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Clinical Trial BillingDocumentation Best Practices11:00–12:30 PM EDT A P R I L

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April 15, 2019

Industry Briefs…2

Drug & Device Pipeline News…7Thirteen drugs and devices have entered a new trial phase this week.

JobWatch…8Job listings, networking events and educational programs.

CenterWatchWeekly

By Leslie Ramsey

P atient involvement shouldn’t end when a trial closes. If you really want subjects to feel they are part of the

team, you need to share the results with them, says one cancer survivor turned trial advocate.

Mary Elizabeth Williams, who wrote about her clinical trial experience in her book A Series of Catastrophes and Miracles: A True Story of Love, Science and Cancer, says sharing trial results with participants builds a sense of pride and accomplishment that can translate into a more positive public view of clinical trials.

Unfortunately, that doesn’t typically hap-pen, Williams said.

“Everything I know about my clinical trial comes from basically my own initiative and

from my work as a journalist, and from my research,” Williams told WCG Clinical Chief Medical Officer Lindsay McNair in a recent interview. And what information she did receive was hard to understand.

“I had to have people explain to me what endpoints are and what ... the charts were and the data was. I wouldn’t have under-stood it myself, and I don’t think any layman would.”

A summary of the trial written for the lay-man would be “amazing,” she said. “To have your hospital or your facility communicate with you, and say, ‘If you would like to read some of the study results, here is where it was published [and] here’s what that means,’ how fantastic would that be?”

Empower Trial Participants by Sharing Results, Author Says

By Brandon May

Collaboration, integration and data-sharing are popular concepts in a life sciences industry focused on finding

novel treatments to meet critical healthcare needs. But are clinical trials really ready to “walk the walk” when it comes to making their data accessible to other sponsors and researchers? Not just yet, say industry experts.

“The industry is a long, long way off from standardizing and collecting and sharing data,” says April Mulroney, chief data officer at WCG Clinical. “Standardizing the collec-tion will enable the standardization of shar-ing, and I think we have a long way to go.”

What is needed are methods of data collection and management that are rec-ognized by the entire clinical research field,

agrees Rebecca Kush, chief scientific officer at Elligo Health Research.

Kush points to the work of the Clinical Data Interchange Consortium (CDISC) as an example. CDISC’s Clinical Data Acquisition Standards Harmonization effort features global and consensus-based data standards available for clinical trials data collection and electronic submission.

CDISC’s approach, which was developed with the encouragement of the FDA as part of its Critical Path Initiative, “streamlines the generation of tables and analysis datasets that meet global regulatory requirements for data,” Kush said. The FDA requires the use of CDISC standards in the electronic submis-sion of applications and data.

Data-Sharing Not a Pipedream, But Still a Challenge, Experts Say

Learn about best practicesto e�ectively manage andexecute clinical trials with the Research Practitioner.

» Earn ANCC contact hours» Maintain nursing certi�cation» Subscriptions at $197

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Caution for NIH's single IRB policy for multi-site researchBy Sue Coons, MA

A tions had to comply with the National Institutes of Health’s (NIH’s) Final

NIH Policy on the Use of a Single Institutional Review Board for Multi-Site Researchpolicy established the expectation that a single institutional review board (sIRB) of record would be used in the ethical review of non-exempt human subjects research protocols funded by NIH that are carried out at more than one site in the United States.

It does not apply to career development, research training, or fellowship awards.

However, some institutions continue to be concerned about their ability to adhere to the policy and the cost of doing so. IRB executives say it is wise to be cautious. Some institutions could spend millions of dollars to upgrade their IRB infrastructure, while some IRBs already have many of the policy requirements in place. In addition, one IRB executive worries that past NIH actions

show that the agency could revise its policy

Emerging trends in clinical research: The need for changeBy John W. Mitchell, MS

T by emerging trends in the clinical trial research sector, consider insight from

a business author and a businessman/politi-cian. In his groundbreaking book, Good to Great: Why Some Companies Make the Leap…And Others Don’t,” Jim Collins wrote:

“Good is the enemy of great. And that is one of the key reasons why we have so little that becomes great. We don’t have great schools, principally because we have good schools. We don’t have great government, principally because we have good govern-ment.”

Secretary Donald Rumsfeld noted that one of the biggest threats to security was driven by factors that fell under the category of “we

don’t know what we don’t know.” In recent issues, Research Practitioner has

explored individual key emerging trends in clinical research, including patient centricity, eMobile advances, and precision medicine. As stand-alone trends, these innovations hold great promise and excitement.

But what insight is gained when all emerg-ing trends are considered in totality? What

clinical research? As Rumsfeld alluded, what might we not know that we don’t know? Are current advancements in clinical research, as Collins asserted, relevant enough to power (great) the sector beyond the status quo (good), given the rapidly evolving medical

ligence (AI), and the expectation of patients?

results on the use of electronic-sourced data, Hugo Stephenson, executive chairman of DrugDev and a physician investigator, observed: “I’m surprised and disappointed there hasn’t been much improvement in the entire clinical trial process, including the

© 2018 CenterWatch. Duplication or sharing of this publication is strictly prohibited. Volume 19, Number 3

Learning Objectives/Outcomes:1. Explain the need for change in clinical research.

2. List key drivers of transformation in clinical trials.

3. Describe examples of clinical trial research trends in action.

4. Discuss why the clinical trial sector will make the jump from trends to practice.

Learning Objectives/Outcomes:1. List the details that local IRBs may know about their

study populations as opposed to a single IRB.2. Describe NIH’s reasoning for going to a single IRB

policy for multi-site research.3. Discuss the impact of using a single IRB on a study’s

direct costs.4. Explain the concern about NIH’s history of choosing

the lowest-cost provider.

42 CE program information

43 Issues in research management

52 SMART IRB

55 CE post-test

May–June 2018

see Emerging trends on page 44

see Single IRB on page 49

o understand the opportunity o�ered

In a 2002 Department of Defense brie�ng,

do these opportunities say about the �eld of

reality of interconnectivity, arti�cial intel-

�ese seem to be fair questions. In a Feb-ruary 2017 CenterWatch article about survey

s of Jan. 25, 2018, research institu-

�is

The most comprehensiveSOP for investigative sites

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WCG | CWWeekly (ISSN 1528-5731)

Leslie Ramsey Editorial DirectorBill Myers Clinical Trials SpecialistRenee Breau ProductionRussell Titsch Business Development Director

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Advertising packages and reprints are available:Email Russ.titsch@centerwatch.com or call (703) 538-7651.

WCG | CWWeekly April 15, 2019 2 of 8

Industry Briefs

FDA RWE Demonstration Project Expands to Live TrialsAn FDA-private sector collaboration that aims to determine the validity of real-world evidence as a substitute for clinical trials has entered a new phase, the partners an-nounced last week.

The RCT Duplicate project — a joint effort by the FDA, Brigham and Women’s Hospital and healthcare startup Aetion — began last spring by comparing the results of 30 completed trials to evidence gathered from healthcare databases to see if both ap-proaches returned the same conclusions.

In the new phase, the project will attempt to predict the results of seven ongoing Phase IV trials, marking the first time researchers are estimating the results of randomized controlled trials that haven’t finished yet. Specifically, it will look to find where RWE can provide “robust estimates” of treatment safety and efficacy for supple-mental drug applications — and potentially fill in for clinical trial results.

“This is going to, hopefully, really give solid evidence of how much we can rely on Big Data to replace the gold standard RCT, which could be really significant both for drug development and for comparative ef-fectiveness studies that support the develop-ment of evidence-based medical guidelines,” Lindsay McNair, Chief Medical Officer with WCG Clinical told CenterWatch Weekly.

The project, which uses Aetion’s software platform to recreate randomized clinical trials through RWE plans to have interim results in mid-2019, with full results by 2020.

Brexit Delayed Again, Trials Still in Limbo The UK has won a further delay from Euro-pean leaders on its exit from the EU, leaving drug sponsors no clearer about the ultimate impact of Brexit on their clinical trials. The departure, originally scheduled for March 29 and then moved to April 12, now is slated for October 31.

UK regulators — who have promised to implement European drug guidelines even if the UK crashes out of Europe without a deal — would be excluded from Europe’s central-ized IT portal for clinical trials and Europe’s single assessment model.

Drug Industry Asks FDA for Clarity on Natural Histories for Rare Disease TrialsThe drug industry is asking the FDA for more clarity around whether natural histories should be included in rare disease trials — and two of the nation’s largest drug trade groups appear to be at loggerheads over the issue.

Among the proposals the FDA is kicking around is allowing natural history stud-ies to substitute as control arms in special cases. Many commenters urged the agency to expand the idea beyond natural histories and to consider such things as patient registries as another way to speed drug development.

Roche, Takeda and BioMarin, among other drugmakers, suggested the agency be more specific about the ways in which natural histories can help speed a drug to market.

But BIO and PhRMA were split on the issue. BIO says it’s worried that the guidance will harden into a requirement that will stall life-saving drugs. Properly done natural histories, the group says, can take several years to complete.

PhRMA, on the other hand, said the draft guidance doesn’t go far enough. Natural

histories “should not be the exception but rather common practice” in rare disease tri-als, the group said.

Read the full comments here: https://bit.ly/2v0aOEd.

Drugmakers Question Exclusion Criteria for Rare Esophageal Disease Trials Two drug companies are pushing back against a proposed FDA guidance that would require drug sponsors to screen out patients in esophageal swelling trials who would respond to an alternate therapy.

The FDA’s draft guidance on eosinophilic esophagitis, published in February, would require clinical trials to exclude subjects who could benefit from proton pump inhibitor (PPI) therapy by holding a two-month pre-trial of PPI dosage.

In comments made public Monday, Adare said the proposal flies in the face of the most recent diagnostic consensus and suggested the agency require trials to exam-ine patient histories to identify and assess those who already have tried PPI.

AstraZeneca noted that the scientific consensus on PPI was still “evolving.” The company currently is working with Chil-dren’s Hospital Medical Center in Cincinnati to recruit 26 esophagitis patients for a trial of Fasenra that’s scheduled to wrap up by next year. AZ won orphan disease status for Fasenra from the FDA last fall.

Read the full comments here: https://bit.ly/2GfWmhH.

© 2019 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW2315

WCG | CWWeekly April 15, 2019 3 of 8

Industry Briefs

Histogenics and Ocugen Agree to MergeA merger agreement has been inked between cell therapy company Histogenics and eye disease drug developer Ocugen, the two companies jointly announced.

The merger would result in a new, clinical-stage biopharmaceutical company under the Ocugen name, with former Ocu-gen stockholders holding around 90 percent of the common stock of the combined company. Current Histogenics stockholders would retain the rest, the companies said.

Ophthalmology programs Ocugen currently has in development include a modifier gene therapy platform, ocular surface disease programs and retinal disease programs.

Histogenics’ technology platform, which has focused on restorative and pain relief

cell therapies, has the potential to be used for a broad range of restorative cell therapy indications.

The merged companies will be head-quartered in Malvern, Pennsylvania, under the leadership of Ocugen’s current manage-ment team.

New Jersey Site Network Ac-quires CNS Research GroupNew Jersey-based sites company Evolution Research Group has acquired Finger Lakes Clinical Research, Evolution officials have announced.

Evolution already owns 13 sites and has a network of affiliates. The company focuses much of its work on early-to-late stage neuroscience drugs. Finger Lakes specializes in central nervous system trials.

Terms of the deal were not disclosed.

J&J Partners with Children’s Research Hospital in D.C.Johnson & Johnson’s life sciences startup subsidiary JLABS has paired up with Chil-dren’s National Health System (CNHS) to develop a large pediatric research facility in the nation’s capital, the two announced last week.

The 32,000-square foot JLABS site will be located on the former Walter Reed Army Medical Center campus — which serves as CNHS’s Research and Innovation Campus — placing it close to academic research centers, universities, federal research institu-tions and government agencies.

The facility will be open to pharmaceuti-cal, medical device, consumer and health tech companies working on new drugs, medical devices, precision diagnostics and health technologies.

Elizabeth Weeks-Rowe

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WCG | CWWeekly April 15, 2019 4 of 8

Features

Data-Sharingcontinued from page 1 “The industry is a long,

long way off from standardizing and collecting and sharing

data. Standardizing the collection will enable the

standardization of sharing...”

—April Mulroney, chief data officer, WCG Clinical

According to Mulroney, there are a bevy of technologies in the start-up phase that may help facilitate greater data-sharing in clinical trials. The costs and hurdles associated with getting these technologies approved and adopted may be the leading reasons why the clinical trials industry has been so slow.

“The biggest barrier for us is who is going to pay for that,” she says. “Pharma has been typically unwilling to pay for data standards and platforms like this because they typi-cally hold their cards close to their chest, and they’re not willing to share their information. I think pharma should treat their scientific IP that they have as private, but I think that the hoarding of information as it relates to sites and patients is just not helpful.”

Despite these sentiments, Mulroney doesn’t believe it’s entirely impossible. “It’s definitely been achieved in other industries that are equally regulated as ours,” she adds, “but the challenge in our industry is that we haven’t even begun scratching the surface.”

The first step is to admit that there is

a problem, Mulroney says, and everyone agrees that drug development can be accel-erated if data are shared. “The second step is sharing in any form or fashion,” she says, “and the third step is to create the standards that make it easier to share.”

“I think the standards for sharing have to be done as an entire industry.”

But it may be up to individual companies to lead the way. “There are a couple of com-panies stepping forward that would like to start sharing results from their clinical trials,” Mulroney says. “The challenge is that it’s one of those things where ‘if everybody doesn’t do it, we’re not going to do it.’ This industry is also a ‘pack mentality,’ and no one wants to be first. Everyone agrees on the value of data-sharing, but no one wants to put their chips on the table.”

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2019Financial & OperatingBenchmarksS E T YO U R S I T E A PA RTBenchmark report helps you know how to accelerate trial growth and maximize e� ciency using current industry trends.

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■ Benchmark data for large sites

■ Comparison data showing di� erences and similarities between small and large sites

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COMPARISON OF SMALL AND LARGE SITES, 2018 21

Sponsor/CRO Improvement Areas

Source: CenterWatch 2019 Financial & Operating Benchmark Survey

In what areas would you most like to see improvement in your relationships with Sponsors and CROs?

% mentioning

2018 Large Sites 2018 Small Sites 2018 Aggregate 2016 Aggregate(n=86) (n=79) (n=165) (n=257)

Contract and budget negotiation issues 37% 25% 31% 14%

Foster a mutually beneficial relationship 23% 34% 28% *

Creation of clear timelines 14% 14% 14% *

Establishment of clear roles and responsibilities 11% 11% 11% 7%

Use of available technology 8% 10% 8% 9%

Remove holdbacks 1% 0% 1% *

Payment turnaround and well-skilled monitors 1% 0% 1% 7%

Additional Master Agreements 1% 0% 1% *

No changes 1% 0% 1% 7%

* Data not collected in 2016 survey.

www.centerwatch.com/FinOps2019

Source: CenterWatch 2019 Financial & Operating Benchmark Survey

In what areas would you most like to see improvement in your relationships with Sponsors and CROs?

% mentioning

2018 Large Sites 2018 Small Sites 2018 Aggregate 2016 Aggregate(n=86) (n=79) (n=165) (n=257)

Contract and budget negotiation issues 37% 25% 31% 14%

Foster a mutually beneficial relationship 23% 34% 28% *

Creation of clear timelines 14% 14% 14% *

Establishment of clear roles and responsibilities 11% 11% 11% 7%

Use of available technology 8% 10% 8% 9%

Remove holdbacks 1% 0% 1% *

Payment turnaround and well-skilled monitors 1% 0% 1% 7%

Additional Master Agreements 1% 0% 1% *

No changes 1% 0% 1% 7%

* Data not collected in 2016 survey.

COMPARISON OF SMALL AND LARGE SITES, 201816

Source: CenterWatch 2019 Financial & Operating Benchmark Survey

In the previous year, how long did it typically take for Sponsors and CROs to pay your investigative site for work that had been invoiced?

Sponsor/CRO Payment Frequency

> 120 days

90–120 days

60–90 days

45–60 days

30–45 days

Large Sites(n=86)

12%

20%

26%

26%

16%

Small Sites(n=79)

3%10%

41%

26%

18%

© 2019 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW2315

WCG | CWWeekly April 15, 2019 5 of 8

Features

Empower Trial Participantscontinued from page 1 “I had to have people explain

to me what endpoints are and what ... the charts were and

the data was. I wouldn’t have understood it myself, and I don’t

think any layman would.”

—Mary Elizabeth Williams

“We live in a culture where all we want to do is know about ourselves.”

Understanding the implications of your trial participation gives you a sense of pride in being part of medical advancements, Williams stressed. “I know how proud I feel of the little teeny, tiny part I played in medical science. I am really proud of that. I am really excited about that,” she said. “To just feel that little bit of my research that my genes, that my DNA, that my blood and my urine all became part of really scientific history, medi-cal history, is an amazing feeling.”

“It’s something that I know I can tell my kids about. Who wouldn’t want that?”

Knowing how their experience was used to help others would change how people look at clinical trials. The result, Williams said, is that more people would be interested in participat-ing. “In a few years, that next patient’s coming down the line who understands more about clinical trials,” and can go into it with a sense of being part of a research team, not just a passive subject.

“You’re part of a team,” Williams said. “That is really empowering, really cool, I would love that. Maybe someday.”

For the rest of the interview, please go to WCG’s website: https://bit.ly/2U9qzqu.

By John W Mitchell

Frustration about clinical trial start-up costs is not unlike Sisyphus’ dilemma. A host of long-standing expense and

inefficiency realities continues to create such uphill headwinds for the clinical trial sector. According to some sources, such problems even threaten the viability of the sector. Sites complain they incur more over-head costs driven by regulatory documen-tation, antiquated data collection and the demands of precision medicine to name a few — all without increased compensation from sponsors and CROs. A recent study supports this concern. Researchers at the Tufts Center for the Study of Drug Devel-opment found that the study start-up phase of five to six months for clinical trials has remained unchanged for the past decade. The study also concluded that CROs mak-ing investments in technology are getting trials done faster. None of this bodes well for smaller, independent players.

“Sites are doing more work for [fewer] patients. We used to enroll about eight pa-

tients per study 10 years ago, and today the number is less than three,” says Jeff King-sley, CEO at IACT Health. His company operates 13 locations in Georgia and a few outside the state in a network of about 100 clinical specialists. “The protocols are lon-ger, and there are more procedures per pa-tient per day. So, you’re doing loads more work, but you’re only paid when you put patients in trials. The averages speak for themselves — you put fewer patients in tri-als today.”

According to Kingsley, the only way to continue this financial model is to partici-pate in three times as many concurrent tri-als. However, he says it’s far more efficient from an overhead standpoint to have one trial that places eight patients, rather than three trials that place eight patients.

Also, at a time when technology is con-quering inefficiencies in other industries, technology is compounding problems in the clinical trial sector. In each of the three studies he cites above, it’s likely that three different electronic health records, tablets

and wearables are used by the different sponsors. When each sponsor uses their own networks and devices for patient-re-ported outcomes, it adds to site workload and time.

“We have no ability to standardize tech-nology,” Kingsley says “We have to do so many trials with so many sponsors, and they have their own decision-making… Our industry suffers from adoptive pho-bia (standardization between sponsors and sites).”

As an example, he cites the advantages of sites adopting electronic platforms such as eSource. Using such a platform could save billions compared to the aggregate cost of monitors flying to sites to review source documents. Clinically, an electronic plat-form also prevents errors such as entering a blood pressure incorrectly or performing a patient procedure out of order from the test protocol.

“We’re trying to convince the industry it’s a huge benefit, but the sites can’t afford

Start-up Costs Can Be an Uphill Slog in Need of Change

© 2018 CenterWatch. Duplication or sharing of this publication is strictly prohibited.

Status Quo is Not a Viable Option: A Host of Challenges Strain Clinical Trial Site Viability

September 2018 A CenterWatch Publication Volume 25, Issue 09

see Site Viability on page 7

By John W Mitchell

C linical trial site finances are a problem that keeps getting bigger. While there’s agreement improvements are on the

horizon — and at least one sponsor is work-ing to shake things up — there’s also a lot of frustration about the slow pace of reinven-tion. A host of longstanding challenges are intensifying. These issues range from slug-

gish payments, contract viability, preferred site status, interconnectivity and something less tangible — a lack of consideration in the relationship.

Payments, which have traditionally ar-rived quarterly, are often now even later. And sites are hiring extra staff to chase down payments— and medical doctors to keep up with the regulatory requirements, says Karri Venn, president of research at LMC Mann

Research, which operates 19 trial and prima-ry care sites in Canada.

According to recent data, 80 percent of sites prefer monthly payments — with 59 percent in-dicating that quarterly payments have a nega-tive impact on trials. Given a choice, they said they’d choose studies that reimburse monthly.

“We learned very quickly that we had to have a floating line-of-credit...[or] we would

see Start-up Costs on page 6

rustration about clinical trial start-up costs is not unlike Sisyphus’ dilemma. A host of long-standing expense and

inefficiency realities continues to create such uphill headwinds for the clinical trial sector. According to some sources, such problems even threaten the viability of the sector. Sites complain they incur more over-head costs driven by regulatory documen-tation, antiquated data collection and the demands of precision medicine to name a few — all without increased compensation from sponsors and CROs. A recent study supports this concern. Researchers at the Tufts Center for the Study of Drug Devel-opment found that the study start-up phase of five to six months for clinical trials has remained unchanged for the past decade. The study also concluded that CROs mak-The study also concluded that CROs mak-The study also concluded that CROs making investments in technology are getting trials done faster. None of this bodes well for smaller, independent players.

“Sites are doing more work for [fewer] patients. We used to enroll about eight pa-

tients per study 10 years ago, and today the number is less than three,” says Jeff King-sley, CEO at IACT Health. His company operates 13 locations in Georgia and a few outside the state in a network of about 100 clinical specialists. “The protocols are lon-ger, and there are more procedures per pa-tient per day. So, you’re doing loads more work, but you’re only paid when you put patients in trials. The averages speak for themselves — you put fewer patients in tri-als today.”

According to Kingsley, the only way to continue this financial model is to partici-pate in three times as many concurrent tri-als. However, he says it’s far more efficient from an overhead standpoint to have one trial that places eight patients, rather than three trials that place eight patients.

Also, at a time when technology is con-quering inefficiencies in other industries, technology is compounding problems in the clinical trial sector. In each of the three studies he cites above, it’s likely that three different electronic health records, tablets

and wearables are used by the different sponsors. When each sponsor uses their own networks and devices for patient-re-ported outcomes, it adds to site workload and time.

“We have no ability to standardize tech-nology,” Kingsley says “We have to do so many trials with so many sponsors, and they have their own decision-making… Our industry suffers from adoptive pho-bia (standardization between sponsors and sites).”

As an example, he cites the advantages of sites adopting electronic platforms such as eSource. Using such a platform could save billions compared to the aggregate cost of monitors flying to sites to review source documents. Clinically, an electronic plat-form also prevents errors such as entering a blood pressure incorrectly or performing a patient procedure out of order from the test protocol.

“We’re trying to convince the industry it’s a huge benefit, but the sites can’t afford

Status Quo is Not a Viable Option: A Host of Challenges Strain Clinical Trial Site ViabilityBy John W Mitchell

linical trial site finances are a problem

gish payments, contract viability, preferred site status, interconnectivity and something less tangible — a lack of consideration in the

Research, which operates 19 trial and prima-ry care sites in Canada.

see Start-up Costs on page 6

In this issue3-4 In Review

Regulatory Update

5 Action Items

Part 4: How to Identify Candidate Fraudulence Early in The Recruitment Cycle By Angela Roberts

Top Considerations for Using Digital Health Devices in Clinical Trials By Vicki Gashwiler

Risk-based Quality Management: The New Normal By Brion Regan

10-11 Pipeline NewsFDA Actions

The CenterWatch MonthlyISSN 1556-3367

Editorial Director Lisa SteinProduction Renee Breau

© 2018 CenterWatch, LLC. All rights reserved. No part of this publication may be distributed or reproduced in any form or by any means without the express written consent of the publisher. Permission requests can be obtained via fax at (617) 948-5101 or emailed at editorial@centerwatch.com. Single-user annual subscriptions start at $399.

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The CenterWatch Monthly September 2018 3

InReview

FDA Signs Off on Treatment for Rare, Adrenal Gland Tumors

The FDA has approved the injectable drug Azedra for rare cancers of the adre-nal glands — the first ever non-surgical therapy OK’d for these tumors.

Azedra (iobenguane I131) is a radiother-apy drug that attacks tumors with a high, specifically targeted dose. It’s designed to treat adults and children (12 and older) with inoperable locally advanced or metas-tic cancers called phenochromocytoma and paraganglioma.

Pheochromocytoma forms inside and paraganglioma grows outside the adrenal gland(s).

Both tumor types release hormones that can cause symptoms including high blood pressure, rapid heartbeat and anxiety.

University of Pennsylvania research-ers gave 68 patients in a trial at least one therapeutic dose of Azedra. The results: 25 percent who received at least one dose and 32 percent of those who received two doses saw their blood pressure drop enough to cut their hypertension meds in half.

“This is a true breakthrough. Until to-day, there were no anti-tumor therapies available for patients with these tumors who were not candidates for surgery,” said principal investigator Daniel Pryma, an associate professor of Radiology and Ra-diation Oncology.

“This therapy not only controls the tu-mors but also the debilitating symptoms caused by their excess hormone produc-tion, meaning it provides dual benefit to patients,” added Pryma, also chief of Penn’s Nuclear Medicine and Clinical Mo-lecular Imaging.

Reported side effects include low white blood cell and platelet counts, fatigue and anemia.

The FDA gave Azedra an Orphan Drug designation, Fast Track status and Break-

through Therapy designation in the U.S. Its license is held by Progenics Pharmaceuticals.

Japan Greenlights Parkinson’s TrialIn the first trial of its kind, Kyoto University

scientists have won approval from Japanese regulators to test adult stem cells as a possible treatment for Parkinson’s disease.

Induced pluriopotent stem cells (iPS) are derived from skin or blood cells and induced back into an embryonic-like plu-ripotent state that can divide into more stem cells or become any type of cell in the body, leading to a potentially unlimited source of any type of human cell needed for therapeutic purposes. They’re consid-ered promising for regenerative research because they can become different hu-man cells and, also, avoid controversy sur-rounding stem cells from human embryos.

Researchers plan to transplant iPS cells into the brains of Parkinson’s patients in the hope they will help repair or replace damaged nerve cells. This is the first trial of its kind to use iPS cells.

Scientists say they’re cautiously optimis-tic after testing the process in monkeys, who showed improvement and didn’t de-velop brain tumors — a much-feared po-tential side effect.

FDA: Focus on Hardest to Treat in Cohort Cancer Trials

The FDA says sponsors considering adaptive studies for early phase cancer treatments should focus on patients with no other options.

The agency notes that so-called expan-sion cohort trials — which start out rela-tively small but expand if a potential treat-ment shows promise--can be a way to get a treatment to market more quickly.

Regulatory Update

see Regulatory Update on page 4

sector. According to some sources, such problems even threaten the viability of the sector. Sites complain they incur more overhead costs driven by regulatory documentation, antiquated data collection and the demands of precision medicine to name a few — all without increased compensation from sponsors and CROs. A recent study supports this concern. Researchers at the Tufts Center for the Study of Drug Devel

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sector. According to some sources, such problems even threaten the viability of the sector. Sites complain they incur more overhead costs driven by regulatory documentation, antiquated data collection and the demands of precision medicine to name a few — all without increased compensation from sponsors and CROs. A recent study supports this concern. Researchers at the

sector. According to some sources, such problems even threaten the viability of the sector. Sites complain they incur more overhead costs driven by regulatory documentation, antiquated data collection and the demands of precision medicine to name a few — all without increased compensation from sponsors and CROs. A recent study supports this concern. Researchers at the

sector. According to some sources, such problems even threaten the viability of the sector. Sites complain they incur more overhead costs driven by regulatory documentation, antiquated data collection and the demands of precision medicine to name a few — all without increased compensation from sponsors and CROs. A recent study supports this concern. Researchers at the

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Drug & Device Pipeline News

Company Drug/Device Medical Condition Status Sponsor Contact

Immatics IMA203 solid tumors Phase I trial initiated enrolling 15 subjects with relapsed and/or refractory solid tumors for which no standard of care therapy is available

immatics.com

Concert Pharmaceuticals, Inc.

CTP-692 schizophrenia Phase I trial initiated concertpharma.com

National Institutes of Health (NIH)

H1ssF_3928 flu Phase I trial initiated nih.gov

Synchron, Inc. Stentrode in combination with BrainOS software

severe paralysis Phase I trial initiated taking place at hospitals in Melbourne, Australia

synchronmed.com

Homology Medicines, Inc.

HMI-102 phenylketonuria (PKU) Phase I/II trial initiated enrolling adults aged 18 to 55 years

homologymedicines.com

NanOlogy NanoDoce (sterile submicron particle docetaxel suspension)

bladder cancer Phase I/II trial initiated dfb.com/nanology-main

Provention Bio, Inc. PRV-031 (teplizumab)

recent onset type 1 diabetes (T1D) Phase III trial initiated enrolling 300 subjects aged 8-17 years with recent onset T1D at 80 centers worldwide

proventionbio.com

Pfizer IBRANCE (palbociclib)

men with HR+, HER2- metastatic breast cancer

sNDA approval granted by the FDA

pfizer.com

Synspira Therapeutics

SNSP113 cystic fibrosis (CF) Orphan Drug designation granted by the EMA

synspira.com

Aggredyne, Inc. AggreGuide A 100 ADP Assay testing cartridge

platelet testing technologies Clearance granted by the FDA aggredyne.com

Spry Health Loop System chronic obstructive pulmonary disease (COPD)

Clearance granted by the FDA spryhealth.com

Viiv Healthcare Dovato (combination of dolutegravir (DTG) and lamivudine (3TC))

HIV-1 in adults who had not received treatment before

Approval granted by the FDA viivhealthcare.com

Amgen Evenity (romosozumab-aqqg)

osteoporosis in postmenopausal women at high risk of breaking a bone (fracture)

Approval granted by the FDA amgen.com

For news on trial results, FDA approvals and drugs in development, Join the LinkedIn Drug Research Updates group!

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