MDA Voices Concern: Will New Drug Guidelines Benefit Medicare Recipients With Neuromuscular Diseases?

by Christina Medvescek

Even though the new Medicare prescription drug benefit — Medicare Part D — won’t take effect until January 2006, decisions are being made now about which drugs will be covered under the plan.

After reviewing the proposed guidelines, MDA has officially registered its concern that the plan doesn’t meet the needs of people with neuromuscular diseases and other rare disorders.

The U.S. Pharmacopeia (USP) is the neutral nongovernmental agency charged with developing guidelines for the formularies (lists of drugs) to be covered by Medicare. USP’s draft guideline, proposing the drug classes and therapeutic categories to be covered, was released in August and immediately provoked protests from groups with widely different agendas.

Doctors, pharmaceutical companies and patient advocates (including MDA) charge that there are too few drug classes and therapeutic categories, which will exclude vital medications for many, especially those with disabilities and complex health care needs.

On the other side of the fence, health plans and pharmacy benefit managers say there are too many classes/categories, which will drive up the cost of drugs by making it difficult to negotiate volume discounts.

Complicating the issue is the legal requirement that plans cover only two drugs within each classification, although more may be voluntarily included. The more categories and classifications contained in the USP guidelines, the more drugs will require coverage.

Although the USP guidelines apply only to Medicare Part D drug coverage, patient advocates fear they may become a model for general health insurance formularies as well.

In a letter to USP protesting the guidelines, MDA outlined three areas of concern:

Lack of appropriate categories for neuromuscular disease drugs

Two problems may arise under the USP proposal: A beneficial drug may be covered but listed under an inappropriate class/category; or a beneficial drug may not fall under any class/category.

In the first scenario, prednisone, widely used to slow the progression of Duchenne muscular dystrophy and other neuromuscular diseases, could be listed under the pharmacologic classes of “adrenal” or “immune suppressant” drugs.

“If Plan D providers choose to adhere strictly to the usage category, coverage for this drug might conceivably be denied for DMD despite medical evidence that it is effective,” warned MDA’s letter to USP, because it would be listed for other disorders, but not for DMD.

Or, it’s possible that prednisone wouldn’t be one of the two drugs listed for each category by some plans.

A similar situation could occur for riluzole (Rilutek), the only approved drug for the treatment of amyotrophic lateral sclerosis (ALS). Riluzole likely would be placed in the proposed pharmacologic class “glutamate pathway modifiers,” but this class exists only in the usage category “memory enhancers — dementia” — a category not typically associated with ALS.

MDA says the second scenario, in which no appropriate class/category is listed in the formulary, “is even more worrisome.” For example, the myasthenia gravis drug mestinon doesn’t fit into any of the proposed classes/categories.

To correct this problem, MDA recommended that USP add the categories “antineurodegenerative agents” and “muscle strength promoting agents” to the guidelines.

Lack of recognition for orphan disease drugs

“Orphan drugs” — developed to treat specific rare disorders — currently aren’t listed in the guidelines.

This omission could have a chilling effect on new orphan drugs, such as a “stop codon read-through” drug currently under development for both DMD and cystic fibrosis by PTC Therapeutics. In recent years, MDA has invested millions of dollars to speed the development of such new therapies.

“If providers who choose to participate under Plan D make no provision to cover these drugs, years of basic research, public donations and groundbreaking legislation will be effectively nullified, leaving those with rare diseases to fall through the cracks,” said the MDA statement.

MDA advocated adding an “orphan drug” category to the Medicare guidelines. This also would give the pharmaceutical industry “an economic incentive to continue to develop treatments for rare but devastating disorders.”

Addition of new drugs

The guidelines don’t stipulate a process for including new drugs on the covered list, or a specific timeline for reviewing and updating the guidelines.

MDA recommended that USP’s guidelines spell out a process and timeline for adding new drugs, categories or classes. The updating process should incorporate public input, MDA noted.

USP will send its final draft of the guidelines to the Centers for Medicare and Medicaid (CMS) in December, and CMS will finalize the rules for Medicare Part D drug plans sometime in 2005.

For the complete text of the MDA letter to the USP, go to www.mda.org/news/040928uspstatement.html. The USP’s August draft of the guidelines can be read at www.usp.org/druginformation/mmg.