MDA States Position on Medicare Drug Guidelines

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MDA States Position on Medicare Drug Guidelines September 28 , 2004

The Centers for Medicare and Medicaid (CMS) is in the process of

determining the classes and categories of drugs to be covered under the

new Medicare prescription drug benefit, which takes effect in 2006. At

the direction of CMS, draft guidelines were drawn up in August 2004 by

the U.S. Pharmacopeia (USP). After reviewing the USP draft, MDA

officially registered concern that the plan doesn’t meet the needs of

people with neuromuscular diseases and other rare disorders.

Below is a copy of the letter sent to USP by MDA during the period for

public comment, which ended Sept. 17, 2004.

Public Comment on Draft USP

Medicare Prescription Drug Benefit Guidelines

September 7, 2004

Submitted by

Muscular Dystrophy Association

3300 East Sunrise Drive

Tucson, AZ 85718

We appreciate the opportunity to comment on the draft USP Medicare

Prescription Drug Benefit Guidelines. The Muscular Dystrophy Association

is a national voluntary health association serving children and adults

with neuromuscular disease. The more than 40 diseases covered in MDA’s

program include neurodegenerative disorders such as amyotrophic lateral

sclerosis and spinal muscular atrophy, genetic muscle-wasting diseases

like muscular dystrophy, and autoimmune muscle diseases. Many of these

diseases are severely disabling and some are fatal. An estimated 25

percent of those served by MDA are Medicare beneficiaries. The Medicare

Modernization Act has the potential to enormously benefit this

population, but the current USP draft guidelines for prescription drug

benefits are inadequate for that purpose.

Upon reviewing the draft guidelines, MDA has concerns about these

issues:

1. Lack of appropriate categories for neuromuscular disease drugs

2. Lack of recognition for orphan disease drugs

3. Lack of detail about the process for updating the guidelines

Lack of Categories for Neuromuscular Diseases

There are two types of problems that may be encountered by those with

neuromuscular disease based upon the current draft formulary: need for

drugs that are covered, but under inappropriate categories or classes,

and need for drugs for which there are no categories or classes.

An example of the former is the immunosuppressant drug prednisone.

Prednisone is the only drug shown to have a clinical benefit in slowing

the progress of Duchenne muscular dystrophy (DMD), a uniformly fatal

genetic muscle-wasting disease that affects male children. The mechanism

of action of this drug in the treatment of DMD is currently unknown,

although new evidence suggests that it does not work through classic

immunosuppression. In the draft guidelines, prednisone 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.

Even if service providers are willing to allow coverage of drugs for

off-label uses, this situation could still lead to problems if plans

choose to include in the relevant class immunosuppressant or

glucocorticoid drugs other than prednisone.

Another example of a drug for neuromuscular disease that might be denied

under the current guidelines is riluzole (Rilutek), the only approved

drug for the treatment of amyotrophic lateral sclerosis (ALS). Riluzole

is a glutamate reuptake inhibitor and as such would likely be placed

into the proposed pharmacologic class “Glutamate Pathway Modifiers,” but

this class exists only in the usage category “Memory

Enhancers-Dementia.” As ALS does not typically cause dementia, the

ability of Medicare users to receive coverage for the only drug shown to

slow the course of this fatal disease might be jeopardized.

An example of the latter situation, in which appropriate drug categories

and pharmacologic classes are altogether lacking, is even more

worrisome. For example, the myasthenia gravis drug mestinon, which

inhibits the breakdown of acetylcholine, does not fit into any of the

categories or classes of the proposed guidelines. It is our

understanding that the guidelines are expected to be populated with all

FDA-approved drugs in the future, but lacking an appropriate category,

we can only anticipate that mestinon will be placed in an inappropriate

category.

Orphan Drugs

In addition to affecting the neuromuscular system, all the diseases in

MDA’s program have in common the fact that they are rare.

Although drugs that are already on the market to treat other disorders

might theoretically be obtained for neuromuscular disease through

off-label prescriptions, new drugs developed to treat a specific rare

disorder, so-called “orphan drugs,” currently have no place in the

guidelines. This omission is of particular concern for MDA, which in

recent years has made multi-million-dollar investments in translational

research to bring new orphan drugs to market for those with

neuromuscular disease. These efforts, along with the benefits of the

Orphan Drug Act, have led more biotechnology and pharmaceutical

companies to develop therapies for unmet medical needs in smaller

populations; however, if providers who choose to participate under Plan

D make no provision to cover these drugs, years of basic research,

public donations, and ground-breaking legislation will be effectively

nullified, leaving those with rare diseases to fall through the cracks.

An example of such a drug currently under development for both DMD and

cystic fibrosis by PTC Therapeutics, Inc., is a “stop codon

read-through” drug that will allow cells to “ignore” certain types of

genetic mutations and generate a functional protein. If the draft

guidelines were to include a category for orphan drugs, new drugs such

as the PTC drug could be added expeditiously, and industry would be

assured of an economic incentive to continue to develop treatments for

rare but devastating disorders.

Addition of New Drugs

The guidelines do not adequately address a mechanism for adding new

drugs to the proposed categories. If the proposed guidelines are meant

to absorb new drugs without modification of categories or classes, then

they are inadequate, as described in the two sections above. Also, the

process of, and time interval for, adding new drugs to the guidelines is

not described explicitly. We believe that the design of the guidelines

must take into account how the guidelines can be modified in the future

and how new drugs will be added to the formulary.

Recommendations

Based on the issues above, we make the following recommendations:

1. Add the following categories: “Anti-neurodegenerative agents,” and

“Muscle strength promoting agents.” At least one drug is available

currently in each of these categories (riluzole for ALS in the former

and mestinon for myasthenia gravis in the latter) and more are under

development.

2. Add a category for “Orphan Drugs” which will insure coverage of new

drugs for rare neuromuscular disease that do not have alternative uses.

Classes listed under the Orphan Drugs category could be disease-based.

3. Develop guidelines for inclusion of new drugs that take into account

the need for new categories or classes and define the timeline for

review and updating of the guidelines. The process of updating the

guidelines should also incorporate public input.

http://www.mdausa.org/news/040928uspstatement.html

MDA Contacts:

Sharon Hesterlee, Ph.D.

Director of Research Development

(520) 529-5433

shesterlee@...

Cwik, M.D.

Medical Director

(520) 529-5496

vcwik@...