At the start of the second day of programming at the 2018 BIO World Congress on Industrial Biotechnology, attendees took in a breakfast plenary discussion as panelists explored biobased materials and how biobased innovations are redesigning markets and fashion trends.

At a conference where biofuels, enzymes, proteins and industrial processes take center stage, some attendees may have been questioning the role industrial biotechnology has in fashion.

However, as moderator Jim Lane, editor of The Digest, emphasized throughout the discussion “there is something here for you.”

Lane comments became the unofficial theme for the day. From workshops designed to help companies grow, a lunch plenary that reflected on World Congress attendee’s contributions to the biobased economy over the past 15 years – while honoring two leaders in industrial biotechnology – and breakout sessions that featured unique topics such as whiskey, Tuesday’s programming was jam packed with unique programming for anyone in industrial biotechnology.

Biobased Materials Redesigning Consumer Markets and Fashion Trends

After reassuring the crowd that industrial biotechnology DOES have a role in fashion, Lane turned over the discussion to experts from several companies that are spearheading biobased threads.

Understanding consumers’ desires for sustainably manufactured products, Christophe Schilling with Genomatica, Inc. dived into his company’s efforts in using recyclable materials to build a circular economy. The segment of the population seeking biobased products is so significant, companies like AMSilk and Okabashi Brands have made it a central focus in their product lines.

“We built the entire brand to go after the demographic that is sustainability focused,” said Kimberly Falkenhayn, President of Okabashi Brands, a company that has been designing and manufacturing flip-flops for more than 30 years.

AMSilk‘s Managing Director and CEO Jens Klein then went on to highlight his company’s work in developing BIOSteel, a strong, durable fiber derived from spider silk that has been used in the production of shoes for Adidas.

Industrial Biotech Thought Leaders: Winners of the George Washington Carver and Rosalind Franklin Awards

As attendees dined in the Grand Ballroom for lunch, the second plenary session of the day kicked off with Dr. Sang Yup Lee from the Korea Advanced Institute of Science and Technology (KAIST) and Krysta Harden, vice president of external affairs and chief sustainability officer at Corteva Agriscience™, receiving the George Washington Carver and Rosalind Franklin Awards, respectively.

In accepting the George Washington Carver Award for Innovation in Industrial Biotechnology and Agricultue, Dr. Lee made sure to give credit to his team of researchers and students back in Korea, who played a critical role pioneering systems metabolic engineering. Dr. Lee left attendees with encouraging words on the overall impact teams like his can have on driving sustainability.

“The achievements our group made is very small,” he noted. “However, if we add up all the achievement you all have made, I can clearly say we are changing the world by providing sustainable chemicals and energy.”

Krysta Harden also had encouraging words for attendees when accepting the Rosalind Franklin Award for Leadership in Industrial Biotechnology and Agriculture by highlighting the progress that has taken place in the last decade in acknowledging the role of women in science – or any field. Harden noted work is still needed for women to receive the same credit as men and dedicated the Rosalind Franklin Award to all women making a difference in their fields.

“This is a great honor for all the women – whether in labs, classrooms, offices or boardrooms – who are making a difference for all of us,” said Harden.

Following the award presentation, award winners joined a group of panelists as they reflected on the past 15 years of industrial biotechnology and the evolution of the biobased economy and looked ahead to what the future may hold.

The Rosalind Franklin Award is sponsored by the Rosalind Franklin Society and the George Washington Carver Award is sponsored by the Iowa Biotechnology Association.

Breakout Sessions

Following the plenary lunch discussion, attendees explored both the exhibit floor and several breakout sessions covering feedstocks for biofuels, whiskey and much more.

At the session “Development of Biorefinery Projects to Convert Forestry Residues to Renewable Fuels,” Phillipp Stratmann spoke on behalf of Velocys, a UK-based renewable fuels company. Velocys is currently developing its first biorefinery using forestry residues as feedstock in Natchez, Mississippi.

“Our process uses 300,000 tons of non-recyclable waste that would otherwise go into landfills,” Stratmann stated. “And for every dollar of feedstock, we’re generating more than $8.00 in fuel revenue.”

Later during the “Nutrigenomics:  Bridging the Gap between Agriculture and Health” breakout session, Fayaz Khazi, CEO of Elo Life Systems talked about how his company is working in partnership with Cargill and other companies to enhance the nutritional profile of agriculture products.

By precisely targeting improvements to food crops, Elo is reducing saturated fat in canola oil, developing protein-rich chickpeas and producing low-calorie natural sweeteners, among other innovations. Representing a One Health mission, Khazi says “it’s really about improving health and wellness through food.”

And as the clock ticked closer and closer to reception time, attendees took in a discussion on the distilling of whiskey and the additional benefits that can be had through the proteins leftover in “pot ale,” the residue left behind in the distillation of whiskey or alcohol.

Workshops & Company and Technical Presentations

In between the breakfast and lunch plenaries, BIO World Congress attendees had the opportunity to attend four sponsored workshops, each outlining a program that could help attendee’s grow their companies.

BIO’s Managing Director of its Industrial and Environmental Section, Rina Singh, participated in a discussion on the U.S. Department of Agriculture’s (USDA) BioPreferred Program, whose goal is to increase the purchase and use of biobased products. Singh provided great context to attendees on the origins of the BioPreferred Program and BIO’s continued commitment and efforts to secure funding for the program through legislation.

Most notably, however, panelists dedicated significant time for attendees to provide feedback and ask questions to USDA representatives on improvements or considerations for the BioPreferred Program.

Of course, as is the case throughout the week, attendees looking to learn about the latest innovations in industrial biotechnology could visit company and technical presentations for an intimate experience with company representatives as they highlighted their new breakthroughs and answered questions.  

Cargill‘s Vice President of Industrials Jill Zullo spoke to a packed room about the company’s successes in replacing petroleum-based products with sustainable bio-industrial systems made from renewable resources. As an example, Zullo pointed to its Cargill Beauty portfolio, a unique range of nature-derived, skin-friendly products with various personal care applications.

“We ask ‘what problems are consumers facing?’ and ‘how are those problems changing in today’s market place,” said Zullo. She went on to note Cargill’s innovations are really focused on “the new natural” to meet evolving consumer demands. But such innovations can’t be created in a vacuum she warns. “No one company can do this alone, so it’s really about partnerships.”

Attendees can look forward to another day of exciting programming tomorrow, Thursday, July 19, as the 2018 BIO World Congress comes to a close.

For those attending the 2018 BIO World Congress in Philadelphia, be sure to be on the lookout for the Iowa Biotechnology Association booth next to registration, where you can learn more about the 2019 BIO World Congress in Iowa. We look forward to seeing you in Des Moines!

Programming for the 15^th BIO World Congress on Industrial Biotechnology kicked off on Tuesday, July 17 at the Pennsylvania Convention Center in Philadelphia.

BIO’s President and CEO Jim Greenwood provided welcoming remarks ahead of the lunch plenary session, highlighting the growth of the biobased economy and BIO’s commitment to fostering innovation in industrial biotechnology.

“I’ve been BIO CEO for more than 13 years, and it has been amazing to see World Congress grow into the biggest showcase of industrial biotech in the world,” said Greenwood.

And the showcase began early, as breakout sessions started at 8:30 a.m. and ran throughout the day.

Breakout Sessions

The breakout sessions gave a glimpse into the not-so-often talked about intersection of biotechnology and industrial and environment. From advancements in the food industry, including flavors and fragrances, algae and enzymes, to industrial biotechnology investments in South America, to biobased products and renewable chemicals, Tuesday’s breakout sessions covered a range of new innovations.

During the “Bio-Industrial Investment in South America” breakout session, panelists looked at the investment climate and national support for bio-industrial solutions in Uruguay. Hank Krakowski with Agrisoma described his company’s work with carinata in the region, an oilseed crop grown to produce renewable, sustainable source of oil, which can be refined into diesel and jet fuel, and a high protein meal for animal feed.

At the “Renewable Chemicals Making Headway into New Materials and Consumer Biobased Products” breakout session, panelists showcased their companies work in developing renewable chemicals that can then be used in partnerships with companies making biobased consumer products. David Sudolsky of Annellotech touched on his companies four guiding principles in partnering on the development of biobased products, including that the product must result in a significant reduction in greenhouse gas emissions, be accessible to large, global markets, and must be reliable and cost effective.

In the “Emergence & Commercialization of Next Generation Ingredients in F&F and other Markets” breakout session, representatives from biotech companies Amyris, Conagen Inc., DEINOVE and Evolva described how biotechnologies such as synthetic biology, advanced fermentation and bioconversion technologies are used by their companies in the development of unique products such as natural sweeteners, cosmetics and skin care – even pest control. And in each of the case studies, speakers emphasized the need to address consumers’ increasing demand for products that are natural and sustainably produced.

To provide for a growing population, “we will need 2.3 planet earths by 2050,” explained Kathy Oglesby of Amyris, which provides more than 2,000 brand products to 250 million consumers every day. “But we only have one planet Earth – biotechnology offers the solution.”

From flavors and fragrances, breakout sessions continued to focus on the trends in production of consumer products, including algae and enzymes for food.

In the “Advancements in Industrial Production of Food Enzymes and Ingredients from New Microbial Platforms” session, panelists examined the bio-processes involved in the manufacturing of novel food ingredients and flavorings.

Henrik Busch-Larsen, CEO of Danish biotechnology company Unibio, explained the conversion of methane and natural gas into protein-based feed additives for livestock. “We’re turning fuel into food, rather than food into fuel,” Busch-Larsen says.

Unibio’s Uniprotein® is produced in an eco-friendly way and is part of the company’s mission to supply the world with innovative, sustainable solutions to overcome the food challenge faced by a growing global population. Watch this video to learn more!

Matt Carr with the Algae Biomass Organization opened the “Algae: A Top Food Trend Today, Potential for Food Security for Tomorrow?” breakout session saying about the intersection of algae and food: “We’ll tell the story to anyone that will listen.” Carr and other panelists went on to highlight the various opportunities for algae in food production, a new trend for the plant that was previously only thought of to advance agriculture and fuel production.

Panelist Jill Kaufman Johnson with Corbion expanded, noting the increasing demand for plant-based protein and the role algae can have. Her lab developed an algae-based goldfish snack that resulted in 30 percent less sodium, 35% less flour and, because of the cell wall that encapsulates the protein, it prevents the cracker from becoming soggy and preserves its “crispiness”.

GreenTech Investor Sessions

Also taking place Tuesday at the BIO World Congress, were GreenTech Investor Sessions, where early stage industrial biotechnology companies had the opportunity to present in front of potential investors and strategists. Included in the GreenTech Investor Sessions were panel discussions in which expert investors and strategists discussed the opportunities and challenges of investing in the industry.

In summarizing the current investment climate, BIO Industrial and Environmental Board Member, and panel moderator, Roger Wyse with Spruce Capital Partners alluded to the challenges the industry has faced as promising technologies have come and gone: “The industry has been through a lot of trauma.”

Underlining previous challenges, Ganesh Kishore, also with Spruce Capital Partners, highlighted the desire of investors to fund companies that make products that are “adaptable” to ensure commercial success.

CRISPR and Gene Editing Tools to Advance Industrial Biotechnology Plenary

Roger Wyse also moderated the lunch plenary on “CRISPR and Gene Editing Tools to Advance Industrial Biotechnology,” featuring a collection of experts and company representatives discussing the future of gene editing technologies.

Most notably, panelists emphasized the need for customer acceptance to allow gene editing to continue progressing and continue to strengthen the biobased economy.

“It’s not rocket science, it’s social science,” said J.J. Jones of Roots & Legacies Consulting during the plenary panel discussion. “Don’t ask ‘what can we do?’ Ask ‘what should we do?”.

Panelist and SR Strategy’s President Sylvia Rowe added that transparency is key. “Transparency is the gateway to trust,” she said. “We’re talking about building a foundation of trust. I think there is a real opportunity to proactively take control of the dialogue.”

Company and Technical Presentations

And not to be missed in the flurry of breakout sessions, investor panels and plenary lunches were company and technical presentations. Several companies presented during Tuesday’s schedule, including Hideyuki Tsukii from Advanced Biochemical Co. in Thailand.

Tsukii presented on his company’s 100 percent bio-based epichlorohydrin (EPI), which when combined with a bio-based BPA will lead to bio-based epoxy resins. During his presentation, Tuskii outlined the benefits of bio-based EPI which is the most sustainable EPI in terms of CO₂ emissions and process environmental performance, and enables downstream industries to reduce its carbon footprint, while increasing the bio-content of finished products.

Read more about Thailand’s presence at the 2018 BIO World Congress here.

With day one in the books, attendees can look forward to even more programming on Wednesday, July 18, with sessions focused on biobased materials, biofuels and the announcement of the 2018 George Washington Carver and Rosalind Franklin Award recipients.

For those attending the 2018 BIO World Congress in Philadelphia, be sure to be on the lookout for the Iowa Biotechnology Association booth next to registration, where you can learn more about the 2019 BIO World Congress in Iowa. We look forward to seeing you in Des Moines!

Listen to the Today’s Homeowner Radio Show for July 7, 2018.

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The facts continue to show that, when it comes to prescription drug costs, the country is heading in the right direction. But this doesn’t mean there aren’t patients who have a hard time affording the medicines they need. In fact, we know:

• Many patients with commercial insurance have experienced a 50% increase in out-of-pocket costs for brand name medicines since 2014;
• The growth in patients’ out-of-pocket costs generally has outpaced the growth in what insurers are spending on health care; and
• Health plans are adopting perverse policies that deliberately inflate patient costs and are discriminating against those with conditions that are more costly to treat.

The insurance industry often claims that these and other schemes are necessary to help keep premiums lower for everyone else. But a new analysis shows that is not the case.

Milliman released the results of a study – commissioned by BIO – that looks at the effects of a California law limiting how much insurers could require patients to pay out of pocket for prescription drugs. The law caps patients’ co-pays at $250 per month for each prescription. Milliman examined how the law impacted premiums for health plans offered through the state’s insurance marketplace.

In a piece published by Managed Care Magazine, the team at Milliman summarizes the results of the study, and they are encouraging to say the least:

• California insurers expected that the cap on co-pays would only increase premiums by 1%;
• The co-pay cap helps patients better manage their drug costs throughout the year, avoiding the financial shock many experience when a new benefit year begins;
• Insurers in California did not expect the utilization or use of prescription drugs to increase because of the co-pay law, which suggests total spending on medicines would remain stable; and
• These results should apply to employer-sponsored health plans offered through the workplace, which is where the vast majority of Americans receive their health care.

These findings show that instead of shifting more costs onto the sickest patients, insurers should be limiting what patients pay out of pocket for medicines and that they can do so without increasing health care costs for everyone else. We know there are positive steps insurers can take right now to do just that. Two insurance companies have promised to pass along to patients the rebates the companies receive from drugmakers to help lower the cost of medicines, and one insurer even stated this commonsense approach would have a “negligible” impact on premiums.

But more needs to be done and more insurance companies need to follow suit. If they won’t, then policymakers need to ask the insurance industry why people are paying more for less coverage, get some real answers and demand better.

The United States Patent and Trademark Office (PTO) is currently considering a rules change that, if enacted, would significantly improve how it conducts patent challenge proceedings. This is a policy change that BIO has long advocated for.

This proposal, entitled “Changes to the Claims Construction Standard for Interpreting Claims in Trial Proceedings Before the PTAB,” will help ensure that patent claims have the same meaning, whether they are reviewed by the PTO, a federal court, or the International Trade Commission. This will reduce abusive practices, such as answer-shopping and repetitive litigation by dissatisfied patent challengers.

This week BIO submitted comments to the PTO in support of the proposed revisions to the standard for construing disputed claim terms in inter partes reviews, post-grant reviews, and covered business method reviews (“post-grant proceedings”).

Read BIO’s Comments

Labeling of GMOs has been in the news a lot lately.  Actually, the issue has been in the news a lot for several years now. People say they have a right to know what’s in their food and how their food is grown. The question is: Why GMOs? Why not other breeding methods like mutagenesis, crossbreeding, or polyploidy? None of them are “natural.” All of them made with man’s intervention. All of them involved changes to DNA.  Why now? GMOs have been on the market in the United States for more than 20 years now – why is there a big push for  labeling GMOs now?

Young man in supermarket comparing bottles of oil, rear view, close-up

Most importantly, should you care if GMOs are labeled? Should you be worried? Should you avoid GMOs, once you start seeing them being labeled? A new study shows that GMO labels reduce peoples fear of GMOs. But does it matter?

GMO Answers volunteer expert Connie Diekman, M.ED., RD, CSSD, LD, FAND, has written a new op-ed in the Kansas City Star that tells what you really need to know about GMOs and labels.

GMO crops have been around since the 1990s. As outlined in the Academy of Nutrition and Dietetics’ review “Advanced Technology in Food Production,” they are grown by farmers in the U.S. and in 28 nations around the globe. The process involves introducing desirable traits in plants, either by selective breeding of healthy strains or by crossbreeding related species.

And 

In 2016, the National Academies of Science released a review of more than 1,000 research publications, which concluded that there was no difference between GMO and non-GMO crops in regards to nutritional value or impact on human health. As a registered dietitian, I found strong confirmation in that review of the benefits and safety of these products.

Further, a 2018 systemic review paper in The Journal of The Academy of Nutrition and Dietetics looked at GE, or genetically engineered, foods – a specialized subset whose genes have been modified using technology – and found their allergenic potential is no different from that of conventional foods.

So while you’re reading these stories about GMO labeling, seeing posts on social media about the issue, remember to stick to the facts and not fall prey to hyperbole and scare tactics.

To learn more about GMOs, visit the GMO Answers website to explore and submit your own questions about the technology.

Next week is the 2018 BIO World Congress on Industrial Biotechnology in Philadelphia. This year’s conference features robust programming for anyone working in industrial biotechnology, including four plenary sessions, 28 breakout sessions across seven tracks, 10 GreenTech Investor Sessions and more than 50 company and technical presentations.

Not to be missed, however, are several sponsored workshops designed to assist attendees of the 2018 BIO World Congress in taking advantage of invaluable opportunities to advance their companies. From learning about finance programs available to help companies develop and commercialize their products to an overview of new technologies revolutionizing the industry, you won’t want to miss any of the sponsored workshops.

The four sponsored workshops will take place Wednesday, July 18, from 10:15 – 11:30 a.m. and include:

A Thriving Bioeconomy: USDA’s Role, Outreach and Finance Programs

Sponsored by USDA Rural Development

A thriving bioeconomy requires research, commercialization, programs and policy to bring biobased products to market. Included in USDA’s Rural Development efforts are research and development, technology transfer-marketing and verification, commercialization and government procurement programs. Additionally, USDA’s grant and guaranteed loan programs finance construction and development of commercial-scale biorefineries and manufacturing facilities and advancement of new and emerging technologies for the production of advanced biofuels, renewable chemicals, and biobased products. At this workshop, you’ll learn about the various opportunities available through USDA to help your company advance in the biobased economy.

Renewable Chemicals and Biofuels Processes Use of Sartorius ambr ® 250 Platform and Umetrics Software for Process Optimization, Scale-Up and Advanced Analytics

Sponsored by Sartorius Stedim

Process development and optimization in industrial biotechnology requires technical solutions which reduce timelines, increase efficiency, and raise likelihood of success. Sartorius’ (booth #317) ambr®15 and ambr®250 fermenters as well as Umetrics Suite of software for advanced analytics has been widely applied to speed up process development and implement advanced data analytics in manufacturing.  Ginkgo Bioworks will detail case studies involving process optimization and scale up from ambr® to 50kL, minimizing developing timelines including an example of taking a shake flask process to economically viable titers in 2 months, scale down from 400kL to 15 and 250mL scale in order perform strain screening experiments, and demonstrating similar performance to 2.5L benchtops when modeling a commercially available process using an insoluble media.  This workshop provides an overview of the technology through product reviews, customer case studies and visions for the future. It wraps up with a Q&A session.

Successful Deals on How to Structure and Close Biobased Economy Financing

Sponsored by Faegre Baker Daniels, Kilpatrick Townsend & Stockton LLP, and New Energy Risk

In this workshop, expert panelists will review the strategies on the first commercial bioeconomy transactions financially closed in 2017 and 2018. Our panelists and moderators constitute the principal investment bankers, lenders, investors and attorneys that structured, funded and/or closed each of these transactions involving biojet, renewable chemicals, biobased products, and renewable diesel projects throughout the country. Each of the transactions were financially closed under different and very sophisticated project financing structures which bodes well for successfully executing new technologies emerging in the respective biobased economy industries now and in the future.

USDA BioPreferred® Program Stakeholder Meeting

Sponsored by USDA BioPreferred Program

During this workshop, you’ll hear from representatives from BIO member companies as they share information about the role of high-value bioproducts in the global bioeconomy, learn about the benefits of working with the BioPreferred program and get the opportunity to provide suggestions and feedback for improvements to the BioPreferred program.

For more information about sponsored workshops, click here. For more information about the 2018 BIO World Congress, click here.

Today, 83 percent of children with cancer survive, compared to just 58 percent in the mid-1970s. Hepatitis C, a once-incurable disease, now has a cure rate of more than 90 percent, thanks to new treatments. And HIV/AIDS – once a death sentence – is now a manageable, chronic condition. This is the power of biopharmaceutical innovation, and it’s been truly remarkable to witness.

This progress would be impossible without the help and leadership of the Food and Drug Administration (FDA) – the hard-working public servants who review, approve, and are ultimately responsible for breakthrough medicines coming to the market in a safe and timely manner. Yet, a recent story by ProPublica takes a cheap shot at the Agency’s personnel, alleging that they are “fast-tracking expensive drugs” to support their budget for scientific reviews – a misleading claim that could not be further from the truth.

A more balanced approach would have highlighted the numerous advances in medicine that have been facilitated by FDA’s work and have resulted in lives saved and quality of life improved for millions of patients, rather than presenting negatives as the norm. A few months saved during FDA review can quite literally mean the difference between life and death for many – though the rigorous evaluation process is still required.  Further, the story fails to note that prescription drug user fees also support significant and important post-market safety activities of FDA, including contributing substantially to the use of the Sentinel database.  This database includes real-world data from tens of millions of patients and provides crucial insights into how those drugs are working once they reach the larger population, as well as safety issues that could not be detected in clinical trials that may need further exploration.

Next, the success of the Prescription Drug User Fee Act (PDUFA) program is hard to understate. As Dr. Janet Woodcock testified before Congress last year, “Before PDUFA’s enactment in 1992, Americans’ access to innovative, new medicines lagged behind other countries. FDA’s premarket review process was understaffed, unpredictable, and slow. The Agency lacked sufficient staff to perform timely reviews or develop procedures and standards to assure a more rigorous, consistent, and predictable process.”

PDUFA has provided FDA the ability to hire more highly qualified scientific and medical staff, to be sure.  However, the increase in staff numbers is only one reason that FDA is able to efficiently and effectively evaluate and make available to patients innovative medicines that are safe and beneficial.

For example, developers of new medicines are able, because of PDUFA, to meet earlier with the Agency, to discuss potential roadblocks, such as possible safety concerns, how best to measure whether the proposed new drug is effective, and how to design clinical trials that have the best chance of answering the key question of whether the potential benefits of a proposed drug outweigh its potential risks.  These discussions help lead to more efficient development programs that are most likely to provide meaningful and valid data.  This has led to a significantly greater chance that an FDA decision about an application can be made in the first review cycle – saving FDA time, company time, and patient lives.  However, neither PDUFA funds, nor meetings with FDA, nor the desire of patients, guarantees success or FDA approval. Every drug approved must meet the FDA gold standards for safety and efficacy. Drug development is risky business – an astonishingly small percentage of investigational drugs make it to the finish line.  In fact, the overall likelihood of approval for an investigational new drug starting Phase I trials remains about 10 percent overall, and only 5 percent for oncology drugs, even with PDUFA’s successes.

PDUFA funds also provide FDA with the ability to meet with the public, including academic experts, physicians and other healthcare providers, consumers, and patients, to discuss creative approaches to developing new drugs.  For example, meetings with the public are occurring to discuss the appropriate use of surrogate endpoints, the evaluation and validation of biomarkers, the use of real-world evidence, and the development of computer models and use of artificial intelligence to identify the most promising approaches to study an investigational medicine’s potential safety and effectiveness.  Such public meetings generally have not been within FDA’s budget capability, so it is PDUFA funds that are allowing these robust and important public discussions to occur.  This influx of funding has allowed the Agency to meet with patients and their advocates and caregivers, and to listen to patients’ first-hand views of their disease.  To hear what patients need, to hear what patients expect, and to determine how best to address this need are crucial aspects of the FDA’s mission.

The ProPublica piece also takes an unfair swipe at expedited approval pathways, the goal of which is to help deliver safe and effective therapies to patients suffering from serious diseases. For instance, the Accelerated Approval program allows for review of a drug based on non-traditional endpoints if it is aimed at treating a serious condition and would fill an unmet medical need. It is a designation that is only judiciously granted.  An approval under this designation can be based on a surrogate or intermediary clinical endpoint, but also requires confirmatory studies to prove the product’s clinical benefit.

Other expedited programs, such as Breakthrough Therapy Designation, simply confer additional Agency resources to an application, to help the most efficient development of products for significant unmet needs and where evidence indicates the potential to provide substantial improvement over current care, including for diseases for which there is no therapy and patients for whom there is little or no hope.

Like biotechnology companies, FDA must stand at the cutting edge of science to meet the needs of patients.  It seems as though nearly every month – or even more quickly – we see new technologies and true breakthroughs coming through the pipeline.  And these high-quality and remarkable technologies are changing the face of medicine. Transformative new technologies such as CAR-T cell therapies for blood cancers, gene therapies for genetic diseases, and next generation immuno-oncology drugs represent real breakthroughs for patients that will change the way we treat disease forever. The developers of such transformative technologies provide strong evidence of safety and efficacy.

We all owe patients a careful and efficient FDA review that will ensure these breakthroughs reach them as quickly as possible.

The social media time capsule was hacked July 4.
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