Tuesday, August 25, 2015

Pharma needs to be bolder to be truly patient-centric

By Jack Whelan

I became interested in clinical trials and how study participants interact with the drug industry after I was diagnosed with a rare and incurable blood cancer called Waldenstrom's Macroglobulinemia.

At the time of diagnosis, all the literature and on-line information indicated about a 5 year outlook after treatment began.  I was told the only treatment options available to me were chemotherapies borrowed from other blood cancers.

But I felt exploring what was available in forward-looking science, specifically clinical trials of molecular targeting agents might be safer and more effective than conventional chemotherapy. 

Seven years on I am still here and I attribute this to clinical trials - I’m currently taking part in my seventh – and my focus on communicating with drug developers as an engaged, educated partner, more commonly known as an e-Patient.

As an experienced trial participant it is my opinion that the biggest challenge facing drug firms today is data collection.

Figuring out how to gather the information needed to prove a new therapy is safe and effective in a way that is timely and complies with the rules that govern clinical trials, particularly those relating to the patient, is difficult because of the out-dated systems, procedures and assumptions about novel targeted therapies.

Many newer targeted therapies generate an immediate response, yet because clinical trial data is reported in “batch mode” rather than in real time we often fail to identify benefits or anticipate emerging adverse events until it is too late.

Although there have been advances, like the use of mobile devices to report trial data in real-time, there remain various out-of-date regulatory requirements that slow the flow of information from patient to drug developer.

As an engaged outsider, I’ve seen far too many examples where privacy and data protection regulations, such as EU Data Protection laws and the US Health Insurance Portability and Accountability Act (HIPAA) regulations, restrict information flow between patients and biopharmaceutical companies. 

Thus I believe the opportunities for more meaningful, more in-depth epidemiological studies have been hampered by too much regulation.

To solve these problems, the industry needs to become much bolder. Rather than being risk averse and trying to satisfy the perceived needs of regulators, drug developers need to instead focus on the patient’s needs.

This most regulated of industries must educate regulators to eliminate many of the out-of-date assumptions and systems that no longer apply to modern medicines.   

Many of the shortcomings of current clinical research methodologies are directly attributable to out-of-date, policies and procedures that the industry follows to achieve compliance and to earn checkmarks on a myriad of now unnecessary check-lists. 

But the underlying goal of all clinical trials remains the same; to determine the safety and efficacy of a drug based on indicators collected from patients.

We urgently need more direct contact and information flow between patients and all those who bring us new therapeutics.

I’m honoured to speak at PCT-Europe on behalf of patients from around the world whose voices might not otherwise have been heard.

PCT-Europe represents one of the best learning experiences for me personally to strive to be a more effective, knowledgeable advocate. I also see the incredible networking opportunities and the vibrant exchange of knowledge and information between collaborators and sometime competitors for the benefit of patients. 

Finally, and again on a personal note, it’s fun to be able to remind life sciences professionals about the nobility of their work and offer a sincere thank you from patients everywhere.

Jack was diagnosed with a rare, incurable blood cancer which research then indicated a five-year outlook for symptomatic patients requiring treatment. Travel with Jack on an intensely personal and oft-humorous bumpy ride on the road to Personalized Medicine still under construction. Encounter some relapsed/refractory detours, conflicting signals and biomarkers, inhibiting regulatory roadblocks and potholes in the healthcare system.  Learn why Jack, not much of beer drinker himself hangs a bottle of Guinness on his chemo infusion rack and speaker’s podium. Using business communications and research skills from his career as a Wall Street Research Analyst in Information Technology (IT) and career Sales VP in IT, he now helps bridge the communications gap between life sciences, medical professionals and patients as a Patient Advocate, Research Advocate and Legislative Advocate.

If you are eager to hear more from Jack, then make sure to register for our upcoming event here

Friday, August 21, 2015

Clinical research: Part art, part science, all communication

By Michael A. Martorelli, Fairmount Partners

I’ve followed contract research for twenty years as an investment analyst and banker. I’ve seen the industry fragment and consolidate, shrink and expand and I’ve used these observations to advise CROs and other service providers of the opportunities and pitfalls.

The most important lesson I’ve learned is that having a clear aim from the outset is the key to success in any drug development project.

The goal of clinical research is to provide regulators with the information they need to make informed decisions about the marketability of new therapeutic products as quickly and efficiently as possible.

To conduct such research in the most effective way requires that sponsors understand the tasks they want their outsourcing providers to conduct empower them to carry them out.

For their part, contractors need to understand and complete their assigned tasks as quickly and efficiently as possible, hopefully on time and on budget.


To achieve these aims, sponsors and contractors need to be led by skilled and adaptable management teams that are capable of establishing the correct clinical programme for each investigative compound.

Clinical research is both an art and a science and its effective management requires leaders who strive for optimal success while being able to adjust to unforeseen difficulties.


The other key party in all effective clinical research is the regulator. It is important that regulators are on top of the scientific and medical advances on which new therapies are based.

In addition, regulators must demand that clinical trials are conducted to high standards while keeping in mind the need to assess the potential marketability of drug candidates as rapidly as possible.

It is also vital that regulators provide sponsors and their contractors with consistent guidance that makes clear their expectations.

Equally, drug developers and their outsourcing providers need to communicate effectively with regulators, remembering at all times that failure to so could result in poorly conceived, poorly executed trials that yield unpersuasive results.


Patients are the ultimate beneficiaries of effective clinical research and it is vital that sponsors, outsourcing providers and regulators share information.

PCT-Europe is an excellent forum for such interaction because it offers workers at all levels of all companies the chance to share experiences and find ways of dealing with common challenges.


By Michael A. Martorelli, Fairmount Partners
Mr. Martorelli came to Fairmount after serving as managing director of research and senior health care analyst at Investec Inc. and its predecessor, PMG Capital. Prior to PMG Capital, Mr. Martorelli spent more than ten years at Janney Montgomery Scott as a research analyst covering companies in various segments of the health care industry.


Tuesday, August 4, 2015

Challenging Industry to Create True Strategic Partnerships

By: Julianne Hull, CEO of WenStar

With outsourcing of clinical trials increasing at a rate of over 40 percent a year, it is critical that partnerships between pharmaceutical and contract research organisation (CRO) partners deliver real value and achieve the desired purpose. This trend has caused an increasing amount of clinical and operational expertise to be developed by CROs, but is this expertise being used to its fullest capability?

It is clear that at the highest levels in partnerships there is usually alignment, but it is questionable whether that alignment permeates through to the Quality Assurance, Operations, Purchasing and Data teams abound.

All to often, strategic partnerships are announced with a great fanfare, but after a few years most of them go quiet – the key question is “why, did they not achieve their original purpose?” Was too much time spent focussing on the partnership – and not on the outcomes they are designed to deliver that will ultimately lead to bringing new medicines safely to market?

There is almost no discussion about those partnerships that end prematurely, or those that are not renewed once they reach their original end date leading to a lack of knowledge sharing and ability to review the full extent of experience gathered.

These issues will feature prominently in the very popular Leadership and Change Management workshops as well as the Outsourcing and Partnership Forum workshops that will be hosted at this year’s PCT Europe in Hamburg.

About the Author: Julianne has worked in the pharmaceutical industry for 20+ years working for Marion Merrell Dow, Wyeth, Pfizer, Biogen Idec and now Ipsen. She holds a BSc in Biochemistry and an MSc in Molecular Virology (Queens University). Julianne has experience in leading clinical operations, clinical data management and vendor management/outsourcing groups globally. Julianne was instrumental in implementing the groundbreaking Wyeth/Accenture strategic alliance in Clinical Data Management. She has been working in varying facets of outsourcing since 1998 including tactical, preferred provider, functional and strategic alliances with responsibilities for governance, vendor outsourcing and operational delivery.

Tuesday, July 28, 2015

Free Webinar: Pharmacoeconomic Assessment through Market Approval and Beyond

Pharmacoeconomic assessment of a drug, medical device, or other healthcare product can take on many forms and occur at multiple points in the development cycle.  Cost-effectiveness analysis, a major component of pharmacoeconomic assessment, has traditionally occurred in the later phases of product development—either as a piggy-back to a phase III or pivotal clinical trial, or peri-authorization.

Join Medpace experts as they explore the application of pharmacoeconomic assessment throughout all phases of clinical trials as well as in observational studies, including registries and other post-marketing data collection.  Save your seat: http://bit.ly/1eucqeB

This discussion will cover topics including:

 ·         How pharmacoeconomic assessment can be envisioned in the context of early phase product development, implemented, maintained to support product marketing, and used to inform future decision-making.
·         Cost-effectiveness analysis at different phases in the product development cycle.
·         Pharmacoeconomic assessment in the post-marketing context.
·         Comparing and contrasting approaches for small/medium product developers relative to larger companies. 
·         Case studies on “real world” implementation.


Lee Walke, Vice President e-Clinical at Medpace, has nearly 25 years of progressive clinical development experience with expertise in collecting real-world outcomes and clinical data, data integration, endpoint derivation, electronic submissions, Electronic Health Records (EHR), and electronic Patient Reported Outcomes (ePRO).

Matthew J. Page, Ph.D., M.P.P., Epidemiologist at Medpace, has a diverse background in academia and research including teaching college courses in epidemiology and biostatistics and working with pharmaceutical and medical device companies to implement numerous pharmacoeconomic methodologies, including cost-effectiveness analysis and budget impact modeling.

Space is limited. Save your seat here: http://bit.ly/1eucqeB

Wednesday, June 10, 2015

Patient-Centered Trial Design: A Real-Life Example

By: Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

The increasing complexity of protocols can cause several inefficiencies in the clinical trials process, such as longer study cycle times, inadequate recruitment and retention rates, and more protocol amendments. Many see the need to simplify such costly and time-consuming trials, but how do sponsors actually achieve this?

At the 2015 Partnerships in Clinical Trials conference, Pablo Lapuerta, Executive Vice President, Clinical Development and Chief Medical Office of Lexicon Pharmaceuticals, presented a session titled, “How Lexicon Achieved Efficiency in Phase 3 with a Patient-Centric Study Design.” In this session, Lapuerta discussed how his company successfully implemented a patient-centric study design for its Phase 3 trial. This oncology study was for a serotonin synthesis inhibitor developed for the treatment of Carcinoid Syndrome, a rare tumor that secretes chemicals into the bloodstream, most often in the gastrointestinal tract or lungs.

Like most rare diseases studies, enrollment was a challenge. In the past, it has taken more than five years to enroll 400 patients with metastatic carcinoid tumors for Phase 3 trials (even with more than 500 sites participating). Lexicon decided to do something different for this study. Patients with carcinoid syndrome who screen failed the Phase 2 protocol would have a new opportunity to be eligible for the Phase 3 study that was designed to focus on the patient. It included the following features to support recruitment and retention:

·         Less frequent visits
·         Home health option
·         Support for travel
·         Short placebo-control period
·         Long open-label phase
·         Less procedures
·         Flexibility on treatment during open-label extension patient focus

In addition to a protocol design that reduced inconveniences for the patient, Lexicon also had an effective CRO partnership. Representatives from both companies visited sites repeatedly in a coordinated fashion, attended scientific congresses together, updated sites on study progress, obtained feedback from investigators on the protocol and patients, and shared best practices in study conduct and patient management.

By maintaining a focus on the patient throughout the trial, this unique Phase 3 study was a success. Lexicon saw both positive site feedback and significant patient interest. Though it still took two years to complete enrollment, there were over 200 total patients in the Phase 3 program. Taking a patient-centric approach means committing to making the patient the priority in choices related to clinical trial design. This includes keeping things simple, holding back on the “wish list” of procedures and measures, and appreciating the value of safety data collected in a setting that reflects actual practice.

Have you been part of a study that incorporated a patient-centric trial design (from protocol design to study conduct)? How did it go?

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina served as the guest blogger covering Partnerships in Clinical Trials 2015. 

Tuesday, June 2, 2015

Life Sciences Shifting Quickly: Is Your Talent Supply Chain Ready?

By Kevin Duffy

When it comes to the emerging trends in human capital management within the Life Sciences sector, senior business leaders are anxious over the competitive landscape and the shrinking supply of skilled talent to help drive their R&D pipelines. What is less understood is that having a strong workforce plan that includes external talent (temporary workers, independent contractors/freelancers, service providers, retirees, etc.) can make or break a company’s ability to achieve its strategic goals. Though the full-time labor force still accounts for the majority of the world’s workers, those numbers are shifting quickly.  Comprehensive plans must re-balance the talent portfolio to account for the opportunity, risk, and cost factors that come with the external workforce.

A contributing factor to this dynamic is that Biopharmaceutical and Medical Device organizations are primarily clustered geographically in the Northeast, mid-Atlantic and northern California regions.  This environment creates incredible competition for talent and places additional pressure on organizations to find the right talent to fit key roles.  Qualified talent is fully aware of the position this creates which can result in movement from role to role, based upon their economic drivers.  To that end, meaningful retention then becomes the issue as talent is being traded back and forth.  To add to the complexity, the capital influences within this industry also have a strong impact on the availability of talent within your supply chain.  It becomes both disruptive and polarizing as we see more mergers, acquisitions, and divestitures on the near horizon as companies position their financial strategies going forward. 

It’s a complex and high-stakes undertaking within this industry therefore forecasting and understanding the need for all talent types is imperative. It’s likely that you already have a higher percentage of external talent then you realize conducting important work on your company’s behalf.  Whether it be in R&D, working in the lab, or positioning you in the marketplace, these individuals can often times form the backbone of your operations.  They are in your talent supply chain, but they’re independent contractors, consultants, and small firms whose people don’t work for you directly. Instead, they essentially act as “companies of one” working for themselves and lending their talents on a series of short-term engagements.

That’s the reality of the new labor dynamic: Modern companies have to adapt to a more flexible, far less permanent definition of work. In doing so, they also have to completely rethink how they acquire, deploy, and engage their workforce—both external and full-time—for maximum business impact.  Companies are now evaluating their legacy policies around how to incorporate external workers into core company processes, such as learning and development, strategic workforce planning, and the like.  This growing reliance on harnessing the power of the entire workforce prompted KellyOCG to sponsor the latest research from Harvard Business Review Analytic Services.

Not surprisingly, more than 70 percent of those surveyed said their organizations are already using the external workforce to meet market demands and maintain efficiency. But more than half also said that using external workers allows them to bring in expertise that their full-time staff lacks, and that non-FTE workers will become increasingly valuable to their organization over the next two to three years.

While this approach may have been a nice to have in the past, it is becoming even more relevant and vital to the health and stability of leading Life Sciences organizations.  By understanding and engaging multiple channels for talent within your supply chain and having a strong grasp on your long term talent agenda, you will move to the front of the competitive landscape to lead the industry’s growth.

To read the full research paper sponsored by KellyOCG on this topic, click here. 

Kevin Duffy is vice president of Global Solutions and Industry Vertical Leader for Life Sciences at KellyOCG.  He has over 30 years of experience in the Healthcare, Clinical Research & Drug Development industries.

Wednesday, May 27, 2015

Show Me Your Data: How Transparency is Driving Drug Development

By: Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

The pharmaceutical industry is in an era of openness and collaboration with the transparency of clinical trial data at the forefront. At Partnerships in Clinical Trials last month, several presentations addressed the data sharing movement.

Inefficiencies in clinical research

There is so much data out there; the struggle is getting more eyes on it. We sit around and complain about how much drug development costs, yet we’re not using what’s already available. We only ever analyze 14 percent of the data from clinical trials and almost always end up starting from scratch with each new study. Throwing away this incredible resource is both expensive and time-consuming. Why not make use of what someone else has already done?

Who benefits from data sharing?

There is a challenge around analytics, as each stakeholder finds different data meaningful. Those who share their data aren’t necessarily the ones who benefit. If a pharmaceutical company shares their data, it’s most often the researchers who can swoop in and do something with it. Being able to combine data from multiple sources enables them to draw new connections. So, who owns the data? It’s curated by sponsors, but is raw data a pubic asset? Sharing the results from clinical trials can benefit the patients who participated. This can help the experience come full circle for them when they can see what resulted from their contribution to the study. Francis S. Collins, NIH Director said, “We owe it to every participant and the public at large to support the maximal use of this knowledge for the greatest benefit to human health.” After all, without patients we wouldn’t have any data.

Should everyone be granted access to clinical trials data?

It seems as though the thought of data being made public is uncomfortable for many people. A large concern is the lack of control. There’s ambiguity around what will be done with that data, and there are serious consequences if data are misinterpreted. When it gets into the hands of the public, it can get very confusing. There will be enormous amounts of noise and contamination, so we must help people understand what data are usable and worthwhile. The industry may consider requiring that the person downloading the data sets show their plan for analysis, so it’s not just a data mining exercise.
Learning from failures

Clinical trials are the most expensive experiment in the world. Half of them fail, and we never release the trial information. Those failed molecules are sitting on the shelf gathering dust. For a molecule that worked in only a quarter of patients, instead of thinking of it as a failed trial, we should be paying more attention to the fact that it was spectacularly effective in that small subset of patients. Sometimes, all it takes is one part of a failed molecule to be reinvented with great results. This wealth of data can help companies to fail and succeed faster.

The drug development landscape as a whole can benefit from data sharing, as there are many downstream benefits. Yet, there are still things we’re figuring out with data sharing. For example, what’s the metric for success – the number of trials we get online? The number of times the data is accessed? Is there value in having these things done in the first place? Time will tell as the practice of data sharing becomes more common, and we’ll realize the value and utility in being more transparent with clinical trial data.

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina served as the guest blogger covering Partnerships in Clinical Trials 2015. 

Friday, May 15, 2015

Source: The website of the National Cancer Institute (http://www.cancer.gov)

Big Data in Clinical Trials: The Opportunities and Challenges

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

At Partnerships in Clinical Trials last month, there were several sessions that hit on the theme of big data and the potential for realizing what truly lies in the vast amounts of data sets from clinical research. The conference was host to several discussions around the opportunities and challenges in harnessing and analyzing existing data.

One session, titled “Can Big Data Cure Cancer?” was a conversation between Atul Butte, MD, PhD, Chief of the Division of Systems Medicine and Associate Professor of Pediatrics, Medicine, Stanford University and Professor Brendan Buckley, Chief Medical Officer, ICON plc. In this talk, an interesting discussion point came up on the role of CROs in the big data revolution.

CROs are sitting on tons of data. Yet, the general belief and practice has been that once the trial is over, the data is invaluable and goes straight to the attic. Most CROs’ spending on R&D is probably close to $0, but for those that see the results of tens of thousands of trials, they could potentially help predict the next best design. This can even be a competitive advantage for them.

CROs may be one way to help achieve better analysis and metrics, with the potential to provide benchmark data on things such as the average time it takes to recruit patients, patterns for lab tests, how long trials took, etc. across sponsors. Of course, there are some concerns, such as ownership as the data belongs to sponsor, the CRO is bound by contract, and relationships with sponsors may be very transactional. However, overcoming these challenges can open the door to a new realm of possibilities. Whether using this example or another opportunity, Butte challenged the audience to use their data to disrupt their own organization and reinvent their business.

In a separate session, titled “Big Data, Big Patient Impact,” the moderator polled the audience, asking what the top big data challenges are within life sciences. Among the top three answers were deciding what data is relevant, lack of big data analytics/scientific staff, and determining the appropriate technology.

The panel discussed that before we can curate and analyze data, the first step is to aggregate the data together since it resides in disparate systems and come from different sources. With a diversity of files and large amounts of multi-structured information, having it all in a standardized format opens it up for further analysis. After all, the data doesn’t make sense unless it all comes together.

In addition, pharma needs to be able to detect small signals and subtle anomalies in these huge sets of data. Seeing the need for a new kind of role, pharma can look to other industries that are more sophisticated in this area. For example, when it comes to fraud detection, banks go through the data available from every single customer at every single location to be able to know when those small signals and subtle anomalies are significant. As we work toward advancing medicine at a faster pace, pharma must better utilize its greatest resource, data, and continue to evolve in this area.

Looking forward to what we can actually do with this data – from understanding why previous protocols failed and acting on already existing data sets to sharing negative trial results – relates to another well-covered topic at #PCTUS: data transparency, which I’ll cover in the next blog post. Stay tuned!

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina served as the guest blogger covering Partnerships in Clinical Trials 2015. 

Friday, May 8, 2015

Patients as Partners – What are the Future Implications of Patient Opinion Leaders on Clinical Development?

By: Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

Patient advocacy is growing in importance, but resources are limited and the shift towards it is not happening fast enough. To talk about how we can accelerate the pace at which it is moving, Partnerships in Clinical Trials offered a keynote panel titled, “Patients as Partners – What are the Future Implications of Patient Opinion Leaders on Clinical Development?” moderated by Jayne Gershkowitz, Vice President, Patient & Professional Advocacy, Public Policy, Amicus Therapeutics. The panel featured two patients, Kaamilah Gilyard, a Lupus clinical trial participant and Alliance for Lupus Research advocate, and Jack Whelan, a cancer survivor and seven-time clinical trial participant.

The panel discussed how patient advocates can better the industry by discovering patients’ unmet needs, creating a feedback loop, designing better experiences for patients, and raising awareness for new clinical trials. It became obvious that the industry can still do a better job of leaning on patient advocacy groups and organizations that have patient advisory boards. They know their patients, including who’s involved and who’s doing what, and can be a valuable resource to point the industry in the right direction. Patient organizations can be the most trusted resource for disease-related information, even over physicians, and thus the key for successful enrollment in clinical studies.

Patients want more communication

Patients want to be involved and provide feedback. The industry must meet the patient where they are and have someone there to answer questions. They want clinical trial content in lay terms – from informed consent forms to study summaries – in language that they can understand. Patients want to speak and to be heard. Sponsors should find out what’s important to them and reflect that in trial design (and then provide them with the findings from the study). Also keeping cultural sensitivity in mind, it’s important to know how to talk to people in different ways and where to go to find them.

The patient perspective on why more people aren’t participating in clinical trials is simple: 95 percent don’t participate because they were never asked. In addition to physicians being disconnected, there are definitely still many myths and misconceptions around clinical studies. Often, people will think of them only as a last resort option, when in reality they’re the first resort for many people. Patients can be a very influential source for increasing awareness. It’s not uncommon for people to be willing to enroll in a trial after hearing a past trial volunteer speak about their experience (in both formal and informal settings).

An audience member expressed his disappointment in the number of empty seats in the room for this keynote panel. For a conference about partnerships, not enough people are considering the patient as a partner. Yet, it became apparent that now more than ever is the time for pharma to embrace patients and patient advocates. The partnerships between patients, physicians, and industry must be strengthened, so together they can collaborate for a cure.

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina served as the guest blogger covering Partnerships in Clinical Trials 2015. 

Monday, May 4, 2015

Starting With a Blank Slate, How Would You Shape an Ideal Partnership?

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

During Partnerships in Clinical Trials last week, a keynote panel titled, “Starting with a Blank Slate: How Would You Shape an Ideal Partnership” featured a group of senior leaders in clinical outsourcing. At a conference about partnerships, it was very fitting to hear them talking about how they would shape an ideal partnership if they could start from scratch. The panelists took a step back to reflect on how they would create an ideal outsourced relationship structure, knowing what they know now.

It starts with alignment on being clear what the problem is that you’re trying to solve. It won’t work if you have a solution yet no understanding of the problem. You must look at the specific problem you’re trying to solve and collectively build a solution around that. One panelist said one of the greatest challenges lies in how to get the best out of the best people to achieve extraordinary outcomes for patients. Another said the thing that stands out is the ability to work together. They must celebrate each other’s strengths and experiences, and collaborate in a way that’s focused on the end goal.

There needs to be a high degree of trust and integration, relying on your partner’s expertise, leveraging what they do best and operating in the most efficient environment. It’s unsurprising that the marriage analogy was use repeatedly throughout this session. One of the panelists joked that you realize there’s probably greater advantages than disadvantages to the partnership, filled with both good times and bad times, and while there are things you’d like to change, they are mostly out of your control. These partnerships are not measured by how everything is going when everything is going well; it’s how things are going when there are challenges. Like any successful marriage, you must work together towards a common goal. The weak ones will get divorced, as there is a lot of churn in industry. When things go wrong, it’s not just one partner’s problems – it’s both – in sickness and in health.

It’s hard to functionally outsource something that you’re not currently doing yourself. It comes down to what activities within larger companies are truly core and strategic in nature that no one else can do better. It’s those that should be kept within the walls of the company. Those that are critical but not core can be given away. You also need to be expert in your own business. If you fail to maintain the expertise, you invite risk. There’s no one solution and it will vary company to company, but it’s crucial to identify goals and objectives and be aligned with your partner.

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina served as the guest blogger covering Partnerships in Clinical Trials 2015. 

Tuesday, April 28, 2015

Examining the Clin Ops Tool Belt: A Discussion on EMR and eTMF

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

During the final day of #PCTUS, in the session, “What’s in Your Clin Ops Tool Belt? Weave New Technology into Clinical Development” a panel discussed different systems that optimize and affect clinical operations, revolving around two in particular: electronic health or medical records (EHR/EMR) and electronic trial master files (eTMF). Moderator Joe Popowicz of Emergent Clinical Consulting facilitated a panel made up of Manny Lazaro of Forum Pharmaceuticals, Catherine Dawson of Aesculap, and Elizabeth Brooks of Decision Driver Analytics.


The panel first discussed the advantages and disadvantages of EMR. Utilizing it for site feasibility and patient recruitment can be useful, as sites can figure out how many people they already have in this system with a certain condition, and can even use it for planning (e.g., find hot spots in the country where certain procedures are done). Yet, there are inconsistencies across hospital in codes and definitions that could really skew results.

The next issue the panel looked at was access to EMR. The panel’s experience seemed to be that about half of sites let monitors go in and access their EMR. Getting monitors access to medical charts – whether they’re certifying copies, or the CRA is sitting next to the CRC working through it would be ideal, but if institutions can’t set it up so that the CRA has access to isolate specific patients, they won’t give them access at all.

Another realization is that EMR is only as good as the data that’s entered into the system. There can be many inconsistencies, incorrect codes, errors, missing info, etc., and it would be dangerous to rely solely on it. There is an obvious need for interoperability between EMRs. We’re victims to the fragmentation of our health care system, as each hospital is so different. It seems there are as many limitations as there are opportunities at the present moment, but with a lot of refinement, we can tap into EMR.


eTMF offers access to do ongoing QC there, and there are obvious benefits to this over having the CRA jump on a plane to spend three days sitting in a dark room going through stacks of documents. It’s helpful for the sponsor – using less resources and time on-site and helps with transparency between sponsor and CRO, but the site’s experience is not enhanced. The general concensus was that eTMF is good during the course of trial, but when the call comes in for audit, you might as well just hit the ‘Print’ button. It ends up being counterproductive if you have to print out the entire TMF for any reason.

These technologies will never fully replace the human factor, but have the potential to support and improve the way we run trials, so long as they’re being used as intended. What’s true with any system is garbage in is garbage out, so adoption and proper training is crucial. These technologies will come and go, so it’s interesting to think what the next tool in the belt will be.

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems​. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina is currently the guest blogger covering Partnerships in Clinical Trials 2015. 

Monday, April 27, 2015

The Ethics and Implications of Compassionate Use (And People Still Don’t Understand the Drug Development Process)

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

The general session, “Fast Tracking Clinical Development in a Moment's Notice: Patient Advocacy, Social Media and the Implications of Compassionate Use” featured Kenneth Moch, former CEO of Chimerix, giving his firsthand experience in dealing with expanded access to investigational drugs.

He recounted the story of Josh Hardy, a young boy who was refused treatment to his company’s experimental drug. Outraged by this, the story was taken to social media and picked up by angry reporters from around the world. As the story blew up, Ken received death threats, and it became clear that people don’t understand the drug development process. He cited an example of someone who thought all patients have a fundamental right to experimental medicines after Phase 1 is complete, because that’s when the sponsor knows all of the effects of the drug (and phases 2-3 are just to make the FDA happy). There are right to try laws which vary by state and give terminally ill patients the right to have access to investigational drugs, but this became a case of compassionate use. The outcome was the FDA helped craft a novel solution: a phase 3 trial that enrolled the boy in this story to the trial within days.

Because the outcry from the social media campaign was so loud, was this a case of special treatment? The ethics are complex, but making exceptions is not ethical. Once the door is opened, you can’t say ‘yes’ to this patient and ‘no’ to everyone else. What would you have done if this boy was your son? Of course, we all want to save the child, but we also want to save people in the future. There are no simple answers to moral dilemmas. The good news is that the boy in the story is alive today, but what would have happened if he died or had a rapidly negative response to the drug? Because there was so much attention on this story, if that was the case, it’s very possible that potential future patients wouldn’t want to enroll in trials.

Ken believes that we as an industry continue to fail to inform the public on how complicated drug development is, and we need to do a better job educating the public now – not as an aftereffect. His experience in dealing with the “mob” of ignorant reporters and ill-informed citizens was done reactively, which he stressed is too late. Rather, we must take a proactive approach to explaining the complexities of the clinical trials process, requirements, regulations, etc. so those who are not in the industry can get the fundamentals right.  

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems​. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina is currently the guest blogger covering Partnerships in Clinical Trials 2015. 

Friday, April 24, 2015

Live from #PCTUS: Wall Street State of the Industry Forecast for 2015

The final session of PCT 2015, "Wall Street State of the Industry Forecast for 2015 - Taking a Closer Look into the Sustainability of the Pharma/CRO Outsourcing Model" was a panel discussion to dive into the state of the industry and diagnose key themes, trends, and challenges.

Moderator: Michael Hay, Managing Director, Sagient Research

- John Potthoff, President and CEO, Theorem Clinical Research
- John Kreger, Partner and Healthcare Equity Research Analyst, William Blair
- Michael Martorelli, Director, Fairmount Partners

One major factor that each panelist mentioned was big data. This comes from all different levels - one key to data capture was as more companies become public, it provides the industry with much more information (growth, profitability, etc..). This information leads us to better decision making across the board.

The discussion continued onward to discuss the challenges that are currently facing this industry...

What needs to change in the industry:
- The challenge is how to keep up with evolving technology
- Clinical trials need to be much more embedded into the healthcare delivery system
- Process inefficiencies

What is the current state of the industry?
- Biotech companies are on FIRE for public investors
- Nontraditional services companies are buying traditional service companies
- Data is king

Thank you for following along with 2015 Partnerships in Clinical Trials! We hope to see you next year in Boston.

Live from #PCTUS: 2 New Patient-Centered Initiatives Announced

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

In some of the sessions that I happened to attend, I was among the first to hear the announcements of two new and separate initiatives to better understand potential clinical trial participants. Both of them strive to address what’s unique by disease, rather than grouping everyone together in one giant all-encompassing patients bucket. They also intend for this information to be used in designing more patient-centric trials.

In the Patient Engagement in Clinical Research track, during the case insight session, “Late-Breaking Results from the Front-Lines of Trial Design: How are Patients Really Engaging in Research?”, Jeremy Gilbert of PatientsLikeMe gave an update to some of his company’s endeavors.

Surveys have been great for generalizing how patients feel about certain things, but Jeremy provided the audience with examples that show just how great patient preferences vary when you break down the answers by disease. Realizing that we need to better understand each group’s unique set of needs and preferences, Jeremy announced that he has teamed up with Ken Getz to create an on-demand patient-centricity resource center, which will be available in the near future. Jeremy said they plan to publish as much of this data and make it more accessible. Getting this information into the hands of people who are designing trials will enable them to learn how specific patient populations think about their disease.

Next, in the Clinical Operations Optimization track, a session by the name of “The Patients 2 Trials Consortium: Breaking News” featured four speakers. Three of them, Lani Hashimot of Novartis, David Leventhal of Pfizer, and Katherine Vandebelt of Eli Lilly, gave back story to the Patients 2 Trials Consortium that brought these three big pharma companies together who share a common struggle – matching patients to trials. Realizing that it does no good for a patient to get matched to their company’s trial if the trial doesn’t help the patient, they knew that by partnering sponsor to sponsor, they could bring valuable clinical trial information to patients.

The consortium agreed on target profiles that define and normalize groups of patients who might enroll for a specific trial. Pablo Graiver of TrialReach, engaged with the consortium to create an infrastructure based on their standard set of data. The result is TrialReach (which is live as of 24 hours ago).

Pablo gave a live demo of TrialReach. One by one, the user answers questions, first by condition, then location, age, etc., and as they answer each question, the list of available trials within X miles of you is narrowed down. By asking simple questions, the algorithm reads eligibility criteria and continuously reads, ranks and refines trials to display results in plain language. The results of trials are split by patient-friendly information (more than what’s available on CT.gov) with the logistics of the trial (specific procedures, number of visits, etc.).

Patient profiles are saved searches, and they have a plan for making this resource accessible to patients – by embedding it on disease-specific websites (where those potential patients already are online). In these instances, people can use the tool outside of trialreach.com, without having to leave the site they’re on. All searches, even from the embedded locations, generate the same snippets of data. Sponsors can the use these queries to show maps of matches and the information from the specific population to design better trials.

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems​. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina is currently the guest blogger covering Partnerships in Clinical Trials 2015. 

Live from #PCTUS: 3 Approaches to Speeding Up Clinical Trials

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

In today’s Clinical Operations Optimization track, a panel discussion titled, “New Approaches to Speeding Up Clinical Trials - What Works and What Doesn’t” was a great challenge/solution session. Craig Coffman of Nektar Therapeutics, the moderator, started the session by emphasizing that running a trial according to original timelines is hard work. Most trials operate in catch up mode, so rather than being ahead of the curve, they’re just trying to stay on the curve. It’s very easy to fall behind, and very difficult to make up any time that’s been lost.

This presentation featured three speakers who gave examples of what they’ve implemented with the goal of saving time on clinical trials.

Making it more convenient for the patient

Charles Drucker of Quest Diagnostics gave an example of a trial that used a phlebotomist service for blood draws. These storefront centers were quick and convenient for the patient and could save the trial time and money. Of course, this method works better for some therapeutic areas than others and isn’t a one size fits every study.

Visiting sites to show sponsor support

Manny Lazaro of Forum Pharmaceuticals talked about taking the time to actually visit and engage with the site (not at the investigator meeting). He’s traveled as far as Serbia to provide support and show that as the sponsor, they care about the site and want to be involved, even when the trial is outsourced to a CRO. Does showing up in person really work? These visits almost always lead to increased site engagement and show an immediate upswing in activities (e.g., 10%-15% boost in recruitment), though they eventually tend to level out again. The sponsors in the room all agreed it’s worth the resources that are required to support site engagement.

The group even talked about sponsors holding “rejuvenation meetings” in which they bring together sites (both underperformers and high enrollers) to learn from each other and have them take time to pause and reflect on what’s gone right, as well as discuss any problems they’re having with getting patients in and consented. Sites appreciate this approach, as opposed to what they’re used to hearing, “When are the patients coming?” over and over again.

Setting goals and committing to bold experiments

Terri Roberson of Abbvie engaged the room with an experiment her company did, called “40 sites in 40 days” which took place between Thanksgiving and Christmas last year. The goal of this challenge was to enroll 40 sites and have them up within 40 days. Having previously taken 90 days on average to get a site up, this was a lofty goal. To save time, they did all feasibilities over phone and offered no contract negotiations. It was a “take it or leave it” offer, where the sponsor was saying we’re committed to you, you need to let us know if you’re committed to us.

The results? All 40 sites were up within 40 days, and they enrolled 100% faster than the non-experiment group of sites. Terri explained how internally they had to get a lot of people behind this effort, which ended up being a lot of work, but a huge accomplishment that really worked for them.

Craig challenged the room not to try just one of these examples, but try a combination of all three, yet recognizing that every situation is different and there’s no one silver bullet to speeding up clinical trials. One of the greatest realizations of this session was that the example methods presented weren’t due to any great new technologies – they were process improvements.

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC at Forte Research Systems​. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina is currently the guest blogger covering Partnerships in Clinical Trials 2015.

Live from #PCTUS: How to Get the Most Out of Your Clinical Data Collection Practices

Linda Tedder, Director of US Clinical Project Management, DePuy Synthes talked with the PCT audience in the final session of the day for the "Best Practices in Medical Device Trials" track. Linda brought a wealth of knowledge on clinical data collection and walked the audience through a step-by-step best practices guide based on her extensive work in the field. Below you will find the key areas to focus in on and questions to raise to ensure the best outcome when planning your very own trials...

What are you required to collect by regulatory requirements?
- FDA, clinicaltrials.gov, Competent Authorities, Notified Bodies, etc...

What do you need to collect regarding you product for safety & effectiveness/performance?
- AEs, SAEs, UADEs, USADEs, device malfunctions, product complaints
- Validated therapeutic measurement tools
- Imaging
- Publications

What additional data are you collecting for costing benefits or because you are worried you might need it later?
- OR times, hospital stay time
- PT requirement/amount
- Return to work status
- Non-validated PROs or therapeutic specific measurement/evaluation tools
- Overall patient satisfaction

CRF Development:
- How will the steps of the study/visits be done at the sites?
- Who will be doing what for the study?
- How will the data be collected?
- What is the evidence strategy?

Opportunities for Improvements:
- Limit free text; use drop down lists or check boxes
- Eliminate duplicate/similar questions, avoid a query cycle
- Allow sites to enter values then select measurement used
- Use dynamic forms in EDC

Data Entry & Collection:
How much data are you collecting?
- Have a plan for how the data will be used

How to avoid pitfalls?
- CCGs; having a usable CRF completion guidelines/instructions
- Site training; SC at SIV & ongoing
- Re-evaluate the database as needed/make changes (poor programming with excessive queries = frustrated sites!)

Other areas to think about:
- Linking vendors & EDC, does it make sense? (ex. core labs, IVRS/IWRS)
- Ensuring ongoing contact with sites for ensuring timely entry

How to ensure data is accurate?
- Program up useful edit checks/auto queries
- Thorough database validation through; completion of multiple test patients, interactive database review, and involvement of clinical operations
- Ongoing data cleaning

- Consider overall SDV requirements & risk-based monitoring
- Provide source worksheets
- Onsite vs. remote monitoring
- Ongoing collaboration with Data Management & CRAs/PMs
- Ongoing quality metrics

Thank you for following along with PCT 2015 & stay tuned for more updates and recaps from Partnerships in Clinical Trials.

Join the conversation on Twitter by following @PartnershipCROs and tracking all on site activity with #PCTUS

Thursday, April 23, 2015

Live from #PCTUS: Picking up the Pieces of Clinical Trial Failure

"From Innovation to Market: Navigating the Mine Fields"

Dr. Joseph Gulfo, Executive Director of the Rothman Institute of Innovation and Entrepreneurship at Fairleigh Dickinson University famously battled the FDA and won over a device in the late 2000s and early 2010s. His product was a melanoma detection device that would better identify melanoma in patients and effectively save more lives. This specific case is famous for the press it got and Dr. Gulfo was here today to explain to the PCT audience his battle plan and how he won the war.

Battle Plan:

·         Build up of arms - peace through strength
o   FDA decisions involve more than your product factors you don't know about
o   Regulatory counsel 
o   BPA; Binding Protocol Agreement (with the FDA) 
o   Money ( He raised nearly 160 million because of the BPA that eliminated regulatory risk for investors)
·         Strategy - sweating every detail
o   AdComm Panel briefing document –
·         Executive summary
o   Best, most knowledgeable doctors and statisticians in the field (mock panel)
o   Adaptable - change according to conditions on the ground
·         Realizing you are in a fight
o   Passion, dedication, determination
·         Define yourself by the outcome
o   Take it to Madison Square Garden (as much PR as possible, the FDA will win in a street fight - the media is your friend) 
o   Unconventional tactics - media and regulatory strategies (citizen petition) 
o   Be a leader, a powerful leader

Thank you for following along with PCT 2015 & stay tuned for more updates and recaps from Partnerships in Clinical Trials.

Join the conversation on Twitter by following @PartnershipCROs and tracking all on site activity with #PCTUS

Live from #PCTUS: Redefining Clinical Trials in the Age of Personalized Medicine

In this morning session, Donald Jones, Chief Digital Officer at Scripps Translational Science Institute led a thought provoking keynote panel discussion titled, "Redefining Clinical Trials in the Age of Personalized Medicine". Donald was joined by Isan Chen, Chief Medical & Development Officer at Mirati Therapeutics, Erynn Gordon, Medical Marketing Director at 23andMe, Niven R Narain, Co-Founder, President & Chief Technology Officer at Berg Health, and Wendy Levin, VP of Clinical Development at Fate Therapeutics, Inc. These industry leaders went back and forth discussing the future of medicine in regards to personalized medicine, their work, and where this is leading us.

Below are the key takeaways from this discussion...

The concept of personalized medicine makes one think that it should be patient specific. However, it can also be about the whole. According to global epidemiology, 600,000 new cancer cases are diagnosed every year and as baby boomers reach retirement age, this cancer explosion is expected to continue to rise.

With the data about expected cases readily available, how do we stop it? Since disease does not happen overnight, it is about identifying the patient population and understanding the full story; everything from sleep patterns, lifestyle choices, exercise history, area of residence, etc... Fundamentally, this collected data would be used to create a map on each and every patient - much like a map of airline routes. By developing these molecular maps, we can identify what is governing the healthy and correspond that with the disease population in order to identify where things have gone array.

So the goal now begins to shift towards, patient prediction. The model needs to be changed so we are no longer reacting to symptoms, but shift the paradigm to being proactive to improve overall health. Erynn Gordon used a specific example from her work at 23andMe and said; A family with a history of pancreatic cancer all seemed to have a specific genetic variant - in their massive genetic database, 127 others carried the exact same variant. 23andMe surveyed these 127 individuals about their family history of pancreatic cancer, cancer in general, and more (as well as 10,000 others as a control) and overnight were able to conclude that the specific genetic variant that this family carried had no connection to pancreatic cancer.

Tests/work like this are helping us shift towards preventative treatment rather than treatment to curve the diseases progression.

The final point brought up what by Wendy Levin and it was about comparative oncology; which provides opportunities to include naturally occurring cancer models in the study of cancer biology and therapy. The comparative oncology tests are done with companion animals with naturally occurring diseases rather than mice who have the diseases artificially implanted - there are 72 million companion animals in the US and they are susceptible to these naturally occurring cancers, and we want to cure these diseases in our pets. In that treatment process, we incorporate the success rates within the samples into new drug development and clinical trials.

Thank you for following along with PCT 2015 & stay tuned for more updates and recaps from Partnerships in Clinical Trials.

Join the conversation on Twitter by following @PartnershipCROs and tracking all on site activity with #PCTUS

Live from #PCTUS: Siteless Studies, Barriers to Patient-Centered Trials, and Recruitment for Rare Diseases

By Kristina Lopienski, Product Marketing Manager, Forte Research Systems, Inc.

Wrapping up today’s preconference activities were the Think Tank sessions. Attendees split up in open roundtable discussions to brainstorm solutions to industry challenges. In the Patient Engagement in Clinical Research track, the three topics up for discussion were “Overcoming Regulatory Barriers to Execute Patient-Centered Trials”, “Patient Recruitment Hurdles in Rare Disease Indications”, and “Exploring Siteless Studies”. Each group discussed one topic at a time, and then reconvened to share highlights and key findings.

It was fascinating to hear these topics being discussed from different perspectives, including sponsor, CRO, site, patient, among others. There were a lot of great conversations, and participants were very engaged in this interactive exercise. Here are some of the conversations that took place.

What does patient-centricity mean to you?

- Driving toward how we can communicate better with patients to help them be more compliant, improve the data we collect, and then share the results of that data with the patient
- Trials that patients are willing to enroll in
- When sponsors are willing to listen to patient feedback
- Bringing the trial to the patient
- Advocacy and engaging with patients as individuals
- Patient-centric trials aim to reduce the burden and increase the benefit to the individual patient, and doing so will improve recruitment and retention
- Creative trial design
- Helping patients with travel, stipends, and providing them with trial summaries (translated into language that’s actually understandable)

What are some regulatory barriers that stand in the way of patient-centered trials?

There are new approaches that are better, and technology is enabling us to do things differently, yet if it’s not validated, we avoid it (we don’t want to be told “no” so we don’t even ask). The FDA will have to move forward to become more patient-centered.  However, some barriers aren’t regulatory, but internal – from outdated SOPs to the excuse, “It’s always been done that way.” Old methods or resistance to change can be overcome, but it won’t always be easy. We have to push for it anyway, because it’s a fight worth fighting.

How would you define a siteless study?

- Patients don’t have to come to a central location; they can do everything remotely (from informed consent to specific procedures)
- A hybrid model where we can minimize or reduce what happens at the site
- Use of ePRO
- “Siteless” sounds like you’re giving something up. Instead, call it “site-free” or virtual site

This topic brought surfaced many other discussions, including the advantages and disadvantages of site visits, debate around if the quality and validity of data entered by a patient or parent is as credible as physician or nurse, and who would have oversight in siteless trials.

Can siteless studies work for every study?

After discussing a model that could work, it’s obvious that it doesn’t fit every study. It may be great for the right studies, but it also doesn’t mean that it’s the end of site studies (at least in the near future).

What is being done to aid in recruitment for rare disease studies?

Because extensive travel is typically involved, it’s not uncommon to use a travel concierge service, or whatever it takes to get the patient there. It’s stressful enough to travel long distances (sometimes overseas) with a sick family member, we don’t want them to worry about flights, rental cars, or where they’re going to stay. Some sites even have a “navigator” who facilitates as much or little as the patient needs.
Having someone there to guide and make arrangements is a great benefit, though it’s very time-consuming and rare outside of rare disease trials.

These topics and discussions were great reminders of how important patient-centricity remains in planning and conducting clinical trials and an excellent end to the first day of the conference. Tomorrow brings several exciting keynotes, as well as a variety of sessions and workshops. Keep following along for more #PCTUS highlights.

About the Author: Kristina Lopienski is the Product Marketing Manager for Overture EDC​at Forte Research Systems​. In her role, she works to bring educational resources to clinical research professionals. She writes on a variety of topics affecting clinical trials on the Forte Clinical Research Blog. Kristina is currently the guest blogger covering Partnerships in Clinical Trials 2015.