Medicare Prescription Drug Benefit Model Guideline

Public Meeting

August 27, 2004

By: Diane Dorman, Vice President, Public Policy, National Organization for Rare Disorders

Good morning. My name is Diane Dorman. I am here this morning on behalf of the National Organization for Rare Disorders (NORD). We are a federation of over 125 patient organizations, and represent the estimated 30 million Americans living with rare diseases. On behalf of those patients and their families, our mission is the identification, treatment and cure of rare disorders through programs of education, advocacy, research and service.

Prior to the Orphan Drug Act of 1983, considered to be one of the most successful health initiatives of the 20th century, there were only 10 therapies specifically developed for rare diseases. Thanks to the incentives contained in the Act, we now have over 250 drugs, biologics, devices and foods helping about 12 million patients lead more fulfilling and productive lives (FDA estimate). Treating rare diseases matters: eighty-five percent of designated orphans are for serious and/or life-threatening diseases; thirty-one percent of orphan designations are for rare forms of cancer.

Between nine and ten percent of the U.S. population are affected by one of the nearly 6,000 known rare diseases (NIH). That translates into 3 to 3.5 million Medicare beneficiaries over 65, and about 550,000 disabled beneficiaries, having a rare disease. So it is easy to see that 250 drugs, biologics, foods and devices are just a start. There is still much to be done.

Rare disease patients are inherently more vulnerable than the typical Medicare managed care population due to age, disability and comorbid conditions and treatments. Yet, there are no provisions to ensure that drugs required for unusual and/or rare conditions will be included in Medicare formularies.

My purpose today is to ensure that Medicare beneficiaries affected by rare diseases continue to have access to orphan drug products. Rare disease patients need these existing drugs, and they will need new ones in the years to come, along with scientifically superior improvements on existing orphan therapies. It is also critical that pharmaceutical companies continue to see the ODA as an opportunity, not an irritation.

With no mandating provision, NORD is very concerned that the proposed formulary classification guideline may delay or ultimately deny access to orphan therapies. It would also be shameful if Medicare became a barrier to the further development of orphan drugs.

We hope the omission was an oversight. At least for rare diseases and orphan drugs, the proposed Medicare formulary guideline cannot be assessed by comparison to categories and classes in existing formularies.

For instance, the proposed formulary classification guidelines include sedatives and hypnotics to help beneficiaries fall asleep. But where is the classification that helps beneficiaries with narcolepsy to stay awake? This rare and lifelong disorder is characterized by chronic, excessive attacks of drowsiness during the day, sudden extreme muscle weakness (cataplexy), hallucinations, paralysis while sleeping, and disrupted sleep during the night. In most cases, narcolepsy is far more disabling than insomnia.

Accordingly, we recommend that USP take appropriate measures to ensure that the formulary guidelines include drugs approved for rare disorders. To accomplish this, USP should establish a category for "Rare Diseases," and within this category a separate class for each rare disease for which an FDA-approved drug exists. Additional classes should be added to this category promptly as new orphan drugs for additional diseases are approved. Without these measures, rare disease patients would have no assurance of actual Part D coverage for the therapies they need.

Secondly, because there may be only two plans in a region, patients with rare disorders may not have meaningful options if the only plans in their region do not include the orphan drugs they need. Therefore, we urge USP to also recommend that CMS assure nationally uniform access to orphan therapies across all plans.

In sum, if USP omits orphan drugs from the proposed model formulary, or makes no provision to ensure that orphan drugs are included, then a wide-ranging class of Medicare patients may be denied coverage and treatment.

Thank you.