The Week That Was team is making our grocery list and checking Black Friday sales, eager to try a new turkey brine and score some electronics deals. Before we fade into a tryptophan-induced slumber, let’s review some of the big announcements from last week:
• President Trump named Alex Azar as the new Health and Human Services Secretary. TWTW sources inside HHS say that Azar was well-liked by politicals and career staff alike when he served as the department’s general counsel and deputy secretary during the Bush administration.
• Meanwhile, Bill Gates committed a $100 million personal investment to Alzheimer’s research, with an additional $50 million going to start-up ventures focused on the disease. While some speculate that the boost in funding will expedite a cure, over the past 10 years, more than 99 percent of therapies have failed to make it to market.
But that’s not all. New blood pressure guidelines may have you skipping the pumpkin pie, while a new digestible “smart pill” may give you more indigestion than Aunt Irma’s ambrosia salad. Read on for The Week That Was...
► BP GUIDELINES: WHAT GOES UP JUST WENT DOWN
The American Health Association and American College of Cardiology released the 2017 Hypertension Clinical Practice Guidelines, which lower the measurement threshold for high blood-pressure, or hypertension, to below 130 mm Hg over 80 mm Hg down from 140 mm Hg over 90 mm Hg. The result? An additional 14% of Americans—around 30 million people—are now likely to qualify as hypertensive just in time for Thanksgiving. The updated guidelines are based on SPRINT, a federally funded randomized trial; according to the study, only about 2% of these newly identified patients would likely need medication to manage the condition.
► OUR TAKE
While the revised metrics suggest a large new crop of hypertensive patients, TWTW asks if the thresholds are really that new. Haven’t most of us heard that a healthy blood pressure target is around 120/80? Skeptics of the guidelines raise questions about SPRINT, even suggesting that some primary care practitioners will ignore the target. Guidelines are critical—but only if those you seek to inform believe in the recommendations. We think AHA/ACC could use some value messaging to better explain why they changed their guidelines and what it means for patients and physicians in the real world. When trying to impact behaviors of stakeholders, clearly explaining the rationale behind the change and its consequences to the day-to-day practice of medicine can reassure audiences and help motivate compliance.
► GENE THERAPY FOCUSED ON RETINAL DISEASE
Gene therapy is starting to pick up, as a new therapy for Leber Congenital Amaurosis (LCA) is heading toward approval. LCA is a rare inherited retinal disease that causes a severe loss of vision, usually resulting in blindness. The therapy, Luxturna, is a single dose of an engineered virus that expresses retinal pigment RPE65, a protein deficient in LCA patients. An FDA advisory committee voted unanimously in favor of Luxturna’s approval, which is expected in January 2018. There are hopes that this therapy will advance treatments for the over 225 known genetic mutations which cause blindness.
► OUR TAKE
While Luxturna’s science is certainly impressive, its anticipated costs are stealing the spotlight. Luxturna developer Spark Therapeutics told investors that there’s a case for valuing the therapy at more than $1 million per patient. The Institute for Clinical and Economic Review (ICER), disagreed, saying that “at a placeholder price of $1,000,000, the high cost makes this unlikely to be a cost-effective intervention.” We’re sure that everyone can agree that this launch will be followed closely as providers, payers and policymakers struggle to figure out how to pay for greater numbers of genetically targeted therapies. By offering a one-time cure rather than years of repeat prescriptions, Spark’s CFO Stephen Webster said gene therapy “creates an unusual conundrum because we are fitting a round peg in a square hole.” We advise our regenerative medicine clients to address pricing early to manage expectations of providers and advocacy groups—and to consider devising risk-sharing models with payers to ensure that patients have access to these ground-breaking therapies.
► TRACK YOUR STEPS, CALORIES AND MEDS? APP-SOLUTELY!
This week FDA greenlighted a pill containing an ingestible sensor that allows doctors to track when patients take their medication. Japan-based Otsuka Pharmaceutical Co. combined antipsychotic medication Abilify with the MyCite sensory component to create the first approved “smart pill.” Once a patient takes the smart pill, a sensor “size of a grain of sand” is activated by interacting with stomach acids. The sensor communicates through a patch worn externally by the patient, which sends medicine compliance information to an app. The sensor tracks the date and time at which the medication is ingested, along with other data like activity level. Can you say Fitbit-meets-pharma?
► OUR TAKE
Despite the ‘wow-factor’ of such an innovative approach to monitoring adherence, there are real concerns surrounding patient privacy. Will HCPs assume the role of a “biomedical Big Brother?” Are doctors expected to intervene if patients don’t take their medicines? And specific to this case, actively tracking the behavior of a patient population that can suffer from delusions or paranoia is particularly concerning. Companies pushing the boundaries of biotech—and bioethics—need to hold themselves accountable to these concerns. Proactively discussing these pros and cons with HCPs and others will help educate prescribers while building drug maker credibility. Companies may also want to consider working with a biomedical ethicist to iron out thorny considerations—we keep one on speed dial just in case.
Until next week,
With that, we’ll be signing off to focus on food, family and football—but not before saying how thankful TWTW team is for our readers. After a brief hiatus next week, we’ll be back in your inboxes on December 4. Happy Thanksgiving to all.
- the Reputation & Risk Management Practice