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Recent announcements that the Hong Kong Stock Exchange (HKEX) plans to allow the listing of pre-revenue biotech companies onto it’s stock exchange has potential to yield significant changes for the global biotech industry. This significant announcement reinforces Hong Kong’s commitment to play a key role in the global biotech ecosystem capable of nurturing companies from development through commercialization stages.

The February 23, 2018 announcement seeking consultation on new rules expanding the listing regime boosts Hong Kong Exchange’s attractiveness for emerging biotech companies. Allowing pre-revenue companies to list on HKEX sends the message that Hong Kong is ready for a dynamic and creative biotech boom.

But what does this really mean for the sector?

After the final round of consultation ends on March 23, 2018, HKEX will publish final conclusions and rulings for pre-revenue biotech listings. A primary benefit of the HKEX ruling will allow for second listings on exchanges. This means all companies that are currently listed on other international exchanges may also seek listing on HKEX. Ultimately this helps companies expand access to public financing.

Garnering investment for biotech projects is challenging for many in the industry regardless of the region. Accordingly, allowing for a second listing may provide legitimacy and aid companies in attracting additional investment to move their product through production. The HKEX ruling supports increasing the collaboration between Chinese and global companies with hopes of expanding the growth that’s already in motion.

But why now?

Over the past 25 years, Hong Kong has been inspired by the Biotech Revolution that swept the early 1980s. Over the past 10-15 years specifically, the Hong Kong biotech investment climate has been particularly challenging. The attrition rate for biotech entrepreneurs seeking financing has been very high with limited success stories.

However, the past 5 years have yielded a new turn for the region’s biotech investment climate. Hong Kong’s biotech industry has become increasingly attractive due to expanding efforts to globalize its industries. More and more, stories of companies finding success after years of financial struggles signaled big changes for the industry.

Hong Kong’s biotech investment climate has improved because there has been a refocused need from government and social enterprises to support healthcare issues. Healthcare has been pushed to forefront and has become increasingly valued across Hong Kong and mainland China.

Another reason for this shift towards biotech has been due to the demands of investors themselves.  With a history of strong investment culture, specifically focused on real estate, more and more investors are looking to diversify. Since entering the biotech sector in the early 1990s, sector growth in Hong Kong has been steady, with steady investment streams from  both public and private stakeholders.

Now in the 21^st century, investors are seeking to diversify and find innovative industries with high potential. Accessibility of pre-revenue biotech companies to list on HKEX allows increased engagement between companies and potential investors. With this new announcement, the strength of its Science & Technology Parks, Hong Kong’s strategic geographic location, and accessibility to the Chinese market Hong Kong is ready for increased biotech business.

Interest in the Hong Kong biotech market is booming and the new HKEX announcement will only aid in developing the sector. With increased investment and increased potential for collaborative partnering, there is great opportunity for Hong Kong’s industry. This potential can already be seen in Hong Kong’s engagement across industry events.

In 2018, as Hong Kong celebrates over 10 years exhibiting at the 2018 BIO International Convention this June in Boston, we look forward to learning more how Hong Kong’s new policy can help fuel biotech innovation both in Asia and abroad. We also invite you to learn more about China’s biotech hubs at the China Summit @ BIO International on Monday, June 4 of the BIO International Convention. Join us for a half-day program to explore China’s regulatory changes, investment trends, and cross-border collaborations in the life sciences sector.

To learn more about Hong Kong’s new HKEX ruling, we hope you tune in to BIO’s President and CEO James Greenwood keynote address to the global investment community on Thursday, March 22^nd at the inaugural HKEX Biotech Summit, as he highlights the global biotech landscape and how collaborations in Hong Kong are helping to make history.

Today we celebrate #NationalAgDay. Started in 1973 by the Agriculture Council of America, National Ag Day celebrates the abundance provided by U.S. agriculture. More recently, the annual national celebration has stretched into a #NationalAgWeek. To help you join in the celebration, we’ve listed below things happening around Capitol Hill all week in honor of American Agriculture:

Tuesday, March 20: Agriculture Secretary Perdue Participating in National Ag Day Events

Agriculture Secretary Sonny Perdue will participate in three events today to commemorate National Ag Day. First, Perdue will introduce Vice President Mike Pence at USDA, who will deliver remarks commemorating President Trump’s National Ag Day Proclamation. Perdue will then head over to the National Press Club to deliver National Ag Day remarks. Finally, tonight Secretary Perdue will be honored with the Abraham Lincoln Vision Award from the National 4-H Council.

Wednesday, March 21: Agri-Pulse Ag & Food Policy Summit

Agri-Pulse holds its Ag & Food Policy Summit this Wednesday at the Hyatt Recency Hotel in Washington, D.C. in honor of National Ag Week. The all-day event will focus on agriculture and food policy and is headlined by some big names in the agriculture sector, including:

• Zippy Duvall, President, American Farm Bureau
• Roger Johnson, President, National Farmers Union
• Ted McKinney, Under Secretary of Agriculture for Trade and Foreign Agricultural Affairs, USDA
• Chairman Pat Roberts, Senate Agriculture Committee
• Senator Debbie Stabenow, Ranking Member, Senate Agriculture Committee
• Sara Wyant, Editor, Agri-Pulse

For more information about the event and to register, click here.

Thursday, March 22: CAST Panel Discussion on Regulatory Barriers to the Development of Innovative Agricultural Biotechnology  

This Thursday, the Council for Agricultural Science and Technology (CAST) will be hosting a presentation and discussion, cohosted by the Association of Public and Land-grant Universities, on the regulatory challenges facing universities and small businesses when it comes to developing innovations. The presentation and discussion will be led by Dr. Alan McHughen, CE Biotechnology Specialist and Geneticist form the University of California, Riverside. For more information and to register to attend, visit the event page here.

Be on the lookout for more content and information about National Ag Day by following #NationalAgDay on Twitter and Facebook.

Puma Biotechnology logo Fred Alger Management Inc. increased its stake in Puma Biotechnology Inc (NASDAQ:PBYI) by 2.0% in the fourth quarter, according to the company in its most recent 13F filing with the Securities and Exchange Commission. The fund owned 190,935 shares of the …

Adding plywood trim is an economical way to convert plain or outdated cabinets to shaker style. Here’s how to go about it.

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.

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.

Danny, Uh oh! My contractor just finished laying 122 square feet of porcelain floors directly over plywood in my kitchen. When I had asked him if he needed me to purchase backer board, he said no, that he could lay it directly over the subfloor. I felt this wasn’t right, which led me to here, […]

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.