[Congressional Record Volume 169, Number 162 (Tuesday, October 3, 2023)]
[Extensions of Remarks]
[Pages E932-E933]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




               RECOGNIZING THE UNMET NEED IN RARE DISEASE

                                 ______
                                 

                       HON. A. DREW FERGUSON, IV

                               of georgia

                    in the house of representatives

                        Tuesday, October 3, 2023

  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

[[Page E933]]

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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