iShares NASDAQ Biotechnology Index logo Maryland Capital Management raised its stake in iShares NASDAQ Biotechnology Index (NASDAQ:IBB) by 200.0% in the 4th quarter, according to its most recent 13F filing with the Securities and Exchange Commission (SEC). The institutional investor owned …

This week, the President’s Cancer Panel published a new report entitled “Promoting Value, Affordability, and Innovation in Cancer Drug Treatment” that examines the price of cancer treatments in America and their value to the patients who need them. With health care costs on the rise, adopting an approach that rewards for quality – not quantity – is garnering support from stakeholders across the board. And as the Panel makes clear, the need to ensure drug prices are aligned with value is “urgent”:

“An ideal framework would integrate information on clinical outcomes, toxicities, impact on quality of life, and costs. It would inform negotiations between drug manufacturers and payers and also could guide development of value-based payment models and benefit designs that promote selection of high-value drugs by physicians and patients. Value assessments also could inform shared decision making among patients and providers and potentially improve patient outcomes,” the group writes.

This is a message that is consistent with what BIO has been saying for some time. In an opinion piece featured in The Hill, Jim Greenwood, BIO’s President and CEO, underscores the importance of a value-based model and discussed the need for expanding this approach across the entire health care system.

“Outcomes-based pricing is already happening”

For starters, Mr. Greenwood explains, doctors and hospitals are increasingly being paid not for the quantity of care they provide, but for the outcome or quality of care patients receive. Our health care system is shifting towards paying for what works, opposed to how much is administered.

“Future treatments will be more accessible and affordable”

Second, because drugmakers are accountable for patient outcomes, a value-based approach encourages insurers to ease coverage restrictions on more costly, innovative medicines. As a result, patients would have access to the affordable treatments they need and greater opportunity to enjoy healthier lives.

“This will bend the cost curve in health care”

Next, by ensuring future cures are accessible and patients adhere to their medications, value-based pricing will lower the trajectory of health care spending. By keeping patients out of the hospital and away for unnecessary doctor visits, we can lower health care costs for everyone.

“Outdated federal policies are standing in the way”

And finally, providing more legal and regulatory flexibility will help expand the use of outcomes-based drug pricing, and more importantly, advance patient health by providing better access to the treatments they need.

While value measurement in health care is not a new concept, groups like the Institute for Clinical and Economic Review (ICER) – who claim to be early advocates for this process – have also proven to lack sufficient input from third-party validators and have not been fully transparent. Echoing similar concerns raised by BIO, the President’s Cancer Panel writes:

“Limitations noted for one or more of these frameworks include lack of patient-centeredness, lack of systemwide perspective, inadequate provisions for updates as new data are obtained, lack of transparency about methodologies, and failure to engage all stakeholders.”

BIO welcomes the efforts by the President’s Cancer Panel and its emphasis to adopt an approach to health care pricing focused on value – not volume. That’s why we continue to highlight the work of the Innovation and Value Initiative (IVI), which recently introduced its Open-Source Value Project. This transparent and holistic approach for assessing value in health care puts patients first, allows for a broad range of perspectives, incorporates the latest available evidence, and considers the full range of scientifically defensible approaches. The promotion of market-based policies will help ensure patients have access to affordable, innovative medicines they need.

A Nigerian- born United States-based Professor of Microbiology and Biotechnology, Prof. Nwadiuto Esiobu, has advised policy makers in the country to embrace the adoption of biotechnology in advancing agriculture, saying that the food security drive of the Federal Government may not be realisable …

One of the leading voices at the intersection of cooking and nutrition is Monica Reinagel or, as active social media users might know her, @NutritionDiva. To kick off National Nutrition Month, Reinagel (a trained chef and nutritionist) wrote a piece about the promise of gene editing, or more specifically CRISPR-Cas9 (“CRISPR”), for the website Food & Nutrition.

As a quick refresher, plants that are genetically edited do NOT carry foreign genes, whereas, the genetically modified foods available in your grocer today are the product of transgenic modification, or the transfer of genes from one organism to another. Not only does gene editing not involve the transfer of genes, but as Reinagel explains, it promises to be even more precise than transgenic modification.

Instead of transferring genetic material from one organism to another, scientists now can edit an organism’s own DNA by deleting a few nucleotide bases (the basic units in DNA) or shifting them slightly. No foreign genetic material is involved. It’s like the difference between using a photo editor to paste one person’s head on another person’s body and editing a few pixels to remove a blemish or fix flyaway hairs in a picture.

And with this new gene editing tool, scientists and farmers alike, are eager about the technology’s potential to enhance crops to solve global issues, like malnutrition.

Genetic researchers working with gene editing, along with farmers and growers, are excited about the potential for CRISPR technology to expedite solutions to a wide array of pressing concerns including climate change, malnutrition and population growth.

Reinagel goes on to note that gene editing, or GenEd as she refers to it, also alleviates concerns shared by opponents of GMOs.

For example, questions about food allergens are largely obviated by CRISPR techniques because no foreign genetic material is introduced in GenEd foods.

Even so, the verdict is still out on how this new technology will be regulated by federal agencies. Reinagel closes with the notion that many scientists agree that the product should be the focus for regulators, not the process for which the product was created.

To the extent that regulations are put in place, scientists are calling for a “product vs. process-based” review system, whereby as new foods are brought to market, they are evaluated according to the attributes and makeup of the food itself, not how these attributes were acquired.

Read the Nutrition Diva’s full piece here.

As we enter a new and exciting era of medicine, there is an important question we need to answer: What if we could measure the efficacy of a medicine, and more broadly, the value a specific medicine will have on patients and society as a whole? To find an answer, efforts like the Innovation Value Initiative (IVI) are underway to develop value-assessment frameworks aimed to assess the worth of pharmaceuticals and other technologies. And as we are learning, not all value assessments are created equal.

In new blog featured in Health Affairs, Peter Neumann and Joshua Cohen of the Tufts University School of Medicine examine the Institute for Clinical and Economic Review (ICER), which has crafted its own way to measure the cost-effectiveness of drugs and technologies, but has come under scrutiny in recent years for their questionable processes and flawed methodology. Here’s why:

Doesn’t Reflect the Full Value of Treatments

Appropriately and accurately measuring value is increasingly important as our health care system seeks to reward quality over quantity and ensure patients have access to the medicines they need. These tools can help us better understand how biomedical innovations can improve the lives of patients and society more broadly, and these findings can help inform future health care decisions. However, ICER’s past ties to the insurance industry make us question who will ultimately benefit from their work. As Neumann and Cohen point out:

“[P]rivately funded assessment … raises questions, and given ICER’s expanding profile, it is important to consider them. A central one is: to whom is ICER accountable? As a private entity with private payers as a key target audience, ICER, however well intentioned, may downplay the “social good” aspect of health care-i.e., spillover effects on caregivers, families, and society at large. For example, treating a person’s cancer, Parkinson’s disease, or substance abuse can have broad benefits for society. Privately-led value assessments tend to ignore or discount such effects, as do private payers, a key audience of ICER’s reports, because the effects fall outside of their budgets.”  

Patient-Stakeholder Perspective Missing

One of ICER’s greatest flaws is its lack of sufficient input from the full breadth of stakeholders and patient advocates, which paints an incomplete picture of value. And while it’s true that ICER has made an effort to incorporate these voices more prominently in their work, the group must do more to ensure these viewpoints are included in the value measurement process. In a recent letter, BIO applauded these efforts as an encouraging step forward, yet groups like the Partnership to Improve Patient Care have noted that ICER’s use of the information these groups have provided is not consistent and their role is not formal.

Barriers to Critical Cures and Medicines Evident

By neglecting the full value and potential of a medicine and ignoring third-party perspectives, ICER’s flawed processes and methodology can detrimentally impact patient care by encouraging insurers to impose higher cost sharing requirements (or outright coverage denials) for some treatments. As Neumann and Cohen explain:

“[B]y identifying cost-effectiveness benchmarks (e.g., $100,000 to $150,000 per QALY) and budget thresholds to which a technology can be compared, ICER weighs in on what level of spending is appropriate. However, judgments about proper spending are better rendered by actual decision makers (payers and their enrollees) in consideration of specific budget constraints, preferences, and tradeoffs.”

Greater Transparency Needed

Believe it or not, until recently ICER had “no formal policy for updating its evaluations based on the availability of important new data,” the Partnership to Improve Patient Care found in a new report. And just this week, A report released by the President’s Cancer Panel highlighted this flaw and others in ICER’s work:

“Some efforts are under way, including those by the Institute for Clinical and Economic Review (ICER) … to develop value frameworks for use in the United States, but none of these is yet widely accepted or used. Limitations noted for one or more of these frameworks include lack of patient-centeredness, lack of systemwide perspective, inadequate provisions for updates as new data are obtained, lack of transparency about methodologies, and failure to engage all stakeholders.”

For a better approach, we should look at value-assessment tools like the IVI’s Open-Source Value Project (OSVP) – a transparent and holistic approach for estimating the value of medical technologies in a way that allows for a broad range of perspectives, incorporates the latest available evidence, and considers the full value of cures and treatments.

Each March, the U.S. celebrates National Women’s History Month, recognizing the great contributions women have made to our nation. It’s a fitting time to announce that BIO is now accepting nominations for the 2018 Rosalind Franklin Award. Every year at the BIO World Congress, BIO honors women who’ve made significant contributions to the field of industrial biotechnology and the advancement of the biobased economy by presenting the Rosalind Franklin Award in Industrial Biotechnology and Agriculture.

While sometimes overlooked, Rosalind Franklin is credited with discovering the structure of DNA. In 1952, while a research associate at King’s College in London, Franklin conceived and captured Photograph 51 of the “B” form of DNA. The photograph was acquired through 100 hours of X-ray exposure from a machine Dr. Franklin herself refined. The discovery was an important advance in modern biology and helped establish the field of biotechnology. Without Franklin’s critical discovery, the biotechnology industry would not be what it is today.

“Rosalind Franklin’s contribution to the discovery of DNA’s structure has been vital to the advancement of the field of biotechnology, but her legacy has often been overlooked,” said Brent Erickson, Executive Vice President, Industrial and Environmental at BIO. “In presenting this award we hope to not only honor Rosalind Franklin’s legacy but also honor and inspire the women working in the field of industrial biotechnology today.”

Nominations for the 2018 Rosalind Franklin Award will be open throughout much of National Women’s History Month, closing on March 28. The Award is sponsored by the Rosalind Franklin Society whose goal is to support and showcase the careers of eminent women in science.

Past recipients of the Rosalind Franklin Award include:

• Vonnie Estes, Independent Consultant, in 2017
• Anna Rath, President and CEO of NexSteppe, in 2016
• Dr. Jennifer Holmgren, CEO of LanzaTech, in 2015
• Dr. Debbie Yavers, Director of Expression Technology, Genomics and Bioinformatics at Novozymes, in 2014

To submit a nominee, or for more information, click here. Also, don’t forget to register for the 2018 World Congress at the Pennsylvania Convention Center in Philadelphia from July 16-19.

This post is part of BIO’s yearlong, bi-weekly series called Flashback Friday, highlighting newsletter stories from BIO’s past.  To learn more about BIO’s history and our 25^th Anniversary visit our interactive historical timeline.

Reprinted from BIONews, February/March 1996

HOW FDA IS CHANGING ITS REGULATION OF BIOTECHNOLOGY

By David A. Kessler, M.D.,  Commissioner, Food and Drug Administration

For the last several years, the Food and Drug Administration (FDA) has been re-examining and reforming some of the ways it regulates bio­logics, including medi­cines made through biotechnology. In the past few months, there has been considerable change in the way FDA deals with these products, and the result will be faster and more target­ed reviews that save biotech compa­nies time and money as they bring needed therapies to the public.

Many of the ideas came from pro­ductive discussion between FDA and the biotechnology community that led FDA to promise certain changes. Today, I can report that we have delivered on those promises. For the changes that have yet to be fully implemented, we are still on target for completion in the time-frames promised.

It is important to remember that everything we do as we modernize our biologic approval process builds on this agency’s fundamental mis­sion: to protect consumers from products under our jurisdiction that are not safe or do not do what they are supposed to do and, at the same time, to promote the public health by getting therapies to consumers as quickly as possible.

This commitment to high standards for safety and efficacy has served consumers well and it has served indus­try well, too. FDA-approved products are the gold standard of the world. Our second responsibility ─ to get products to patients expeditiously ─ requires that we strip away unnecessary regulatory and procedural hurdles. This not only allows industry to move promising products from the bench to the bedside efficiently but also enables FDA to use its limited resources well.

So, what have we done to achieve this? The changes started early last year when the Center for Biologics Evaluation and Research (CBER) addressed two of industry’s concerns. First was the licensure of small-scale or pilot plants. For some biologic prod­ucts, indust1y had to actually build a large-scale production plant before it could receive marketing approval. That led to a few costly mistakes when con­struction started before the drug was shown to be safe and effective. Now, for well-characterized biotech medicines, the pilot plant can be sufficient to win marketing approval.

CBER’s second decision was to sep­arate the filing of a product license application and an establishment license application-PLAs and ELAs. In the past, the FDA required that PLAs and ELAs always be submitted together from the same company, a policy that prevented indust1y from submitting one or the other application when it was ready. We modified that policy so that the two applications could come in at separate times. This could allow a company to receive earlier feedback from FDA on an application than it would have previously.

After additional study, we an­nounced several new measures in November that represent the most sig­nificant overhaul of the regulation of biotech drugs that the FDA has ever attempted. As biotechnology became more sophisticated, it became possible to characterize some biotech drugs as accurately as a traditional chemical­based medicine, suggesting that the two types of drugs could be regulated in the same way. As a result, the agency decided that it could drop the ELA requirements for well-character­ized biologics while not compromising the quality of these products. The pro­posed rule dropping that requirement was published on January 29 of this year. It must still go through a com­ment period and final rulemaking, but the process is well on its way.

In addition, FDA promised to consolidate the 21 different product and establishment license application forms that were used for biological products into a single form that could be used by both CBER and the Center for Drug Evaluation and Research (CDER). The draft form is now available for use for well-characterized biotech drugs. It is now available through CBER’s fax-on­demand system. It, too, will be going through clearance for publication in the Federal Register.

At this point, if a company has a well-characterized biologic for which it is ready to seek a PLA, and the agency agrees that the compound is well characterized, then the company only has to file the new single form. CBER is working with companies to make this transition period as smooth as possible. It is even likely that companies just entering the review process may never need to think about ELAs because they will have been eliminat­ed by the time the product is approved. Of course, if some problem shows up in the rulemaking, ELAs may last longer than any of us expects.

We made several other announce­ments in November including the decision to no longer require biotech com­panies to seek approval to release each lot of a well-characterized biotech product, and the elimination of the need for companies to submit all of their promotional labeling to FDA for pre-approval before they launched a new product. Final rules need to be written, but the process is well along.

Lastly, we promised in November that we would respond to new information submitted by companies in response to a clinical hold within 30 days. We are now doing that. Both CBER and CDER have committed to that response time and are now work­ing on needed internal procedures.

As the discussion between the agency and industry continues, we need to think beyond terms like ELAs and PLAs and focus on the science and the medicine, and then ask what we need to know to determine whether it is safe and effective, and what is the most cost-effective way for the company to submit its infor­mation so we can make good, fast decisions.

The notion that one regulatory struc­ture fits all biologics is outmoded. We simply cannot assume that the ways we have regulated biologics in the past will work for the future. And we no longer are assuming that. I think you will find that this agency recognizes that we need to be flexible enough to, as much as possible, tailor our policies to specific product lines so that safe and effica­cious medicines reach consumers as quickly and efficiently as possible.

In honor of today being International Women’s Day, and March representing National Women’s Month, I’d like to profile a few of the influential women working at the intersection of science and food and agriculture.

Allison Van Eenennaam

Allison Van Eenennaam is a specialist in animal genomics and biotechnology working in the Department of Animal Science at the University of California, Davis. She is a vocal proponent of using biotechnology in agricultural to drive innovation and frequently showcases her expertise and passion on the issue through education and public events. She was the recipient of the 2014 Borlaug CAST Communication Award and was prominently featured in the film Food Evolution. Allison is so adamant about raising public awareness around agriculture issues, such as research funding and consumer acceptance of technology, that her and her team of scientists have created some very entertaining videos to educate the public. Fast forward to 2:33 in her team’s Gene Shop video, a parody of Macklemore’s Thrift Shop, to see and hear Allison’s solo performance.

Emma Naluyima Mugerwa

Emma Naluyima Mugerwa is a smallholder farmer and private veterinarian from Uganda who has advised other local farmers, as well as Uganda’s President, on improving livestock through genetics. She may be best known for her role in the film Food Evolution, where she worked with scientists to educate African communities on the humanitarian benefits of biotechnology in agriculture, specifically gene modification. Emma’s approach to enhancing food using biotechnology boils down to the idea that “everyone needs to eat”.  During her formative years as a student, Emma was taught how to farm for both food and income by her teachers and grandmother. In honor of her work, Emma was asked to give the keynote address at the 2014 World Food Prize, where she discussed her work in Uganda and how we can continue to feed the growing world by leveraging biotechnology.

Mary-Dell Chilton

We profiled Mary-Dell Chilton in our 25^th anniversary piece looking back at her work with  agrobacterium. The discovery of this natural method of gene modification has led to countless innovations in the field of agriculture, contributing to the 4.58 million acres of GM crops sustainably grown around the world today. In 2013, Chilton was honored with the World Food Prize for her groundbreaking work. Like Emma Naluyima Mugerwa, Chilton views the benefits of agricultural biotechnology through a humanitarian lens. In response to winning the Award, Chilton noted “With biotechnology, we are working with nature on a higher level to more precisely determine the outcome of crops. Through ongoing research, we can continually improve their quality and productivity and do this in a way that will allow future generations to provide for their needs as well.”

Nina Fedoroff 

A molecular biologist by training, Fedoroff has long been involved in regulatory issues surrounding genetic modification of organisms (GMOs), serving on countless scientific advisory bodies including the National Science Foundation and the National Academy of Sciences (NAS). Dr. Fedoroff was an author of a 1987 NAS Council White Paper which first articulated the principle that GMOs should be regulated based on their properties, not on the method by which they were derived. In 2007, President George W. Bush awarded her the National Medal of Science. She has contributed to education and public policy pertaining to recombinant DNA and genetic modification of plants.” She lectures all over the world and is a frequent media contributor on the history and science of genetically modified organisms, science diplomacy and the future of food production.

Tamar Haspel

While Tamar Haspel is not a scientist or researcher by trade, she is an influential figure in the food and agriculture space because of her extensive reporting on food and science for the Washington Post’s Unearthed blog. Her blog addresses many of the questions, and subsequently much of the confusion, around science and food. As a journalist she is careful to consider both sides of the debate when writing about biotechnology, or using GMOs, in food production. And, Tamar doesn’t stick to her day job. According to her website, when she’s not “knee-deep in the public food conversation” she gets “dirty” raising chickens and oysters, growing her own vegetables and trying “to stay connected to the idea that food has to come from somewhere.”

The use of fearmongering around genetically modified (GM) crops by the organic industry has led to ballot initiatives in several states and counties looking to ban the technology. Arguably, the most famous example was highlighted in Food Evolution when Hawaii outlawed GM crops only to reverse course and rely on the technology to save one of the state’s most popular exports, the papaya, from being wiped out by disease.

In a recent piece for the Daily Camera, Mara Abbott looks at a similar case currently taking place on the U.S. mainland in Boulder County, Colorado. Abbott writes about Boulder County’s Cropland Policy, which was passed in 2016 and bans the use of GM crops on county-owned open land. As a result, many farmers that lease land from the county are prohibited from growing GM crops.

Spearheaded by county commissioners Elise Jones and Deb Gardner, the underlying goal of the ban was to decrease the use of glyphosate, a pesticide that is commonly used on GM crops. As part of the ban, county officials were to conduct research on alternatives to GM crops to help farmers who leased county land and were now on the wrong side of the law. However, as Abbott points out, the county looked to uneven the playing field in their favor during the bidding process for the research project:

The original research project vision, the Sustainable Agriculture Research and Innovation Initiative – SARII – was scrapped last fall due to accusations of a fixed process in the first bid attempt and a lack of interest from viable tenants in the second.

Fast forward more than a year later and inaction by the county allowed field bindweed to start taking over the land that was to be researched. And, as a result:

Acting on a desire to reduce the use of pesticides, the county commissioners held a piece of land out of production for over a year, hoping to use it for a research project that failed before it even started, during which time enough weeds grew on the property that the county staff had to treat them with the very chemical Jones and Gardner wished to avoid.

Pretty ironic, right? But, what’s even more ironic? While glyphosate is continuing to be used by county officials, farmers looking to lease county land to grow GM crops – which have “allowed the local farmers to be more targeted and effective with pesticides, reducing [glyphosate] use compared to previously grown conventional crops by 80 percent” – are restricted from doing so.

As Abbott writes in closing:

The GMO ban overly simplified the challenges of a complex industry.

Read Mara Abbott’s full piece here.

In recognition of BIO’s 25^th anniversary, BIO CEO and President, Jim Greenwood sat down with two early champions to talk about what triggered the formation of the organization and what we can learn from the past to help shape the future. That future is envisioned by BIO’s current chair, Dr. John Maraganore in a third video.

First up is Fred Frank, the first life sciences specialist in investment banking. In a career spanning nearly 60 years, Frank served as the lead underwriter in more than 125 initial public offerings. He negotiated more than 75 mergers and acquisitions, including some of the largest and most important transactions in the history of biotechnology.

Next Greenwood looks back to 1993-the year BIO was established-with Robert Beckman, one of the founding members.  Beckman is currently a managing partner at The Channel Group which provides management consulting services to life sciences companies.

After talking about where BIO came from, Greenwood talked about where we are going with Dr. John Maragaore, BIO’s current chair and CEO of Alnylam Pharmaceuticals, Inc. Maraganore has led a career pursuing therapies to address unmet medical needs. At Alnylam Pharmaceuticals, Maraganore is helping lead the development of RNAi therapeutics which aim to address genetic medicines, cardio-metabolic diseases and hepatic infectious diseases.