You're Already A Guinea Pig. Here's How To Be A Better One.

By | March 14, 2019

Not long ago, I received a treatment so unproven my insurance company wouldn’t pay for it. A sports medicine specialist injected platelet-rich plasma (PRP) into my pubic symphysis, a joint in the front of my pelvis. The injection made me distressed not because of the pain associated with it (which was substantial), nor because of its uncertain impact on my health, nor even because of its certain (and significant) impact on my financial wellbeing. Instead, I was distressed because, even though I received the treatment at a leading academic medical center, my body’s response to the treatment was not monitored in a manner that will yield scientifically valuable information about the benefits or risks of the therapy for future patients.

It doesn’t have to be that way. Third party payers have the power to persuade clinicians to provide promising but unproven treatments within the context of scientifically useful clinical trials.

Seven months before my PRP injection, I had undergone surgery for impingement of my left hip. The manipulation of the hip during the procedure disrupted my pubic symphysis, causing pain so severe that I couldn’t roll over in bed for most of the next two months, with each cough or sneeze feeling like it would rip my pelvis in half. Eventually the joint pain subsided, thanks to a (well-proven) steroid injection and use of a (scientifically established) sacroiliac belt. But the symphysis remained unstable, with half of my pelvis dropping several millimeters with each step I took.

Hoping to avoid the need for surgical fusion of the joint, I came across a family of treatments known as prolotherapy. Prolotherapy treatments are based on the theory that injecting irritants into injured tissues will stimulate inflammation that promotes healing. Early in its history, most prolotherapy involved the injection of dextrose, a type of sugar, into joints, although some providers used alternative irritants. Over time, the theory around prolotherapy has evolved where some believe the primary goal should not be to irritate tissues but, instead, to promote healing without generating excessive inflammation. That’s why today some physicians inject growth hormone or stem cells or PRP into tissues, rather than more strictly irritative substances.

Both theories sound plausible, but little science backs up the validity of either approach. Despite the paucity of evidence on the effectiveness of prolotherapy, lots of clinicians are making lots of money off these treatments by charging money to desperate patients, like me, who can afford to pay for the interventions. And most of these therapies are beyond the current regulatory power of the FDA, because of peculiarities in the way the agency oversees certain categories of biologic products. Moreover, the NIH does not have the resources to fund clinical research on these kinds of interventions. We have a system, then, that gives providers little incentive to test whether their treatments work. In fact, collecting rigorous data in such circumstances could backfire, by discrediting these lucrative interventions.

Without pressure, encouraging clinicians to collect data about the benefits and risks of unproven treatments, we’ll be no further along in understanding the value of these therapies ten years from now than we are today. That’s why third-party payers should use economic leverage to nudge clinicians to gather data that will inform us about whether these treatments are effective. In the case of prolotherapy, payers could join with clinicians to create a consortium of providers who would agree to follow standard protocols for how to treat and monitor specific health conditions. Instead of different clinicians injecting different concentrations of different cocktails into different body parts, the consortium would agree upon the most plausible therapies for specific injuries and test them.

The consortium model could be used to evaluate promising but unproven treatments like prolotherapy. For example, a consortium could be formed to coordinate the use of stem cell therapy, for its many purported indications, or for unproven off-label uses of pharmaceutical products.

The consortium model could also be extended to unproven but covered services – healthcare interventions that because of tradition, lobbying, or peer pressure are covered by most third-party payers but are, plausibly, ineffective for the “indicated” uses. For example, in the 1990s a series of court cases forced insurance companies to pay for bone marrow transplant in the setting of advanced breast cancer, despite no evidence this intervention improved patient outcomes. It was many years before randomized trial evidence proved bone marrow transplant to be ineffective. Such evidence would have potentially accumulated earlier if payment for such treatments had been dependent upon investigators collecting meaningful data on outcomes. In fact, Medicare has recently agreed to pay for bone marrow transplant in adults with sickle-cell disease, but only in the setting of a randomized trial designed to determine whether patients benefit from this treatment.

One benefit of a consortium is that clinicians would feel pressure to join it, because those who refuse would be at a competitive disadvantage – patients would have to pay out-of-pocket for non-consortium clinicians while patients seeing consortium clinicians would have part of the expense reimbursed by insurance.

But would payers have any incentive to create such a consortium? Payers would benefit to whatever extent the evidence accumulated through the consortia disproved the benefits of currently covered services. However, any private insurance company joining a consortium would risk facing adverse selection, whereby its willingness to pay for currently uncovered interventions, like prolotherapy for musculoskeletal disorders or bone marrow transplant in sickle-cell disease, would encourage patients with relevant conditions (and accompanying expenses) to selectively enroll in that company’s plans, thereby driving up the price of its premiums relative to competitors.

Because Medicare does not face such competition, it should take the lead in establishing such consortia. The federal government could also consider requiring consortia participation for insurance plans participating in the Affordable Care Act exchanges, as long as federal administrators recognize that such efforts will lead to steeper insurance premium increases, and thereby require that many enrollees receive larger federal subsidies.

The economic and political costs of forming these consortia are potentially worthwhile, given that well-designed programs could rapidly advance our knowledge about which healthcare interventions show promise and which do not. We all strive for a healthcare system where tests and treatments are based on evidence of safety and effectiveness. It is time we leverage the power of the purse to incentivize clinicians to provide and monitor unproven therapies in ways that yield evidence of what does and doesn’t work.

Forbes – Healthcare