The Power of the Patient Lobby: FDA Approves Muscular Dystrophy Drug

September 27, 2016

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

September 19, 2016

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.

Article: Sugar Industry and Coronary Heart Disease Research

SKMC Leaders in Design Thinking

September 12, 2016

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 Nut Addiction, A Public Health Threat with Economic-Driven Pushback

September 6, 2016

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

Mylan Attempts to Quell EpiPen Price Backlash

August 29, 2016

Amidst a growing furor over the skyrocketing price of the EpiPen (an auto-injecting epinephrine administrator used by people with life-threatening allergies), Mylan (the manufacturer of the device) has expanded its financial assistance program. Many question whether these coupons will be enough to counteract the backlash Mylan is currently receiving, online and in Congress.

Although prices for the device have been steadily and steeply climbing for years, these increased prices have recently come to the forefront with higher deductibles and out-of-pocket costs for users of the device. Many link the drug’s price hike to the imminent arrival of a generic version.

Article: Mylan to Offer Some Patients Aid on Cost of EpiPens

Our Physician Leaders Next Door: The Rothman Institute

August 22, 2016

In less than two weeks, the NFL season opener kicks-off and the physicians at Rothman Institute are busy preparing the Philadelphia Eagles for the new season: helping players recover from multiple injuries including one hairline rib fracture and ACL, hamstring, and quadriceps injuries. Additionally, this past month, the US Woman’s Gymnastics Team, also cared for by the Rothman Institute, took home the gold medal at the Rio Olympics. Beyond being a leader in sports medicine, physicians at Rothman Institute are leaders in orthopedics clinical research and innovators in advanced modalities—no small feat considering orthopedics is a competitive and inventive field. One article describes new, trans-disciplinary work published in orthopedics this July, covering diverse and exciting topics including: bariatric patient care, use of magnetic resonance imaging to predict postoperative injury, opioid addiction risks in adolescent athletes, and progress towards lab-grown cartilage for patients with hip injuries (to name a few). As SKMC students, we are extremely fortunate to have the chance to learn from the internationally recognized Rothman Institute.

The leader of this orthopedics powerhouse, Dr. Alex Vaccaro, is speaking this Tuesday at 5:00 p.m. in Forderer Auditorium (snacks will be provided)! Dr. Vaccaro will discuss the foundational strategies and skills that it takes to run an accomplished practice as a physician leader. He is extremely successful inside and outside of the operating room, having authored over 500 peer-reviewed articles, served as President of the American Spinal Injury Association, and led the largest orthopedic center in Philadelphia since 2014. The Rothman Institute is consistently ranked as the finest orthopedic practice in the Delaware Valley. Come to this event and learn about being a physician leader, running a practice, and the intersection of clinical medicine, research, and business!

Article: July 2016 Briefing- Orthopedics

Students Seek to Eliminate Step 2

August 16, 2016

Thousands of medical students, at over 100 medical schools, are currently campaigning to have the Step 2 licensing exam (a standardized test administered in the 4th year of medical school) eliminated. The exam, which has been required for over a decade (after previously being required only for graduates of non-US medical schools), recreates clinical encounters to evaluate students’ interaction with patients and problem-solving abilities. The problem? It is administered in only 5 cities, which students must travel to, and costs $1,275. A student movement and petition to end the exam, citing needless expense to students, began at Harvard Medical School this spring, and has gained national prominence and support, including from the American Medical Association.

 Article: $1,300 to take one test? Med students are fed up.

Cupping: A Common Trend in 2K16 Summer Olympics

August 9, 2016

Cupping, an Eastern medicine technique from the 18th century, has been widely used in this year’s Summer Olympics, including by swimmer Michael Phelps. Cupping consists of applying negative pressure to skin to separate tissues and increase blood flow to the area. The technique leaves characteristically round bruises on the skin due to the rupture of capillaries during the process.

Cupping is becoming increasingly popular not only amongst Olympians, but also in the general population. Physical therapists, athletic trainers, and massage therapists provide cupping, along with other alternative medicine techniques (such as acupuncture and massage). A cupping device can even be found online for at-home-use (the safety of which is undetermined).

While cupping is popular, there is little scientific evidence it confers actual physiological benefit. According to studies, such as one study investigating the effect of cupping on knee arthritis, the benefits perceived after cupping— such as reduced swelling and increased healing rate— are likely due to the placebo effect. Further studies separating physiological and placebo effects are required to better understand the cupping technique.

Article: What Are the Purple Dots on Michael Phelps? Cupping Has an Olympic Moment