Monday, August 29, 2016

Preventing Inconsistencies in Study Conduct and Protocol Noncompliance Across Sites

By: Shree Kalluri

Clinical trials are more complex than ever and it’s important to make sure a study is being conducted in the same manner across sites. However, ensuring consistency across multiple sites for any aspect of a clinical trial can be more difficult than one would expect. Some inconsistencies at the site-level are unknown to many sponsors, resulting in both efficiency and quality problems in the industry.

One example of this industry challenge stems from having each research site transform the schedule of events, also known as the time and event schedule, into their local clinical trial management system (CTMS). This transcription requires sites to review and interpret the protocol document and build it out as a protocol calendar in their CTMS. The protocol calendar, a schedule of treatments and tests to be completed by a participant on a study, is one of the most important aspects of a clinical trial, and any deviations from the calendar can have detrimental consequences. The calendar is connected and tied to other downstream activities and uses within the site’s CTMS, making it more powerful than a static spreadsheet or PDF.

This process can be replicated from ten to a hundred times based on the size of the trial and the number of participating sites. With so much duplicated effort, this method is unnecessarily complicated, administratively expensive, and fraught with the potential for costly errors.

Friday, August 26, 2016

Personalized Medicine and the Future of Healthcare

"Personalized medicine is having an incredible impact on the way we think about drug development and clinical trials." Isan Chen MD, Chief Medical and Development Officer, Mirati Therapeutics

Advances in basic molecular science are paving the way for increasingly targeted therapies that address the unique biological mechanisms involved in a patient’s illness. Ultimately, medicines may become truly “personalized,” allowing for a fully customized approach to health care that takes into account an individual patient’s medical history, disease risk, and pathology, and selects the most promising course of treatment based on these and other characteristics.

This vision for the future may be a long way off, since translating scientific advances into targeted therapies has not proven to be quick or easy. However, taking advantage of innovative clinical trial designs could lead to more efficient clinical trials that do a better job of matching treatments to specific patient populations and speed the development of targeted therapies.

From Big Data to Genomics testing and targeted therapeutics, Dr. Chen discusses the disruptive approach to next generation clinical development.

To learn more on personalized medicine, targeted therapies and more, join us at Partnerships in Clinical Trials US in Boston, MA - October 5-7. Click here for more information.

Thursday, August 25, 2016

The Expectations & Realities of Clinical Trial Evolution

Joining the Partnerships in Clinical Trails US speaking faculty is Greg Sweatt, Senior VP of Life Sciences at ePatientFinder. As a co-founder, Greg was able to use his background in Health Care IT and apply it to the complex problem of matching the right patients with the right clinical trials. A loved one’s battle with Alzheimer’s, and physicians being unaware of existing clinical trials, was part of the impetus to partner with EHR vendors and their physicians to compliantly use patient health information in the consideration of clinical trial recruitment.

At the upcoming event, Greg will be presenting a session titled "The Expectations and Realities of Clinical Trial Evolution - Digital to EHR Data". As a lead in to this exciting talk, Greg joined us for exclusive Q&A to discuss the major trends, concerns, disruptors, innovation and his key to long term success in the clinical trials industry.

Wednesday, August 24, 2016

Big Data in Clinical Research

By Norman M. Goldfarb

According to Wikipedia, “big data” consists of “data sets that are so large or complex that traditional data processing applications are inadequate… The term often refers simply to the use of predictive analytics or certain other advanced methods to extract value from data, and seldom to a particular size of data set.”

IBM has proposed a four-dimensional model of big data: volume, velocity, variety and veracity. The biggest big data set is very large, grows very fast, includes numerous types of data from multiple sources, and provides the most accurate and complete picture.

In clinical research, the term “big data” is often used loosely to refer to large data sets, non-relational datasets, or advanced methods of visualization. For example, a “large simple trial” with 25,000 patients conducted 20 years ago would probably be considered an exercise in big data today, even though the data set is large only in comparison to other clinical trial data sets, is static in size, includes very few types of data (which can be captured in a relational database), and presents a very narrow, albeit accurate and precise, picture.

Tuesday, August 23, 2016

The Orphan Drug Act: A Boom for Business

Drug prices for orphan drugs had increased by 37%

An important new review of the impact of the Orphan Drug Act has found some surprising results. Since the act was approved over 30 years ago in the US, more than 500 drugs have been approved in previously forgotten conditions.

By definition, an orphan or rare condition affects fewer than 200,000 people in the US. The act paved the way for other regulatory authorities to follow, including the European Union, Australia and Canada, which similarly provides many benefits to orphan drug makers.

A recent analysis of the impact of the act from 2012-2014 by America’s Health Insurance Plan (AHIP), the US trade association of health insurers, found that drug prices for orphan drugs had increased 37% on average in non-orphan indications. This is possible as many medications approved for orphan conditions may also be used for on-label or off-label use to treat other, more prevalent non-orphan indications.

Monday, August 22, 2016

‘Too much weight placed on FDA approval of wearables,’ says UK health research expert

In a rapidly evolving digital landscape, the disruptive innovation of mobile technology, applications and so-called ‘wearable’ technology could be a complete game-changer for the healthcare sector. However, the potential for wearables and mobile apps to be used in clinical trials is raising all kinds of questions about legality, privacy and accuracy.

Matthew Simmons, Head of the Drug Development Unit at the Sarah Cannon Research Institute, believes that too much focus has been placed on the need for U.S. Food and Drug Administration (FDA) approval of these devices and applications.

“Clinical trials are based on analyses of often large numbers of patients, where trends and comparisons between groups are more important than an individual’s data. If you look at, for example, the heart rate on a particular day – the same margin for error applies to everyone, making it a ‘precision vs. accuracy’ problem that can be solved by statistics,” said Simmons, who will be a panellist on disruptive innovation at this year’s Partnerships in Clinical Trials Europe conference.

Friday, August 19, 2016

A patient's view of clinical trials - cancer patient and theatre maker Toby Peach (pt 2)

Watch part 1 of Toby’s story here

After being diagnosed with Hodgkin’s Lymphoma at the age of 19, Toby Peach was part of a clinical trial to test if ABVD could be given without radiotherapy. He now performs a theatre production - The Eulogy of Toby Peach – based on his experiences with cancer. We spoke to Toby to get a patient’s view of a clinical trial, which for him was overwhelmingly positive.

 Toby explains: ‘I was on a trial randomised to not having radiotherapy which was a blessing for me at that time…because I then didn’t have the scarring of radiotherapy on my neck.’

More broadly, Toby is well aware of the benefits of clinical trials, particularly for cancer:

‘The thing with clinical trials is they are good for the future - for the next generation who may be diagnosed. It is for us to carry on developing and improving everything that we can to ensure we give more people a chance to come through cancer.’

Watch the interview with Toby above, and catch up on part 1, where Toby shares his full story, here.

Toby Peach will join over 120 other industry leaders speaking at Partnerships in Clinical Trials Europe in Vienna on 16-17 November 2016. Find out more at

Thursday, August 18, 2016

The State of the Clinical Trials Industry | An Interview with Scott Sawicki, Director of Clinical Procurement Management, Novartis

In this exclusive interview, Scott Sawicki, Director of Clinical Procurement Management at Novartis, joins the Partnerships in Clinical Trials US team to discuss the current trends, biggest concerns, major disruptors, and the role of innovation in clinical trials - as a lead up to the event on October 5-7, in Boston.

Scott is a member of the PCT US advisory board and helped shape this exciting program that brings together the best curated content on clinical trials outsourcing, procurement, operations, and innovation as well as the disruptive technologies that will make your trials faster, better, and more cost-effective. Take a look at Scott's thoughts on the current state of the industry below...

Wednesday, August 17, 2016

Industry Trends: Clinical Trials & Development

Steven Whittaker, Executive Director, Avoca Quality Consortium and Senior Consultant, The Avoca Group joins the speaking faculty at the upcoming Partnerships in Clinical Trials US conference. The state of the industry is constantly evolving, and Steven sat down with the PCT US team to say a few words on the current industry trends, his biggest concerns, and what he feels the secret to long-term success is, prior to his talk this October in Boston.

Tuesday, August 16, 2016

PCT Europe 2016 Interactive Brochure Launch

As the word cloud above shows, research, pharma, patient, clinical and outsourcing are unsurprisingly some of the words that appear most often in the Partnerships in Clinical Trials Europe agenda for 16 - 17 November 2016.

We have now released the beautiful interactive brochure for the conference for you to explore the 120+ influential industry speakers and 450+ companies joining over 1000 attendees in Vienna.

Some of the highlights of the agenda include:
  • - 8 conference tracks.
  • - A choice of 5 new pre-conference focus days on The Outsourcing and Partnering Forum,  Patient Centricity, Budgets and Cost Modelling and Improving Communication and Managing Conflict.
  • - Professor Kenneth Getz from Tufts will highlight the major trends in the drug development and CRO landscape and their impact on outsourcing strategy.
  • Dr Mark Porter, a GP and media medical correspondent, will debate with a senior industry panel on current challenges in the clinical arena and new trends across the industry.
  • - A physician-scientist that is studying a deadly illness and battling it as a patient: Insights into the importance of collaboration in biomedical research with David Fajgenbaum, University of Pennsylvania and Castleman Disease Collaborative Network.
  • - Diagnosed with brain cancer at 21 and given six months to live, concert pianist and branding veteran Matthew Zachary is the Founder at Stupid Cancer, the largest charity comprehensively addressing young adult cancer.
  • - Fresh from Rio, Sir Steve Redgrave will depict the challenges of living with diabetes whilst becoming the only British athlete to win five consecutive Olympic gold medals.
  • - Toby Peach talks through his award winning theatre show The Eulogy of Toby Peach, which is a discovery of self-mortality and explores a difficult subject in a refreshing, insightful and humorous way. 

Monday, August 15, 2016

Being Careful What You Wish For

As my short bio at the end of this post says, I am a cancer mom, in fact, I am a two-time cancer mom which is quite rare in the world of rare things (pediatric cancers are classes as rare diseases, in case you didn’t know). Despite being told, perhaps as far as even assured, in 2006 that the cancer diagnosis of our first born son at only five-months of age, which claimed his life two months later, was random or ‘bad luck,’ it turned to not be the case. In 2014, our then seven-year old son was diagnosed with the same cancer.

Gene mutation

While the focus immediately following our son’s diagnosis was 100% on getting him treatment at the best hospital possible for his diagnosis (neuroblastoma), there of course was the lingering sick-feeling that we were not fouled by bad luck, but rather, bad genes. And so started a nearly 18-month long odyssey to solve our medical mystery.

Despite there being no shortage of genetic specialists and even relatively new genetic markers and blood tests, getting true attention onto this issue from our care team(s) took a lot of effort on our end. We were twice cautioned that sequencing his tumor would be costly and likely not yield any helpful or actionable information. We did not care. And ultimately this test did provide the clue that ultimately unlocked the mystery: a p53 mutation.

Friday, August 12, 2016

"If I wasn’t the one doing the fighting, who was it that saved my life?” Cancer patient and theatre maker Toby Peach's story (pt 1)

Toby Peach was diagnosed with Hodgkin’s Lymphoma at the age of 19. At the time he had no real idea of what cancer was or what happens when it is treated.

His experience gave him a new perspective on cancer and made him realise how misunderstood the disease often is: ‘It slowly became apparent that I wasn’t the one who had beaten this thing. When I realised that it was me fighting me – it was a rebellion, then who saved me? If I wasn’t the one doing the fighting, who was it that saved my life?’

From this experience he created a theatre production - The Eulogy of Toby Peach - that is part celebration of life, part exploration of the complexities of being a cancer patient. As he explains, ‘If one in two people are going to have cancer, should we not know what we are going to face? What is this thing we hear so much about?’

We spoke to Toby about his story and you can watch part one of the interview above. The second part is coming next Friday and explores Toby’s encounters with clinical trials.  

Toby Peach will join over 120 other industry leaders speaking at Partnerships in Clinical Trials Europe in Vienna on 16-17 November 2016. Find out more at

Thursday, August 11, 2016

Navigating the Maze of Development Technologies: Some Best Practices

by Frances Grote, Practice Lead, Clinical Sourcing Advisory, ISG 

Anyone who has recently attended an industry conference, browsed through a trade publication or, for many of us, opened an email might well feel overwhelmed by the proliferation of technology solutions in the drug development space. Parts of the development process have been relying on technology as their primary means of conducting business for some time now, but it’s beginning to feel like every activity you might think about doing has inspired the creation of a device or a digital solution. It’s a rare clinical vendor who doesn’t offer technology to support their services.

Although this rapid growth of options brings the promise of better and faster drug development, the extensive choices in the marketplace can also put sponsors at a disadvantage. Even the most cost effective technology solution is not cheap. In addition to the hard dollars that have to be invested to purchase or subscribe, there are substantial behind-the-scenes costs: systems integration, training and organizational change management, grandfathering ongoing work in legacy systems, and maintaining end-user support. And those are the costs if the application does what it’s supposed to do pretty much out-of-the-box. Investments of time, money and human resources can skyrocket if the technology solution is not yet as ready for prime time as everyone predicted.

Additionally, sponsors making a decision to invest in new technologies are required to be ever more sophisticated in their selection process. The playing field now includes non-traditional providers who are finding ways to bring their experience around cloud, digital and big data into the drug development space. And the lightning fast pace of innovation can make it ever more challenging to ensure adequate protection of patient privacy, data integrity and regulatory considerations.

Tuesday, August 9, 2016

Where’s the Buzz in Clinical Research?

By Norman M. Goldfarb

Technology is hot. Big — or just lots of — data, visualization, the cloud, investigator site files, patient recruiting, and eConsent are prominent. Wearable technology is treading water without more professional-grade products. The challenges of prioritizing, selecting, operationalizing and integrating technology remain.

Building relationships, improving communications, growing networks, broadening product lines, and collaborating in every direction — including among competitors — are major trends, albeit far from a smooth road. Consolidation — often in unexpected ways (e.g., IMS plus Quintiles, LabCorp plus Covance, WCG Group plus CenterWatch, ICON plus PMG Research, PPD plus Synexus, and BRANY plus CITI) — continues as the ultimate expression of these trends.

Consolidation in the IRB space appears to be largely complete, with six major players, but stay tuned as the NIH requirement for single review undercuts the rationale for local review of industry-sponsored studies.

Continue Reading

Monday, August 8, 2016

REPORT | Industry Voices: The Impact of Brexit on Clinical Trials in the UK

Six weeks on from the initial shock of the EU referendum result, the dust has begun to settle in Britain and industries across the four countries are starting to assess the situation they now face.

For those companies, individuals and bodies involved in clinical trials it’s likely to be a tricky break-up, for when it comes to clinical research, the UK and EU are deeply coupled. That’s clear from the pre-referendum voices of senior figures; GlaxoSmithKline CEO Andrew Witty for example: ‘Over the last 15 or 20 years, Europe has gone from 27 fragmented, independent, not-talking-to-each-other regulatory authorities in the healthcare space to one. That's a big deal.’ That’s clear also from the statistics; the UK has the second highest number of ongoing global clinical trials among EU countries, and in 2015 provided the most senior advisors to the European Medicines Agency (EMA).

However, over the last month and a half there has been an acceptance of the referendum result and many of the industry’s leading figures are already looking towards what the best moves are now. As Kieran Doran, Senior Healthcare Ethics Lecturer at University College Cork, rather theatrically puts it: ‘There are two schools of thought; either this is the end of the world as we know it, or this is a great opening that will present many exciting opportunities.’

In this report, leading industry figures assess the situation and suggest the best way forwards with regulations, movement of staff and new international partnerships.

Join the debate with over 120 influential industry speakers and 1000 attendees at Partnerships in Clinical Trials Europe in Vienna on 16-17 November 2016. Find out more here.

Friday, August 5, 2016

Increasing Patient Partnerships

"Patient partnerships are increasing in the clinical development/drug development processes. That's an evolution of the fact that patients are more astute and sophisticated. And what I've seen as having the biggest contributor to this is the internet. Back in the day, we didn't have the internet and people were calling the patient organizations to say, "Here is my diagnosis, what can we do? What is the current research? Is there a trial available?". And you had that opportunity to really engage and educate - while that still exists, it exists differently because of the internet. People are now able to get online and start that search for information themselves." said Jayne Gershkowitz, VP of Patient & Professional Advocacy, Amicus Therapeutics in a recent interview with the Partnerships in Clinical Trials team. 

She continued to say, "Patients will see who is doing research from the academic side, the industry side, they find the patient advocacy organizations and begin calling around. By getting the information at the early part of the journey, when they start to investigate on their own, they begin to become educated and empowered, taking things into their own hands.

The power of the internet has allowed patients to connect with others like them all over the world, through the online communities, social media, and developing their own pages - these reinforce the physical communities; i.e. conferences and meetings."

Continue Reading

Wednesday, August 3, 2016

Pharma "should be thinking about patients as ‘healthcare consumers’ instead of patients"

We spoke to Matthew Zachary, the outspoken Chief Angry Officer of Stupid Cancer, about how patients are treated in clinical trials

After surviving brain cancer as a 21-year-old, Matthew Zachary founded Stupid Cancer in 2007 to address the 72,000 new diagnoses of young adult cancer each year. It has since grown in to the largest US charity providing advocacy, research and support for the often neglected group of 15 - 39-year-olds.

We spoke to the self-described ‘global healthcare disruptor and Chief Angry Officer’ about how patients are currently treated in clinical trials and how that could be improved.

How well do you think the industry currently works with patients in clinical trials?


“90%of all clinical trials worldwide fail to enroll patients within the target amount of time and must extend their enrollment period.” Source: Transforming Clinical Research in the United States: Challenges and Opportunities

As an industry, the answer is a system-wide failure of goals vs. working with patients to achieve them. Either the goals are wrong, the way industry works with patients is wrong, or both.

Tuesday, August 2, 2016

WEBINAR: Is adaptive design the future of clinical trials?

In recent years, the development of novel compounds has slowed down, due in part to rapidly escalating costs and the increased complexity to get approval from Health Authorities. 

To reverse this trend, the Food and Drug Administration released the critical path initiative in 2004 and the critical path opportunity list in 2006. There is clearly a need for advancing innovative trial designs, and adaptive clinical trials is one of the solutions. Coupling adaptive designs with other initiatives for improving clinical trial quality and efficiency shows strong promise in addressing the challenge of improving R&D performance. 

Isabelle Naëije, Clinical Trial Head at Novartis, will be leading a free webinar on the benefits and limitations of adaptive design based on her own extensive experience. She will begin with a definition of what adaptive design actually means, before going on to describe the different types, their benefits and the concerns around them. 
  • When? Wednesday 10th August 2016 2pm - 3pm BST
  • Who? Isabelle Naëije is currently based in Basel, Switzerland. She is the Clinical Trial Head in charge of a Phase I-II study in the field of Renal Cell Carcinoma sponsored by Novartis, in collaboration with Merck. Before taking this role, she worked for 5 years at Actelion Pharmaceuticals Ltd, Switzerland as Project Manager in the Clinical Pharmacology department.

Friday, July 29, 2016

Patient Centricity and Improving the Clinical Trial Process

The Partnerships in Clinical Trials team were able to sit down with Jack Whelan for an exclusive interview. Jack will be joining the speaking faculty at the conference in October and is a very active research advocate determined to help improve the clinical trial process. Jack himself has participated in seven clinical trials, and is an eight year survivor of a rare incurable blood cancer. The unique patient perspective that Jack is able provide is one you won't soon forget. It is time for some fresh new thinking about clinical trials.

Continue Reading...

Tuesday, July 26, 2016

Effective Vendor Oversight: A Fundamental Element of Drug Development

By Frances Grote, Practice Lead, Clinical Sourcing Advisory, ISG

Of all the potential drugs being developed today, it’s likely no more than five percent will make it to market. On top of this high failure rate, complex protocols and stringent regulatory requirements--while necessary to ensure safety and efficacy—have greatly increased the burden of process, documentation and data collection. With an industry-wide spend of nearly $50 billion a year on outsourced services, almost all BioPharma companies depend on vendors to conduct some of their most complex and sensitive development activities. The operational and clinical expertise of those vendors is crucial to success.

Clearly, both sponsors and vendors care deeply about getting the maximum value from these relationships. But value is not the same thing as cost. Financial analyses can only give you one part of the picture. Sponsors also need to take a hard look at what they’re investing in vendor oversight.

For starters, many sponsors struggle with helping their internal staff make the transition from study management roles to performing vendor oversight. The ability to oversee outsourced activities in a way that delivers optimal performance while minimizing risk requires an entirely different set of strategies and skills than running a clinical trial. And once a sponsor internal team has the right training, they’re still going to need the metrics and leading indicators to proactively identify risk and reliably predict timelines and costs. Without these tools there is no way of knowing if the value being achieved meets the objectives that drove the decision to outsource in the first place.

Monday, July 25, 2016

WATCH: Partnerships in Clinical Trials Highlights

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.

Last year's event was a fantastic, with unique solution-oriented sessions designed to showcase real insights to foster real changes – unfiltered, unedited and unscripted. Plus, it was all under one roof – sessions, keynotes, roundtable discussions and exhibitors showcasing the latest and greatest clinical advancements.

A very big thank you to everyone who attended and participated. Did you make it into the highlight video? Try and spot yourself! And don't miss out on PCT 2016, October 5-7 in Boston, MA. Click here for more information about this year's event!

Friday, July 22, 2016

Ultra Rare Diseases and the Collaboration Between CROs & Pharma

At the last Partnerships in Clinical Trials in Boston, MA, we sat down with Mike Collins, VP of Global Clinical Operations at Alexion Pharmaceuticals for an interview. At last year's event, Mike presented a session "CRO and Pharma Collaboration to Meet the Need of Patients with Ultra Rare Diseases". Stemming from the topic addressed in his presentation, during this exclusive interview we asked Mike about ultra rare diseases, the treatment options, development of drugs, the research around these diseases and much more.

Every rare disease study is unique. Working with patients in rare diseases poses new and different challenges to both clinical trial sponsors and their CRO partners to ensure successful trial execution starting with the most critical factor – understanding the needs of the patient. Hear from Mike as he sheds light on how a sponsor and CRO can align efforts to successfully execute a global rare disease study.

Wednesday, July 20, 2016

Maximizing Sponsor/Provider Relationships for Longer-Term Success

Joining the Partnerships in Clinical Trials speaking faculty this October is Jason K. Casarella, SVP, Business Development & Marketing at Advanced Clinical. Jason was kind enough to sit down with the PCT team and address some of the major trends and the biggest challenges that are impacting the industry today, as well as how you can maximize sponsor/provider relationships for long term success.   

Monday, July 18, 2016

The Future of Clinical Trial Recruitment is Already Here

You would think from some of the discussions at this year’s DIA Annual Meeting in Philadelphia that the digital promise to clinical research – offering among other things, deeper patient insights, faster recruitment, greater retention and cheaper, quicker, more successful trials - was all a bit fanciful; an idea that would take decades to roll out.

Speakers at a number of different events (mainly from sponsor companies) noted the ethical, organisational and process hurdles to be overcome, including the need for much greater standardisation of data handling systems that study sites were required to use, for real progress to be made.

I was more encouraged, though, by other speakers and exhibitors at DIA who have seen the potential for innovative study design and operation, and are either implementing it themselves already or commissioning it from partners; from patient-friendly, online study materials, to web-based advertising, and relationship-building with patient advocacy groups – particularly those with an online presence. 

Friday, July 15, 2016

The Greatest Challenges Facing Clinical Research Development Today

Expert Opinion:
What is the greatest issue or challenge facing clinical research development today and how do you think it should be addressed?

"The single greatest challenge in the industry today is that the field has thought of itself as being commoditized" -Atul Butte, Director of the Institute for Computational Health Sciences, UCSF

The Partnerships in Clinical Trials team were able to sit down with many industry experts to get their professional opinion in regards to the greatest challenges facing clinical research development today. We were joined by;
  • Atul Butte, Director of the Institute for Computational Health Sciences, UCSF
  • Cathy Dawson, Director of Medical Scientific Affairs Group, Aesculap
  • Isan Chen, Chief Medical Officer, Mirati Therapeutics 
  • Jayne Gershkowitz, VP of Patient & Professional Advocacy, Amiscus
  • Mike Collins, VP or Global Clinical Operations, Alexion Pharmaceuticals
  • Gary Thompson, Senior Director of Data Sciences, Eli Lilly & Co.