An Approval Process for Services

I am working on a telemedicine project and have been involved in telemedicine since 2004.  Telemedicine is obviously a solution to many aspects of care – people are admitted to the hospital for “observation” when, clearly, with sensors and video we could “observe” patients in their homes more efficiently and at dramatically lower cost, for example.  Telemedicine research is ongoing and is proving (sometimes for a second or third time) that telemedicine works.  The obvious question is, when do we consider telemedicine to be proven effective?  I realized recently that we finally have an answer to this question, and it’s in a partnership between Medicare and a group called the National Quality Forum.

For decades, the process of commercialization of a new drug has been well understood.  I’ve worked in both drug and device development and in each case, the process was clear and reasonably predictable.  The US Food andDrug Administration sets the rules for drug approval, and for the most part, it is straightforward.  When a lab discovers that some substance, a molecule or cocktail, has therapeutic potential, they can start to engage the FDA.  Typically, this involves first proving that the drug is safe for human use (after preliminary data suggests that the answer will be yes), then studying the drug in a series of progressively larger studies to demonstrate that it is effective.  Once researchers complete phase III – typically two large randomized, controlled, and blinded studies – the FDA gives its stamp of approval and the drug can be shipped to the pharmacy.

At this point, the drug is not just approved for use but there is also the expectation that it will be used.  If studies have shown that this drug is more effective that standard therapy, people may claim that to not use the drug constitutes harm.  If your doctor doesn’t offer you a prescription for a new, FDA-approved drug that has been demonstrated to be more effective than other options, you might be able to claim that you did not get good care.  If your insurance company didn’t cover it, you might complain to regulators.  Many doctors and patients want to use a new drug, even if the evidence that it is better than other options is ambiguous.  The FDA process isn’t perfect, but it is good enough that people are willing to invest in it.

In contrast, there are many models of care and therapeutic approaches that are known to be effective but, because they have no regulatory process, they never become the standard.  At the simplest level, a neurologist from the University of Pennsylvania has convincingly shown that referral to a neurologist for patients with Parkinson’s reduces hip fracture, nursing home placement, and mortality by about 20% each.  If there were a drug that did this, we would be shocked to hear a doctor didn’t offer it to his or her patients, but we readily accept that approximately half of Parkinson’s patients do not receive regular neurologist care.

There are other consequences, too: clinical research on drugs changes – often, decreasing dramatically – after FDA approval, freeing up funds to pursue the next breakthrough.  Once a model of care of mode of care delivery is demonstrated effective, the amount of research often increases, as other clinicians want more information and not to just rely on what they read in a scientific paper or heard at a conference.  In telemedicine, many bright academic pioneers have been slow to scale up, while entrepreneurs commercialize their innovations.  This happens for two reasons: first, without a regulator-established risk tolerance for approval, entrepreneurs and expert clinicians will have different perspectives on when a therapy is sufficiently established, and second, research dollars continue to be available even after the therapy is quite well established.

A relatively new organization offers us the chance to change this.  In the early 2000’s, largely driven by Medicare, there was a desire to promote the delivery of high quality care.  After some false starts working with professional societies, Medicare partnered with NQF to form the Measure Application Partnership, an initiative to identify pay-for-performance metrics.

In doing this, NQF developed a process to review and assess proposed measures of quality care, including process and outcome measures, and variously applicable to individual providers, to facilities, and to health systems.  With appropriate measures adopted, Individual providers might track whether they query about sleep disturbance in their Parkinson’s patients, facilities could measure inappropriate use of dopamine-blocking antipsychotics, and heath systems could track whether physical therapy referrals go to PT’s trained in LSVT-Big.

Although there are typically no restrictions against using a care delivery model without approval, there are good reasons to hope that NQF approval could simplify the process of getting a new model of care adopted.  This will be very important to healthcare in America as we recognize that the greatest challenge facing the American healthcare system is much less in the development of new treatments, and much more in access to the ones that currently exist.

There are good reasons to hope that the NQF could finally give us an FDA-like organization for approving health services.  I hope that we will see that not only will NQF approval create the expectation that a process or model of care can be made available, but also that, after NQF approval of a measure, research funding will move on to new questions (including a class of “post-approval” questions as with drugs) rather than continuing to support replication of prior studies.