Problem
There are approximately 25 million men, women, and children in the United States suffering with rare disorders, many of whom are denied access to quality healthcare coverage, because managed care organizations (MCOs) may deny benefits due to low demand, unfamiliarity with standards of care for unusual diagnoses, and/or the high cost of care. When benefits are denied and there is no appeals process, patients continue to suffer unnecessarily because there are no safeguards to insure the fair and unbiased resolution of appeals.
Solution
The Patients' Bill of Rights must be enacted by Congress to ensure that all Americans are guaranteed the right to timely and appropriate medical care.
Policy Position
Men, women and children in the United States who are afflicted with rare "orphan diseases" must have access to specialist physicians and other healthcare providers who are knowledgeable about the diagnosis and treatment of unusual disorders.
Health plans must provide for appropriate choices of doctors and hospitals; access to knowledgeable and experienced specialists; optional use of specialists as primary care providers; continuity of care; and guaranteed coverage of emergency room care established on a "prudent layperson" standard based on a reasonable determination by the patient that there is a medical emergency.
Orphan drugs must be given special consideration by health insurers because of the small populations they are designed to help, and their unique therapeutic value. Patients must have access to pharmaceuticals, biologics, medical devices and medical foods deemed medically necessary even if they are not on the insurer's formulary.
Newly introduced orphan drugs that demonstrate improvement in safety, efficacy and/or quality of life should receive immediate approval by MCOs for payment - with evaluation occurring at 12 months - to avoid delays in treatment.
There must be a legally binding external appeals process, with specific timelines for resolution in place, when a patient is denied coverage for a service or product that his or her doctor feels is necessary.
If the plan does not abide by a ruling of an external appeals panel, patients should have legal recourse.
Insurers must obtain patient permission to release private medical information about any patient.
Insurers should pay for routine medical costs associated with government-approved clinical trials if patients have a serious or life-threatening illness for which no standard treatment is effective.
Plans that cover prescription drugs and have a restrictive formulary, must have participation of doctors and pharmacists in developing the formulary, must disclose the formulary restrictions to patients, and must provide for exceptions to the formulary where the doctor determines a certain drug is necessary for the patient.
Health plans must provide, in layperson's terminology, understandable information to patients about what is covered and what is not, about the plan's appeals process, and about any other conditions that may apply.
Whom We Represent
NORD is a unique federation of approximately 140 not-for-profit health organizations and thousands of individuals and medical professionals serving people with rare diseases. As a voluntary health agency dedicated to helping people with rare disorders, NORD is committed to the identification, treatment and cure of rare disorders through programs of education, advocacy, research and service.
For More Information
Contact Diane E. Dorman, Senior Director for Public Policy, 301.421.0018. ddorman@rarediseases.org
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