P

atients and patient advocacy groups are demanding faster approval for treatments they and their constituents need, and need right now. Groups more generally interested in reducing government control over drug approval are accompanying them in this mission. The target? The US Food and Drug Administration.

Getting the FDA to approve drugs faster is seen as one way to get the access that many patients and their families want. Vice President Pence and many other legislators seem to have been persuaded that the FDA is the roadblock. It isn’t. The real barrier is payers of prescription drug benefits, such as health insurance companies and self-insured employers.

The premise that the FDA needs to speed things up worked in the late 1980s when AIDS activists and cancer groups successfully pressured the FDA to make the drug approval process faster. But this won’t work today because payers, which weren’t nearly as influential 30 years ago, now regulate access to drugs. To speed access to new treatments, then, groups need to incorporate payers into their strategies.

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Payers of prescription drug benefits have become crucial in controlling access to drugs over the last three decades. During that time, tens of millions of people gained coverage for prescription drugs. This has improved access to drugs for those with coverage. But it has also taken the decisions that individual patients once made about whether or not to pay for prescriptions and aggregated them under the authority of payers.

Payers make decisions that balance the needs of the covered group as a whole against their organizational objectives, rather than focusing on individual patient situations. Coverage for the group often comes at the cost of an individual patient.

In the early days of prescription drug coverage, payers mostly paid the bills as pharmacies submitted them. But as prescription drug benefit plan costs soared from expanding use and escalating prices, payers began narrowing coverage policies and applying aggressive utilization oversight. Now payers are demanding evidence of drug safety and effectiveness that goes far beyond what the FDA requires for market approval as a basis for prescription drug coverage.

Many payers have also begun assessing the “value” a prescription drug offers as a basis for coverage. This means that payers may decide not to cover the cost of a drug that has a price they believe is too high for the benefits it provides to individual patients and to the overall group they cover. There may not be enough data showing the benefits of a drug to an individual patient. Or, in the case of unambiguous benefits to patients, the price may be higher than the patient benefit can justify, especially if covering this drug requires reducing coverage of other drugs for other patients, or raising premiums for everyone in the plan.

The net effect of payers’ actions has been to reduce access to prescription drugs. Recent examples of FDA-approved drugs directed at important unmet medical needs that have met stiff resistance from payers include eteplirsen (Exondys 51) for Duchenne muscular dystrophy, nusinersen (Spinraza) for spinal muscular atrophy, and the new anti-hepatitis C agents.

Getting the FDA to relax approval standards, or pushing the FDA aside altogether, will not clear the way for new and promising treatments when payers act as a barrier to access. And payer requirements for drug coverage are only getting more demanding.

Groups looking to speed access to new therapies need to work with payers to identify clinical benefits and economic value at the same time that they push for rapid regulatory approval. This is not easy during the pre-approval, clinical trial period. However, patient groups and pharmaceutical developers could use their expanded access programs and patient registries to generate the evidence that payers need to evaluate new drugs.

Access to new treatments will not improve just by beating on the FDA. It will mean cooperating with payers, too.

Russell Teagarden is a member of the NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access. Arthur L. Caplan, PhD, is director of the division of medical ethics at the NYU Langone Medical Center.

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