Monday, March 30, 2015

Wanted: Guidelines for Access to Experimental Drugs

Critically ill patients need new medicines, but what if this slows FDA approval for future patients?

By Kenneth I. Moch. This post was originally published on the Wall Street Journal      
A year ago this month a social-media crisis thrust a set of complex ethical questions and dilemmas onto life-science companies. The family of a 7-year-old boy turned to patient advocates and social media to pressure Chimerix, a small biotech company of which I was CEO, to provide access to an experimental drug, brincidofovir, to fight his life-threatening viral infection.

Over five days Chimerix became the focus of a social-media wave that rapidly spread into the global mainstream media. Josh Hardy would get the drug, but the patient advocate who led the campaign has said he would have “destroyed Chimerix” if Josh hadn’t.

Expanded access, more commonly called “compassionate use,” provides an experimental medicine to treat a seriously ill individual who has no other options. Because these requests generally occur prior to completing clinical studies designed to determine safety and efficacy, there may be only limited understanding of the drug’s risks and benefits given the patient’s specific medical situation. Moreover, a patient may want the medicine for a different disease or condition than the one for which it is being developed.

Requests for expanded access increased 92% in 2014, a trend that will continue. In the aftermath of Josh Hardy’s story and others, eight state legislatures have enacted and over 20 are considering “right to try” laws, under which terminally ill individuals are deemed to have a “fundamental right” to receive experimental medicines and devices via expanded access. Last month the Food and Drug Administration took steps to simplify the expanded access application form for physicians.

Life-science companies now bear nearly the entire weight of deciding requests for expanded access. Management teams must balance the near-term needs of the individual versus the longer-term needs of the population, with little to guide them.

Should pressure from social media or special connections play a role in whether a patient receives an experimental medicine? Should a company avoid any risks to a development program in order to help a larger number of patients in future years? If there are a limited number of doses available, how should a company choose which patients receive the medication?

What if a critically ill patient gets worse or dies for reasons unrelated to the experimental drug, but because of an ensuing uproar other patients decline to participate in clinical trials? Who is advocating for future patients who, because of slower clinical-trial enrollment or unexpected events, might not receive a needed medicine because FDA approval is derailed or delayed by even a month?

These ethical decisions should not rest solely on corporate leadership. Instead, there needs to be a focused effort to create a more equitable approach to expanded access. Here are several proposals:

• Life-science companies should publicly state their policy on expanded access. It must be recognized that a company has the right not to make an experimental medicine available if it believes the greater good is served by this decision.

• Regulatory guidance should provide a framework so companies can consider the risks to the drug development timeline and approval pathway in the face of unforeseen or unfortunate consequences from granting expanded access.

• Especially for smaller companies whose drug or device may be their only product, there should be an optional system to provide support for these complex decisions. Last August New York University bioethicist Arthur Caplan and I proposed an independent “Expanded Access Institutional Review Board,” which could help companies weigh multiple factors, including equitable access and availability, cost, and the short- or long-term risks and benefits to a development program.

Josh Hardy received brincidofovir, but not under expanded access. With extraordinary collaboration from the FDA, Chimerix initiated a new Phase 3 clinical trial that has recently shown the drug’s potential to reduce the mortality rate of the virus that was killing Josh. He was the first patient enrolled in the trial, rapidly responded to the drug, and now is approaching his 9th birthday.

This positive outcome should not lead anyone to assume that there is a simple, monolithic solution to expanded access. Each drug is different, the testing and data required for FDA approval are different, and patient populations are different. Expanded access is not a substitute for clinical trials.

Our current system is rife with ethical dilemmas and is not equitable. If we want critically ill patients to receive experimental medicines as a last resort, we need to find a balance between the immediate needs of individual patients and the equally important needs of future patients.

About the Author: Mr. Moch has been co-founder or CEO of four companies developing therapies for life-threatening diseases and most recently was the president and CEO of Chimerix In.

Thursday, March 26, 2015

Building Clinical and Economic Evidence for Mobile Medical Applications

Est. 1997: Mobile Medical Applications and the FDA

The FDA describes mobile apps as “software programs that run on smartphones and other mobile communication devices. They can also be accessories that attach to a smartphone or other mobile communication devices, or a combination of accessories and software.” The FDA defines mobile medical applications (MMAs) as “medical devices that are mobile apps, meet the definition of a medical device and are an accessory to a regulated medical device or transform a mobile platform into a regulated medical device.”

The FDA’s MMA guidance document describes three categories of MMAs:

While the FDA’s MMA guidance document is relatively new, in 1997 the FDA first cleared the RhythmStat XL System, a Class II medical device MMA that telephonically received and recorded electrocardiograph (ECG) data from a cardiac event recorder and transmitted it to a Palm device. Between 1997 and 2011, when the first MMA Draft Guidance document was published, approximately 50 MMAs were cleared by the FDA. Since 2011, this number has more than doubled. Most MMAs are Class II medical devices, follow the pre-market notification pathway and are predominately used in chronic disease management, ECG and remote patient monitoring. They have begun to emerge in image diagnostics and in medication management and adherence. Within the last decade, numerous MMAs have become game changers in delivering improved outcomes and enabling cost-effective health care. For example:

Launched in 2012, the AliveCor Heart Monitor enables health care providers and patients to record, display, store and transfer single-channel ECG rhythms. AliveCor has continuously evolved since its launch, adding new features and mobile operating platforms, and obtaining additional FDA clearances in 2013, 2014 and 2015. The evolved versions are able to display ECG rhythms and detect the presence of atrial fibrillation. One reason for AliveCor’s early market success is that it was able to utilize, from launch, existing Current Procedural Terminology (CPT) coding widely covered and adequately reimbursed by traditional payers (i.e., third-party insurers). This enabled physicians to adopt the technology without financial penalties (even under the traditional fee-for-service reimbursement environment).

AirStrip Technologies develops remote patient monitoring MMAs. As with AliveCor, AirStrip MMAs have evolved since their launch in 2004 as AirStrip OB, intended to be used by obstetricians to remotely review critical real-time data from labor and delivery (e.g., fetal heart tracings, maternal contraction patterns). As with AliveCor, AirStrip has added features and additional modules, expanding to a total of 11 cleared MMAs to date. AirStrip’s success is due to its ability to demonstrate improved clinical outcomes as well as improved financial performance to end users and payers (which, in the case of a technology used during inpatient hospitalizations, are one and the same).

WellDoc develops MMAs that function off of its diabetes management platform system. One of its MMAs, BlueStar, was the first mobile prescription therapy cleared by the FDA for adults with type 2 diabetes. At the system’s foundation is an integration of technology, real-time data, analytics, ease-of-use and collaboration that ultimately drive improved metabolic outcomes and reduced health care costs. In addition to covering BlueStar as a pharmacy benefit, many traditional third-party insurers have embraced WellDoc’s MMAs (e.g., DiabetesManager) as a part of their own disease management programs. WellDoc achieved market success through the conduct of rigorously designed, randomized and controlled clinical trials to demonstrate improved clinical outcomes and improved cost-effectiveness in the management of type 2 diabetes. These studies went beyond what was required for FDA regulatory approval, which facilitated early adoption and coverage by third-party insurers.

MMAs: Evidence and Success
As demonstrated by the cases above, MMAs regulated by the FDA follow a life cycle that is typical of a medical device. As such, it’s not surprising that regulatory, clinical and reimbursement strategies are the critical success factors for an MMA to gain marketing approval as well as widespread market utilization. As with pharmaceutical, biotechnology and other medical device products, MMA developers seeking widespread coverage and utilization by the health care ecosystem must understand market access and reimbursement factors for their particular site(s) of service and utilization scenarios. They must also go beyond the minimum requirements of establishing safety and efficacy in pre-market clinical studies to obtain evidence of effectiveness under conditions of actual use and cost-effectiveness according to the payer’s perspective (noting that the “payer” may be a traditional third-party insurer, a health care provider or facility or a patient).

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.

Wednesday, March 25, 2015

Study Identifies Best Practices for Lean Outsourcing of Clinical Development

By Marc Dresner, Senior Editor, IIR

We can now add clinical development to the list of functions at major corporations benefiting from the Lean movement pioneered by Toyota.

According to a study conducted by The Avoca Group on behalf of Purdue Pharma, Lean Models for outsourcing clinical development have by and large been achieving desired results at major pharmaceutical companies where they have been deployed.

“It was interesting to hear how well these models were working from so many different people at so many different companies,” said Dr. Denise Calaprice, Senior Consultant at the Avoca Group, in a recent interview with Inside Clinical.

Big pharmacos have been adopting Lean Models to accommodate greater demand for efficiency, reduced headcount and non-traditional, “nimble” product development strategies.

Denise Calaprice
Calaprice noted Lean and even “virtual” clinical outsourcing isn’t necessarily new; small pharmacos and biotechs have always done it out of necessity.

Dr. Mitch Katz
But in recent years, large pharmaceutical companies have been exploring and adopting Lean Models for outsourcing clinical development to accommodate greater demand for efficiency, reduced headcount and a drive toward non-traditional, “nimble” product development strategies.

Until now little was known about the extent or effectiveness of these implementations, which concerned Purdue’s Head of Medical Research and Drug Safety Operations Dr. Mitchell Katz when the company began considering a Lean strategy as part of a restructure last year.

“We had undergone a headcount reduction and our pipeline was lean…A model that would minimize the level of internal resources for CRO oversight made sense.”
– Dr. Mitch Katz, Purdue Pharma

“We [at Purdue] had undergone a headcount reduction and our pipeline was lean,” Katz told Inside Clinical. “For us, a model that would minimize the level of internal resources for CRO oversight—the Lean Model—made sense.”
(It’s worth noting that this wasn’t simply a belt-tightening measure; Katz said that while Purdue is shifting some focus from its pain franchise, the Lean Model will factor into plans to expand its CNS work.)
Purdue enlisted The Avoca Group to canvass companies—large and small—that had implemented or evaluated Lean Models in order to compile best practices and standards.

The study uncovered seven key areas companies should explore when considering adopting a Lean Model as part of its clinical development strategy.

The study uncovered seven key areas companies should explore when considering adopting a Lean Model as part of its clinical development strategy, spanning such things as which competencies to retain in-house (staff and/or process), the number and types/sizes of CROs to engage and even the types of trials to be outsourced (phase, TA, region).

“Companies had to ask themselves these questions in order to really make sure that they were behaving in a smart way with respect to these models,” Calaprice said.

“When they did go through that process of analysis and made the right decisions for their company—the decisions weren’t always the same—the model seemed to work really well for them,” she reported.

“It was interesting to see how certain contractual models—like risk-sharing—and metrics, CRO relationships and expectation manuals played such a significant and effective role,” Katz added.

In this episode of Inside Clinical—the Partnerships in Clinical Trials podcast interview series—Katz and Calaprice share findings from the study and discuss implications for sponsors and CROs.

Editor’s note: For more on this topic, Drs. Mitch Katz and Denise Calaprice will be speaking at the 24th Annual Partnerships in Clinical Trials Conference running April 22nd through the 24th in Boston.
For more information or to register, please visit
For more information about the study or The Avoca Group's customized research services, contact

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 publication for market researchers in the pharmaceutical industry. He may be reached at Follow him @mdrezz.

Tuesday, March 24, 2015

Is Risk Based Monitoring the Future of Clinical Trials?

By: Sathya Sankarji, Clinical Data Manager II at Eclipse

When and why to opt for RBM, an increasingly popular approach to clinical trial site management.

Site monitoring is one of the most costly factors of clinical trials.  Traditionally, clinical trial teams have had to allocate equal resources to all study sites, regardless of risk.  When adopting a Risk Based Monitoring approach, you work to identify the sites with the greatest risk and need and allocate resources accordingly.  This intelligent strategy allows you to use your team’s time more efficiently, and often lowers the costs associated with site management.  RBM has become increasingly popular, as regulatory agencies in the U.S. and Europe have recently expressed support for the tactic.  

The Benefits of RBM

Source Data Verification (SDV) is expensive, but the conventional approach has been to perform 100 percent SDV.  Literature states that SDV can consume about 25 percent of a trial budget. A lot of this cost goes to verifying data that are not critical.  Focusing time and effort on non-critical data verification can affect the quality of review and source verification of critical data.  CRAs can use their time more effectively if the monitoring is targeted, and they focus on quality rather than quantity.

A quality by design approach which involves determining the risk indicators and a well-designed monitoring plan will result in faster and smoother trial conduct and ultimately a successful regulatory submission.  Additionally, integrated technology that provides a holistic view will aid in early trend analysis and help you make informed decisions early in the conduct of the trial.

Some questions to ask when considering a RBM solution:

·         Does it offer real-time information on site performance and data quality?
·         Does the solution provide ways to identify error ahead of time?
·         Is the solution flexible enough to allow mid-study changes?
·         Do you have a trained team to leverage the various tools that technology offers in the optimal way?
·         How do the errors detected by monitors on a 100% SDV compare to those detected with reduced SDV?

RBM may prove an optimal site management strategy for your next clinical trial, potentially saving you time and money.  Ensure you understand the benefits of the approach and the details of the specific RBM program you’re considering so you can make the right decision for your team. 

INFOGRAPHIC:  Caption: Risk Based Monitoring can be defined in a number of ways

Friday, March 20, 2015

Spring into PCT Savings!

Spring into Savings with a 30% off the Standard Rates to Attend Partnerships in Clinical Trials*!

But Hurry! This flash sale is only valid thru Monday, March 23rd!

Be sure to use code “SPRING30” to save, register here.

*Qualifications, rules and regulations:

Offer cannot be applied retroactively to confirmed or pending registrations from today's date and priorThe 30% discount cannot be combined with any team discounts or other promotions and/or discount offers.Discount is only valid from March 20-March 23rd, 2015All registrations are subject to IIR approval.

Wednesday, March 18, 2015

Transparency is Needed in the Future of Clinical Trials

“Telling the truth is a relatively new thing in clinical medicine and well as clinical research.” This quote, which came from Dr Phil Hammond, a GP and Broadcaster, who was chairing a panel at last year’s Partnerships and Clinical Trials Europe event, gives an insight into the changes that are being implemented in the pharmaceutical industry. The discussion chaired by Hammond discussed the topic ‘Clinical Trials in 10 years’ and a key point that stood out was the need for transparency.

As the quote at the beginning states, transparency, i.e. the truth, is relatively new in the field; however the need for it is becoming critical for companies within the industry. It has been rather shunned in the past due to wanting to preserve patient privacy and safeguarding volunteers in trials, as well as not giving anything away to competitors.

However, Anders Persson, Hub Director of Clinical Operations at AstraZeneca highlights there is now a need to share data more widely and make sure it is available for everyone. Jay Kaminski, Corporate Vice President of Global Clinical Research and Developmental Operations at Celgene believes that transparency helps to get the patients more involved with their own data and point of care. Having data in the public domain can help to educate patients but he believes it needs to be done in a consistent manner from company to company.

Brendan Buckley who is the Chief Medical Officer at ICON stressed that patients should be viewed as partners. By interacting on a partner level means more involvement for the patient, who at the end of the day, are the most important part of the trial. Transparency across these partnerships will keep the patient engaged and informed, otherwise they might head elsewhere for knowledge. Patients often now talk to each other and engage with one another on platforms such as social media, forums and chat rooms. So being open and transparent with the patients will let them have the information to interact with one and other, which ultimately keeps them more informed with decisions and help with future trial loyalty.

I think the transparency should go beyond the clinical trials; in a recent post I discussed Collabobeat which is a system designed to share doctor’s notes with patients and create a more open relationship between physician and patient. Transparency at a higher level could help these relationships as well, as doctors can then share data from clinical trials to their patients to help persuade them to try new drugs or trial drugs to potentially improve their health.

Transparency within clinical trials is deemed something that is important in the next 10 years. A willingness to share participant level data and results may be important in keeping patients engaged and informed which may help with future trials.

Check out the full video below:

For information on the 24th annual Partnership in Clinical Trials conference taking place in Boston this April, click here:

About the Author: Harry Kempe, a marketing intern at IIR USA, who works on various aspects of the industry including social media, marketing analysis and media. He is a recent graduate of Newcastle University who previously worked for EMAP Ltd. and WGSN as a marketing assistant on events such as the World Architecture Festival, World Retail Congress and Global Fashion Awards. He can be reached at

Monday, March 16, 2015

What is the Role of the Patient in Clinical Trials?

Last year’s Partnerships in Clinical Trials Europe event asked the question ‘what is the role of patients in the clinical trials industry?’ We asked many different attendees at the event what they thought the role of the patient was within their company and the general consensus that came from answers was that the patient is by far the most important thing to think about when developing new drugs. Katarzyna Dulna, who is the Clinical Research Manager at Clinmark gave a succinct response that sums up to me the role of the patient – ‘the patient is the most important, it is in the front of the mind and in the back of the mind.’

Companies in the pharma industry are becoming more patient-centric in their approaches to clinical trials. Meeting the needs of the patient before anything is paramount when developing new drugs. Aaron Mazze, the Vice President of Marketing at Merge eClinical stressed the need to look ‘for ways to help organizations get their drugs on the market faster’. New technologies and innovations in the development and production lines will help to produce drugs faster and thus be of aid to patients who are in urgent need of them.

Sasha Sonnenberg, the Vice President of Commercial Operations at Marken goes on to say that the care and quality standards need to stay high even after the critical stages of development. He mentions that ‘it’s not about the first shipment, it’s about each and every shipment that needs to be handled in the same important way and needs to be handled with care’. The drugs after production need to get to the physicians to give to patients. If a shipment is lost or damaged then that could cause serious ramifications down the line, potentially being life threatening for patients.

Jack Whelan, author, research advocate and cancer survivor/warrior gave a compelling keynote at last year’s event which gave mention to the fact that patients today are ‘more educated, empowered and e-connected’, which he believes should be highly encouraged. Patients today, especially with diseases such as cancer are far more connected with one and other through platforms such as chat
rooms and educational forums. Industries need to encourage such platforms so that firstly patients and other affected people such as family or friends can learn as much as possible about dealing with diseases and secondly industries can look at these forums to see exactly the needs of the patient. Patrice Hugo, the Associate Vice President and Chief Scientist at Labcorp Clinical Trials highlighted that companies need to be more ‘exposed to the patients reality.’ which can be done through studying forums and other similar sites.

The role of the patient for clinical trials companies is incredibly important. By getting the needs of the patient at the forefront of development, pharma companies are able to produce new drugs that are more patient-centric. The production from start to finish has to be carried out in the most efficient way possible and so new technologies and techniques are constantly being looked at to minimize time taken to get the drugs to patients.

Check out the full video below: 

For information on the 24th annual Partnership in Clinical Trials conference taking place in Boston this April, click here:

About the Author: Harry Kempe, a marketing intern at IIR USA, who works on various aspects of the industry including social media, marketing analysis and media. He is a recent graduate of Newcastle University who previously worked for EMAP Ltd. and WGSN as a marketing assistant on events such as the World Architecture Festival, World Retail Congress and Global Fashion Awards. He can be reached at

Thursday, March 12, 2015

Relationships are Key to Startup Partnerships

Partnerships between pharmaceutical companies are vital for the development and growth of startups within the industry. Jodie Morrison of Tokai Pharmaceuticals spoke at the Partnerships in Clinical Trials Conference in 2014 and discussed in an interview the challenges that small, speciality pharma companies face and how big CRO’s should be interacting with them.

Morrison, the CEO of Pharmaceuticals and who has huge experience in clinical affairs, believes that ‘smaller companies are reliant on partnerships’. Smaller specialty pharma industries often struggle to make an impact in the industry due to them not having the same resources and funds that the larger companies do. Forming partnerships gives an opportunity to boost productivity and get past financial roadblocks that could halt R&D and clinical trials before they even reach the human trial phase. Partnerships have to be made in a way that is mutually beneficial and Morrison believes that they need to be ‘appropriately built.’

A theme that is prevalent throughout the interview is one that people are incredibly important, in that having staff and a team that are happy and work well will ultimately boost turnover. Morrison noted that often there can be a lot of travel involved for the ‘front line’ employees such as program managers. They deal with sites in different locations and small things such as putting them up in a nice hotel or paying for presents for relatives they may be staying with, can go a long way for keeping teams happy.

Partnerships between pharma companies, especially for the smaller companies are very at risk of failure and there is very little room for error. The partnerships, as mentioned before should be ‘appropriately built’ and often in startups, the bulk of the staff on a non-management level is through partnerships. Forming new partnerships should be looked at as if they are hiring someone, as in the long run, partnerships are the driving force of the company. So to minimize the chance of failure, as previously touched upon, having a strong and happy team to work with and drive the company forward will ultimately mean a stronger chance of success. Morrison, mentioned that she was fortunate enough to have never lost an employee as a result of them wanting to leave which was down to a great team environment and ultimately was beneficial to the company.

The crucial element that I believe came out of the interview and is the key to success in pharmaceutical partnerships is the importance of all people within the company. From top to bottom, having staff feel incentivized and mentored, but not in a competitive environment with each other means better collaboration and in the long run better productivity for the company.

To watch the entire interview, check out the video below:

For information on the 24th annual Partnership in Clinical Trials conference taking place in Boston this April, click here:

About the Author: Harry Kempe, a marketing intern at IIR USA, who works on various aspects of the industry including social media, marketing analysis and media. He is a recent graduate of Newcastle University who previously worked for EMAP Ltd. and WGSN as a marketing assistant on events such as the World Architecture Festival, World Retail Congress and Global Fashion Awards. He can be reached at

Wednesday, March 11, 2015

CRF Health eCOA Solution to Support Flexibility & Compliance in Global Diabetes Trials

Recently, CRF Health, a global provider of eCOA solutions for the life sciences industry, released a TrialMax eCOA solution for diabetes clinical trials. Developed with clinicians and patients to optimize usability and compliance, the solution enables investigators to collect patient reported outcome assessments and deliver defensible data, while minimizing patient and site burden.  

"CRF Health's experience in eCOA data collection in diabetes studies gives us a wealth of knowledge and experience in helping customers implement solutions that are not only patient-focused, but that ensure the highest levels of quality data,” said Katie Garner, Therapeutic Areas Manager, CRF Health.

Today, Diabetes trials tend to have complex protocols which can lead to reduced compliance by overburdened patients. Fortunately, CRF Health's diabetes TrialMax Touch eDiary leads patients through data collection in an intuitive and fast way. It has been designed to easily fit into patients' lives while providing more compliant data, as well as increased patient retention. The customizable solution enables features to be added or removed depending on the needs of a particular trial, while the reference diary includes 5/7 data collection points which can easily be adapted.

"Our complete solution for diabetes trials is already being used in four impressive trials due to its unique capability to integrate patient diary entry and blood glucose monitoring, helping to ensure studies run as smoothly as possible, while delivering reliable results,” said Garner.

TrialMax Touch eDiary integrates with the Entra Health MyGlucoHealth wireless glucometer, enabling automatic transfer of measurements to a patient's eDiary. The integration ensures complete and accurate data while reducing the risk of transcription errors. Specifically, alerts can be set-up to remind patients to take their medication and test their blood glucose level, while also providing new basal dose directions from the physician in between site visits.

Further, sites are also made aware of hypoglycemic events via CRF Health's TrialManager, email or SMS in near real time which provides greater opportunity to take appropriate clinical action. The system also benefits from the ability to integrate with CRF Health's instrument library, allowing sponsors to electronically incorporate common diabetes instruments into the solution.

About the Author: Amanda Ciccatelli, Social Media Strategist of the Marketing Division at IIR USA, has a background in digital and print journalism, covering a variety of topics in business strategy, marketing, and technology. Amanda is the Editor at Large for several of IIR’s blogs including Next Big DesignCustomers 1stDigital Impact, STEAM Accelerator and ProjectWorld and World Congress for Business Analysts, and a regular contributor to Front End of Innovation and The Market Research Event,. She previously worked at Technology Marketing Corporation as a Web Editor where she covered breaking news and feature stories in the technology industry. She can be reached at Follow her at @AmandaCicc.

Friday, March 6, 2015

Communication Breakdowns Threaten Co-Development of Drugs and Companion Diagnostics

Drug Companies, Diagnostics Makers and Labs Must Align or Risk Millions in Development Delays

By Marc Dresner, Senior Editor, IIR

Drug companies are increasingly co-developing and embracing early evaluation of companion diagnostics in drug development to potentially reduce clinical trial costs and improve study outcomes,
but the way most companies go about it introduces a serious new risk that could potentially frustrate the entire development effort.
David Jackson, PhD

That’s according to Arno Therapeutics VP of Diagnostics and Companion Diagnostics Dr. David Jackson, who says there’s typically no coordination between the sponsor, the manufacturer of the kitted CDx product contracted by the sponsor and the lab that actually deploys it.

“The industry is still keeping these relationships separate.”

“I’m only aware of a handful of relationships that incorporate all three pieces in synchrony,” said Jackson. “By and large, the industry is still keeping these relationships separate.”

That’s a potential liability, Jackson says, because when issues related to the test or to the trial design or enrollment, respectively, crop up, the absence of a collaborative structure between the sponsor, the CDx manufacturer and the lab almost guarantees a costly delay—possibly to the tune of millions per day.

Changes can have “a very serious ripple effect.”

Jackson stressed that due to the less flexible approach for developing a diagnostic—because it is a device not a compound—any changes can have “a very serious ripple effect.”

Moreover, Jackson observed that drug developers are frequently ignorant to problems that can arise around regulatory requirements for companion diagnostics and don’t adequately understand the function of the clinical trial lab supporting development of diagnostics intended to subsequently support the drug.

“The company developing a targeted therapy needs to become more knowledgeable internally,” he said. “There are issues that come up during development of the diagnostic that are influenced heavily by trial design and by anything that may come up with the FDA.”

Editor's note: Last July, the FDA issued new guidance 
to help companies identify the need for companion diagnostics at an earlier stage in the drug development process and to plan for co-development of the drug and companion diagnostic test.

Issues may also occur at the laboratory level.

“Issues may also occur at the laboratory level as they see specimens come through for a trial that necessitate a change to the diagnostic test, or worse, a change to the trial from an enrollment perspective,” he added.

Jackson urged drug developers to stop siloing partnerships with CDx makers, labs and, where applicable, CROs, because there are too many interdependent factors that can cause a delay with a profound financial impact.

“More than half of the cost of developing any compound to approval is associated with the value of time,” Jackson noted. “For just a half-billion-dollar product, for example, every single day lost toward approval amounts to more than $2 million in lost revenue.”

In this podcast for Inside Clinical—the official interview series of the 24th Annual Partnerships in Clinical Trials conference—David Jackson makes a case for a new partnering model between drug developers, CDx makers, labs and CROs, and offers tips for each party.

Editor’s note: David Jackson will be speaking at the Partnering with Central Labs Summit co-located with the 24th Annual Partnerships in Clinical Trials Conference running April 22-24 in Boston.
For more information or to register, please visit

For more information about the co-located Partnering with Central Labs Summit, click here.  

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 trade publication for market researchers in the pharmaceutical industry. He may be reached at Follow him @mdrezz.

TransCelerate Highlight Collaboration as the Future for the Pharmaceutical Industry

Last year’s Partnerships in Clinical Trials Europe, featured a panel discussion centered on TransCelerate BioPharma Inc. TransCelerate is a non-profit organization that focuses on developing innovation in research and development, improving patient wellbeing and pinpointing and solving R&D problems that are common today. The talk was with three leading members of the pharmaceutical industry - Dalvir Gill, CEO of TransCelerate BioPharma Inc., Jonathan Zung, Vice President, Head Global Clinical Sciences & Operations, UCB, and Thor Voigt, Senior Vice President, Global Clinical Operations, Boehringer Ingelheim.

The discussion, which was titled ‘The Collaboration Between Major Stakeholders’, looked at how collaboration is key to driving down costs in the pharmaceutical industry. Drug development costs are increasingly on the rise despite application efficiencies, companies merging and the introduction of new technologies, to name a few. A few years ago some R&D leaders from major pharmaceutical companies came together to look at problem areas in the industry that need improving. Clinical Trial Execution was seen as an area that had very outdated methods that was very costly and needed a revamp.

Gill, the CEO behind the organization, pinpointed the future focus of clinical trials needs to be aimed at big data, connected interfaces and more patient-centric trials. Companies when undertaking projects should look at whether they enhance previous investments; fit in with their roadmap for the future, if they will realistically deliver and it the venture will fit their day to day practice.
Zung, the Head of Operations detailed how TransCelerate had decision making meetings every other week that involved huge players in the pharmaceutical industry. He stressed that consensus decision was crucial for making a difference within the organization, rather than just giving precedent to one company’s agenda over another’s. The focus is on how a difference can be made rather than just letting companies do what they please. On similar lines of more consistency between companies, Voigt, of Boehringer Ingelheim highlighted the need for standardization across the industry. He believes there is a need to standardize definitions of indications and diseases plus in and exclusion criteria.

The more encompassing nature of TransCelerate helps to bring companies together in a transactional network that lets members purchase active comparators and placebos from one and other. The non-profit body also is launching a CRO forum that is open to all CRO’s in order to engage with each other to help push development in clinical trials. The point that stood out to me the most from this discussion was TransCelerate’s move to engage regulators in the pharmaceutical world. This is not just the FDA but globally; for example a week after the talk, the organization was engaging with PMDA, the Japanese regulatory body. The aim is to make sure both parties understand each other so there is no contradictory guidance in the trial processes.

TransCelerate though still a relatively new entity, have really pushed to improve collaboration within the pharmaceutical industry and are developing solutions to drive costs down and improve productivity. To hear the more in-depth discussion and further innovative industry ideas, watch the whole video below:

About the Author: Harry Kempe, a marketing intern at IIR USA, who works on various aspects of the industry including social media, marketing analysis and media. He is a recent graduate of Newcastle University who previously worked for EMAP Ltd. and WGSN as a marketing assistant on events such as the World Architecture Festival, World Retail Congress and Global Fashion Awards. He can be reached at

Tuesday, March 3, 2015

Developing Technologies Payers Will Adopt

Despite the tremendous contributions medical devices have made to medicine, historically physicians viewed devices with suspicion and were wary of machine-produced information. Today, the U.S. medical device industry is the largest in the world with a market size of approximately $110 billion and is expected to reach $133 billion by 2016.1 Despite the industry expansion, many of the challenges medical devices faced in the past continue to affect modern-day markets. While new technologies are developed at a rapid pace, providers and payers may remain slow to adopt them.

Sufficient data demonstrating effectiveness, safety and value from multiple perspectives is key to the adoption of new devices. For health care providers, devices should be practical and meet recognized clinical needs. For payers, a device’s combined clinical and economic value must be clear before coverage is provided. Addressing the needs and concerns of payers and end users can be challenging.

Seeing Value Through the Payer’s Eyes

In the U.S., when we refer to “payers,” we traditionally mean government payers, such as the Centers for Medicare & Medicaid Services (CMS) or private payers such as the Blue Cross Blue Shield Association. More and more, health care facilities (e.g., hospitals) directly purchase medical devices without receiving reimbursement from traditional payers. In these cases, facilities act as payers, too.

The expanding role of health care facilities as payers is spurred by health care reform, the creation of Accountable Care Organizations (ACOs) and a shift in emphasis from volume-based to value-based reimbursement. For innovative medical device adoption and success, identifying appropriate payers and market strategies revolving around those payers’ perceptions is crucial.

It is critical for medical device manufacturers to remember that traditional health care payers are not impressed with the safety and efficacy data collected in clinical trials for regulatory approval; payers seek evidence of real-world effectiveness as compared to currently available treatments. Furthermore, while often not explicitly stated, cost considerations are implicit in payer decisions to cover innovative technologies.

Payers and providers are also moving together toward value-based reimbursement where payers offer financial incentives to providers for improvements in population health. However, because value-based reimbursement is not yet the norm, manufacturers must design studies to collect sufficient short- and long-term data to demonstrate device safety, real-world effectiveness and economic impact from the traditional volume-based payer perspective as well as from the myriad value-based reimbursement perspectives still evolving.

Finally, when conducting research it is important to remember the role of hospitals and other health care facilities as direct purchasers of some technologies. Hospitals also want information on how an innovative technology will affect their patients’ health as well as their bottom line.

Paving the Road Ahead

Demonstration of long-term clinical and economic value from the payers’ perspective is necessary to gain coverage and adoption. It is crucial for every step of the development process to take place with the technology’s value story in mind. The right research and a well-formulated market access strategy will save substantial time and money and lead to earlier market adoption and widespread usage of medical devices and other technologies.

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.

SelectUSA. The Medical Device Industry in the United States. Accessed November 25, 2014.

Friday, February 20, 2015

Webcast Offers Glimpse into Partnerships Topic

What a great turnout we had for our co-hosted webcast Change Investigative Site Relationships for the Better. And thanks to Partnerships in Clinical Trials, our co-host. The webcast featured presenters from IMS Health, who spoke to using data in choosing investigators and sites for a clinical trial, and David Green, Executive Director of United Therapeutics presented “Building and Maintaining Clinical Site Relationships and Loyalty.” At the Partnerships event, Green and his colleague Hassan Movahhed, Senior Vice President, Global Development Operations, United Therapeutics will go in-depth on what was presented in the webinar in their presentation Pharma—Investigator Collaboration: Meeting Needs Through Motivation.

Partnerships and Applied Clinical Trials chose this webcast to highlight a very important topic in the clinical research enterprise—the needs to understand and address the clinical investigative sites needs. While April Lewis and Ying Jiang at IMS Health provided information on data used for feasibility, qualification, negotiation (contracts and Fair Market Value), they also urged sponsors and CROs to share this data with the investigators to start a supportive dialogue to address their needs.

Why would this matter? Well, Tufts CSDD recently reported the decline of investigators—experienced investigators listing their increased burdens, as well as the novice investigators who conduct one trial and leave. Neither is good for maintaining clinical research in the long run noted Green. Green also agreed with Lewis that data be the foundation to very important discussions that lead to true collaborations.

Applied Clinical Trials is going to show up in force at the Partnerships in Clinical Trials conference. We are sponsoring both the Partnership Hall of Famer Award, as well as the Clinical Operations Optimization track for the event. We look forward to seeing you there!

Tuesday, February 17, 2015

Recent trends in Pharmaceutical R&D

Citeline have released their 2015 Annual Review regarding the trends in pharmaceutical R&D. Ian Lloyd, the reviewer for the past 25 years gives insights into new trends and the implications of them. In this blog I shall summarise the main points that are discussed, however for more detailed analysis the whole whitepaper has to be read. 

One of the main focuses of the paper is total pipeline size and this year the figure has once again risen. The increase of 8.8% from 2014 takes the number of drugs in the development pipeline up to 12,300. The study then broke down the pipeline into different clinical stages and found that the phase with the highest number of drugs was in the preclinical phase. 6061 drugs were found to be in the pipeline in 2015 in the preclinical phase compared to 5484 in 2014. The mostly sought after data is from the three stages of clinical development. For the three stages there were increases across the board; for phase one, two and three there were increases of 4.9%, 7.0% and 8.6% respectively.

The report goes on to break down the pipeline figures into which companies are doing the best in terms of pipeline size. The top 25 is given but I have picked out the top 3:
  1. 1. GlaxoSmithKline – 258 drugs in the pipeline (2015)
  2. 2. Novartis – 245 drugs in the pipeline (2015)
  3. 3. Roche – 234 drugs in the pipeline (2015)
GlaxoSmithKline remains top of the pile despite slipping from 261 drugs in 2014 with the two next companies trading positions from 2014. A notable point in the table is that UK based AstraZeneca took 4th spot after fighting off a huge takeover bid from US based Pfizer who could only make 7th. However, how long can AZ fight off the Pfizer onslaught? More takeovers are likely because as it stands there are 3,286 companies with active pipelines. 56% of these companies are very small and Lloyd believes they could easily be gobbled up. The majority of the companies are found to be from the United States; 47% of R&D companies have headquarters in America, only losing 1% to the UK from 2014.

In terms of what therapeutic areas are dominating the pipelines, anticancer still leads the way with biotechnology a close second, despite not strictly being a therapeutic group. The worst performer was in the neurological area which only had a 4.5% increase from 2014. In terms of individual diseases, cancer types lead the way with 15 out of the top 25 being forms of cancer. The highest number of active drugs which are non-cancer are for rheumatoid arthritis and type 2 diabetes that came 4th and 5th respectively in the study.

Of all the drugs in the pipeline, the vast majority came from a synthetic chemical source. 6771 came from that origin with second being biological protein antibody with 1267. Out of all these drugs the study showed that almost half were injectable (47.4%) and the next most being oral at 37.6%.

As can be seen, there are very positive things happening in the pharmaceutical R&D world currently with the number of drugs increasingly year on year. Increasing numbers of companies are getting involved in the creation of new drugs which is very important for beating and helping patients in need. To read more in detail about what has been happening please read the full article. 

About the Author: Harry Kempe, a marketing intern at IIR USA, who works on various aspects of the industry including social media, marketing analysis and media. He is a recent graduate of Newcastle University who previously worked for EMAP Ltd. and WGSN as a marketing assistant on events such as the World Architecture Festival, World Retail Congress and Global Fashion Awards. He can be reached at

Friday, February 13, 2015

Presidents Day Sale 25% off | See who is attending Partnerships 2015

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.

IIR's Partnerships in Clinical Trials continues to bring together clinical leaders to identify, engage and enter into strategic relationships that drive their clinical research forward.

Who are some of the companies that will join us?
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* 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* Inovio Pharmaceuticals* InVivo Therapeutics* Johnson & Johnson* Johnson & Johnson - Janssen Biotech, Inc.* Juventas Therapeutics* Lexicon Pharmaceuticals* Mallinckrodt Pharmaceuticals* MEI Pharma* Merck* Mirati Therapeutics* Momenta Pharmaceuticals* National Center for Advancing Translational Sciences (NCATS)* Nektar Therapeutics* Novartis* Novartis Institute for Biomedical Research* NPS Pharmaceuticals* Otsuka* Pfizer* Pfizer China* Purdue Pharma* Regeneron* Roche* Roche Romania SRL* Sanofi* Shire Pharmaceuticals* 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* And more!*

Are you ready to join Clinical Leaders from these companies and more on April 22-24, 2015 in Boston? Register to join us with priority code PRESDAY and save 25% off the standard rate.

Find out more about this year’s event.

Have any questions? Email Roxana Siu.

*This promotion is only valid through Wednesday, February 18th at 11:59 PM. Offer cannot be applied retroactively to confirmed paying registrants or registrants that are pending payment. Cannot be combined with any other discounts or promotions. All registrants and guests are subject to IIR approval.