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Institute of Medicine: Delivering High-Quality Cancer Care: Charting a New Course For a System in Crisis; Part IV: The Evidence Base for High-Quality Care

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Welcome to part IV; part I addressed the current landscape of cancer care, part II addressed patient-centered communication and shared decision-making and part III addressed the work force caring for patients with cancer.  Part IV, to come, will address the evidence base for high quality care.  The IOM says that the current evidence base is inadequate in that it does not address many pressing clinical questions.  While there is a problem in medicine in general, the complexity of cancer care makes the problem particularly bad in our world.


Clinical trials generate most of our evidence base.  There are concerns that the FDA’s medical device approval processes are less rigorous than those for drugs.  Many (I’d say most) of our clinical trials are funded by pharmaceutical companies.  These trials are often narrowly focused on demonstrating the efficacy of a particular drug in order to improve patient care, influence packaging labels or expand market share.   Many critical questions are not investigated because they do not fit in with this agenda.  For example, trials focused on using generic drugs in different combinations or for different situations are challenging to fund.  The relative effectiveness of already approved drugs is rarely compared.  It is hard to conduct trials combining drugs from different manufacturers (I can personally attest to this).  Some populations, such as the elderly, are routinely under studied.


One particularly important type of research is comparative effectiveness research (CER).  IOM defines CER as follows (cited verbatim from chapter 5):

  1. The objective is to inform a specific clinical question
  2. It compares at least two alternative interventions, each with the potential to be a “best practice.”
  3. It addresses and describes patient outcomes at both a population and subgroup level.
  4. It measures outcomes that are important to patients, including harms and benefits.
  5. It uses research methods and data sources that are appropriate for the question of interest.
  6. It is conducted in settings as close as possible to the settings in which the intervention will be used.


CER is not just for clinical trials—it can also take the form of observational research and reviews of existing research.  The American Recovery and Reinvestment Act of 2009 appropriated $1.1 billion for CER.  IOM was asked to identify CER priorities and six were in cancer: screening technologies for colorectal and breast cancer, management strategies for localized prostate cancer, imaging technologies for diagnosis, staging and monitoring of all cancers, and comparing treatment strategies for liver metastases.  While the national cancer institute (NCI) conducts important CER research, financial constrains substantially limit it.


In order for research to be meaningful, it must be conducted on subjects who are representative of the real population affected by the disease.  Currently, only 3% of adults with cancer participate in clinical trials.  Members of racial and ethnic minorities, individuals with comorbidities, older patients, low income persons and rural patients are underrepresented.  The affordable care act (ACA) requires insurers to pay for the parts of care on a trial that would otherwise be needed anyway and this may help some.


The report particularly focused on the lack of participation of the elderly in clinical trials.  In one study (Javid 2012) when trials were available and patients were eligible for enrollment, physicians discussed trial participation with 76% of patients under 65 years but only 58% of older patients.  The problem is even worse in registration trials (the ones that lead to FDA approvals of new drugs): although patients aged 65 or older represent 60% of the cancer population, they constitute only 36% of registration trial participants (Talarico 2004) (in reviewing the report, it’s even worse for the older old: for patients at least 70, the rate was 20% and for patients at least 75 the rate was 9%).  Further, the older adults who do participate in trials may be healthier than the average older patient, leaving clinicians without a strong evidence base for optimal treatment of a real-world population.  We need new definitions of aging that go beyond chronological age and extend to physiological characterization of patients.  The IOM notes that granting pharmaceutical companies 6 months of additional patent exclusivity in exchange for conducting pediatric research has led to useful information on the care of children and suggests that a similar approach to older patients and patients with multiple comorbidities might help address the problem.


Patient-reported outcomes or PROs are just what they sound like—the patient’s take on her own condition.  PROs can include cancer symptom severity, severity of side effects, quality of life, functional status and other outcomes.  They do not always agree with clinician data and are complimentary to it.  In 2008 the NCI issued a contract to develop a PRO version of the standard terminology for adverse events (CTCAE) called PRO-CTCAE; it continues to be developed and may help advance research.  I will say that PROs are already improving clinical research and will increasingly do so.


Thanks for reading; stay tuned for part V on “A Learning Health Care Information Technology System in Cancer Care.”

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