Personalized Medicine Education and Advocacy

Thought leadership in personalized medicine

Turning a Corner: How This Month’s Decisions at FDA Inform the Debate on Drug Costs


Personalized medicine is turning a corner. FDA approved four new indications for personalized medicine in July of 2015, a record for the agency and the field. As you may remember, 20 percent of FDA’s 2014 drug approvals were personalized medicines. At this rate, that percentage will likely be matched or exceeded this year.

But what does that mean for the field?

It means policymakers throughout the health care ecosystem have to play catch up, because ethically and scientifically there is no going back. Science has led us here because personalized medicines save and extend lives. They keep people at work, enjoying their families and contributing to society. They reduce morbidity and the adverse health events associated with older treatments. And for some patients they extend lives long enough to allow for participation in promising clinical trials, which can extend lives even further.

For evidence of personalized medicine’s potential, look no further than this month’s approvals (see table below). While the list covers a variety of disease conditions, it is clear that we are making significant headway in turning deadly diseases into chronic conditions, and in some cases, finding functional cures. For example, through significant investments by industry and the patients they serve, we now have treatments for cystic fibrosis that allow young people with the disease to live longer, fuller and more fulfilling lives.

Table 1. July’s Approved Indications for Personalized Medicines

Drug Indication
FDA approved IRESSA (gefitinib) as a first-line indication for metastatic non-small
cell lung cancers with mutated or overactive EGFR
Technivie ®
(ombitasvir, paritaprevir & ritonavir)
FDA approved Technivie (ombitasvir, paritaprevir and ritonavir) for treatment of chronic hepatitis C genotype 4
Daklinza ®
FDA approved Daklinza (daclatasvir) for treatment of chronic hepatitis C genotype 3
Orkambi ®
(lumacaftor & ivacaftor)
FDA approved Orkambi (lumacaftor and ivacaftor) for treatment of cystic fibrosis in patients
who have a specific mutation, the F508 del mutation, which is the leading cause
of the disease

It is incumbent upon decision-makers to ensure that health policy and delivery systems support the use of these important medicines so that they can continue to deliver on their promise.

Reimbursement policies are part of that discussion. As noted in a recent article from 118 oncologists that was published in Mayo Clinic’s academic journal, Mayo Clinic Proceedings, current trends in coverage policies can result in individual patients being required to make co-payments that are as high as 30 percent of the total cost of a prescribed therapy. That is not sustainable, but the factors that lead to these unfortunate circumstances are not linear. They involve multiple actors. And so should the solution.

In this context I encourage all the field’s stakeholders to work together toward ensuring that personalized medicine makes us healthier, because as FDA’s recent decisions make clear, personalized medicine is no longer just a hypothetical paradigm.

It is our current reality.

Amy M. Miller, Ph.D.
Executive Vice President
Personalized Medicine Coalition

4 thoughts on “Turning a Corner: How This Month’s Decisions at FDA Inform the Debate on Drug Costs

  1. Pingback: Are Statins Bad For Me? Personalized Medicine Can’t Yet Say |

  2. Pingback: Are Statins Bad For Me? Personalized Medicine Can’t Yet Say

  3. Pingback: Are Statins Bad For Me? Personalized Medicine Can't Yet Say - NPR - ClinicPress

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