Just 10 percent of the drugs that get to the Food and Drug Administration’s Phase 1 testing process make it to market. Innovation is difficult, and it is costly, but still U.S. scientists and researchers persist. As a result, we are in the midst of a biopharmaceutical revolution. Research that began a generation ago is finally paying dividends with researchers developing a breathtaking number of treatments and cures for diseases and chronic illnesses that were previously untreatable.
And there’s more: firms headquartered in the U.S. are conducting more than 61 percent of the clinical programs in the world, meaning these companies will generate the treatments and cures of tomorrow. We highlight some of this work in the news stories and posts below, but email us if you need more info like this for a story, or have questions. We look forward to working with you.
Patrick O’Connor – Executive Director
Rosemarie Calabro Tully – Communications Director
TWEETS OF THE WEEK
Other countries that have adopted value-based #DrugPricing models demonstrate why @icer_review’s proposed #QALY model would harm US #patients: Britain's @NICEComms has denied coverage of medicines that would benefit about 16,000 #cancer patients annually. https://bit.ly/2DyKK7K Click here to RT.
“It is extremely concerning to see numbers assigned to value, or worse, devalue the life of a person with a disability or chronic condition.” Why else is @icer_review's proposed #DrugPricing model cause for concern? Read more via @MorningConsult: https://bit.ly/2IYwfxC Click here to RT.
.@PioneerBoston's Dr. William smith explains how #QALY systems like those proposed by @icer_review threaten the health and quality of life of “millions of people waiting for cures.” Read here via @statnews: https://bit.ly/2VchD0s Click here to RT.
.@icer_review has proposed using #QALY systems in determining #DrugPricing. But 82% of American voters find these kinds of QALY systems concerning. Why are #patients, doctors & others in the health care industry against value-based pricing? Read here: https://bit.ly/2ZiCX7q Click here to RT.
THE STORIES THAT DIDN’T GET ENOUGH ATTENTION
An Update On Drug Innovation In America. IQVIA, a resource for health research data, released a report this week that provides an overview of pharmaceutical innovation in America in 2018. The Axios headline heralding the study says it all: “Prescription drug R&D is thriving” in the United States. We explore IQVIA’s results. Read the full blog post here.
Arnolds’ Funding Research Outcomes In States. The debate on healthcare costs extends far beyond Washington and into state legislative chambers. Just as this debate extends beyond the Beltway, so does the influence of John and Laura Arnold, and Arnold Ventures. Read the full blog post here.
In case you missed them, here are some other recent posts on the APMI blog:
WHAT WE’RE READING
How Do We Lower Drug Prices? Address The FDA Approval Process. That’s what Mercatus Center Senior Research Fellow Robert Graboyes argues in The Hill. Graboyes says, “We can lower costs. … The biggest key likely lies in a speedier, less costly, more predictable FDA process. Achieving that, however, will require a long, concerted effort and some tough political decisions. Much tougher than placing faith in simple, quick solutions.”
What Won’t Work? “Value” Based Assessments. In an op-ed at Morning Consult, Randall Rutta from ConnectHealthLLC writes, “Comparative and cost-effectiveness analyses by third-party groups are a key component of socialized health care systems. … The use of such assessments to determine the value of treatments could significantly limit access to treatments not determined to have adequate ‘value’ under these broad, population-level studies. In an era of patient-centered, personalized medicine, resorting to such tools at the expense of the nation’s most vulnerable is inappropriate.” At the American Thinker, Patients Rising Executive Director Terry Wilcox also argues against so-called value assessments.
Innovation Stops Cancer? Maybe. According to STAT News, Arrakis Therapeutics has “devoted the past two years” to creating “a platform capable of discovering compounds that work against RNA.” As a result, the company has announced an “early breakthrough” in its quest to develop a pill that will block “RNA machinery needed to make some well-known, cancer-causing proteins.”
Innovation Prolongs Life: Study Says New Drug Can Be Used To Slow Kidney Disease. As The Associated Press reports, nearly 30 million Americans have diabetes, and most cases are Type 2, which “can damage the kidneys over time, causing disease and ultimately, failure.” That’s why the wire service says “it’s hard to overstate the importance of” a new study that found a drug that can “help control blood sugar in people with diabetes” and “help prevent or slow kidney disease.”
Innovation Prevents Disease: Ebola Vaccine Is Working, And New Malaria Vaccine Has Just Come Online. STAT News reports “the experimental Ebola vaccine being used to try to contain the outbreak in the Democratic Republic of the Congo is protective 97.5% of the time, according to new data released by the World Health Organization. The data … suggest a ‘very impressive’ performance by the vaccine.” Additionally, NPR reported this week that the world’s first malaria vaccine is now being used in sub-Saharan Africa.
Innovation Saves Lives: New HIV Drug Will Help More Patients Manage Disease. org, a global resource for information and educational resources on HIV and AIDS, reports the FDA has “approved the first complete HIV treatment regime containing only two antiretroviral drugs,” an advance that means “treatment-naïve individuals can benefit from a highly effective regime with fewer component drugs, reducing their lifetime cumulative exposure to different drugs and potential long-term toxicities.”
Innovation Cures: After Decades of “Painstaking Research And Heartbreaking Setbacks,” Gene Therapy Cures 10 Newborns. The Washington Post reports gene therapy has been used to cure ten newborns with the rare genetic disorder called “bubble boy” disease. The Post says, “The treatment appears to have completely rid the babies of their immune disorder with no side effects or complications — a result scientists have sought for decades through painstaking research and heartbreaking setbacks.”
The Cost Of Developing Gene Therapies. In a Morning Consult op-ed, the American Consumer Institute’s Dr. Joseph Fuhr explains the promise gene therapies offer for the 25 to 30 million Americans living with rare medical conditions. Innovation is time-consuming and costly, though. Dr. Fuhr explains, “The R&D investment required to create a novel gene therapy will likely exceed the $1.3 billion to $2.6 billion needed to develop a new biologic drug. Likewise, the failure rates in the fledgling gene therapy market are expected to surpass those of the pharmaceutical industry as a whole, where fewer than 1 in 10 drugs that reach clinical trials ever make it to market.”
QUOTATION OF THE WEEK
The Wall Street Journal editorial board notes the U.S. Department of Health and Human Services has the authority to allow drug importation. But hasn’t – perhaps because it’s not safe:
“Health and Human Services has had the authority since 2003 to allow importation, but the secretary must certify the practice would pose ‘no additional risk’ to the public’s safety, and ‘result in a significant reduction’ in cost for the ‘American consumer.’ These are high bars. No secretary has ever made such a judgment.”
At PennLive, Patricia A. Epple, CEO of the Pennsylvania Pharmacists Association, explains how pharmacy benefit managers are driving drug prices higher (by the way, PBMs are reportedly spending a record amount on lobbying these days):
“Discounts provided by pharmaceutical companies, often up to 30-40 percent, are negotiated with PBMs supposedly to lower prices for patients through their insurance plans. PBMs then negotiate with pharmacies on what they will actually pay them for the drugs to be dispensed to their customers. Yet, when just three PBMs control 85 percent of the market, such negotiations are often one sided and local pharmacies are getting squeezed out of business. … With nearly five billion prescriptions being dispensed each year and near monopoly control, PBMs have quietly asserted themselves as key drivers in the cost of drugs paid by consumers.”
UPCOMING EVENTS TO WATCH
Tuesday, April 30, 9:30 a.m.: U.S. House Energy and Commerce Committee
Location: 2322 Rayburn House Office Building, Washington, D.C.
Topic: Prescription Drug Coverage in the Medicare Program
Contact: (202) 225-5735