITICO Global Policy Lab has been looking at ways to turbocharge cancer research in Europe while unraveling the thorny issues of prevention and personalized medicine. In this brainstorm white paper, we lay out the key questions facing policymakers and industry leaders as they look for new ways of treating cancer.
The problem: Europe is behind the U.S. when it comes to getting new drugs to the market. Researchers and investors say brainpower is not the problem. But fragmentation, confusion about data rules and limited public funding make it hard to get new therapies off the ground. German researchers say the challenge of getting public funding for clinical trials amounts to a “valley of death.”
The question: What are the biggest barriers facing cancer researchers today?
The problem: Randomized controlled trials have historically been considered the gold standard for testing a new therapy. However, designing a valid study is much harder when only a few patients have a disease. When theres no established treatment for a particular cancer, theres heavy pressure to provide access to experimental medicines, even if their safety and efficacy is unproven.
The question: Are clinical trials, authorization and pricing and reimbursement processes up to the task in an era of increasingly personalized treatments? What is the role of so-called real-world data, collected after patients start using a drug?
The problem: An ounce of prevention may be worth a pound of cure to health systems, but with industry funding a high proportion of R&D, theres not much profit motive to study ways to catch cancer early or improve patients lives short of a cure. A recent analysis of published cancer research argued that the EUs largest countries — including Germany, France, Spain, Italy and Poland — under performed on research into screening and palliative care. Research into genomics and epidemiology, however, is robust.
The question: Whats the right balance of research emphasis between prevention and treatment?
The problem: Medicine is often a competitive, market-driven industry, with companies and even academic institutions reluctant to pool resources and cooperate on a common goal. Governments may be in the best position to bring together the best minds to focus on a major societal problem, independent of its money-making potential. With this in mind, European Research Commissioner Carlos Moedas has proposed a series of “missions” in the EUs post-2020 budget for research funding, and he said cancer would be a logical domain.
The question: When it comes to cancer, whats the best target for a “moonshot” in Europe?
The problem: For many cancers, the ability to participate in clinical trials is tantamount to access to treatment. But European citizens who dont live near a major academic hospital or affiliated clinic may be out of luck. Kids are also at a disadvantage: A European Commission report last year found that incentives to get drug developers to test cancer drugs made for adults in children arent working — even though many rare childhood cancers are treated with medicines made for a completely different cancer in adults.
The question: What are the unique sources of disparities in cancer research, treatment and long-term care, and how do we address them?