July has been an active month at the US Food and Drug Administration when it comes to personalized medicine.
The agency approved four molecularly targeted drugs this month. The agency kicked off July by greenlighting the cystic fibrosis drug Orkambi (lumacaftor and ivacaftor) for patients with the F508 del mutation, which is the most common cause for the disease. Then, AstraZeneca's non-small cell lung cancer therapy Iressa (gefitinib) returned to the US market. Finally, the agency approved two chronic hepatitis C drugs, Technivie (ombitasvir, paritaprevir, and ritonavir) for genotype 4 and Daklinza (daclatasvir) for genotype 3.
These back-to-back approvals is a record for the agency and signals that "personalized medicine is turning a corner," writes Amy Miller, executive VP of the Personalized Medicine Coalition, in the non-profit's blog Education & Advocacy.
In 2014, 20 percent of drug approvals were personalized treatments by PMC's count. "At this rate, that percentage will likely be matched or exceeded this year," Miller predicts, urging the rest of the healthcare ecosystem to catch up. "Because ethically and scientifically there is no going back."
According to the PMC, the spate of new precision treatment options shows that drugmakers and researchers are having success in transforming life-threatening diseases into chronic conditions. The new therapies for cystic fibrosis that target the underlying molecular cause of the disease is a prime example of this.
However, if patients are to benefit from these new types of treatments, other parts of the healthcare system must also evolve. Healthcare professionals often decry the system of reimbursement and cost of cancer drugs as a major barrier for patient access to precision care advances.
Earlier this month, 118 oncologists wrote in the Mayo Clinic Proceedings of how coverage policies are putting too heavy a burden on cancer patients by requiring them to make co-payments as high as 30 percent of the total cost of a prescribed drug. The authors suggest mechanisms for establishing "fairer" prices through a post-approval FDA review mechanism, by enabling Medicare to negotiate prices, and by getting groups like the Patient Centered Outcomes Research Institute to factor in drug cost in value assessments.
Reimbursement policies resulting in high copayments for patients aren't sustainable, the PMC acknowledges, and encourages disparate healthcare stakeholders to work together to find solutions. "The factors that lead to these unfortunate circumstances are not linear," Miller writes. "They involve multiple actors. And so should the solution."
This post has been updated to reflect that PMC's comments refer to reimbursement issues and not drug cost.