Friday, January 30, 2015

Planning Ahead to Stay Ahead: Integrating PROs and Economic Data in Oncology Trials

The era of moving from regulatory approval to automatic worldwide adoption of a new treatment is long gone. This can be particularly true in oncology, where treatments are often expensive and can potentially be just as harmful as the disease. In the crowded oncology marketplace, collecting patient-reported outcomes (PRO) and economic data during or alongside clinical trials establishes an oncology treatment’s value to regulatory agencies, as well as to governments and private payers, maximizing your return on investment. Moreover, the rise and refinement of ePRO technologies and processes (e.g., smartphone apps, wearable devices and automated data collection/analysis) are making patient-reported data more accessible and cost-effective to collect.

Importance of PROs and Economic Endpoints in Oncology Clinical Trials
The inclusion of PROs in oncology clinical trials enables a better understanding of overall treatment effectiveness, including a more complete risk-benefit profile. In fact, numerous organizations, including the Food and Drug Administration and the American Society for Clinical Oncology, believe that PROs are essential components of clinical research in oncology.

To enable comparison of findings and ensure consistency across trials, the National Cancer Institute’s Symptom Management and Health-Related Quality of Life Steering Committee has recommended that all oncology trials include PRO measures of a core set of 12 symptoms. Using this core set simplifies the comparison of PRO findings across treatments and can facilitate the process of determining treatment value based on PRO findings. Use of additional PRO measures should also be considered to further demonstrate product value. But how do you determine the best measures? Should they be cancer-specific or based on country-specific guidance? Other factors to consider include: the goals of the clinical trial, the type/stage of cancer, existing treatment alternatives and commercial (reimbursement) targets for the product.

As the development of innovative, yet more expensive treatments in oncology continues, reimbursement agencies and other payer groups are increasingly challenged to manage budgets and provide the best possible treatment to patients. In Canada, Australia and many countries in Europe, economic evidence is required for market access and reimbursement of a new product. While such evidence is not explicitly required in the United States, health care decision makers examine treatment-associated medical resource utilization (MRU) in budgetary impact modeling and other reimbursement-related activities. Thus, as with PROs, it’s important to collect these data up front to ensure they are available for health economics analyses before market launch and for discussions with the appropriate market access agencies.

Ensure Success: Work With Health Economics and Outcomes Research Experts
Health Economics and Outcomes Research (HEOR) experts are intimately familiar with PRO-specific regulatory guidelines, as well as specific health economic, costing, modeling and other methodologies needed to satisfy diverse payer and reimbursement agency demands.

Whether HEOR expertise is available in-house or through outside consultants, it’s important to leverage proven intelligence in clinical, regulatory and marketing discussions related to oncology products, no matter the phase of development. This will ensure all PRO-related activities, including PRO selection, training and analyses, are conducted in compliance with the appropriate regulatory guidelines and follow the appropriate methodologies specific to where trials are being conducted globally. Similarly, to maximize reimbursement and product pricing success, clinical development teams should work with HEOR specialists to make sure all health economic activities are initiated early so that all necessary MRU endpoints are included in the clinical trial and the appropriate methodologies and costing techniques have been selected in advance based on the requirements for submission to appropriate agencies.

Including PROs and MRUs in oncology clinical trials should not be an afterthought. In today’s competitive health care marketplace, thoughtful planning of a clinical trial in consultation with HEOR experts can facilitate regulatory approval, lead to a PRO label claim and provide the data needed for market access and reimbursement. Incorporating PRO and MRU endpoints into a clinical trial can save you considerable time and maximize return on investment. Early preparation in consultation with experienced HEOR experts is the best plan for long-term product success.

About Decision Driver Analytics
Founded in 2006, Decision Driver Analytics provides a suite of services that covers the full range of health economics and outcomes research (HEOR) as well as the outreach materials needed to reach investors, health care providers, payers and the public. Utilizing veteran health economists, epidemiologists, biostatisticians, medical writers and reimbursement experts, DDA’s services include complete product life cycle value analysis, strategy and planning, clinical economic study services, predictive modeling analytics, definitive analysis studies, communication and sales training.

Decision Driver Analytics is an Associate Sponsor at this year's Partnerships in Clinical Trials.  Would you like to join them in Boston April 22-25, 2015?  As a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off current rates!

Wednesday, January 28, 2015

Five Steps for Improving Processes in Clinical Trials

Author: John R. Wilson, Jr., PhD, MPH, is Senior Vice President at Beaufort, LLC.

Nobody would accuse the clinical research enterprise of being a paragon of efficient processes. It’s not that we don’t want our processes to be efficient; it’s just that the work needs to get done, no matter how inefficient the current processes are. After an initial burst of enthusiasm, process improvement projects seem to lose steam and then are quietly set aside for more pressing priorities.

The good news is that there is a proven, five-step process for improving business processes. This article will provide an introduction to the five steps, using risk-based monitoring (RBM) as an illustrative example.

But first, let’s define what we mean by “process.” In essence, a process is a standardized series of steps taken to achieve a goal. Processes simplify, streamline and regulate our activities. Formal processes are known as procedures (“standard operating procedures (SOPs)”), with well-documented steps. In our business, procedures are frequently designed to comply with governmental regulations and guidelines. Informal processes, on the other hand, just seem to exist as customary practices that are passed along by word of mouth, along with the forms and other artifacts of the process.

The Japanese method of kaizen (continuous improvement) is not limited to incremental changes. Sometimes, major changes are required to fix an obsolete or broken process. RBM, for example, requires major changes that cannot be implemented in tiny steps. Successful organizations must therefore be able to make major process improvements.

The five basic steps to significantly improve a process are: (1) map, (2) analyze, (3) redesign, (4) assign resources, and (5) implement improvements. Only after completing the first four steps can we productively move on to the fifth step, in which a process is actually improved.

For details, see John Wilson's article, “Five Steps for Improving Processes in Clinical Trials,” in the Journal of Clinical Research Best Practices.

This article is presented by Partnerships in Clinical Trials partner The Journal of Clinical Research Best Practices.  PCT will take place April 22-24, 2015 in Boston,  As a reader of this blog, when you register to join us and mention code XP2000LI, you can save $100 off current rates.

Friday, January 23, 2015

Calling Industry Influencers! PCT Award Nominations Due 2/9

The 24th Annual Partnerships in Clinical Trials Conference, April 22-24th, is looking to honor individual(s) working to advancing clinical research. In appreciation of your contribution, award winners will receive a Gold Pass* to attend the conference on April 23-24 valued at $3,495!

2015 Award Categories:
  • • Woman of the Year: A leader in the clinical trials industry, a mentor and an advocate for other women in the organization.
  • • Clinical Innovator of the Year: This award honors an individual or a company with a disruptive technology platform or new way to optimize clinical development that has increased the efficiency of clinical trials.
  • • Partnership Hall of Famer: This award honors two individuals and/or companies engaged in a partnership that moves beyond the traditional transactional model to further drug development. This award can be applied to non-traditional partnerships including academia, patient organizations, etc.
Nominations are due February 9th. Don't wait, submit your entry now!
    All award recipients will be notified four weeks before the event and honored in front of their peers during a ceremony on April 23rd.

    As a reader of this blog, when you register for PCT 2015, you can save $100 off the current rate when you mention code XP2000BL.

    Thursday, January 22, 2015

    Web Seminar: Change Investigative Site Relationships For The Better

    Live Webinar: Thursday, February 19, 2015
    Time: 11:00 am - 12:00 pm EST
    • - David Green, Executive Director, United Therapeutics
    • - Hassan Movahhed, Senior Vice President, Global Development Operations, United Therapeutics
    • - Lisa Levin, Product Education, IMS Health Moderator:
    • - Lisa Henderson, Editorial Director, Applied Clinical Trials (Moderator)
    Register Free at

    Un-enrolling sites. Slow study start-up. Patient drop out or non-compliance. All of these issues--and more--have been targeted as contributors to high clinical trials costs. But what else do they have in common? They involve principal investigators and site staff. As concern grows, solutions have emerged to help physician researchers better conduct research. Other solutions are aimed to help sponsors identify issues early at the investigative site, to intervene and influence positively when necessary.

    Applied Clinical Trials and Partnerships in Clinical Trials join together to bring a group of experts to discuss the importance of the investigator in clinical trials, and how to select and engage them in a true partnership.

    Key Learning Objectives
    • • Understand the emotional or “soft” issues behind investigator engagement and loyalty
    • • Understand how data underpins feasibility and investigator support
    • • Learn how investigators perceive both qualitative and quantitative approaches from sponsors
    Who Should Attend
    • • Clinical operations, project managers, directors
    • • Investigators, CRAs, site staff
    • • Patient recruitment specialists

    Presented by: 

    In partnership with:

    Tuesday, January 20, 2015

    Getting Started With Social Media Listening in Clinical Research

    Today's guest post comes from Rahlyn Gossen. She is the founder of Rebar Interactive, a clinical trial patient recruitment and digital marketing company. Rahlyn also maintains a blog, newsletter, and Twitter profile focusing on digital strategy for clinical trials.

    Social media is changing the world. According to 2014 Pew Research data, about three out of four online U.S. adults use social media. Popularity varies in other countries, but overall social media adoption rates are rising worldwide. And with rising popularity comes dramatic change in consumer behaviors and expectations. No longer are people satisfied with merely being consumers of information. They also expect to share and create it. This democratization of information distribution and creation is reshaping entire industries.

    Healthcare is no exception. Social media has been a key enabler in what many describe as the epatient movement. Healthcare providers are no longer the only source of healthcare information. Increasingly, patients are using social media to consume, share, and create healthcare information. And this democratization is shifting power from traditional healthcare authorities to patients. Now peer-to-peer healthcare conversations are influencing patient opinion about everything, including clinical trials.

    Are you listening?

    These conversations can provide key operational insights for your clinical trial. Many sponsors have shown an interest in using social media to communicate their message, particularly around patient recruitment. But listening, in this case, is at least as powerful as talking. Furthermore, listening generally carries less complexity than a social presence, particularly when it comes to regulatory issues. Sponsors who put down the bullhorn and pick up the earphones can be rewarded with valuable information, including insights into:
    • • Patient characteristics, attitudes, and behaviors
    • • Patient healthcare journey
    • • Patient engagement and recruitment opportunities
    Listening can take many forms and vary in sophistication. For example, a simple Twitter search can be considered social media listening. Click here and scroll through some tweets. Congratulations, you have just listened to Twitter conversations about Alzheimer’s. On the more sophisticated spectrum, a variety of technology tools are available to help you collect, filter, and organize these conversations. However, even the most sophisticated technological tools require significant human analysis by someone knowledgeable about social media.

    Social media listening is of course not without difficulties. For example, it can take some time to learn social media listening tools and become proficient at separating signal from noise. In addition, available listening tools are not perfect, so it’s good to have an understanding of where technology weaknesses are so you don’t inappropriately place trust in the technology. And lastly, for the data to be useful, you need someone that understands the digital and social landscape enough to contextualize it. In short, social media listening is not “plug and play.”

    To learn more about the practicalities of social media listening, check out “Social Listening Intensive – Why Now, Lessons Learned, and Connections You Need” at Partnerships in Clinical Trials April 22-24 in Boston. Representatives from Shire, Novartis, and Eli Lilly will help you:
    • • Learn how social listening can help you understand community attitudes and behavior
    • • Explore the benefits and drawbacks of using social listening as a tool for patient engagement
    • • Gather input that may shape future research
    Our ability to attract patients to clinical trials will increasingly depend on our commitment to listening to them. Social media is a valuable and accessible means to do that.

    Thursday, January 15, 2015

    Social Media "Compassionate Use" Crusade Sets Unsettling Precedent

    Former Chimerix CEO Ken Moch Warns Drug Makers to Prep for “Compassionate Use” Campaigns

    By Marc Dresner, Senior Editor, IIR

    Back in March the drug industry got a jarring demonstration of social media’s firepower when a grassroots campaign to get a seven-year-old cancer patient pre-approval access to an experimental anti-viral medicine captured national attention and made headlines worldwide.

    The #SaveJosh campaign—named for patient Josh Hardy, now eight—generated sufficient pressure to force Durham, NC-based Chimerix, a small biotech, to create a 20-patient open-label study for brincidofovir (CMX001)—in Phase 3 trials—in order to accommodate this very sick little boy.
    Josh Hardy

    Without question, the drug saved Josh Hardy’s life and the good news is that his condition has dramatically improved.

    But the case raised some very troubling ethical questions around “compassionate use” in an age when “who shall live” decisions can in effect be crowdsourced via social media.

    It also introduced a new dilemma for drug manufacturers in that the advent of compassionate use crusades almost overnight and on the massive scale facilitated by social media can potentially impede development efforts.

    Kenneth Moch
    According to former Chimerix CEO Kenneth Moch—who received death threats and had to be placed under security detail during the emotionally-charged ordeal—had the outcome been different and Josh Hardy’s response less favorable, development of brincidofovir could’ve been delayed or even derailed, consequently preventing the larger prospective patient population from accessing a potentially life-saving drug.

    “This was not just about Josh Hardy; it was about the many ‘Joshes.’” - Ken Moch

    “This was not just about Josh Hardy; it was about the many ‘Joshes,’” said Moch in an interview with Inside Clinical.

    “The issue is how to balance providing immediate access to a patient in dire need against the goal of making a drug available to as many patients as possible by getting it approved,” Moch emphasized.

    For smaller biotechs like Chimerix—with just 50 employees and no other product in market—the financial strain and demands placed on personnel by compassionate use interventions can compound the problem by siphoning scarce resources that would’ve otherwise been dedicated to moving a drug through the development and approval process.

    “This was the first time that social media had been used so intensely around a child who looked and was so needy.”

    “This was the first time that social media had been used so intensely around a child who looked and who was so needy,” Moch noted.

    “The decision for making a drug available pre-approval currently rests with the company developing the drug,” he added. “And smaller companies—particularly smaller biotech companies—may not have the ability to respond to these significant social media pressures.”

    It’s worth pointing out that the mainstream media—Fox News, in particular—depicted Chimerix as a Big Pharma Goliath with deep pockets, an image that served to incite public outrage and ratchet up the pressure.

    Helping a gravely ill child get access to a life-saving drug that had been denied him by a heartless drug giant is a compelling and dangerously irresponsible narrative to spin, and the emotions it elicited at the time appear to have made a reasoned dialogue about the complexities involved in making compassionate use decisions impossible.

    According to Moch, Josh Hardy was one of hundreds of patients who had not been eligible to participate in the brincidofovir SUPPRESS trial and who had petitioned Chimerix for pre-approval access but had been denied the medication for reasons related to the company’s Phase 3 development efforts.

    Social and mainstream media pressure enabled Josh Hardy to get the drug and to survive, but the event set a troubling precedent by relegating the decision to the whims of public opinion and inequitable influence.

    How do you decide who can and cannot have access to an experimental drug? Contacts? Money? Social media?

    “How do you decide who can and cannot have access to an experimental drug? Is it through social media? Is it through contacts? Is it through money? Is that fair and equitable? I think not,” said Moch.

    The Josh Hardy case has forced the industry and regulators to scrutinize expanded access to experimental drugs. BIO and PhRMA have since responded and the FDA has formed a task force.
    But Moch emphasized that companies must be prepared to address social media campaigns for pre-approval access to drugs they’re developing head on or face potentially disastrous consequences to their development efforts.
    In this podcast for Inside Clinical—the official interview series of the 24th Annual Partnerships in Clinical Trials conference—Ken Moch discusses the issues around “compassionate use” and what manufacturers can learn from his experience.
    Listen to the podcast and download a transcript here!

    Author's note: Ken Moch co-authored a great post on this subject with NYU's Art Caplan on the Health Affairs blog. Check it out here.

    Editor’s note: Ken Moch will present “Fast Tracking Clinical Development in a Moment’s Notice: Patient Advocacy, Social Media and the Implications of Compassionate Use” at the 24th Annual Partnerships in Clinical Trials Conference running April 22-24 in Boston.

    For more information or to register, please visit  

    Marc Dresner is IIR USA's senior editor and special communication projects lead. He is the former executive editor of Pharma Market Research Report, a confidential newsletter for marketing researchers in the pharmaceutical industry. He may be reached at Follow him @mdrezz.

    Wednesday, January 14, 2015

    See who is attending Partnerships 2015 | Last chance to save up to $800 on PCT

    Partnerships in Clinical Trials 2015 is the world’s foremost event series for clinical development leaders and practitioners. This event draws 1100+ leaders from pharma, biotech & medical device companies, CROs, and third-party services providers in to network, learn, strategize and advance next practices in clinical trials across the globe.

    Here’s a sneak peek of the who’s attending PCT 2015 so far:
    23andMe * Abbvie * Actavis Plc * Aesclup * Alexion Pharmaceuticals * Alliance for Lupus Research * Amgen * Amicus Therapeutics * Astellas * AstraZeneca * Baxter Bioscience * Bayer Vital GmbH * Berg Health * Biogen Idec * BioMarin Pharmaceutical Inc. * BioStorage * BlueBird Bio * Boehringer Ingelheim * Boston Scientific * Bristol-Myers Squibb * Cadila Pharmaceuticals * Celerion * Cubist Pharmaceuticals * DePuy Synthes * Dimension Therapeutics * Eisai * Eli Lilly & Co. * EMD Serono * Forum Pharmaceuticals * Genentech * Genzyme * GlaxoSmithKline * Grunenthal GmbH * ICON plc * Inovio Pharmaceuticals * InVivo Therapeutics * Johnson & Johnson * Janssen Biotech, Inc. * Juventas Therapeutics * Lexicon Pharmaceuticals * Mallinckrodt Pharmaceuticals * MEI Pharma, Inc. * Merck * Mirati Therapeutics * Momenta Pharmaceuticals * National Center for Advancing Translational Sciences (NCATS) * Nektar Therapeutics * Novartis * Novartis Institute for Biomedical Research * Novartis Pharma AG * NPS Pharmaceuticals * Otsuka * Pfizer * Pfizer China * Purdue Pharma L.P. * Quintiles * Regeneron * Roche * Roche Romania SRL * Sagient Research * Sanofi * Shire Pharmaceuticals * Stanford University * Sunovion Pharmaceuticals Inc. * Takeda Development Center, Asia * Takeda Oncology * TESARO Inc. * Tokai Pharmaceuticals * Transparency Life Sciences * Trevena * Tufts University School of Medicine * UCB Pharma * United Therapeutics * Valeritas * Vistakon, J&J Vision Care * Voyager Therapeutics
    Partial Attendee List (as of 1/13/15)

    Partnerships in Clinical Trials will take place April 22-24, 2015 in Boston, MA. This is the last week to save up to $800 when you register and mention code XP2000BL to save your $100 off the current rate. Register here.

    Tuesday, January 13, 2015

    Is the FDA driving the development of drugs instead of science?

    Yes according to Joseph V. Gulfo, MD, MBA, in a recent post at The Hill.  According to the article, 41 new drugs were approved in 2014 and 40% of them were orphan drugs.  The Orphan Drug act is a big part of this - pushing exclusivity and driving profits. Gulfo sees this as a problem - because they see drug approvals but the public continues to fewer drug approvals that drive health.  Instead those being approved and their high costs are seeing profits that lean on blockbuster drug levels.

    So to conclude his article, Gulfo points out what the Orphan Drug Act has taken away from - science and medicine are no longer driving development for health.  Instead it's the policies of that the FDA set forward for drug approvals.  Do you agree?

    Dr. Gulfo will be joining us at Partnerships in Clinical Trials on Thursday, April 23 for the presentation Picking up the Pieces of Clinical Trial Failure: How I Battled the FDA and Won.  For more information on this session and the rest of the program, download the agenda.  If you'd like to join us, as a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off current rates - lowest rates of the year expire Friday, January 16.

    Monday, January 12, 2015

    Patient Organizations Needed! Complimentary Conference Attendance

    Calling all non-profit patient representatives and organizations! Leading with the patient voice a top priority for the 24th Annual Partnerships in Clinical Trials Conference (April 22-24th in Boston, MA) which is why we're looking for representatives to be a part of our event community. New for 2015, patient organizations and representatives will have a home base in our "Patient Pavilion". Qualifying organizations will receive 2 conference exhibit hall passes and a table top stand.

    About the Conference: Partnerships in Clinical Trials is the world’s foremost event series for clinical development leaders and practitioners. This event draws 1100+ leaders from pharma, biotech & medical device companies, CROs, and third-party services providers in to network, learn, strategize and advance next practices in clinical trials across the globe.

    Interested in participating or know a Patient Organization who would be? Please email Program Director, Marina Adamsky. Hurry, spots are limited and filling up fast!

    Friday, January 9, 2015

    Reminder: Looking For Thought Leaders In The Central Labs/CDX Space!

    The Institute for International Research is looking for speakers from sponsor companies for our upcoming Partnering with Central Labs Conference to be held in conjunction with the 24th Annual Partnerships in Clinical Trials Conference -- April 21-22, 2015 in Boston, MA.

    DEADLINE EXTENDED! We are currently recruiting pharmaceutical/biotech/medical device professionals in the procurement, outsourcing, relationship management with Central/ECG/Imaging relationship management, quality and category leads. Plus, Companion Diagnostics Program Directors and Heads of Translational Medicine.

    Topics include:
    • • Innovation in Microsampling
    • • Strategies to Get to First Patient In and Site Initiation Quicker
    • • Interpret Testing for Drug Induced Liver Injury
    • • Managing Ancillary Vendors at a Small to Midsized Pharma Company
    • • Delve into the Shipping Costs Between Labs + Shipping Companies
    • • Examining the Regulatory Requirements for a Successful Submission for CDx
    • • The Development of Regulated Next-Generation Sequencing-Based Clinical Trial Assays and CDx
    • • Lab Selection and Qualification Criteria
    • • Use of a Local vs. Global Lab in Complex Clinical Trials
    • • New Developments in Biomarker Testing
    • • Sponsor/CRO/Lab Relationships – Using Metrics to Benchmark Success
    • • Best Practices in Lab Data Integration
    • • Contracting with Central Labs - What to Put in Your Contract
    We invite you to submit a proposal* for a speaking opportunity directly to Marina Adamsky.

    Thank you and we look forward to welcoming you to Boston in April!

    *Note: Proposals are subject to review IIR to ensure the overall quality of the conference program. Please note that due to limited speaking slots, preference is given to abstracts from those within pharmaceutical and biotech companies, regulators, and those from academic centers. A select number of suppliers providing services to the industry are offered presentation opportunities in conjunction with an event sponsorship. Please email Patricia Rose at

    Thursday, January 8, 2015

    Clinical Development Innovation- Participate in 2015 Avoca Industry Survey – Results to be Unveiled at PCT

    We invite clinical trial industry professionals with valuable knowledge and insights to participate in Avoca's 2015 Annual Industry Survey. The topic of this year's survey is Innovative Approaches to Clinical Development.

    For a complimentary copy of the 2015 Avoca Report on Clinical Innovation, please complete the survey today.

    For more than 10 years, The Avoca Group has surveyed industry executives and managers to gain a better understanding of key trends affecting the conduct of outsourced clinical development. The insights gained from this research have proven valuable to industry leaders who are working to strengthen relationships and enhance R&D productivity. Your participation is instrumental in facilitating these improvements.

    In addition to receiving an Executive Summary of key findings from our research, upon completion of the survey you will be entered into a drawing for one of two Amazon gift cards valued at US $500. As always, your personal information will remain confidential and will not be associated with your survey responses in any manner.

    The results of this research will be unveiled at the 2015 Partnerships in Clinical Trials Conference. We greatly value your feedback and thank you in advance for your participation.

    Click the the URL to access the survey:

    If you have trouble accessing or completing the survey, please email us at

    Partnerships in Clinical Trials 2015 will take place April 22-24, 2015 in Boston, MA. As a reader of this blog, when you register to join Avoca at the event with priority code XP2000BL, you’ll save $100 off the current rate. The lowest rates of the year expire next Friday, January 16. Find out more and register here.

    Have any questions about the Partnerships in Clinical Trials event or want to get involved? Feel free to email me at

    Tuesday, December 23, 2014

    It doesn’t show signs of stopping… Let is sale! Let it sale! Let it sale!

    It doesn’t show signs of stopping…
    Let is sale! Let it sale! Let it sale!

    ‘Tis the season! As our holiday gift to you, here’s 30% off the standard rates when you register for Pharma and Healthcare events from now through Wednesday, December 31! Use the code “Holiday30” at checkout.

    This event was specially selected for your interests:

    Partnerships in Clinical Trials
    April 22-24, 2015, 2015 in Boston, MA
    >Accelerate your speed to market by leveraging new partners, new technologies, and new business models at the must-attend clinical event of the year!
    Find out more about the event
    Register to join us.

    For a full list of all events you and your colleagues can save on this holiday season, visit the Future of Biopharma blog.

    Have any questions? Email Jennifer Pereira.

    Monday, December 22, 2014

    Considerations for conducting trials in low-cost countries

    Rick Morrison, Founder and CEO of Comprehend Systems recently took and in-depth look at some of the major considerations an company should look at before they venture into low-cost countries for clinical trials.  With the steep cost of clinical trials - Astra Zeneca faced a cost of $11.7 billion for each of the five drugs it brought to market from 2007-2011 - it's important that companies find efficient, low cost and ethical ways to bring drugs to market faster and safer.

    Morrison suggests looking into expanding clinical trials beyond domestic trials to reap the benefits of diverse patient populations, the shrinking of cost and time to market for successful drugs and the ability to test the products in the countries and patient populations to which they'll be sold.    Not only are these benefits clear within themselves but the number of patients presented in a global clinical trial allows to companies to bring drugs to market an average of 6-7 months faster.

    What are some of the benefits you see from expanding clinical trials beyond the traditional borders?

    This April at Partnership in Clinical Trials, we will host Effects of Globalization on Clinical Development a full day workshop to kick off the 2015 event.  Presentations will look at global data, streamlining outsourcing, patient recruitment and more.  For more information on this session and the rest of the program, download the agenda.  As a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off the current rate.  We're also giving away a few free passes on Twitter.  Find out how you can win.

    Friday, December 19, 2014

    Biotechs provide critical link to innovations for Pharma companies

    Innovation in the life science industry is evolving.  With over 90% of innovations occuring outside of big Pharma's research and development labs, it's important that big pharma be in tuned with what's going on in and leverage the expertise across the value chain.  In a recent interview at partnering 360®, Jochen Maas, the General Manager of Research and Development at Sanofi Germany sat down and shared his thoughts on capitalizing on external innovation with partners and identifying the real experts in the Pharma and Biotech industry to get products to the patients as fast as possible.

    Watch the interview here:

    Partnering with experts in the field a company trying to excel in - especially Pharma and Biotech - is critical. What do you see are the benefits of working with the innovative biotech industry for clinical trials?

    This April at Partnerships in Clinical Trials, Annalisa Jenkins, Dimension Therapeutics will join us at a session to look further into opportunities for innovation within the biotech sector. Would you like to join her April 22-24 in Boston? As a reader of this blog, you can register to join us with code XP2000BL and save $100 off current rates. Or you can enter for a chance to win a free pass when you retweet this tweet!

    Wednesday, December 17, 2014

    America's Highest Paid Female Executive and Her Unusual Path to Pharma

    Today's guest post comes from Rahlyn Gossen. She is the founder of Rebar Interactive, a clinical trial patient recruitment and digital marketing company. Rahlyn also maintains a blog, newsletter, and Twitter profile focusing on digital strategy for clinical trials.

    “Futurist, pharma tycoon, satellite entrepreneur, philosopher. Martine Rothblatt, the highest-paid female executive in America, was born male. But that is far from the thing that defines her. Just ask her wife. Then ask the robot version of her wife.”

    While reading the introduction of an online New York magazine profile of Martine Rothblatt, I wondered how accurately it reflected the content of the full article. As someone who reads a lot on the Internet, I’ve come into contact with my share of sensationalistic and hyperbolic hooks. But by that point my curiosity was piqued, so I continued reading. And the article did not disappoint. Martine Rothblatt is every bit as fascinating a person as the snippet above would lead you to believe.

    In the 1990s Martine’s youngest child was diagnosed with primary pulmonary hypertension, a rare and fatal disease. Martine took action. She started the PPH Foundation, educated herself about pulmonary conditions, and founded a pharmaceutical company. Her company, United Therapeutics, went public in 1999 and received approval for a PPH drug last year. Martine’s daughter, whose condition inspired her foray into pharma, turns 30 this year and works for United Therapeutics.

    Prior to founding United Therapeutics, Martine was already quite accomplished and even considering retirement. She had founded Sirius Radio and took it public in 1994. Prior to Sirius, Martine founded GeoStar, a GPS-based navigation system. Martine began her career as a communication satellite lawyer after earning a combined law and MBA degree from UCLA.

    Currently, Martine is expanding United Therapeutics and promoting a new book titled Virtually Human: The Promise—And Peril—of Digital Immortality. Martine considers herself a transhumanist, which according to the article, is “…a particular kind of futurist who believes that technology can liberate humans from the limits of their biology—including infertility, disease, and decay, but also, incredibly, death.” According to Martine, artificial intelligence will be the primary vehicle to enable this new future, where even the dead can be reanimated as digital beings.

    The description I’ve provided is a tiny sample of the fascinating details contained within the New York magazine article. Give it a read, or better yet, see Martine Rothblatt speak in person at the 24th annual Partnerships in Clinical Trials conference taking place in Boston on April 22-24, 2015. Martine will keynote the Women’s Clinical Leadership Forum, and I have no doubt she will be a highly engaging and informative speaker.

    Thursday, December 11, 2014

    The state of clinical research on COPD

    COPD is a disease that is quickly growing to be one of the biggest expenditures in healthcare.  According to the NIH, it's predicted that by 2020, the disease will cost the United States $49 billion.  In 2010, it cost the state of Florida $2.5 billion.

    There is no cure, and the UK's NIHR points out that the only preventable cure for this disease is to quit smoking.  Over 35,000 patients have participated in research and between 2008 and 2009, there were 157 new studies that began. See World Startup Report's research here.

    What other measures are being taken in COPD research? Laura Runkel, Associate Director of CNS, Autoimmune/Inflammation & Ophthalmology, Citeline, will be joining us on December 17 to look at the trends developing in clinical research for this disease. Register to join us here and mention priority code XP2000W2BLOG.

    Wednesday, December 10, 2014

    Catch up: Partnerships in Clinical Trials Europe 2014

    Last month more than 950 of the industry’s senior professionals in clinical research and outsourcing gathered in Barcelona for the 13th Annual Partnerships in Clinical Trials Europe conference. With a packed agenda from plenary sessions to collaboration zones and a lively exhibit floor, there was something for everyone.

    This year we focused on many new topics such as:
    • - Transparency:  Greater visibility, data standards, identifying trends, data privacy and anti-corruption
    • - Disruptive technologies:  How they're being used and how it’s being taken forward
    • - Patient-perspectives
    • - Impact of social media on clinical trials

    Attendees heard exclusive industry updates:
    • - TransCelerate’s plans for 2014
    • - Is disruptive innovation working?
    • - Private equity in outsourcing: impact on CRO and clients
    • - Advances in patient recruitment
    • - Risk-based monitoring and compliance issues

    Spotlight: The Patient Perspective 

    “I like to see competitors  be in a forum that allows them to collaborate. They can share information in a very informal way. “  -Jack Whelan, Cancer Warrior & Survivor

    Jack Whelan, Author, Lecturer, Research Advocate, Cancer Warrior & Survivor, ePatient
    One of the most talked-about keynote sessions was Jack Whelan, Author, Lecturer, Research Advocate, Cancer Warrior & Survivor, ePatient. He gave attendees the perspective of the patient, which is invaluable. Whelan’s very personal talk examined the impact of a heavily regulated environment on sharing meaningful patient data and patient reported outcomes. He asked important questions such as ‘What steps can be taken to facilitate a follow up system that encourages continued monitoring of clinical trial participants?’ which fostered many discussions throughout the event.

    But, don’t take my word for it. Watch the video below to hear what attendees and exhibitors had to say about the event and get a glimpse of the even itself.

    Here is a sample of the hard work, inspiring speakers and partnership networking we saw last month in Barcelona. Check out the full album on flickr! Find yourself and your colleagues in the photos and share with your networks to keep the conversation going until next year in Hamburg! You can follow us on twitter @PartnershipsCT and use #PCTrials to make even more connections!

    flickr                  twitter          linked in
     Collaboartion Zone  Collaboartion Zone  Wine Casino
     Jack Whelan  PCT Bar  Networking

    Find out more about what’s coming in 2015! 
    Check out for the most up-to-date information.

    18-19 NOVEMBER 2015

    If you want to register today – contact Michael Dunnet,

    Tuesday, December 9, 2014

    Investigator data could be the key to unlocking clinical trials

    Clinical trial data varies between each company.  The companies also have a habit of always returning to the same clinical trial sites and investigators instead of branching out and finding the best site and investigators for their trials.  Because with out the investigators, there are no patients, recruitment costs will skyrocket, and cost and wasted time continue to grow.  But as European Pharmaceutical Contractor points out, a streamlined data system across all sites could easily open up more sites and promising investigators to conduct clinical trials faster.  By streamlining data, sponsors could see success rates of past trials, recruitment rates over periods of time and have a world of investigators open up.  This article dives into how the Investigator Databank took on the challenge and has seen success since it was implemented in April 2014.

    Four key areas are identified where the streamline of data and reports can help improve the reporting and collaboration of clinical trial sites:
    • -Access to more investigators
    • -Reduction of non-performing sites
    • -Decrease in time for feasibility and site identification
    • -Expand access for clinical researchers to research opportunities
    Have you participated in streamlining your clinical data?  What are some of the benefits you see in addition to the four identified by EPC?

    Data transparency is rising to the top of many conversations - streamlining investigator sites is just one.  This April at Partnerships in Clinical Trials, we'll bring together some of the key companies in Pharma including Boehringer Ingelheim, Pfizer, UCB Pharma, Center for Medicine in the Public Interest and GlaxoSmithKline during the panel Show Me Your Data: How Transparency is Driving Drug Development to look at how streamlining and sharing data can improve clinical trials throughout the drug industry.  For more information on this session, download the agenda.  If you'd like to join us April 22-24 in Boston, as a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off the current rate!

    Friday, December 5, 2014

    Is the answer to advancing cancer treatments giving more patients access to clinical trials?

    What is the key to accessible clinical
    trials for patients?
    Only 10% of all cancer patients in the United States participate in clinical trials.  In a recent article from Reuters, they point to accessibility to clinical trials as one of the major barriers to participating in the trials.  In the article, Dr. Matthew D. Galsky, Icahn School of Medicine at Mount Sinai in New York, states that clinical trials are the sources of many of the major advancements in treatment for cancer, so efforts should be made to increase this number.

    Not only do clinical trials for cancer treatments advance the science and lead towards cures but they also often provide better overall care for the patients.  Patients who participate in trials receive follow up, more treatment guidelines and are better able to manage treatment for their disease.  They also have frequent interaction with healthcare professionals who are closely  monitoring their disease as well as hope that traditional treatments might not provide.  By finding new and innovative ways to allow patients to participate in clinical trials, this allows patients who may not live near a trial site to access in a potentially life saving treatment.

    In your opinion, what is one of the major setbacks to creating a more accessible clinical trials for patients with difficult to treat cancers?

    Connecting the Innovation Dots: Solutions to the Disconnection Between the Pace of Technology and Traditional Approaches to Clinical Trials presented by by Donald Jones, Chief Digital Officer, Scripps Translational Science Institute will look at how trials can be rethought and give the opportunity to more cancer patients more access to participate in clinical trials.  For more information on this session and the rest of the Partnerships in Clinical Trials program, download the preliminary agenda.  If you'd like to join us April 22-24, 2015 in Boston, as a reader of this blog when you register to join us and mention code XP2000BL, you can save $100 off the current rate!

    Thursday, December 4, 2014

    Latest clinical developments showing hope in Alzheimer's research

    As the population of the United States continues to age, the search is on for a cure - or even a treatment to slow down this debilitating disease.  A few doctors are making headway in the search. What types of treatments are showing promise in clinical trials?

    • -Biogen Idec is working on a a treatment that works to reduce beta amyloid levels in the brain.  As announced yesterday, Their drug BIIB037 is designed to eliminate the plaque formed by the beta amyloids.  The levels were reduced in a dose- and time-dependent fashion.  Find out more about this treatment showing success in the early Phases at the Wall Street Journal.
    • - Dr. Eva Feldman, researcher at the University of Michigan, has been working with stem cell therapies.  Fetal stem cells were injected into the brains of mice that were engineered to develop Alzheimer's.  Early studies show that the mice that received the treatment did not show the cognitive degeneration that those who used the placebo did.  Find out more about this trail at The Detroit News.
    • -The National Institute on Aging has developed a new blood test that could prove to identify Alzheimer's Disease up to ten years before symptoms are exhibited. According to USA Today,  Tests are showing 100% accuracy with all those who have tested so far.  It has only been tested in 174 individuals so far, but early tests show promise.  By identifying the disease earlier, it would allow for more individuals to participate in clinical trials before their disease progresses beyond help.

    Later this month, we're pleased to team up with Citeline's Laura Runkel, Associate Director of CNS, Autoimmune/Inflammation & Ophthalmology, as she looks at other treatments that are in the pipeline to better identify and treat Alzheimer's disease.  Register to join her web seminar on December 17.  Mention priority code XP2000W3BLOG.

    Wednesday, December 3, 2014

    Clinical trial patient enrollment partnerships for HIV treatment

    In Citeline's most recent report Collaborations in HIV treatment trials: a help or hindrance for patient recruitment?, Doro Shin, MPH, Principal Analyst, Infectious & Genitourinary Diseases looks at the enrollment rates sites and programs for HIV clinical trials in relationship to number and type of sponsors.

    Here are a few of the key statistics on enrollment for the HIV trials studied:
    • -Highest trial enrollment rates were from a single industry sponsor.  Higher trail enrollment rates were not observed with a collaboration
    • -Government agencies were the only group who benefited from higher sight enrollement rates when collaborating with another party for AIDS clinical trials
    • -Site enrollment rates increased as the number of sponsors increased
    • -Collaborative trails saw a higher enrollment rate than single sponsors
    • -Collaborations saw higher sight enrollment rates across all trial sizes

    See how one government organization, NCATS, is collaborating with industry to speed up drug development in the presentation How a Collaboration between the National Center for Advancing Translational Sciences (NCATS) and Transparency Life Sciences is Accelerating Drug Development this April at Partnerships in Clinical Trials.  For more information on this session and the rest of the program, download the agenda.  As a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off the current rate.

    Wednesday, November 26, 2014

    Cyber Monday starts early! See details how you can save on Partnerships in Clinical Trials!

    To get a head start on the holiday season, as a member of our online  Partnerships in Clinical Trials community, we’re giving you an extended chance to take advantage of our Cyber Monday sale. Register with code CYBER2014  and receive 30% off the standard rate.

    Are you interested in attending on of our other events?  Head to our sister blog Future of BioPharma for a complete list of events you can save on through Monday, December 1.

    *This promotion is only valid Wednesday, November 26, 2014 until 11:59PM on Monday, December 1 , 2014. Offer cannot be applied retroactively to confirmed paying registrants and cannot be combined with any other discounts or promotions. All registrants and guests are subject to IIR approval.

    Tuesday, November 25, 2014

    Six Questions to Determine FDA Clinical Trial Site Compliance

    Drug accountability management is one of the key items that can ensure that all aspects of running a clinical trial - from data integrity, staying on a the clinical trial time line, managing costs and more.  In a recent white paper from Cenduit focusing on electronic drug accountability systems.

    What are the six questions that a site can ask to ensure compliance?

    1. 1.  Who is authorized to administer or dispense the investigational drug?
    2. 2. Has the investigational drug been supplied to any unauthorized person?
    3. 3. Can the records for investigational drug inventory be reconciled, i.e., the quantities shipped, received, used, and returned or destroyed?
    4. 4. Can drug shipments, dispersals, and returns be verified?
    5. 5. Is the drug stored in the manner mandated by the protocol?
    6. 6. Does the storage of drugs with the potential of abuse meet the federal regulations for controlled substances?
    In the white paper, they focus on the different aspects of keeping records to verify the questions above.  As trials evolve, they call for the evolution of record keeping as well and set forth options for the systems to keep these records electronically providing electronic drug accountability.  A simple error in a paper based system is hard to detect and trace.  As accountability becomes more complex, considering a more dynamic system is important and can help trace and keep companies compliant by doing the following:

    Interactive response technology (IRT) systems are used in many clinical trials for a myriad of tasks, from patient randomization to drug supply management and allocation. IRT is ideal for drug accountability because it tracks drug dispensing units by warehouse, depot, and site location, and by batch, bulk lot, packaging step, label group, and patient allocation.

    Do you use an IRT system for your drug accountability management?  What's the biggest benefit of doing this electronically?

    Data keeping is just one way to optimize clinical trials.  This April at Partnerships in Clinical Trials, the track Clinical Operations Optimization will focus on how operations, including  speeding up clinical trials and the tools to do so will be covered.  For a preview of the sessions in this track, download the agenda.  As a reader of this blog, when you register to join us and mention code XP2000BLOG, you can save $100 off current rates.

    Tuesday, November 18, 2014

    Web Seminar: Global Rise of Chronic and Age related Diseases: Trends in Clinical Development Globally

    Global Rise of Chronic and Age related Diseases: Trends in Clinical Development Globally
    Presenter: Laura Runkel, Associate Director of CNS, 
    Autoimmune/Inflammation & Ophthalmology, Citeline
    Join us for a webinar on Dec 17, 2014 at 2:00 PM EST.
    Register now!
    Mention priority code XP2000W3BLOG

    About the web seminar:
    Chronic and age related diseases are on the rise globally. Given the demographics of an aging population and life style changes in the high income countries, this marked rise in chronic diseases is projected to continue into the future. Clinical trial activity in these diseases has increased globally as treatments are sought to address the substantial unmet need. Case studies for drug and clinical trial activity in two serious diseases, chronic obstructive pulmonary disease (COPD) and Alzheimer’s disease, will profile the current therapeutic landscape to gain key insights into successful programs and to gauge future prospects.

    Several novel fixed-dose combination (FDC) therapies were approved in 2013 for COPD maintenance therapy. The analysis of study design and timelines provides a current snapshot of the COPD landscape and a look at the future for pipeline FDC competitors still in development. Standard of care for Alzheimer’s disease includes cognition enhancing drugs that provide limited symptom relief and have no impact on disease progression. Hope for a treatment that halts or slows the progression of Alzheimer’s disease lies with the development of disease modifying therapeutics. The early clinical disappointments for beta amyloid antagonists have created an opportunity to improve clinical trial design and AD diagnosis. A paradigm shift is reflected in current AD clinical trial design by top industry sponsors.

    What you will learn:
    • • The utility of Citeline’s integrated drugs, trials and timelines solutions for analysis of clinical development status/trends
    • • Key insights into the success of COPD FDC clinical programs
    • • The current competitive landscape for pipeline FDCs in COPD
    • • How treatment of earlier stage Alzheimer’s disease is occurring for disease modifying drugs
    • • What are the top industry sponsors, drugs, and clinical timelines for pipeline Alzheimer’s disease therapies

    This web seminar is produced in coordination with Partnerships in Clinical Trials. 

    Wednesday, November 5, 2014

    Steps towards Risk Based Montioring

    In the November 2014 International Clinical Trials Journal, Andrew Newbigging, the Senior Vice President of Research and Development at Medidtat outlined many of the elements of risk based monitoring, how traditional procedures are holding things back and how the new, updated approach can bring costs down on a monitoring budget.

    To get a RBM project started, Newbigging outlined these steps:

    • 1) Identify attributes of a clnical protocol
    • 2) Identify key risk areas
    • 3) Categorize risks into an Integrated Quality Risk Management Plan

    After these things are set into play, a trial can then be monitored by both operational data and and trial data.  KRIs can then allow the monitors to identify the risks of each trial and change them from afar.  By using RBMs and the three criteria above, fewer sight visits will have to be made and will reduce the over all cost of site monitoring.  It will also improve the quality and security of the projects in the long run.

    Read the full article here.  Do you feel that the high cost of implementation for risk based monitoring will be outweighed by the benefits that central monitoring brings to a clinical trial?

    This April at Partnerships in Clinical Trials, we will have the full day working group dedicated to Risk-Based Study Management.  For more information and a preview of this study, download the agenda.  As a reader of this blog, when you register to join us and mention code XP2000BLOG, you can save $100 off the current rate.