2017 was an amazing year for biomedical advances, with 46 novel drug approvals, a near record – more than double the number in 2016 and the highest since 1996.
Beyond the sheer number of novel new drugs approved, 2017 also stands out as a year of many firsts, with the approvals of the first CAR T-cell immunotherapies from Novartis and Kite Pharma as well as the first gene therapy that targets a disease caused by a specific genetic defect, Spark Therapeutics’ Luxurna. As the Wall Street Journal explains in a recent editorial:
Luxturna uses a virus as a vehicle to insert a normal copy of the gene directly into a patient’s retinal cells. According to the FDA, patients who received Luxturna in a trial “demonstrated significant improvements in their ability to complete [an] obstacle course at low light levels as compared to the control group.”
Gene therapy has shown promise in early trials for other disorders caused by monogenetic mutations such as hemophilia, sickle cell disease and Duchenne muscular dystrophy. It will be more difficult to treat maladies caused by multiple genetic mutations, but companies are investing billions in curative therapies.
The editors also explain the technology behind the new CAR T-cell treatments:
Researchers are also making strides using cell-based gene therapy to treat persistent cancers. The FDA this year approved two CAR T-cell therapies for blood cancers, which in trials produced nearly miraculous results for patients who had failed to respond to other treatments. The therapy re-engineers a patient’s white blood cells by combining the defensive properties of B-cells with the offensive machinery of T-cells. Patients receive infusions of CAR T-cells, which target an antigen unique to cancer cells. As a bonus, CAR T-cell infusions produce less debilitating side effects than traditional treatments.
In a clinical trial of patients with large B-cell lymphomas, Yescarta by Kite Pharma (now owned by Gilead) produced an overall three-month response rate of 82% with 54% of patients experiencing complete remission. Fewer than 10% of non-Hodgkin lymphoma patients respond completely to traditional therapies. Likewise, 83% of pediatric patients with acute lymphoblastic leukemia responded within three months to Novartis’s Kymriah CAR T-cell therapy.
But as the Journal’s editors note, these breakthroughs didn’t happen in a vacuum – smart public policy played an important role in bringing these technologies through the complex and expensive development process necessary to bring a new medicine from a discovery in the lab through to FDA approval:
A 2012 law encourages development of treatments for pediatric orphan diseases by allowing the FDA to award priority-review vouchers that pharmaceutical companies like Spark Therapeutics can later redeem for fast-track review of other drug treatments. The vouchers have boosted research in rare diseases.
And while we’ve seen a number of other smart policies enacted over the past few years, including the 21st Century Cures Act and reauthorization of the Prescription Drug User Fee Act (PDUFA), continued innovation will equally require that bad public policies – such as exposing American patients to potentially counterfeit, unsafe drugs from abroad or imposing restrictions on which medicines Medicare beneficiaries may use – as well as flawed methodologies for valuing new medicines – be unequivocally rejected.
The way to continue the success of 2017 in the next year is clear: build upon the policies that are proven to work to ensure more patients have access to the innovative cures and treatments they need.
Read the full WSJ piece here.