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Tag: Parkinson’s Disease

The FDA’s Culture: Should Safety Dominate All Practices?

By STEVEN ZECOLA

An organization’s culture is an internal set of shared values, attitudes and practices. The cohesiveness of the organizational culture will affect whether the entity will meet its vision, purpose, and goals.

One type of organizational culture is hierarchical in nature.   Unlike a risk-taking culture, this structure features policy, process and precision. It is best suited for mature and stable organizations.

The disadvantage of a hierarchal culture is that its stability and control can turn into rigidity. In many cases, the organization develops a negative attitude towards ideas supplied by third parties. It paints itself as having the perfect answer for every issue, no matter how large or small.

My interactions with the FDA suggest that its cultural practices are focused on safety, seemingly to the exclusion of all other issues.  This practice may be appropriate in the regulation of food, but not for drug research where flexibility and creativity are required to cure complex diseases.

Over the past decade, I have witnessed an excessive adherence to its existing practices in the context of BRCA1-related breast cancer, metastatic cancer, precision medicines, “Big Data” and Parkinson’s disease. While the rulings were directed at me, the FDA’s position on these issues has impacted millions of people for the worse.

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The Case to Realign Parkinson’s Disease Research

By STEVEN ZECOLA

If asked, the leaders of the research organizations working on Parkinson’s disease would say that they have made tremendous progress and are optimistic on finding a cure for the disease. 

In truth, this viewpoint understates the magnitude of the challenge and results in insufficient resources being devoted to PD. Given the size of the challenge versus the available resources, most research studies today don’t even include finding a cure for PD as part of their objective.

The time is ripe to get everybody on the same page when it comes to the objectives, resources, and timelines for PD research.

What We Know About Parkinson’s Disease

Parkinson’s disease (PD) is a chronic, progressive movement disorder that affects the lives of almost one million Americans. Roughly 50,000 of the inflicted people die each year, often by injuries from falling.  The incidence of PD is expected to expand to 1.6 million in the U.S. by the year 2037.

The characteristic motor symptoms of PD are tremors, stiffness, slowed movement and impaired balance. Over time, people with PD also experience non-motor symptoms including changes in mood, problems with attention and memory, sleep disturbances, fatigue, and changes in bowel and bladder function.  PD has a considerable impact on the quality of life.

The cost to treat PD has been estimated to be $50 billion a year, split equally between the direct cost of care and the indirect costs of lost opportunities for the patients and caregivers.

PD is a complex disease which is thought to result from an interaction between genetic and environmental risk factors.  More than 20 genes have been identified as having an impact on the onset of PD.  However, genetic variation is estimated to contribute only about 25% to the overall risk of developing PD. Moreover, like the majority of neurodegenerative disorders, little insight is available on how specific sequence variations contribute to disease development and progression.

In short, the exact cause of PD is unknown.  However, we know that that there is more than one manifestation of the disease. We can also reasonably conclude that more than one single element or therapy will be required to cure the disease.

What We Know About Parkinson’s Disease Research

PD was first discovered and described by James Parkinson in 1817 in London, England.

In 1911, the efforts of Kazimierz Funk, a Polish biochemist, paid off with the identification of Levodopa as a potential treatment.

By 1970, the FDA approved the use of Levodopa combined with Carbidopa for the treatment of PD. Since then, this combination has remained the gold standard for treatment.

During the last 50 years, many attempts have been made to improve this treatment and avoid its long-term complications.  While several enhancements have been approved by the FDA and have helped patients, no treatment has cured or slowed the progression of the disease.

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The Perfect Storm: When Parkinson’s Patients Enter the Hospital

By HOOMAN AZMI MD, FAANS 

When a patient enters a hospital either in an elective or more urgent manner, the main focus of the care team is to address the chief complaint. Other diagnoses, while important, may not receive as much attention. While this may not affect patients in most circumstances, it can be very impactful in patients who have Parkinson’s disease (PD). Studies have demonstrated that when patients with Parkinson’s disease enter the hospital, they are more susceptible to developing hospital related complications. Patients with Parkinson’s disease have a higher length of stay (LOS) than those entering the hospital for the same diagnosis without PD and can develop complications such as dysphagia, confusion and falls, impacting their outcomes and increasing their LOS.

Awareness about PD and its treatment and implications thereof are critical in ensuring reduced risks for this patient population. People with PD are very dependent on their medication, and timing of this medication is critical to maintaining good symptomatic control. In the outpatient setting, the main goal of medication management for these patients is to provide as much ON time as possible while minimizing side effects of the medications, such as dyskinesia. ON time describes a period of time when the medications are working and symptoms are controlled. Patients with advanced PD may have considerable difficulty with motor fluctuations if they transition from the ON state to an OFF state when the medication effect has worn off and they are symptomatic. The fine tuning of the medication regimen is pain-staking and often the result of multiple office visits and telephone calls to arrive at the best schedule customized for the patient.  This can often result in seemingly unconventional timings (sometimes on the quarter after the hour) and at time q3 or even q2 intervals. Deviations from these regimens, even as little as 15 minutes delays, can have deleterious effects on patients with PD, as detailed above.

When patients with PD enter the hospital, attention is seldom paid to the exact timing of medication administration.  If a patient takes a particular medication six times daily, ordering the medication six times daily in the hospital defaults to standard timings that often are different from the patients’ own regimen, causing timing errors.  Almost 75% of PD patients who enter the hospital have delays in their medications and more than 60% of these patients can have complications during their hospitalization because of these delays.

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