Gene therapy has made all the difference for young patients at Children's National Hospital in Washington, D.C., according to Eric Manuel Balmir, PharmD, vice president of clinical ancillary services and chief pharmacy officer.
In simplistic terms, gene therapy alters a patient's genome through the removal and replacement of strands of DNA. It's being used at Children's National to treat several diseases, including spinal muscular atrophy, a genetic disorder with symptoms similar to that of muscular dystrophy.
Gene therapy is able not only to treat a disease, but to cure it, according to Balmir. One child with spinal muscular atrophy came into the hospital in a wheelchair and walked out, he said
"Pharmacy has gone through an evolution," Balmir said. "Twenty-five to 30 years ago we used drugs to treat symptoms. Then we shifted from symptoms to treatment."
Hypertension and diabetes treatment is saving lives.
But now, Balmir said, "we're able to treat diseases." However, these gene therapies come at a high cost.
Spinal muscular atrophy is a leading genetic cause of infant mortality, according to the Food and Drug Administration, which in 2019 approved
onasemnogene abeparvovec-xioi, marketed as Zolgensma, to treat it.
Onasemnogene abeparvovec-xioi is priced at $2.1 million per patient, according to nature.com.
Other recent FDA approvals in December include Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years and older.
By 2030, 54 approved gene and cell therapies are expected in the FDA pipeline.
As life-saving gene therapies come on the market, there needs to be a national pathway for coverage, Balmir said. Other countries have made gene therapies accessible. Children's National is working with lawmakers to support coverage for these expensive therapies.
WHY THIS MATTERS
To smooth accessibility to gene therapy, Children's National Hospital recently announced a partnered with Clearway Health, a specialty pharmacy solutions company born out of Boston Medical Center.
Clearway acts as project manager, navigating payer approvals and managing access for the gene therapy drugs, Balmir said.
Its model supports health system access to gene therapies and accelerates the payer, legal and procurement pathway, Clearway said.
The model was used when Children's National Hospital treated its first pediatric patient with ZYNTEGLO, a one-time, potentially curative gene therapy treatment for transfusion-dependent beta-thalassemia, a rare blood disorder requiring regular red blood cell transfusions.
Clearway Health said its model makes it possible to absorb the high cost of a one-time, lump sum payment while protecting the financial integrity of the institution.
"Many health systems are not set up to coordinate the procurement of a novel gene therapy. There are an incredible amount of steps and considerations involved in the process," Balmir said in a statement. "Clearway Health's framework allowed us to quickly and efficiently address these concerns, positioning our hospital to provide the latest pharmaceutical advancement."
THE LARGER TREND
Clearway Health helps hospitals and health systems build in-house specialty pharmacies, which it said provides integrated, patient-centered care and improves financial revenue.
The company has made several investments in its industry affairs team, in order to help health systems build relationships with manufacturers, payers and pharmacy benefit managers to negotiate costs, procure and dispense limited distribution specialty medications and improve access to payer networks.
ON THE RECORD
"Novel gene therapies represent the advancement of emerging therapies addressing the possibility of untethering patients from a lifetime of burdensome, costly, chronic care with a single treatment. While very promising, the full clinical impact of these treatments is not yet clear, driving questions on coverage, cost efficiency and delivery," said Shawn Francis, PharmD, director of specialty pharmacy at Clearway Health.