Clinical information tends to be more complex, comes from multiple sources in different formats. As a result, clinical data submission has become time-consuming, costly and error-prone. CDISC® (Clinical Data Interchange Standards Consortium) established new data standards to speed up data-review and improve clinical data exchange, storage and archival. Our technology edge combined to our experience in standards implementation allows us to develop tailored CDISC solutions to accelerate your FDA review. Clinovo introduced a new opportunity to learn these recognized clinical data standards!
Clinovo’s new “CDISC Standards: Theory and Application” class is an 8-week training program starting in June 11th, 2013. The TechTrainings are technical hands-on classes for entry-level or experienced clinical trial professionals designed to help them reach the next step in their professional career. The class will be held in Palo Alto at Dentons Offices or remotely.
Taught by Sy Truong, President at Meta-Xceed and author of award-winning papers, this new course will give an overview of CDISC standards: ODM, SDTM, ADaM and Define.XML. Students will learn how to transform legacy data into these clinical standards through real-life examples. Case studies will include data exchange, archival, and electronic submission to regulatory agencies such as the FDA.
Clinovo will continue to offer the “Base Clinical SAS Programming” class to help entry-level programmers prepare the Base SAS certification, as well as the “Advanced Clinical SAS Programming” class to tackle advanced real-world SAS programming challenges. Clinovo offers $50 gift cards for referrals.
More information on the class can be found on clinovo.com/techtrainings.
Olivier Roth launched the TechTrainings by Clinovo in 2012, a series of hands-on courses for clinical trial professionals, leveraging his company’s years of on-field experience and industry expertise. He is the Marketing & Communication Coordinator at Clinovo, a CRO based in Sunnyvale, focused on streamlining clinical trials for life science companies through technology solutions. Olivier helps managing Clinovo’s marketing and communication from marketing strategy to partnership management, lead generation, event planning and new business opportunities. Prior to Clinovo, Olivier was working as a Strategic Marketing Consultant at VivaSante, an international consumer healthcare company based in Paris.
2012 was a good year for the life science industry, with a 7% growth in R&D pipelines and 35 new drugs approved by the FDA, delivering some truly innovative, needed therapies. Challenges faced include the feared patent cliff as well as passing of the medical device tax set forward in 2010.
This year, the FDA approved 35 new drugs during the fiscal year, which ran from October 1, 2011 to September 30, 2012. The approval rate in 2012 matched that of 2011, both reaching higher rates than previously seen.
Many of these new drugs are successfully going through the review process as they are addressing unmet needs for deadly diseases. Additionally, the FDA met their deadlines 34 of 35 times, surpassing their PDUFA goal of 90%. Since the close of the fiscal year, no less than 3 new drugs have already been approved.
However, the Sequester may affect the FDA approval rates and goals. The Sequester, passed in August 2011 as part of the Budget Control Act (BCA), is a package of automatic spending cuts projected to total $1.2 trillion, scheduled to begin in 2013 and end in 2021. Part of these automatic spending cuts will be geared towards FDA budgets, which could be forced to slash jobs and thus lose some of its capacity to approve drugs for 2013.
On the basis of third-quarter earnings, Ernst & Young estimates combined sales at the global top 13 drug companies dropped by close to 4% this year from about $557 billion in 2011.
With the patent cliff taking its toll on some of the best selling drugs ever known to our industry, the biggest blockbuster of 2012 is Humira from Abbott Laboratories, totaling $9.48B. Initially approved for the treatment of rheumatoid arthritis, the money maker is drawing on the expanded treatment of 5 additional indications. In total, the top 15 selling drugs generated $95.55B in revenue (and counting).
In addition, 6 companies found themselves in the Top 10 most charitable organizations over the last year. Taking the top spot was Pfizer with over $3.06B in cash and products donated. Merck (#3), Abbott Laboratories (#7), Johnson & Johnson (#8), Eliy Lilly (#9), and Bristol Meyers Squibb (#10) round out the list with all 6 companies donations totaling $6.9B.
The Patent Cliff
The patent cliff has taken a large effect this year, and by 2015 will have an estimated $250B in lost sales for the industry. The once mighty Lipitor has fallen from the top spot to number 10 this year, with a 42% sales drop in the first quarter of 2012. Four other best-sellers lost patent protection this year as well, affecting $22.8B worth of retail sales for 2012.
Capital Market and Merger And Acquisitions (M&As)
As of the third quarter 2012, 14 U.S. life sciences companies completed IPOs, raising almost $950 million. Twelve of the 14 U.S. companies are in drug discovery and development, and California companies account for half of the IPOs in 2012.
The $146B M&A market this year found some much watched participants, including the acquisition of Human Genome Sciences by GlaxoSmithKline. However, overall M&A fell by 35% in 2012, down $79B from last year. According to the 2013 Baybio 2013 California Biomedical annual report, As of November 2012, 53 M&A transactions were recorded of California companies of which 33 were biopharmaceutical, 18 diagnostics and two R&D companies. The top M&A deal was the acquisition of Amylin Pharmaceuticals for $10.6 billion by Bristol-Myers Squibb in collaboration with AstraZeneca.
According to sources, multi-billion-dollar acquisitions were outnumbered by more modest research pacts and partnerships in which drug companies acted to add promising compounds to their pipelines. The early weeks of 2012 brought a flurry of research pacts focused on oncology, typical of a more targeted approach. Takeda Pharmaceuticals, Merck & Co., Eli Lilly & Co., and AstraZeneca all gained access to compounds that biotech firms were developing for cancer.
Overall, U.S. life sciences (biotechnology and medical devices) investment for the first three quarters of 2012 is down 19 percent in dollars and 12 percent in deals from the same time period in 2011.
Investments in emerging markets increased by 65% this year, reaching a total of $20B, with China as leader of the pack. While the US will remain the largest market, China’s growth of 14 – 18% over the next 2 years will give it a firm place at #2.
Market presence is expanding to developing countries due to the need for higher medical demand, use of generics, and growing populations. Latin America, North Africa, and other parts of Asia are seeing increased attention from the industry while Brazil, Russia, India and China (BRIC) continue to grow.
Biggest Marketing Settlements
2012 saw 4 of the largest marketing settlements ever handed out to pharma. Not only did it see 4, but they ranked among the first 7, including the #1 spot, totaling $6.462B. The winners, or losers in this case, are GlaxoSmithKline ($3B), Abbott ($1.5B), Johnson & Johnson ($1.2B), and Amgen ($762M). All of the settlements shared a common thread of unlawful promotion with a few having kickbacks as reasons for judgment. The increase in penalties can be related to the Justice Departments continued outrage for misbehaving, going so far as suggest future penalties be given to executives for irresponsible corporate behavior or taking away patent rights.
This year also saw the banning of pay-for-delay deals, where brand name drug companies made deals for their generic competitors to delay the release of the competition. The Federal Trade Commission led this mission as these delays cost taxpayers $3.5B a year in higher drug prices, as well as violating trade laws.
Medical Device Tax
Effective January 1st, 2013, the Medical Device Excise Tax went into effect, generating an expected $29B in revenue over the next 10 years. Part of a 2010 healthcare law, 2012 saw the attempt, and fail, for repeal and has since become finalized by the IRS. The tax, applied to most tools used by medical professionals (including those used for humanitarian efforts), has garnered much criticism with some reflecting that the tax will prevent researchers from innovation, ultimately costing lives for patients who need these tools. As the tax will apply to all companies, regardless of profit, companies have to navigate the new arena for funding, R&D, and expansion. Some companies who will be hit hardest, such as Stryker, have already started reducing costs by reducing work force.
How the medical device tax will affect the industry remains to be seen, as well as the patent cliff. 2013 is projected to be another year of growth with a strong pipeline of potential new blockbusters under development. Will big companies look to acquisition as a way of bolstering their offerings? Will the rising stars, such as Onyx, be purchased by the industry giants? Will Alzheimer’s treatments catch a break? Only time will tell what the future holds.
- “Life Science Trends 2012”, Alexander, D., McMerty, B., Frey, K., Waddell, A., Carlyle & Conlan, 2012
- “FY 2012 Innovative Drug Approvals”, U.S. Food and Drug Administration, December 2012
- “Pharma R&D Annual Review 2012”, Llyod, I., citeline, 2012
- “The 15 Most Generous Companies Of The Past Year”, Sprung, S., Business Insider, July 26, 2012
- “The Best Drug Companies Of The Past 15 Years”, Herper, M., Forbes, February 9, 2012
- “Startups struggle with impending device tax”, Garde, D., www.fiercemedicaldevices.com, November 2, 2012
- “These are the top 10 payers of the medical device tax, says Moody’s”, Parmar, A., Med City News, March 8, 2012
- “Pharma’s Top 11 Marketing Settlements”, Staton, T., Palmer, E., www.fiercemedicaldevices.com, June 26, 2012
- “Beyond the Patent Cliff”, By Rick Mullin, December 10, 2012. http://cen.acs.org/articles/90/i50/Beyond-Patent-Cliff.html
- “The Sequester, explained”, By Suzy Khimm, September 14, 2012. http://www.washingtonpost.com/blogs/wonkblog/wp/2012/09/14/the-sequester-explained/
- “California Biomedical Industry 2013 Report”, By BayBio, CHI and PWC, January 7, 2013. www.CaliforniaBiomedReport.com
- “A Banner Year for New Drugs”, The 2012 Burill Report, http://www.burrillreport.com/article-a_banner_year_for_new_drugs.html
- “Big pharmas eyeing innovative targets”, October 25, 2012. http://www.acquisitionsdaily.com/2012/10/25/big-pharmas-eyeing-innovative-targets/
Marketing Manager at Clinovo
Associate, Business Development
Extract from the 2013 California Biomedical Industry Report
A BayBioreport, in collaboration with CHI and PwC: http://www.californiabiomedreport.com/
The 2013 report found California to be:
As the center of biomedical innovation in the U.S, California’s biomedical industry is a national treasure. But the pace of R&D productivity and its global leadership position hang on the availability of capital to fund future innovation and a regulatory framework that is based on consistency and innovative technologies.
The 2013 Biomedical Industry Report found:
The FDA’s mission is to protect public health by assuring “the safety, efficacy and security” of drugs. Indeed, the FDA has the tremendous challenge of ensuring sponsor companies deliver efficient treatments to patients while meeting the highest possible safety requirements. FDA reviewers always need to thoroughly weight risks versus benefits. This sometimes has dramatic consequences: The FDA admits that in the United States 100,000 people die every single year taking FDA-approved pharmaceutical drugs. In addition to that, two million people a year suffer from serious adverse events, which include stroke, heart attack, and permanent neurological damage.
The FDA is increasing efforts to improve patient safety and identify potential side effects. It is more and more demanding with healthcare companies, trying to ensure qualified processes are in place . Indeed, while the number of FDA approvals per 1,000 US-based clinical trials has declined from 7.5 in 2004 to 3.1 in 2010, industry experts are facing increased complexity and cost when managing clinical trials. A 2010 PhArma report argues that between 2000 and 2007, the median number of procedures per clinical trial increased by 49%.
For a drug or a medical device, everything starts or ends one day in the walls of the Food and Drug Administration. What make the difference between failure and success are your clinical data and the way you deal with the FDA. This article intends to help you understand the secrets behind a successful FDA submission.
Provide regulatory compliant data
One of the new FDA expectations includes using standard format for clinical data for their submission. The FDA is thus gradually implementing CDISC standards. CDISC® (Clinical Data Interchange Standards Consortium) established these data standards to speed up data-review and improve clinical data exchange, storage and archival. CDISC standards have been acknowledged as recognized standards by the FDA for years now. They are gaining momentum and undoubtedly are an asset to accelerate the FDA review process. By 2016, CDISC standards are expected to be mandatory for any drug submission.
Understand and follow the FDA’s transformation
Staying up-to-date on any new initiatives is fundamental in order to always anticipate FDA expectations. The FDA is adapting to therapeutic-based clinical trials or personalized medicine. On the same note, the rise of orphan drugs forces the FDA to develop specific and shortened review processes. The FDA is continuously adapting to these life sciences developments. Christine Conroy, Vice President of Regulatory Affairs and GCP compliance at Affymax, points out that with the growing number of very particular compounds and patient-specific therapies, it has become sometimes difficult to provide data the FDA asks. The FDA sometimes lack experience in new fields such as biomarkers. Indeed, there is no reference point as everything is new, so it happens that the FDA brings up issues not always relevant in that field.
Find creative study design strategies to meet your endpoint
Small companies often lack financial or human resources to oversee CRO activities and analyze the quality of the data. This can be overcome by being more creative in the sponsors’ design strategy to reach the endpoints. Sponsors can for instance rely on open source based eClinical Systems (such as CDISC Express or ClinCapture marketed by Clinovo) in order to avoid expensive licensing fees and meet tight budget requirements.
Partner with the FDA
Transparency is an absolute pre-requisite in partnering efficiently with the FDA. It is critical to be detail-oriented and to think upfront to provide and anticipate the information the FDA will need and require. Having internal checking prior to the actual FDA submission is critical because “if you have questions or doubt about your data, the FDA will too”, explains Sandra Nino-Siddens, Executive Director of Regulatory Affairs at Geron Corporation.
Sandra Nino-Siddens claims that “sponsors should be straightforward in presenting rationale and steps followed to develop a safe product.” She outlines the importance of building good relationships with the FDA from the beginning and to keep forth-coming interactions with them. Sandra Nino-Siddens states that “the FDA should be seen as a as a long-term partner, and not be seen as the police, nor as a consulting company.”
Olivier Roth, Marketing & Communication Coordinator at Clinovo
If you liked this article, we recommend you to read: What is the prescription drug user fee act in a nutshell
CDISC® (Clinical Data Interchange Standards Consortium) is establishing data standards to speed up data-review and improve clinical data exchange, storage and archival. Today, 60% of FDA submissions are already done in CDISC standards. The FDA is getting more and more involved into CDISC standards, a meaningful signal for the industry. Theresa Mullin, Director of Office of Planning and Informatics within CDER, claimed that “the FDA is committed to using CDISC standards for the foreseeable future”. These data standards are expected to be mandatory by 2016 for every drug submission.
CDISC standards hold the clinical data to a greater level of readability and compliancy in regards to FDA requirements. Carey Smoak, Senior Manager of SAS Programming at Roche Molecular and CDISC Device Team Leader, points out that “a submission without CDISC standards can have a review period twice as long as one under standards”. Indeed, they facilitate the FDA review process since they are known and understood by reviewers.
A 2009 study conducted by Gartner in collaboration with the CDISC organization shows that the overall clinical trial duration is divided by two when using CDISC standards. Thus CDISC standards ultimately speed up time to market.
So if the benefits of using CDISC standards are so obvious, how can we explain that so many sponsor companies are still not adopting them?
Converting legacy data to CDISC standards is expensive
Clinical data standardization is no simple process: It is time consuming and proves to be tedious. However, a few open source CDISC conversion tools have been launched to address this problem. One successful example is the OpenCDSIC validator software, recognized by the FDA and freely available. CDISC Express, Clinovo’s free SAS-based SDTM mapping tool, has been downloaded 600 times.
In the future, standards can be adopted smoothly if the industry works harder at incorporating them earlier in the process. Indeed, the next challenge is to push CDISC standards upfront in the clinical trial process. CDISC experts agree the best timing to implement CDISC standards is the database built.
CDISC standards are still evolving
Standards are still being built and are in constant evolution. The CDISC organization is still releasing new versions of its clinical standards. Sponsors companies are often scared that if they convert their clinical data to a format, it will be obsolete a year later. Clinical trial experts still state however that sponsor companies should shift to CDISC standards as soon as possible.
Companies often lack the internal expertise
In order to be efficiently used and maintained, Carey Smoak points out that “the wiser choice is to hire people with expertise on CDISC standards”. Companies should educate themselves on this topic and exclusively hire experts from CDISC Registered Solutions Providers organizations.
Ale Gicqueau, CEO at Clinovo
The Prescription Drug User Fee Act (PDUFA) was originally enacted in 1992, and authorizes the federal Food and Drug Administration (FDA) to collect fees from pharmaceutical companies seeking FDA approval of new drugs and biologics. In return for collecting fees, the PDUFA requires the FDA to meet performance benchmarks with the overall intention of making the New Drug Application review process more efficient. Since 1992, user fees collected by the FDA under the PDUFA have permitted the agency to expand its NDA review staff and considerably reduce a product’s review time while still assuring the safety of human drugs. Congress is required to reauthorize the PDUFA every 5 years by September 30th in order to ensure the continuity of the user fee programs.
Austin, TX – 18 April 2012 – The Clinical Data Interchange Standards Consortium (CDISC) is pleased to announce today at the CDISC European Interchange Conference in Stockholm, Sweden, the release of the first iteration of a Protocol Representation “Toolkit” for clinical research. The purpose is to make it easy for authors of the research plan or protocol to reap the benefits of the Protocol Representation Model (PRM), which has been developed over the past decade by global clinical research experts from academia, industry and government. Using such a model can save time and resources for research studies by enabling electronic re-use of protocol information for other purposes such as clinical trial registration, study tracking, regulatory information and study reports. The current release of the “Toolkit” includes a standard Study Outline Template in MS Word format, a standard list of Study Outline Concepts, and a complete mapping of the Study Outline Concepts to both the Biomedical Research Integrated Domain Group (BRIDG) model and the CDISC Study Data Tabulation Model (SDTM) Trial Summary (TS) Domain. Read more »
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