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What, precisely, is the precision medicine initiative?

First of all, it’s a big deal, high on President Obama’s agenda and with its own page on the White House Web Site

Near term goals

One immediate goal of the Precision Medicine Initiative will be to significantly expand efforts in cancer genomics to create prevention and treatment successes for more cancers.

But the long term goals are broader

the Initiative will 1) support a national network of scientists who possess the talent and skills to develop new approaches for answering critical scientific and medical questions and 2) launch a national cohort study of a million or more Americans to propel our understanding of health and disease. The goal is to set the foundation for a new way of doing research that fosters open, responsible data sharing with the highest regard to patient privacy, and that puts engaged participants at the center.

Precision Medicine Initiative logo

You’ll soon recognize the PMI logo

Some specific research questions would be to:
  • Identify genomic variants that affect drug response
  • Assess clinical validity of genomic variants associated with disease
  • Identify biomarkers that are early indicators of disease
  • Understand chronic diseases and best management strategies
  • Understand genes/pathways/factors that protect from disease

In the process, we will learn about EHRs, mhealth, patient engagement and new research methodologies.

Transparency

Workshops conducted by the PMI Working Group are open to the public if space is available, and can be viewed on webcasts. The recent workshop on Participant Engagement and Health Equity (July 1 and 2, 2015) was phenomenal, and worthwhile catching the video.

The Learning Health Care System

Two meetings in one week with a strong emphasis on the learning health care system – an indication that ideas are taking hold.

At ISPOR 2015

The first plenary session, ‘Taking stock of the learning health care system: What have we achieved and why does it matter?” highlighted the issue with four speakers, two from PCORI, one from UnitedHealth Group, and a fourth from CareMore/Anthem. The session was full, and many of us listened from the overflow room. Penny Mohr (PCORI) presented this encouraging graph showing adoption of electronic health records (EHRs) by physician offices since 2001.

learning health system

Adoption of EHRs in the US

At Stanford’s Big Data in Biomedicine Conference

Held over a three day period last week, and live streamed, this was only the third annual Stanford Big Data in Biomedicine Conference, and it was sensational. They closed with a session on Learning Health Systems, and featured four speakers: Amy Abernethy, Flatiron, Julia Adler-Milstein, U Mich, Chris Longhurst, Stanford, and Rob Merkel, IBM Watson Group. A very different group than the one at ISPOR, and very interesting to compare the approach and emphasis taken at both conferences in the same week.

Although all four speakers were excellent, I’d like to highlight the work of Chris Longhurst, co-author of the first published use of aggregated EHR data to influence a patient care decision in real time. When EHRs work smoothly, they can be queried by practicing physicians to learn from patient experience. This is not a clinical trial, and not even a retrospective cohort study, but it makes use of clinical experience to inform a discussion. Their most recent publication, “Bringing cohort studies to the bedside: framework for a ‘green button’ to support clinical decision-making” in the Journal of Comparative Effectiveness Research outlines the approach clearly, and the executive summary offers these succinct comments:

  • Electronic health records (EHRs) from past patients are a source of information, which reflects patient’s treatment choices and their effects as they happen in actual clinical practice.
  • This source of information is readily available and can be queried at the point-of-care to aid in decision-making for individual patients.
  • Real-time querying tailored to individual patients requires: EHR-based phenotyping; quantifying inter-patient similarity; optimal cohort selection; cohort visualization; automated confounder control and integrating results with clinical guidelines and existing evidence from clinical trials.
  • Real-time querying also requires real-time validation, an important open area of research. While bias from measured confounders can be minimized using automated propensity scores techniques, bias from unknown or unmeasured confounders can still threaten the validity of results. Evaluating results from cohorts with known estimates can increase confidence in these methods.
  • Results from point-of-care EHR-based cohort studies should not be looked at in isolation but be presented in the context of existing clinical guidelines and any available evidence from clinical trials.

They are beginning to implement a learning health care system, and set a model for how this might be achieved in other settings.

The challenge of pediatric Patient Reported Outcomes (PROs)

Think about it. We want to know how the child experiences his or her illness and therapies, and we can just ask, right? Well, some children are too young to even understand or respond to questions, infants and young toddlers, others go through a “no” stage and answer “no” to every question, and others may be unable to respond to questions for a range of other reasons.

Pediatric patient reported outcomes (PROs)

The recent IOM workshop, Comprehensive Cancer Care for Children and their Families, touched on some key issues around use of PROs in children with cancer, and much of what was said is applicable to children with other diseases and conditions. I’m hoping that video of the presentations will be posted (they often are).

The research imperative to develop pediatric Patient Reported Outcomes (PROs)

Try presentation by Lillian Sung, The Hospital for Sick Children, Toronto has the bland title “Academic Perspective on Clinical Research”, but is a hard hitting overview of the issues around implementing Patient Reported Outcomes (PROs) in pediatric cancer patients, and includes a full list of action items and recommendations. She notes the FDA 2009 Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims and its statement,

We discourage proxy-reported outcome measures for this population (i.e., reports by someone who is not the patient responding as if that person were the patient). For patients who cannot respond for themselves (e.g., infant patients), we encourage observer reports that include only those events or behaviors that can be observed.

Pediatric Patient Reported Outcomes (PROs) to improve estimates of toxicity and adverse events

Bryce Reeve made a critical point in favor of use of PRO’s in pediatric cancers. He presented evidence that there is a low correlation between clinicians’ report and patient report for a range of symptoms. The consequence of relying on clinicians’ report for identifying adverse events is an underestimate of adverse events, something that can be corrected by broader use of PRO’s in cancer research. Presumably this is relevant to other diseases, as well.

Other resources

The current measles outbreak and the importance of vaccines

An unnecessary, completely avoidable outbreak of Measles in the United States.

First, the science.

Measles vaccination protects against measles.  The measles vaccine, and so many other vaccines, are some of the biggest successes in public health, leading to almost 100% decreases in the incidence of a wide range of fatal and disabling infectious disease.

Measles vaccinations

Measles cases in the United States, 1950-2001, CDC

 

Parental concerns

Some children have medical conditions that preclude vaccination (see CDC “Who Should Not Be Vaccinated”), but this is a small group. Some parents of healthy children believe (without any basis) that measles vaccines (and others) cause a range of negative health effects in children and, the current generation of parents born in the 1970’s, 80’s and 90’s, don’t remember how deadly infectious diseases can be. A third group of parents have religious beliefs opposed to vaccination.

The law

Most states require vaccinations before entry to the kingergarden, but may allow unvaccinated children to attend pre-school. Some states provide exemptions for religious or other objections (see the CDC database for state immunization laws).

 Current Measles vaccination behaviors

Because vaccinations have been so successful in eradication of infectious diseases, people have forgotten how terrifying and harmful the diseases were, and have focused their anxieties on the vaccines instead of the disease. This figure shows a downward trend in measles vaccine coverage in the United States, between 1980 and 2013, and the decline from initial high levels of 97 and 98% down to current levels of 91% is enough to affect herd immunity and allow the current outbreak of measles.

measles vaccinations

Measles vaccination rates in the US 1980-2013, WHO

 

The result – an unnecessary, completely avoidable outbreak of Measles in the United States

On the upside –

a vigorous discussion and flood of information reminding us all about the value of vaccines and the importance of vaccination, including a wide range of articles, stories and commentaries giving voice to the message.

Great overview in the The New York Times

Articles and cartoons in The New Yorker (not sure it is helpful to call people “idiots”), but an indication of the widespread involvement in the discussion.

http://www.newyorker.com/cartoons/daily-cartoon/daily-cartoon-monday-february-2nd-measles-disneyland

http://www.newyorker.com/humor/borowitz-report/zombie-jonas-salk-rises-grave-hunt-idiots

Roald Dahl’s account of his 7 year old daughter’s death from measles.

HEALTHy 2015!

Happy and healthy new year to all.

Looking forward, looking back, resolutions, predictions. There’s so much of that as the old year winds down and the new year comes in, and I decided to take my pick.

I’m looking forward to more collaborations in 2015 – both in the sense of projects to work on, but also, in the larger sense of increased collaboration health research cutting across sectors of the health community. I’ve been noticing a tendency for initiatives to involve more and more stakeholders and sectors, to create larger databases, to increase enrollment in studies, to obtain patient input, and for all the other good reasons out there.

An example is the Optum Labs collaboration to pool health care data, and translate research findings into improvments in patient care. Some partners include AARP, Harvard Medical School, the Mayo Clinic, Pfizer – you get the picture. What a welcome change from only a few years ago, when these organizations worked in isolation from each other!

Another massive collaborative health research effort is the Observational Medical Outcomes Partnership (OMOP), a public-private partnership to develop methods for using observational healthcare databases to study medical products. Partners include PhRMA, FDA, and the Foundation for the National Institutes of Health. The effort has led to another collaborative, Observational Health Data Sciences and Informatics (OHDSI, pronounced “Odyssey”), a multi-stakeholder, interdisciplinary collaborative to bring out the value of health data through large-scale analytics.

Collaborative health research is definitely in the air, and that is a fantastic change in the way we do things.

The UCSF blog put together its “Top Trends in Health and Science for 2015”, and second on their list is “Breakthroughs in Teamwork” with this quotation from Matthew State, MD, PhD, chair of Psychiatry,

One of the major drivers of recent progress has been a wholesale shift in culture. Investigators who were once fierce competitors are now finding ways to collaborate with one another in large-scale, multi-site genomic studies.

If someone else besides me is saying it, it must definitely be true! UCSF began an initiative in 2013 to promote team science, and they are taking serious measures to alter the academic climate and foster team approaches,

While scientific research is collaborative in its core, the traditional academic model rewards investigators who get primary credit for their research successes, including first or last authorship in published articles.

Here’s to more teamwork and collaboration in 2015, and lots of high quality science.

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