The Food and Drug Administration recently approved eteplirsen, the first drug available for use against Duchenne muscular dystrophy, the debilitating muscle wasting disease affecting young boys. The drug works through exon-skipping and is only applicable to certain forms of the disease. Currently, similar methods of gene therapy are also being developed against other forms of the disease.
Expert advisors to the FDA had voted against approving the drug, citing the relevant drug study’s small size (12 boys) and inadequate control group, which they claimed provided insufficient evidence of the drug’s efficacy. A decision on the drug’s approval was delayed for months, and the FDA, despite granting approval, is requiring additional confirmatory efficacy trials. The drug’s ultimate approval is seen by many as the direct result of an impassioned lobbying campaign by patients and their advocates. (Such advocacy groups for muscular dystrophy have historically been highly vocal and organized.) Further, with the approval of this drug, its cost has skyrocketed, adding another layer of complexity to the ethical issues surrounding this case. Many see this drug’s approval as setting a precedent for the influence of patient groups and pharmaceutical companies on medicine, with widespread disagreement over whether this is a good or a bad development.
Article: FDA Approves Muscular Dystrophy Drug That Patients Lobbied For
Not So Sweet: The Influence of Big Sugar on American Perceptions of Diet and Heart Disease
An article published in this week’s JAMA Internal Medicine
outlines the ways in which the sugar industry shifted public discourse about heart disease in the 1960s. A team of UCSF researchers analyzed documents from Sugar Research Foundation, which paid a modern-day equivalent of $50,000 to a team of Harvard researchers in the 1960s to focus scientific blame on saturated fats in the pathogenesis of coronary artery disease. Of course, we now know that both fat and sugar intake play key roles in CAD; however, corporate interests still carry immense weight (pun totally intended) on food and public policy in this country. Just this week, the soda lobby sued the city of Philadelphia
over an approved volume-based soda tax, which was issued to combat epidemic levels of obesity
in the city.
Design thinking, defined as: “a human-centered approach to innovation that integrates the needs of the people, the possibility of technology, and the requirements for success,” has been recently recognized as a powerful tool to address major shortcomings in healthcare. Through the implementation of design thinking, care delivery, medical provider training, and overall healthcare experience can all be improved. Recently, the New England Journal of Medicine recently identified Sidney Kimmel Medical College (SKMC) as a leading innovator in implementing design thinking within medical education.
SKMC is going beyond supplying medical students with an arsenal of memorized facts and is training students to use design thinking to improve healthcare. JeffDESIGN is the first design program that has been implemented in a medical school. In this program, students work with a variety of mentors to solve real problems in healthcare. Success of implementing design thinking in medical education is evident in the outcomes observed at SKMC: an improved patient room, a smartphone app for patients, and a restructured medical curriculum (which was awarded 2nd place at the AMA Medical Education Innovation Conference). Currently, two student groups are filing for medical device patents. Further, design thinking was the basis behind the creation of our very own PEL— which is designed to fill gaps in medical education, better preparing SKMC physicians to become healthcare leaders.
Design thinking, although not novel to the art of medicine, is being re-discovered at SKMC as a feasible way to solve significant problems in healthcare and to develop physician leaders prepared to apply this approach in medicine.
Article: Making Design Thinking Part of Medical Education
Betel nuts are an immensely popular, addictive, and deadly snack in Taiwan, India, Myanmar and other regions of Asia. Chewed by nearly 10% of the world’s population, betel nuts are the 4th most commonly used psychoactive substances (behind tobacco, alcohol, and caffeinated drinks). These nuts, known as Taiwan’s chewing gum, yield a buzz akin to several shots of espresso, or amphetamine.
Alarmingly, this snack gives more than a buzz, being associated with a high incidence of grotesque flesh-eating tumors. In Taiwan, 90% of the 5,700 oral cancers diagnosed each year are in patients with a betel nut chewing habit. Statistically, the cancer will kill 40% of these patients.
As with many addictive substances, kicking the habit is not easy, and faces economically driven pushback from producers and vendors dependent on the significant betel nut industry. Despite these challenges, Taiwan is working to confront this deadly public health threat with new regulations and education efforts on the dangers of betel nuts.
Article: Hard times for ‘betel nut beauties’ as Taiwan tries to kick deadly addiction