Express authorization of medicines, a double-edged sword

Cristina Roure

The approval of Aducanumab by accelerated route, by the Food and Drug Administration (FDA), for the treatment of Alzheimer's has been carried out against the unanimous opinion of its Advisory Committee on this matter, which has raised enormous dust. It is the first drug that attacks a supposed cause of the disease instead of its symptoms and has generated great expectations in a therapeutic area in which the gap is notable.

Make Evidence-Based Medicine Great Again!

Cristina Roure
 

“Make Evidence-Based Medicine Great Again!” is the provocative title of a conference that Dr. Domenico Pagano delivered at the 56th Congress of the American Society for Cardio-thoracic Surgery, calling for the need to restore professionalism, integrity, and trust in data and evidence in medicine. The conference is long, it lasts 55 minutes, but Pagano makes a brilliant presentation on the increasing manipulation that has led us to the swamp in which evidence-based medicine (EBM) currently inhabits.

Parallels between pandemics and the bystander effect

Cristina Roure
 

I remember how, just a few weeks before the declaration of the state of alarm, we looked first at China and later at Italy with astonishment and disbelief, as if what was happening there was not with us, as mere spectators. Perhaps we thought that borders could protect us, or at least give us time to anticipate and do better than the Chinese or the Italians. When we took action, our still fresh health contingency plans were pulverized in less than 48 hours by a tsunami that drove us straight to the ground of improvisation.

Nutrition and health: confusion is served

Cristina Roure

Red and processed meat, so reviled for years in all healthy eating guides, is back to happy days. Recently, the United States Department of Agriculture abolished the initiative promoted by Michelle Obama that proposed the inclusion of fresh fruits and vegetables in school cafeterias. The reason was to defend the freedom of the individual and the educational centres to offer a more nutritious meal following the preferences of the American population.

Should drugs cost "their worth"? What is the fair price?

Pedro Rey

 

There is a significant tension between the promise of medical innovation and affordable access to such innovation. The introduction of new drugs on the market at ever-increasing prices is perceived as the main inducer of the uncontrolled increase in healthcare spending in most developed countries, putting the viability of proper healthcare at risk and creating access barriers. This trend, which had been moderated in recent years by the exhaustion of the development of new products through chemical synthesis, has been reactivated with the new biotechnological products. In many countries, not only in less developed ones, the cost of treatment for diseases such as cancer can be unaffordable, either because it falls entirely on the patient or because the public system that finances it cannot afford it. Similarly, when a potential cure for hepatitis C became available recently, even high-income countries found themselves rationing treatment and looking for ways to guarantee access.

Lessons from the opioid crisis, a case of overtreatment with devastating consequences

Cristina Roure
 

If you are regulars of American series such as House, The Affair or This is us, you will be familiar with the classic protagonist who, after an episode of acute pain, ends up becoming addicted to prescription opioids such as Vicodin®, OxyContin® or Percocet®. Do not think that this is a writer’s exaggeration.

In 2015, life expectancy in the United States changed the trend and began to decline for the first time since the First World War. Among the causes, the epidemic of deaths from opioid overdoses, which multiplied by six between 1999 and 2017 (1), surpassing deaths associated with AIDS at its worst or those related to the Vietnam War. The epidemic was declared a national emergency by the United States Department of Health in 2017 and, despite the campaign deployed to combat it (2), 130 people still die in that country every day from opium overdose. If you are interested in the subject and want to delve into it, read the supplement that Nature dedicated to it in September last year.

Precision medicine, hope or hype?

Cristina Roure

Fifteen years after the human genome has been deciphered, after 3 billion dollars and after 13 years of intense network research, the cost of sequencing the entire genome has been reduced a thousand times and more than 20 million people already get concrete results. In a few weeks, from a simple saliva sample, these people will receive comprehensive information about their genetic material for less than $100 thanks to companies such as 23andMe or Ancestry. In the United States, through an alliance between Genomind and the Albertsons pharmacy office chain, patients treated with psychoactive drugs can receive advice from their pharmacist so that their doctor prescribes a pharmaceutical-genetic test and perform it in the same pharmacy office. It’s spectacular that a series of emerging diagnostic technologies target healthy people to predict or detect diseases early: immune footprint to predict various types of cancer and infectious diseases, biomarkers for Alzheimer's disease, or exhaled breath tests for early detection of lung cancer.

Escape the biomedical bubble

Cristina Roure

One of the most recurrent citation in clinical management is the Porterian concept of health value, which must be established based on the interest of the patient and not the rest of agents, read professionals, managers, researchers, administration, biomedical industry, etc. According to this concept, the ultimate purpose of research and innovation in health should be to provide maximum benefit to patients and society. However, as I commented in my last blog post, I often prioritized research and the real needs of patients, but society goes in different ways.

For a research based on value. The failed model of antibiotics

Cristina Roure

As we have commented on some occasion in this blog, neither the price nor the volume of investment in R&D of the medicines corresponds to the value they provide. Antibiotics, along with vaccines, have saved millions of lives, have allowed to address challenges such as transplants and complex surgeries with guarantees of success and, if this were not enough, they also add enormous value to the productivity of the agricultural sector.

The health care footprint on the planet, one more reason for right care

Cristina Roure

According to the Health and Climate Change Commission of The Lancet, the greatest opportunity in 21st century health is an adequate response to climate change. I have to confess that, had I been asked, I would surely have answered that the next revolution in health would be technological, probably connected to personalized medicine, 3D printing or electronic health.

Non-pharmacological prescription, the first choice treatment

Cristina Roure

Regular physical activity, directed exercise, social prescription or cognitive therapies don’t perform miracles, but neither do the medications that are prescribed daily in consultations and instead we accept them as a necessary evil, allocating 1 in 5 euros that is spent on health to pay for medicines that are not always taken, that don’t always produce the expected benefits when they don’t cause more harm than good.

Surprisingly the medical community find it hard to accept the role that non-pharmacological interventions and particularly physical activity should have not only in the prevention and treatment of chronic diseases, but also to combat frailty in an aging population like ours which moreover, lives submerged in a lifestyle that, after the incessant search for comfort, avoids any effort.

The slow progress of clinical safety, a problem of "many hands"

Cristina Roure

In this section we usually discuss overdiagnosis and overtreatment, but today we will talk about the difficulty in achieving safe care environments for patients.

Some will remember the publication in the year 2000 of the report “To err is human. Building a safer Health System” by the Institute of Medicine's Quality Care, which created a big impact in the media due to the alarming figures of deaths caused by medical errors in the USA. (1). Since then, a lot of work has been done in order to improve patient safety, and a lot of progress has been made for the dissemination of the clinical safety culture, and a great deal of effort and resources have been devoted to the implementation of safe practices for reducing the risk of medical errors.

The myth of lack of adherence

Cristina Roure

It's not that they don’t know or don’t want to know, it's that they can’t

I recently read of a doctor complaining that when he began his career, he assumed that if a disease was treated, the patient would improve, but in reality the results were far from expectation. It isn’t surprising if we remember that adherence to advice and treatment in chronic patients is less than 50%, as shown by a recent survey conducted in Spain in a sample of 1,400 chronic patients.

Despite attempts to change the attitude and pardon the patient, such as calling those that don’t comply or adhere, the truth is that systems to increase adherence to treatment always focus on changing patients’ attitudes or aptitudes. Lack of adhesion is rarely viewed as a system problem.

Measuring the value of anti-cancer drugs

Cristina Roure

Anti-cancer drugs, especially palliative drugs, are toxic, costly and sometimes of little benefit, as a result their value to the patient and society are often questionable. It’s also true that significant improvements in the survival rates are threatened by the difficulty in accessing them, due to their unsustainable cost.

I do therefore I am or the bias of intervention in medicine

Cristina Roure

This week, thanks to a post of Sergio Minué in the blog "El Gerente de Mediado", I discovered the recent publication of "Ending Medical Reversal" The book refers to situations in which new studies more robust than the pre-existing ones contradict the standards of commonly accepted practices, which have now proved ineffective or even harmful.

Some will recall such vivid examples as the use of protein C activated in sepsis, high dose chemotherapy combined with autotransplant of stem cell in metastatic breast cancer, the aprotinin in cardiac surgery, or hormone replacement therapy in postmenopausal women. All of them were used for years until proven harmful.

I have not been able to read the book yet, but I read a report by the same author; Dr. Vinay Prasad in Mayo Clinical Proceedings exposes his vast research. Over ten years, 363 studies evaluating established practices were published, out of which 146 (40%), between 12 and 19 per year, were revoked (1). The author explains in the following video that such revocations usually occur after the precipitous adoption of new therapies based on incomplete or inadequate studies.

Precision medicine, personalized medicine and person-centred medicine

Cristina Roure

In recent years we have frequently heard about personalized medicine referring to the use of our growing understanding of genetic variability in medicine for prevention strategies, more accurate and safer diagnoses and more effective treatments for each individual.

I must confess that the use of the "personalized" adjective referring to the individualization of treatments based on the genetic characteristics of each person has always caused me some discomfort, because I think that a person is much more than a set of genetic information, however accurate it may be.

For this reason I was happy to hear the term precision medicine in relation to the new initiative of the Obama Administration announced in his discourse of State of the Nation on January 20th, 2015.

Risk savvy according to Gerd Gigerenzer

Gerd Gigerenzer, Director of Harding Center for Risk Literacy at the Max Planck Institute for Human Development in Berlin, has published "Risk Savvy. How to make good decisions" (Penguin 2014). It’s a book that addresses the difficulties of making decisions in uncertain environments and the need to know how to communicate risks in an understandable way. According to the author, according to his own experience, 80% of doctors do not understand the meaning of a positive result in a diagnostic test and, following this line, in an Australian study, from the 50 doctors surveyed, only 13 responded that they understood the concept of "positive predictive value" (which is the probability of having a disease if a specific test is positive), but only one of them finally was able to explain it properly. In a post on this blog, "Too much mammography or the mirage of screenings", Cristina Roure said that a woman's risk of breast cancer after showing a suspicious lesion on a screening mammogram is 10%, when most doctors believed to be 90%. After this introduction, we can understand why we ought to consider Gigerenzer’ book as essential in medical practice in a world of probabilities.

From the Triple Aim to the Quadruple Aim

Cristina Roure

Readers of the blog Advances in Clinical Management will be familiar with the term Triple Aim coined by Donald Berwick from the Institute for Healthcare Improvement of the United States(1) which recognizes those clinical projects that simultaneously achieve the triple objective to:

1. Improve the patient experience (satisfaction and quality)
2. Improve clinical outcomes in the population
3. Reduce health care per capita costs

Knowledge chain sanitation, a difficult undertaking

By Cristina Roure 

In one of my posts  from "Pantone" series, August 17, I was talking of the need for crystal clear knowledge sources so we can make appropriate decisions, but the point is that our sources are contaminated by a number of biases that permeate the decisions we make as citizens, as patients, as health professionals or managers, and this leads to over diagnosis and overtreatment. Faced with these biases, Gerd Gigerenzer and Muir Gray, in the book "Better Doctors, Better Patients, Better Decisions" propose some sanitization measures, to which I have allowed myself to add some homemade ones:

1. How to make research more relevant to patients

a) Regulatory agencies, such as the FDA or the EMA, should require studies demonstrating the superiority of the new drugs compared to the best treatments available, not placebo.
b) Independent sponsors should promote research on simple, beneficial and patient relevant treatments and practices that do not involve the use of drugs or technology, practices that for this reason lack sponsorship (exercise, healthy diets or checklists to improve clinical safety).
c) I would add that the so called Patient Reported Outcomes should be included in clinical trials, which unlike conventionally employed variables, report on the effectiveness of treatment perceived by the patient.

Crystal clear knowledge for decision making

By Cristina Roure 

Excellent health care is one in which patients, professionals and managers apply the best available knowledge in decision making. Only well-informed agents can take the most appropriate decisions for an effective, safe and efficient health system. We often think that the big problem of the system is the lack of money, and only devoting more resources will solve the problems, but lack of knowledge is much more worrying than it may seem at first glance.

Sir Muir Gray, Chief Knowledge Officer of the National Health Service (NHS), uses an analogy that I find very spot on: "It takes knowledge as crystal clear as the water we drink if we want to take the right decisions." But the current reality is very different because we drink from contaminated sources of knowledge.