The drugs are currently being reviewed or have recently been approved by the Food and Drug Administration.
One is Risdiplam, the first oral therapy to treat spinal muscular atrophy (SMA), the report said. Risdiplam is a gene-splicing intervention designed to correct a genetic defect that results in a reduction of the survival motor neuron, which leads to weakness and disability.
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Another is Viltolarsen, which is in development to treat specific forms of Duchenne muscular dystrophy. DMD is a rare genetic disorder that affects young boys. It is characterized by progressive muscle deterioration and weakness. The onset of symptoms occurs between three and five years of age and worsens over time. Progressive muscle weakness leads to confinement to a wheelchair by the early teenage years. Later, patients experience life-threatening heart and respiratory conditions, with death commonly occurring in the late teens or twenties.
The third is Trodelvy, which was approved earlier than anticipated on April 22 to treat adult patients with metastatic triple-negative breast cancer (TNBC) who have received at least two prior therapies for metastatic disease. TNBC is an aggressive breast cancer that accounts for up to 20% of all breast cancer cases.
The term "triple negative" in TNBC means that this cancer tests negative for the three most common cancer causes – estrogen receptors, progesterone receptors and human epidermal growth factor receptor 2 protein.
Trodelvy is a novel antibody-drug conjugate (ADC), which combines both biologically engineered antibodies and traditional chemotherapy drugs.
Additionally, the report identifies an industry trend relating to antibody-drug conjugate category drugs.
Antibody-drug conjugate drugs are a refinement or extension of precision medicine, aimed at maximizing therapeutic benefits while minimizing undesired side effects for an individual patient, the report said.
Precision targeting allows antibody-drug conjugate (ADC) drugs to deliver chemotherapy that would otherwise be much too powerful to use on a stand-alone basis. The chemical payload is 100 to 1,000 times more toxic than traditional anticancer agents. But precision-guided ADCs can be highly lethal to the targeted cancer cells, while leaving healthy cells unharmed.
Other conjugate "payloads" will go far beyond hunting cancer cells. Various manufacturers are exploring how to leverage the ADC approach to produce vaccines, radiological treatments, immunosuppressive, cardiovascular and more.
WHY THIS MATTERS
Innovations in drug therapy have the potential to save lives, improve outcomes and reduce costs.
For example, Duchenne muscular dystrophy is associated with substantial sickness and death, affecting approximately 6,000 males in the U.S.
THE LARGER TREND
In addition to the eight ADC drugs approved by the FDA since 2010, there are nearly 100 investigational ADCs currently in preclinical and clinical trials for more than 35 different types of cancer in nearly 20 organs and tissues.
OptumRx is United Health Group's pharmacy benefit manager and is a pharmacy care services company.
As noted in the Q1 2020 issue of the Optum Rx report, the company expects the emphasis on orphan drugs to continue this year, making up nearly 40% of newly approved drugs across all disease categories. Orphan drugs, or pharmaceuticals developed solely for the treatment of orphan diseases, are commercially underdeveloped due to the fact that they are often not profitable to produce.
ON THE RECORD
"What is new is that we now are starting to see the development of orphan drugs become more competitive, increasing the potential for reduced costs and broader patient accessibility," said Sumit Dutta, chief medical officer for Optum Rx, United Health Group's pharmacy services company and pharmacy benefits manager.
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Improving the patient experience is a central focus for providers, but health systems still have a long way to go to change the traditional role of physician as consultant telling patients what they need to do. Decision-making must be shared, says Andrew Neuner, CEO of the Health Quality Council of Alberta, Canada. For more on this topic, click here to watch the complimentary webinar "Health System Transformation: Improving Population Health After COVID-19," on June 16 at 11 a.m. CT.