Physicians collaborating to cope with drug shortages

by Kimberly McGhee
Business Development & Marketing

As patients, we don't think too much about where our drugs come from. We just expect them to be available when we need them.

But what if a new, potentially life-saving drug for your condition were not yet available for human use because its development had become bogged down because of financial or other pressures? Even worse, what if a standby drug, one long used to successfully treat someone with your condition, were suddenly not available?
MUSC is working hard on both fronts to ensure that the drugs you need will be available. As two articles in the April/May issue of Progress Notes show, MUSC is working both to discover and develop the new drugs of tomorrow and to ensure an adequate and safe supply of today's standby medications.

Drug Discovery

Patents on drugs last only 20 years and it can take 8 to 12 years to bring a drug to market, leaving a pharmaceutical company only 8 to 12 years to commercialize the drug. In that time, it must try to recoup its initial investment, estimated to be about $1.3 billion for a new compound. With many of its "blockbuster" patents expiring, the pharmaceutical industry has become wary of investing that much time and money into the development of a novel compound that could prove to be a dead end. Instead, it has begun to collaborate more with academic medical centers and biotechnology startup companies to do the initial legwork.

MUSC is now able to perform most of the functions typically performed by a drug company, including identification of a target (a molecular or cellular entity important to the pathology of a disease that can be influenced by a drug), identification and optimization of a compound, preclinical studies of the compound's efficacy and safety and clinical trials in humans. The further down the development pipeline that a researcher can take a promising compound, the more "de-risked" and therefore attractive it will be to a pharmaceutical company. Because commercializing a drug is prohibitively expensive, the project is usually passed off to industry before any phase 3 trials (large multicenter trials) are undertaken.

Industry benefits from this collaboration with academia by assuming a project after some of its risk has been removed, and academic medical centers like MUSC benefit by seeing their research translated more rapidly into patient care and by recouping their research investment. And, of course, patients benefit by having access to cutting-edge treatments more quickly.

Ensuring Safe, Ample Supply

In the best of times, the profit margin for manufacturers of generic drugs is narrow. Recent changes to Medicare reimbursement have further narrowed or erased that margin, leaving generic manufacturers with little incentive to continue producing some drugs.

Injectable drugs, which can be more difficult and therefore expensive to manufacture because sterile conditions must be maintained, have been the most affected. These include standard-of-care oncology, anesthetic and antiviral medications, many of which are currently in critical shortage nationwide.

Manufacturers either shut down production of such drugs to focus on more lucrative medications or dedicate only one production line to them. Mergers between pharmaceutical companies are occurring more frequently, meaning that duplicate production lines can be shut down and overall capacity to produce the drug is decreased. Factories housing production lines for generics—sometimes the single production line for a given generic medication—are sometimes not properly maintained or updated, meaning that the shortage of a critical injectable drug is only a manufacturing hitch or bad inspection report from the US Food and Drug Administration away.

MUSC physicians are collaborating to cope with such drug shortages. They monitor carefully the drug needs of patients and the current supply of such drugs, finding appropriate alternatives where necessary. They are also acting nationally to address drug shortages. Michelle Hudspeth, M.D., chief of Pediatric Hematology/Oncology at MUSC's Children's Hospital, testified before Congress about the consequences of drug shortages for patient care and some potential underlying causes, and Heather Kokko, PharmD, director of Pharmacy Services at MUSC, helped write an expert panel recommendation on how to cope with such shortages.

That report warned against hoarding drugs, which could cause a patient in need at another hospital to go without the necessary treatment and against buying from grey marketers, who sell drugs of questionable origin and efficacy. Such marketers capitalize on shortages to charge exorbitant prices for drugs that could be stolen, counterfeit or lacking in efficacy due to improper storage.

MUSC is working hard both to design the new, more effective drugs of tomorrow and to make sure that the drugs we depend on today are safe and available when we need them.

To read more about drug development at MUSC and other stories, visit the April/May issue of Progress Notes, available at http://www.MUSChealth.com/progressnotes.

Editor's note: "Progress Notes" is a bimonthly publication produced by Business Development & Marketing Services and sent to all physicians licensed in South Carolina to inform them about clinical and research innovations at MUSC.

Friday, June 1, 2012