Recognizing the Unmet Need in Rare Disease

Floor Speech

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Mr. FERGUSON. Mr. Speaker, I rise today to speak about the unmet need in rare disease. Less than 5 percent of rare diseases have a Food and Drug Administration-approved treatment. For families affected by many of these diseases that lack such treatments, the suffering is exponential. Fortunately, advances in biomedical research based on the human genome is creating hope for millions of Americans affected by life-threatening genetic disorders.

Gene therapy, which in many instances are single treatment course interventions, is now a reality to either satisfy this unmet need or improve the standard of care for conditions that have had treatment options. Since 2019, FDA has approved gene therapies for spinal muscular atrophy, biallelic RPE65 mutation-associated retinal dystrophy, cerebral adrenoleukodystrophy, beta thalassemia, hemophilia A and B, Duchenne muscular dystrophy, and dystrophic epidermolysis bullosa. Estimates suggest that FDA is likely to approve another 20 gene therapies for rare genetic disorders by 2030.

Georgia is playing a leading role in this rapid innovation as well as its ultimate access. For example, Emory University Hospital in Atlanta is a clinical trial site for a sickle cell disease gene therapy that is currently under review for marketing authorization at FDA, as well as gene therapies for Fabry disease, phenylketonuria, and hemophilia. The Hemophilia of Georgia Center for Bleeding and Clotting Disorders at Emory will be a site for the administration of the most recently FDA- approved gene therapy. ROCTAVIAN, which is indicated for adults with severe hemophilia A, is an outpatient infusion in which a functioning factor VIII gene is transferred to the patient's liver to allow for the patient to produce normal levels of factor VIII on their own. Prior to this treatment option, severe hemophilia A patients exclusively required prophylactic disease management, which is extraordinarily expensive and currently consists of infusions of blood clotting factors three times per week or weekly injections of monoclonal antibodies. This gene therapy is expected to significantly reduce this burden, improve health outcomes, and save millions of dollars per patient.

As a member of the GOP Doctors Caucus, I am concerned that payors, particularly Medicaid plans, may hide behind flawed coverage criteria recommended by state pharmacy and therapeutics committees or drug use review boards because of the cost of gene therapy. That simply should not be the case. Patients must have access to this innovation. Indeed, if a medical professional prescribes a medicine for an individual patient and attests to its medical necessity for such individual, all payors, including Medicaid, should pay for it without subjecting it to step therapy or placing any other restriction relating to the use or prescribing of such drug, unless such requirements or limitations are specified in the ``Indication and Usage'' section of its label. With Georgia recently establishing a rare disease advisory council (RDAC), it is my hope that the patients, caregivers, clinicians, scientists, and officials comprising the council will be able to appropriately ensure this access by influencing Medicaid drug coverage policies, among other important objectives regarding rare disease family needs.

The ``medical necessity'' of a rare disease therapy should be determined by the prescriber, not the state Medicaid plan. Timely access to prescribed therapies for these patients is consistent with the mission of Georgia's RDAC and should also be a goal for us in Congress, especially considering the unmet need already affecting the community. Timely access to therapy is especially critical in rapidly progressing, fatal pediatric disorders, such as cerebral adrenoleukodystrophy, Batten disease, cystic fibrosis, Hunter syndrome, metachromatic leukodystrophy, Sanfilippo syndrome, and spinal muscular atrophy type 1, conditions that have a limited treatment window, such as achondroplasia, or diseases with painful, life-threatening episodes, such as sickle cell disease, epidermolysis bullosa, and hereditary angioedema. For several of these conditions, FDA either recently approved its first medicine, or are soon expected to approve its first medicine, so access to this innovation without delay must be a priority.

Access to medicine is the lynchpin to driving continued innovation. Without clear pathways to access, the investment in transformative therapies for rare disorders will erode. Complex, fatal genetic disorders with low prevalence will become less attractive for biopharmaceutical engagement if payors continue to limit access and Congress continues to pursue anti-innovation price controls. We must come together to explore solutions that promote access to innovation.

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