Clinical Trials Key to Keeping New Drugs Safe

Patient safety is a core component of our medical system. In order to assure that physicians are prescribing the best and safest treatments, we rely on research, clinical trials and thorough analysis of data. 

One area that is in the process of changing health care is in the biotechnology industry. It's enormously exciting for researchers in the field and also - of course - for patients afflicted with horrific diseases like cancer, renal disease, multiple sclerosis and HIV/AIDS.  

With many of these chronic diseases, health disparities are a real issue; but there is hope if we make sure that minority populations are included in clinical trials.  Ensuring that the data and research our physicians rely on is representative of the entire population will improve the quality of care these patients receive. 

Unfortunately ethnicity, socio-economic status, geography, physical disabilities, and age are all too often limiting factors in clinical trials.  Researchers, seeking to achieve accurate data with limited funds, often ignore the need to include a broad sampling of patients when engaging in clinical trials.  Different people will react differently to medications.  These differences are magnified when dealing with persons of different genetic makeup, compromised immunities, or challenging co-morbidities.

Biologics is a new way of treating patients with some of the most serious illnesses and are chemically distinctly different from other drugs.  Because each drug represents a large, three dimensional molecule - unlike small molecule drugs that we are all used to - biologics are much more vulnerable to even slight changes in their manufacturing.  And as the U.S. Food and Drug Administration (FDA) does more work to ensure biologics are safe, we need to make certain that a wide variety of people, including minorities and people with disabilities, are included in safety tests.

On November 2, the FDA will hold a hearing to identify the steps necessary to establish a process for how biosimilars - which are similar to biologic versions of drugs, but not exactly the same - will be approved in the United States. The FDA hearings will be a first step in laying out the rules of the road for pharmaceutical companies, biotech researchers, physicians and the public at large. 

Throughout the process, FDA should keep patient safety as its guiding principle; understanding that everybody is different, representative clinical trials must be an essential part of guaranteeing that biosimilars are safe for patients.  If everyone is on the same page in ensuring the safety of the medicine supply, good policy decisions will follow.

Two key issues should dominate the conversation.  The first is that unlike with simple drugs, complex, large-molecule biologics are impossible to duplicate.  Different companies will use different raw materials and different processes.  Even slight variations in the biosimilar's makeup could have huge impacts on the drug's potency and safety.  Only after robust clinical trials should biosimilars be able to meet the FDA's mandate for public safety.

And that leads to the second major issue: tracking information and data, which is known as pharmacovigilance.  Pharmacovigilance is science that tracks, assesses, and hopefully prevents adverse side effects of medicine.  It's important that every drug, including generics and biosimilars be subject to the same robust pharmacovigilance as innovator and traditional drugs.  That way, we all can see and identify any harmful trends affecting patient health.  It's the only way to ensure our nation's medicine supply is safe and is accomplishing the goals we expect.   

Understandably, there is a movement to expedite the biologic/biosimilar process.  But in medicine that always opens up the possibility of corner-cutting and public endangerment.  This process must be determined by doctors and researchers - not politicians, bureaucrats or businessman.

In time, biosimilars may become as commonplace in our health care system as traditional generic drugs are today.  But history has shown that indulging in scientific leaps without looking first where we'll land does more harm than good.  We have to move forward with biologics, but we have to do it right.

Dr. Gary Puckrein, president and CEO of the National Minority Quality Forum.


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