Advancing Healthcare: From Testing to Treatment

ON AVERAGE, ONLY 25 new experimental drugs were approved by the FDA’s Center for Drug Evaluation and Research (CDER) every year during the past decade. Taking more than 10 years to go from testing to treatment, the healing properties of these drugs help countless numbers of patients, sometimes providing life-changing and lifesaving results.

And, it all begins in the lab. After pre-clinical studies, a drug moves into the human clinical testing stage. But, as our article “The Perfect Storm for Patient-Focused Clinical Trials” explains, getting patients to participate in trials has proved challenging, causing them to falter in staggering numbers. Fear, access, cost and awareness are cited as the major barriers to trial participation. What can be done? The federal government is investing in developing a national patient-centered clinical research network to consolidate and share data, and new enacted legislation mandates the inclusion of patients in earlier stages of development, which spurred the pharmaceutical industry to develop its own approaches to encourage participation. In addition, many patient organizations are looking at ways to educate patients, make them feel more empowered and make it easier for them to access clinical trials.

PANDAS, a rare neurological disease, is one example of how research to find new treatments is often limited by small participation numbers. Dr. Rodney Lusk, author of the article “PANDAS Treatment,” discusses several case reports that looked at whether tonsillectomy and adenoidectomy might more successfully treat and resolve the symptoms of PANDAS than current treatments. While the results are mixed, more importantly, they reiterate the need for more accurate and thorough studies.

Another challenging area of research centers on autoimmune disorders (ADs), some of which are treated with high doses of human plasma-derived immune globulin (IG). Made from a limited resource, IG is highly expensive to manufacture. Fortunately, as our article “Autoimmune Disease: A More Effective Treatment on the Horizon?” details, a molecular discovery known as sialic acid could pave the way for IG to more effectively treat ADs in smaller doses. Even more exciting is that sialic-switch technology could be used with a laboratory-made molecule to reduce the need for the plasma-derived product.

In fact, research conducted to develop treatments for diseases at the molecular level is a major focus today. In “Precision Medicine: A Seismic Shift in Treatment Strategy,” we look at research being conducted to develop treatments targeted to individual patient needs based on genetic, biomarker, phenotypic or psychosocial characteristics. Precision medicine combines information technology with the field of medicine by creating a biobank of patient data research showing which drugs attack diseases at the chemical and genetic level. Physicians can access this biobank to find therapies that might best treat an individual, minimizing the trial-and-error portion of the treatment process.

The value of new treatments can’t be overstated. As author and blood expert Keith Berman states in his article “The Future Has Arrived: A Wave of New Products Is Redefining Hemophilia Care,” treatment for hemophiliacs has undergone a radical transformation. Berman catalogs the history of coagulation factor products, bringing us up to date on the current extended half-life products that have reduced the number of required infusion sessions and resulted in improved treatment compliance.

As always, we hope you enjoy this issue of BioSupply Trends Quarterly and find it both relevant and helpful to your practice.

Helping Healthcare Care,

Patrick M. Schmidt

Publisher