Back to the Future in the Quest for Affordable Cancer Care

Liam Davenport

July 13, 2016

BARCELONA — Oncologists will need to drive the development of personalized medicine and consider the repurposing of noncancer drugs, alongside the use of biosimilars, if cancer care is to remain affordable, experts said here at the 18th World Congress on Gastrointestinal Cancer.

Paul Cornes, MD, from the Comparative Outcomes Group and European School of Oncology and an oncologist based in Bristol, United Kingdom, told delegates that oncologists need to take the lead on controlling costs in cancer — otherwise, "we will be led by someone else."

He emphasized that, while time and investment are needed "to turn the promise of personalized medicine into reality," elements such as inexpensive, rapid genome analysis; the use of blood sampling rather than repeat biopsies; and a dramatic increase in targeted drugs "are falling into place."

Crucially, Dr Cornes said that there are "savings from within our current budgets that we can access to support innovation without harming patient outcomes," primarily by saving on "futile and marginal treatments."

He added that previously ignored inexpensive potential therapies, including repurposing drugs such as aspirin and cimetidine, should be reinvestigated, and that nations need to learn from each other to "copy the best" and drop "the worst" practices.

Session chair Mario Dicato, MD, from the Haematology-Oncology Service at the Luxembourg Medical Center, Luxembourg, told Medscape Medical News that the cost of cancer care "is going to be a major issue."

Discussing the presentation, Dr Dicato said that while he does not see, at present, any potential in repurposing cimetidine, there are "more and more studies showing that aspirin can be very useful as a preventive measure in cancer," particularly in patients with colon polyps.

Another "major possibility," he said, is the use of biosimilars. He explained that there has been an "explosion in cost over the past couple of years," especially in patients treated with immunotherapies. He said that immuno-oncology is a major advance in treatment, but it has come with an unprecedented cost explosion. However, many other targeted drugs are now coming off-patent, and so the increasing use of biosimilars could result in price drops of around 30%.

"The other thing, obviously, is to restrict the treatment only to patients where you can predict that it will work," Dr Dicato said. "If you have a marker of a patient's response, there's a possibility that will narrow down the usage of the drug to a half of the patients or a third of the patients."

He continued: "This is being done, and obviously you save a lot of money rather than giving it to everybody, where half of them will not benefit. That will waste time and their disease will grow because they will not respond."

Most Devastating Economic Impact

Dr Cornes began his presentation by highlighting that cancer has the "most devastating economic impact of any cause of death in the world," with an estimated total worldwide cost of cancer-related premature death and disability of $2.4 trillion.

However, cancer deaths have declined substantially in recent years, with a 19.4% reduction in the United Kingdom alone over the past 20 years and an increase in median cancer survival from 1 year in 1971 to 10 years in 2010. Moreover, up to 60% of the increase in cancer survival since 1975 can be attributed to novel medicines.

The number of novel drugs has also increased exponentially, he said, with an additional 43 launched in the past 5 years alone and almost 2000 cancer medications in development at any one time.

Dr Cornes added that the advent of targeted, precision medicines for specific forms of cancers is "transforming cancer care," resulting in a tripling of survival in, for example, some leukemias and gastrointestinal stromal tumors.

This has led to the possibility of patients receiving combination therapies that target different pathways within a disease, with the potential of achieving a cure.

However, this comes at a price, Dr Cornes noted, adding that the cost of cancer drugs has increased exponentially since the 1970s, at a rate five times faster than in other classes of medicine.

Moreover, the compound annual growth rates of cancer care in the United States, United Kingdom, and Australia are between 3.6% and 5.2%. The result is that, with an average monthly cost of a branded oncology drug in the United States is now approximately $10,000, a combination of three drugs will result in an average annual drug therapy cost of $360,000 per patient per year. Consequently, he said that "dramatic budget increases" will be required by the middle of the next decade to pay for cancer care.

The financial crash in 2008, however, has resulted in an ever-widening gap between the economic output and debt burden of industrialized countries, Dr Cornes said. Add in the aging population, with its substantially increased cancer burden compared with previous generations, and the increasing costs of modern cancer care will be unaffordable.

There also is a huge disparity between the healthcare spending on, for example, colorectal cancer between many developed nations, yet with often little difference in mortality rates, while countries such as the United Kingdom and Australia spend similar amounts but have an absolute difference in colorectal cancer survival of 9%.

Dr Cornes emphasized that, alongside efficacy and effectiveness, an important consideration when evaluating a drug is its efficiency, or cost-effectiveness. Pointing out that that money can only be spent once, he said that much of current healthcare spending does not improve outcomes and that better use of available funds to the most cost-effective interventions could potentially save hundreds of thousands of life-years.

There needs to be a shift in strategy away from evidence-based medicine, with its focus on overall survival and quality of life, to value-based medicine, in which a drug will be judged on its cost-effectiveness, he said.

As an example, Dr Cornes discussed the European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, which aims to provide a rational, structured, and consistent approach to stratifying a drug's clinically meaningful benefit.

As reported by Medscape Medical News, this scale emphasizes the precedence of cure over death, the importance of direct endpoints, and the validity of disease-free survival in curative as opposed to noncurative disease. However, Dr Cornes pointed out that it does not consider the cost of a therapy.

He said that the "only long-term, viable option, if comprehensive healthcare is to remain affordable," is to align spending to the long-term growth of the economy as a whole, which will require a realistic approach, reliance on quantitative information, and "difficult choices."

This aligns with the World Health Organization's World Medicines Situation Report, which includes as part of its definition of the rational use of a medicine the proposal that patients receive an appropriate medicine "at the lowest cost both to them and the community."

Citing the example of monoclonal antibody trastuzumab (Herceptin, Genentech), Dr Cornes pointed out that it can be difficult, over the short term, to determine the true value of an innovation. However, improvements in the precision and cost-effectiveness of personalized medicine should mean that the number of nonresponders to therapy should decrease.

Dr Cornes believes that budgetary savings could be made from increased use of generic drugs, and of biosimilars, which are copies of patent-expired medicines that have the same indications, quality, safety, and efficacy of the reference medicine but a lower cost.

He highlighted a recent report that estimated that the use of biosimilars in the five biggest European Union markets and in the United States could result in cumulative potential healthcare savings of more than €50 billion over the next 5 years, and reach as much as €100 billion.

Dr Cornes also suggested that noncancer drugs, such as aspirin and cimetidine, could be repurposed for novel uses in oncology and that academic organizations such as ESMO could take advantage of fee reductions from the European Medicines Agency to become a drug developer.

Furthermore, simple measures, such as timing treatments during the day so that they cause the least amount of toxicity, putting patients in rooms with a view, and putting plants and flowers in their rooms, could have a significant effect on hospital stays, the use of analgesics, rates of adverse events, and patient satisfaction.

Summarizing, Dr Cornes said that there is only "one key rule" for access to innovation, which is that the only drug that works is one that "we can afford to give."

How Is Benefit Assessed?

For Dr Dicato, a key aspect to the evaluation of a drug is how its benefit is assessed. "There is something called statistical significance, and then there is clinical significance," he noted, adding that a drug that improves progression-free survival "might not show any advantage in overall survival."

Describing this as a "major issue," he pointed out that much of the marketing for novel cancer drugs in recent years has been based on progression-free survival.

In an interview with Medscape Medical News, Dr Dicato said, "All in all, what you can say is that, if you take the five diseases that are the major cause of cancer mortality in Western countries, which is lung, breast, colorectal, prostate, and pancreas cancer, cure is rare if the surgeon is too late."

"You are not gaining very much by spending a lot of money on end-stage situations of disease," he continued. "You are getting, at the most, a couple of months by spending a large amount of money, and if you can shift the patients to an earlier stage by doing screening or whatever, you are going to save a lot of money."

Dr Dicato stressed: "It's the issue between statistical versus clinical significance. If you take 1000 patients, you are able to show that a few weeks of survival is going to be statistically highly significant, but you are going to have to treat a lot of patients who will have side effects of the drug to get a few weeks of survival. You really have to think, then, what you are doing, and the money you are spending is fantastic. Both ESMO and ASCO [American Society of Clinical Oncology] are working on scoring systems to improve value and possibly limit costs."

At the same time, healthcare systems in Europe and the United States USA are developing innovative approaches to financing cancer care, including "pay for performance'" arrangements with manufacturers, he noted.

He cited the example of bortezomib (Velcade, Millennium Pharmaceuticals) for the treatment of multiple myeloma, which was quickly shown to be effective in at least half of patients. However, it was, and remains, "costly." As a result, the United Kingdom's National Health Service agreed to pay for the drug only for patients who respond.

Dr Dicato said that the concept has been picked up in some Scandinavian countries, and a commission in the Benelux countries (Belgium, the Netherlands, and Luxembourg) is also considering it. "It means you pay for performance, which is a very good idea: value, rather than cost will be the master concept."

Dr Cornes disclosed that he has received a salary from the United Kingdom National Health Service and has received honoraria from Accord Healthcare, Amgen, Bernstein, the British Medical Journal, the European Generics Association, the Global Academy of Health Sciences, Hospira, Janssen, Lilly, Merck Serono, Napp, the National Cancer Society Malaysia, the Pharmaceutical Association of Malaysia, Pfizer, Roche, Sandoz, and Teva. Dr Dicato has disclosed no relevant financial relationships.

18th World Congress on Gastrointestinal Cancer (WCGC). Presented July 2, 2016.

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