Monday, May 28, 2018
Calls for change on anti-abortion law in Northern Ireland
Sunday, May 27, 2018
Ebola vaccinations begin in rural Congo on Monday: Ministry
Friday, May 25, 2018
Newborn Screening – How to Fill the Gap to End Diagnostic Odysseys
Many of the over 7000 rare diseases identified so far are not obvious at birth or may come with confusing, non-descript symptoms. Unfortunately, the currently available diagnostic algorithms are unsatisfactory. If a patient's condition remains undiagnosed, the miraculous and rapid advancements in pediatric rare disease therapies mean nothing. The truth is that some families search for more than ten years for the correct diagnosis, which often translates to irreversible damage or even death for pediatric patients. It is essential to find these babies and ensure that they live their healthiest life by receiving the care they need as early as possible.
The painful, costly and sometimes traumatic diagnostic odyssey that patients live through is a result of a few key factors. First, there are not enough newborn screening tests available. A good newborn screening test has a very low false negative rate, a low false positive rate and costs about $1.00. There are newborn screening tests for about 70 conditions available today. This falls well short of the 200 or so rare diseases that can affect newborns that have FDA approved therapies. Second, newborn screening is not consistent. While newborn screening is one of the most successful public health programs in the United States, each state decides what conditions to screen, creating inconsistencies. A baby can be undiagnosed simply because they are born a few miles away in another state that does not screen for a certain condition. While the promise of the Recommended Uniform Screening Panel (RUSP) is to standardize screening panels across states, each state has a different process for adding conditions to their state panel and for providing the resources for additional testing. Finally, outside of the US, there is a baby born every minute that goes undiagnosed because many countries have little or no public newborn screening program, even for conditions for which there are very inexpensive therapies.
A company located in Durham, North Carolina called Baebies, is in the business of advancing comprehensive newborn screening and pediatric testing. The company name comes with an extra "e," because it believes "everyone deserves a healthy start." Finding babies with rare disease using expanded newborn screening is the overarching goal of Baebies which sees the diagnostic odyssey as unnecessary and costly for patients and families. A discussion of how newborn screening reduces rare disease diagnostic odysseys will be held during the BIO International Convention: Newborn Screening: Ensuring All Babies Live Their Healthiest Lives, will be held on June 6 at 11 a.m. in Room 209, Level 2. The session is facilitated by Nicole Boice of Global Genes and the panel includes Richard West (me), representing Baebies, alongside Mark Dant of the EveryLife Foundation for Rare Diseases and the Ryan Foundation, Max Bronstein of Audentes Therapeutics, and Paldeep Atwal of genomeMedical. Let's move towards ending the diagnostic odyssey.
Richard West, Founder & CEO Baebies
With nearly 20 years of life science CEO experience, Richard leads the strategic direction for Baebies. Richard is currently on the board and executive committee of NCBIO, is a member of the Blackstone Entrepreneur Network, and serves on a number of private company boards. Richard is an Adjunct Professor at Duke University, where he teaches entrepreneurship. He has an Engineering degree from Duke University, an MBA from the University of West Florida and completed all requirements except dissertation for a PhD in management from the Peter F. Drucker Graduate School of Management.
Wednesday, May 23, 2018
On the President's Drug Cost Plan, Three Important Concerns
Part 1. Restricting access under Medicare Part D
The Trump administration is out with its plan to lower the cost of prescription drug for America's patients. The plan rightly rejects the flawed schemes we hear regularly from pundits and the media - importation and direct government price negotiation in Medicare - that together would weaken the safety of our nation's pharmaceutical supply chain, reduce patient access and do little to lower costs.
The plan contains some positive ideas, such as encouraging value-based pricing agreements, fixing the flaws in the 340B drug discount program, limiting pharmacy gag clauses, and passing rebates through to patients in Medicare Part D and capping their out-of-pocket costs. However, buried in the details are proposals that could harm the ability of patients to get the medicines they need both now and in the future.
Today, BIO is starting a blog series that will highlight three proposals in particular that could pose significant challenges to patients. As the first in a three-part series, this blog will look at proposals in the president's drug pricing plan that would affect a popular prescription drug program for America's seniors.
Restricting access under Medicare Part D.
The Medicare Part D prescription drug program has been a remarkable success for both seniors and taxpayers.
- The program cost $349 billion less over 10 years than initially expected;
- Average premiums dropped 3% in 2018 to less than $34 per month; and
- Hospital admissions for seniors have also dropped by 8%.
It's no wonder then that nearly 90% of seniors are satisfied with the program.
Medicare Part D generally covers medicines that patients buy at a retail pharmacy and take themselves. The private health plans administering the Part D program are required to provide at least two drugs for each class of drugs that exists. Then there are six "protected classes" of drugs for patients facing particularly devastating diseases like cancer, HIV and mental illnesses. Health plans are required to cover "all or substantially all" medicines that fall within these protected classes.
Current policies help provide seniors and people with disabilities with choices, and more choices means more competition that can lead to lower costs. However, under the Trump administration's proposal, health plans would only be required to provide one drug per class, down from the current requirement of at least two. The administration may also eliminate some of the protected classes altogether. These changes would leave doctors and patients with fewer options, forcing beneficiaries to pay more if the medicines their doctors prescribe aren't covered.
Health plans would also be allowed to increase the use of so-called utilization tools – such as "step therapy" or "prior authorization." These bureaucratic hurdles are already far too common, and while these "tools" may help insurers protect their bottom line, they also bury doctors in paper work and make it harder for patients to get the right medicines at the right time.
The nation's biopharmaceutical innovators support taking steps to strengthen the Medicare Part D program. That effort should focus on lowering seniors' out-of-pocket costs and ensuring they have broad access to the medicines they need.
Stay tuned for the second part of this three-part blog series, which will look at proposed changes to the Medicare Part B program.
Saturday, May 19, 2018
Friday, May 18, 2018
2017 Rosalind Franklin Award Recipient: CRISPR has the potential to address important challenges
As we've covered here before, gene editing holds tremendous promise in advancing modern agriculture. And it's not hard to see why. With gene editing tools like CRISPR, researchers have the capability to engineer plants to be, amongst other things, better for the environment, more nutritious and disease-resistant.
BIO's 2017 Rosalind Franklin Award for Leadership in Biotechnology recipient Vonnie Estes is an agtech innovation consultant who is tuned into the current conversations happening at farms and agricultural businesses across America. And a lot of those conversations revolve around the new gene editing technology.
In a blog for Ag Funder News, Estes entertains the question: Should You Be Using CRISPR for Your Ag Business? In addressing this question, Estes starts by outlining the "number of challenges" that must be addressed before CRISPR becomes widely adopted, including the regulatory landscape:
One of the biggest restrictions to the global commercial use of gene editing is uncertainty around regulation. The USDA just ruled it would not regulate plants that could otherwise have been developed through traditional breeding techniques, as long as they are not developed using plant pests. This is a good start, however, there are a limited number of improvements that can be made within these criteria. If Agrobacterium is used as a delivery method or a greater number of genes are edited - which will be needed for complex traits - it will be considered GMO and more highly regulated. Most crop genetics and food production companies operate globally: shipping seeds, plant materials, or final foods worldwide. The EU has not yet ruled if gene edited crops fall under the genetically modified organism rule, although there is some indication that may change. Until this is clear, many crop genetics companies will not risk the potential trade barriers of using gene editing technology.
Vonnie Estes, Agtech Innovation Consultant
Estes then goes on to note that because of the contentious debate around GMOs and the public's confusion around the difference in the science behind the two technologies, gene editing will be susceptible to similar pushback. However, Estes believes the younger generation of consumers will be receptive, especially if the benefits are clearly defined and explained:
Consumers are more likely to accept the technology if they get a direct benefit, such as better taste or higher nutritional value. Food producers are right to be cautious, but I'm hoping the younger, tech-savvy, more demanding food consumers will be accepting of gene editing technology.
Nevertheless, the potential for the technology to solve some of the world's food challenges is encouraging and the science community is enthusiastic:
As the world's population rapidly approaches eight billion, modern food production methods will need a radical transformation. Gene editing can certainly help meet that challenge of production, along with making food more flavorful and nutritious. The path to widespread usage is currently complicated – from the science to consumer acceptance. But with the tremendous enthusiasm of the research community, gene editing technologies will improve rapidly along with the all of the techniques and technologies needed to support crop use.
Read Vonnie Estes full piece at AgFunderNews.com.
Tuesday, May 15, 2018
Food Evolution Writer/Producer Trace Sheehan to Keynote Food & Ag Program at #BIO2018
Trace Sheehan, writer and producer of the highly-acclaimed documentary film Food Evolution, will be a keynote speaker at BIO's International Convention in Boston, the Biotechnology Innovation Organization (BIO) announced today.
Since its premier in November 2016, Food Evolution has contributed to a robust discussion about science, facts, and food. The film focuses on the GMO debate because the filmmakers found it to be emblematic of the public misunderstanding about the science of food and food sustainability. In his keynote on Tuesday, June 5, Sheehan will share his experience in making the film and explain how the process influenced his own opinions along the way.
Other food and ag-related programming to note:
On Monday, June 4, BIO will host "One Health Day," a half-day of speakers and panel discussions focusing on how the One Health concept – the interconnectivity of human, animal and environmental health – can help address today's greatest challenges. The program starts with a keynote presentation and panel introducing the One Health concept before diving right into an exploration of the business case for One Health with a panel of senior leaders from the private sector. The program also looks to global perspectives on One Health policy development and insights into U.S. government funding and research decisions.
On Tuesday, June 5, a second half-day of programming is dedicated to food and agricultural innovation in our "Food, Health, and Environmental Future Day." This program, featuring Keynoter Trace Sheehan, will include this series of panel discussions:
- A Two-Part Food Innovation Dialogue: Cutting-Edge Food, Health, & Environmental Products and Tools…Coming to a Grocery Store Near You(?)
Part 1 looks at the landscape of innovation now and on the horizon, including applications of genome editing in animals and plants, microbes for soil health, and more…
Part 2 brings the food value chain together in a conversation about consumer preferences and trends around sustainability and transparency, and how the innovations discussed in Part 1 will fit in today's (and tomorrow's) marketplace. - Biotech in Our Backyard: New England's Booming Food and AgTech Innovation Ecosystem
- AgTech Investment: Opportunities for Plants, Animals, Microbes, and Beyond…
On Wednesday, June 6 and Thursday, June 7, BIO's 2018 International Convention also features a track dedicated to Genome Editing, including programming about Engaging Our Nation and Our World in the Era of Gene Editing and genome editing as The Next Frontier in Agriculture.
Held in conjunction with BIO, CRISPRCon: Conversations on Science, Society and the Future of Gene Editing will take place on June 4-5 at the Boston Seaport Hotel & World Trade center (Separate registration is required).
The BIO International Convention, the world's premier life sciences event, will take place at the Boston Convention & Exhibition Center, June 4-7. Hosted by BIO, the 2018 event is expected to attract more than 16,000 attendees and 1,800 exhibitors from 74 countries.
Advance media registration is now available for qualified reporters working full time for print, broadcast and web publications with valid press credentials.
