Monday, June 8, 2009

What is Comparative Effectiveness Research?

One of the fascinating (and much debated) new programs contained within ARRA (American Recovery and Reinvestment Act) is 1.1 billion dollars to be spent on comparative effectiveness research (CER). CER immediately became a much debated topic with government supporters strongly in favor of it and many coming out just as strongly against it. Naturally, like most controversial topics, there has been minimal discussion of what it is. According to the department of health and human services CER is:
“Comparative effectiveness research is the conduct and synthesis of systematic research comparing different interventions and strategies to prevent, diagnose, treat and monitor health conditions.”
Right, so in lay speak, that means that CER will evaluate procedures and medicines and see which ones are going to have the most value to patients. It is a program that is designed to cut waste from the process of patient care.

HHS also lists the criteria on which medicines and treatments will compared...
  1. Potential impact (based on prevalence of condition, burden of disease, variability in outcomes, and costs of care).
  2. Potential to evaluate comparative effectiveness in diverse populations and patient sub-populations.
  3. Uncertainty within the clinical and public health communities regarding management decisions.
  4. Addresses need or gap unlikely to be addressed through other funding mechanisms.
  5. Potential for multiplicative effect (e.g. lays foundation for future CER or generates additional investment outside government).
Or to put it into simpler terms...
  1. Will this significantly help a serious problem?
  2. Will this have an impact across a broad range of population?
  3. Will this make treatment decisions easier?
  4. Will this help where no one else is helping?
  5. Will this help future research on CER?
So why do we need this? Well, in traditional clinical trials medicines are compared only against placebos (a.k.a. nothing). The idea behind CER is that we'll be able to make better decisions if these medicines are tested against each other. In theory, we will be able to determine which medicines and treatments are most effective and we will be able to determine if specific medicines should be targeted for specific patients. The goals of CER are to provide better care at a lower price. Whether it can do that is up for significant debate.

Why do we need the government to do this? One of the primary reasons why the government would be involved in this is because the private sector lacks sufficient incentive to engage in this research. Any discoveries regarding the comparative effectiveness of medicines could have unpredictable benefits and costs across industry. It's difficult to incentivize a business to do research which could show their product is the least effective one on the market. This leaves the government, in its role as representative of the public interest, as the most neutral entity to do the research.

But what does this all mean in the end? One major concern is that each party in the health care field is going to have a very different definition of what constitutes waste; and the costs and benefits of an expanded CER program are going to depend entirely on which definition of waste you use.

There is the potential for very real problems with CER. It has the potential to be as wasteful and bureaucratic as any of the worst government programs (Medicare I'm looking at you...). Also, HOW you run the program (and not the program itself) is going to determine whether CER is a miracle or a monster.

We'll examine these key criticisms - and any others you suggest in the comments section - on Thursday.


  1. CER throws up so many Qs, that its really quite mind boggling. So, I'll just ask a few :
    1.Is the FDA evaluation process for new drugs going to be revised?
    2.Will CER funds be given out in addition to the funds for every grant (drug development or otherwise)?
    3.How do PIs apply for CER funds? Will there be a CER section in the grant application?
    4.How will the "results" of CER studies be implemented? Govt. Programs ( Medicare/ Medicaid, VA...) Private Insurers
    5.What effect will this have on R&D spending on new/better drugs by big Pharma?

  2. 1 and 4. CER is not so far advanced that it will be used to make regulatory changes... yet. That may occur in the future, but right now this is an exploratory program. There are a lot of concerns about this implemenatation phase which I discuss in the follow up post.

    2 and 3. CER is a different program rather than additional funding to existing research. The details have not entirely come out although the NIH plan for implementation is available here.