3 ways biomarkers are changing drug development

Michael D. Dalzell

Targeting a biomarker in drug development addresses the age-old problem of one-size-fits-all drugs working in half or fewer of patients, because response rates of drugs in patients with a corresponding biomarker are generally much higher. Here are three ways molecular discoveries are driving change in the way drugs and biologics are developed.

1Biomarkers and drug-diagnostic combinations.  According to a 2010 report from the Tufts Center for the Study of Drug Development, in some therapeutic areas, half of active drug trials involve some sort of biomarker. The fruits of this research are beginning to hit the market. In 2011 and 2012, three personalized medications — defined as requiring the use of a companion diagnostic to identify appropriate patients — received Food and Drug Administration approval. In 2013, four such products had been approved by November 15. The speed of approvals can be expected to continue to increase; Roche, for one, has said that by next year, at least half of its approvals will be for drugs requiring a companion diagnostic.

Drug-diagnostic approvals in last 3 years

AWP = Average wholesale price

Sources: Manufacturers’ package information; Medical Marketing & Media, UBC/Express Scripts, Nature news blog, Seeking Alpha,

2Unmet needs.  The advent of biomarkers has re-invigorated research into treatments for disease areas where therapy has not changed for a long time.

Medications in development for conditions with no approvals in 10 years
Drugs in developmentTherapeutic area
Ovarian cancer158
Amyotrophic lateral sclerosis61
Small cell lung cancer41
Cervical cancer28
Septic shock26
Sickle cell disease19
Myasthenia gravis7
Source: “The Biopharmaceutical Pipeline: Evolving Science, Hope for Patients.” Pharmaceutical Research and Manufacturers of America, 2013

3Repurposing medications.  The average wholesale prices in the timeline above suggest that the cost of developing new personalized medicine is unsustainable. That, coupled with the potential for biomarkers to make treatment more effective in specific patients, led the National Institutes of Health to launch an initiative to see if old drugs or abandoned molecules could have utility in people with specific molecular profiles.

For a pilot NIH program, eight drug manufacturers donated 58 molecules that have undergone preclinical and, in some cases, human testing. Last June, NIH awarded nine grants totaling $12.7 million to see if these molecules can provide new benefits in certain subpopulations.

Diseases targeted under the NIH “Discovering New Therapeutic Uses for Existing Molecules” initiative

  • Alcoholism
  • Alzheimer’s disease
  • Calcific aortic valve stenosis
  • Duchenne muscular dystrophy
  • Lymphangioleiomyomatosis
  • Nicotine dependence
  • Peripheral artery disease
  • Schizophrenia

Source: National Institutes of Health–National Center for Advancing Translational Sciences, June 2013

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