Compliance In Focus

Recently, the United States Department of Health and Human Services and the National Institute of Health (NIH) issued two proposed rulings that may have a profound impact on how results of clinical trials are made more transparent to the public.  However, before we discuss these proposals, we should discuss briefly how the public has access to clinical trials.

In March of 2014, we wrote about Compassionate use in Unapproved Medical Devices. To briefly summarize, compassionate use refers to the use of an unapproved device or drug in a single patient or small group where the condition of the patient/group is serious and there is no alternative treatment. The FDA can use regulatory discretion to determine if an investigational drug/device can be used and FDA approval is required prior to use.  This clause has come under the spotlight recently with the treatment of two American health care workers and a Spanish priest with an experimental drug, ZMapp, developed by the pharmaceutical company MAPP Pharmaceuticals. ZMapp is a serum-based drug that previously had not been clinically tested in humans.  It represents a number of drugs and vaccines that are currently being developed  to combat Ebola, a single-stranded RNA virus that is highly pathogenic with a fatality rate of up to 90%. According to the World Health Organization, as of 16 August 2014 there have been 2240 reported cases and 1229 deaths from this disease spanning four countries in West Africa (Guinea, Liberia, Sierra Leone, and Nigeria).

Recently we have reviewed the role of direct-to-consumer genetic testing  within the sphere of personalized medicine.  Personalized medicine can refer to tailoring disease treatment, therapy, or prevention to a particular individual, often utilizing some of the genomic signatures that make each of us unique.  Today we will look at a recent FDA news release which looks at personalized medicine from the perspective of the health care professional in respect to two areas: in vitro diagnostic (IVD) companion devices and laboratory developed tests (LDT).

On August 5th we introduced the notion that there is an overwhelming abundance of information available at the fingertips of the consumer.  In Part One we took a closer look at the role of the FDA in Personalized Genomics and direct-to-consumer genetic tests. Now in Part Two we will take a closer look at the role of the FDA in regulating mobile medical applications.

On August 5, 2014 we introduced that the field of personalized medicine has grown rapidly and has encompassed both the field of genetic testing and mobile health apps.  Today we take a closer look at direct-to-consumer genetic testing.  It took thirteen years for the first draft of the human genome to be completed.  In the eleven years since this monumental accomplishment the field of genomics has grown exponentially.  And the fruits of this growth are multi-faceted: tests for disease association, risk, diagnosis, treatment; the list goes on and on.  However the implications for the results of these tests are far-reaching and have a direct affect on both the individual person and on the greater collective.  In the wake of this rapid development of genomic knowledge and increasingly more powerful computers and equipment capable of processing this information, many companies have formed to provide direct to consumer, relatively low cost personalized genomic information.  And with the creation of these companies, the FDA has taken a closer look at the information being disseminated to the consumer.

The amount of information an individual has at their fingertips can be staggering. This can lead to a great deal of empowerment for the consumer in medical, consumer, and social interactions.  In the medical field, improvements in technology have led to a dramatic decrease in the cost to process and analyze an individual’s DNA which has resulted in an influx of genetic tests and information available to an individual. Furthermore, the prevalence of mobile devices/apps (including mobile health apps) has grown exponentially giving consumers an awe-inducing number of choices at the buffet of instant information. As one would expect, this rapid progression has not come without growing pains.  Both the FDA and the companies creating/providing this information have had to consistently re-evaluate how and if this content falls under the auspices of Federal Regulations.

On Friday, May 23, 2014 the Federal Register announced a notice that in an effort to increase the quality and efficiency of clinical trials, the Food and Drug Administration (FDA) was planning to grant a one-year, $7,500,000 grant to Duke University’s Translational Medicine Institute (DTMI) (renewable up to a total of five years, $37,500,000). This grant would primarily go to fund the public-private partnership Clinical Trials Transformation Initiative (CTTI).  CTTI  was originally created as a partnership between the FDA and Duke University in 2008, but now includes more than 60 organizations with representatives from government agencies, industry, patient advocacy groups, professional societies, investigator groups, academic institutions, and other interested parties.

Having a sick child can be one of the hardest jobs of parenting.  Having a sick child that requires treatment and care beyond a quick visit to the local doctor or nurse, or light fluids and lots of rest can cause the worry to rise exponentially.  And what if treatment involves a procedure involving an experimental medical device?  A recent guidance published by the FDA on March 24, 2014 attempts to clarify the department’s thinking on how to effectively protect the pediatric population.  This guidance comes after the FDA issued a finale rule on January 10, 2014 amending PMA regulations to require inclusion of information relating to pediatric subpopulations that suffer from the disease or condition that a device is intended to treat, diagnose, or cure, and the number of affected pediatric patients.  This guidance caps off a series of rules and guidance’s issued over the last 13 years when, in 2001, the FDA issued an interim rule to comply with the Children’s Health Act of 2000.  This interim rule mandated that all pediatric research in the US offer additional protections to ensure pediatric patient safety.

Currently hypertension affects 30.4% of the American population and is projected to cost the nation 91.4 billion dollars a year in 2015.  A subset of hypertensive patients have what is referred to as resistant hypertension.  This refers to a patient receiving three or more anti-hypertensive medications without receiving adequate benefit.  Because of this arbitrary definition, the number of people afflicted with resistant hypertension has varied between 5% in general medical practice and 50% in nephrology clinics. As an alternative to additional anti-hypertensive medication, renal denervation has been used as a therapy to combat high blood pressure.