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, Forbes.com

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

Managed Care’s Top Ten Articles of 2016

There’s a lot more going on in health care than mergers (Aetna-Humana, Anthem-Cigna) creating huge players. Hundreds of insurers operate in 50 different states. Self-insured employers, ACA public exchanges, Medicare Advantage, and Medicaid managed care plans crowd an increasingly complex market.

Major health care players are determined to make health information exchanges (HIEs) work. The push toward value-based payment alone almost guarantees that HIEs will be tweaked, poked, prodded, and overhauled until they deliver on their promise. The goal: straight talk from and among tech systems.

They bring a different mindset. They’re willing to work in teams and focus on the sort of evidence-based medicine that can guide health care’s transformation into a system based on value. One question: How well will this new generation of data-driven MDs deal with patients?

The surge of new MS treatments have been for the relapsing-remitting form of the disease. There’s hope for sufferers of a different form of MS. By homing in on CD20-positive B cells, ocrelizumab is able to knock them out and other aberrant B cells circulating in the bloodstream.

A flood of tests have insurers ramping up prior authorization and utilization review. Information overload is a problem. As doctors struggle to keep up, health plans need to get ahead of the development of the technology in order to successfully manage genetic testing appropriately.

Having the data is one thing. Knowing how to use it is another. Applying its computational power to the data, a company called RowdMap puts providers into high-, medium-, and low-value buckets compared with peers in their markets, using specific benchmarks to show why outliers differ from the norm.
Competition among manufacturers, industry consolidation, and capitalization on me-too drugs are cranking up generic and branded drug prices. This increase has compelled PBMs, health plan sponsors, and retail pharmacies to find novel ways to turn a profit, often at the expense of the consumer.
The development of recombinant DNA and other technologies has added a new dimension to care. These medications have revolutionized the treatment of rheumatoid arthritis and many of the other 80 or so autoimmune diseases. But they can be budget busters and have a tricky side effect profile.

Shelley Slade
Vogel, Slade & Goldstein

Hub programs have emerged as a profitable new line of business in the sales and distribution side of the pharmaceutical industry that has got more than its fair share of wheeling and dealing. But they spell trouble if they spark collusion, threaten patients, or waste federal dollars.

More companies are self-insuring—and it’s not just large employers that are striking out on their own. The percentage of employers who fully self-insure increased by 44% in 1999 to 63% in 2015. Self-insurance may give employers more control over benefit packages, and stop-loss protects them against uncapped liability.