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see Lack of Training on page 6 »

see Market Value on page 5 »

May 27, 2019

Industry Briefs…2

Up and Coming…4

Drug & Device Pipeline News…8Sixteen drugs and devices have entered a new trial phase this week.

CWMarketPlace…9 Industry service provider profiles.

CenterWatchWeekly

By John Mitchell

A t its very core, a clinical trial is a mea-surement system. But unlike other fields that rely on measurement and

data reporting, the clinical trials industry has not focused enough on training for everyone involved, from the investigators all the way down to the patients.

Lack of training, particularly for patients, creates variability detrimental to trial find-ings, says Nathaniel Katz, founder and chief science officer of WCG Analgesic Solutions.

“People have had magical thinking about the way clinical trials generate data,” Katz told the audience of a recent WCG webinar. “There’s this strange belief that if you give some people the treatment and (others) the placebo ... the trial will generate an

observed effect size that somehow will ac-curately characterize the pharmacology of the treatment,” Katz said.

That attitude, he stressed, has resulted in trials ending in expensive failure. Some of those failures could have been avoided with training to eliminate key causes of variability that undermine the scientific process.

The problem is that clinical trial research has not set a high enough training standard.

“Training is not really even viewed as a scientific topic in the world of clinical trials,” Katz said. “Training has been viewed as a checkbox activity or something to do to please regulators, but not something that has a direct impact on our ability to achieve our scientific objectives in clinical trials.”

Lack of Training Compromises Trial Results, Expert Says

By Brandon May

Establishing a fair market value for a clinical trial that both sponsors and sites can agree on often is easier said

than done. There is little or no consensus on the “fair” price to be paid for clinical trial services. Instead, sites should focus on pro-viding a solid defense for each expense they expect a sponsor to foot.

“Often, people say they can’t pay more than fair market value,” says David Vulcano, vice president of research compliance and integrity at the Hospital Corporation of America, “and that’s great, but not every-body agrees on the dollar amount of what fair market value actually is. Instead of fair market value, we should probably use the term ‘defendable market value.’”

“First and foremost, sites need to have a better understanding of how to defend their costs,” Vulcano continues. “There’s probably a fair market value “safe harbor,” a range that we can work comfortably in. But in general, sites often are not willing or capable or knowledgeable to push back and obtain the funding they require.”

According to Vulcano, many sites arrive at a crossroads when faced with sponsors that won’t pay more than a certain amount on a line item, basing their limit on a value that too often has been set in deals with less experienced sites. “If a sponsor ... sees a median rate of $100 for a line item, they might offer it at $80 because they know some people might try and negotiate up,”

Market Value Should be “Defendable,” Not “Fair,” Experts Say

A Note from CenterWatchCenterWatch Weekly will not be published Monday, June 3. The next issue will be published Monday, June 10.

PipelineNews

FDA Actions

Company name Drug name Indication FDA action

Lumendi, LLC DiLumen C2 second-generation endoscopic accessory indicated to ensure complete positioning of an endoscope in the large intestine and assist with optical visualization, diagnosis and endoscopic treatment

510(k) clearance granted

Varian Calypso Anchored Beacon transponder

tumor detection 510(k) clearance granted

Conavi TM Medical Inc. Novasight Hybrid System simultaneous imaging of coronary arteries with both intravascular ultrasound (VUS) and Optical Coherence Tomography (OCT)

510(k) clearance granted

Prisyna, the oral care division of Synedgen

Moisyn product line xerostomia 510(k) clearance granted

C4 Imaging LLC HDR MRI Marker use prior to high dose rate (HDR) brachytherapy to accurately locate the position of the applicators that guide the placement of radioactive sources for the treatment of multiple cancers

510(k) clearance granted

Amerigen Pharmaceuticals Limited and Dipharma S.A.

Miglustat 100 mg capsules Adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option

aNDA � led

Sage Therapeutics intravenous formulation of brexanolone (SAGE-547)

postpartum depression (PPD) NDA � led

Veloxis Pharmaceuticals A/S

de novo indication of ENVARSUS XR (tacrolimus extended-release tablets)

Prophylaxis of organ rejection in kidney transplant patients

sNDA � led

Jazz Pharmaceuticals Xyrem (sodium oxybate) oral solution

cataplexy and Excessive Daytime Sleepiness (EDS) in pediatric narcolepsy patients

sNDA � led

Abeona Therapeutics Inc. ABO-102 AAV-mediated gene therapy for the treatment of San� lippo syndrome Type A (MPS IIIA)

RMAT Designation granted

Immusoft Corporation Immune System Programming MPS I (Mucopolysaccharidosis type I) Orphan Drug Designation granted

Stealth Biotherapeutics elamipretide Leber’s hereditary optic neuropathy (LHON) Orphan Drug Designation granted

P� zer Inc. TRUMENBA (Meningococcal Group B Vaccine)

Active immunization to prevent invasive disease caused by Neisseria meningitides group B (MenB) in children ages 1 through 9 years

Breakthrough Therapy Designation granted

MeiraGTx Limited AAV-RPGR X-linked retinitis pigmentosa (SLRP) due to defects in the retinitis pigmentosa GTPase regulator (RPGR) gene

Fast Track Designation granted

The following is a sampling of FDA regulatory actions taken during the previous month, compiled from CenterWatch and third-party sources including the FDA and company press releases. For more information on FDA approvals, visit www.centerwatch.com/drug-information/fda-approvals/.For custom drug intelligence reports, email marketresearch@centerwatch.com. Join the LinkedIn Drug Research Updates group!

9:45 AMiPad3

Addressing Barriersto Entry and Retention

By Sony Salzman

B eing a principal investigator (PI) is tough. So tough, in fact, that more than half of new investigators give up er

thei rst FDA-regulated drug trial.Studies by the T s Center for the Study of

Drug Development (CSDD) have revealed a variety of challenges for rst-time investigators.

e rst — and some would say worst — challenge is that the path to success for PIs is murky, said Gerrit Hamre, project manager of CTTI and co-author of the group’s paper on the study. Although there are training programs and resources for investigators, most new investigators are unaware of their existence (outside of GCP training), and un-derestimate the infrastructure, st ng, bud-geting, contracting, and operational skills necessary to succeed.

e second overwhelming obstacle for edgling investigators is administra-

tive burden. Complex protocols, rigorous reporting demands and multiple technol-ogy platforms place a huge strain on inves-tigators and sta .

e third major obstacle new PIs face is the lack of accolades and/or s cient nancial reward to make all of that e ort worthwhile.

With mounting evidence that the prob-lem isn’t getting better, sponsors and CROs are rethinking the site selection process and trying to develop new ways to support inex-perienced investigators.

Sponsors and CROs are doubling-down on their top-performing sites — a trend that has helped fuel industry consolidation, says Ken Getz, director and associate professor of CSDD.

“At the same time that sponsors are look-ing for scaled, experienced sites and site networks, the CROs are buying them,” said Getz.

He predicts that in the short term, the in-dustry will continue to see the largest and most expensive sites and site networks build more share of the market.

Yet sponsors can’t rely solely on vetted academic medical centers and large com-mercial sites for all of their study partici-pants; the burgeoning elds of rare disease and oncology research in particular require them to cast a wider net for patients.

Experts agree that rst time investiga-tors can take some steps to bolster success — including nding a mentor, hiring an ex-perienced research coordinator, learning to budget appropriately and being technologi-cally savvy and platform-agnostic — but most also agree that ultimately, sponsors and CROs need to step up if they want their PIs to thrive.

“I’m sympathetic to investigators because you don’t know what you’re getting into,” says Hamre, adding, “I do think investiga-tors very en bite o more they can chew, but they don’t realize it. In that regard, I think it’s the sponsors and the CROs that have a bit more responsibility.”

Getz added that sponsors must “take ownership for site selection practices that

Industry Tries to Stem Investigator Dropout

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

CRA/CRC Shortages Slow Clinical Trial Pace

June 2018 A CenterWatch Publication Volume 25, Issue 06

see CRA/CRC on page 8

Industry DevelopingStandardized TrainingBy Daphne Butas

As clinical trials continue to change, the evolving roles of CRAs and CRCs are exacerbating the problems sponsors and

CRO nding well-trained candidates.“What’s going to really get interesting is how

much these roles are going to evolve as more new initiatives come forward, like eConsent,

ePRO and risk-based monitoring,” said Jim Kremidas, executive director of the Association of Clinical Research Professionals (ACRP). As risk-based monitoring has taken hold and more monitors are analyzing data from a central loca-tion rather than traveling to the sites, sponsors and CROs are looking for CRCs with more of a robust math background, he said. And as the sponsors’ and CROs’ relationships with sites becomes ever more important, sponsors and CROs are looking for CRAs who excel at forg-ing friendly, supportive connections with sites.

“I think we’re going to see a morphing of these role into subspecialties — a data analyst and site-relationship manager type of CRA, along with the traditional role that the CRA has always had,” said Kremidas. “With CRCs, I think we’ll begin to see a quality assurance focus, someone who oversees the input of the data into the eCRFs. I think we’ll see a tech-nology type of specialist for patients to call in order to get help logging on. And I think the traditional role will be there, too.”

see Investigator Dropout on page 6

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WCG | CWWeekly May 27, 2019 2 of 9

Industry Briefs

FDA Plans Clinical Trial Regulatory ChangesThe FDA has unveiled its agenda for upcom-ing proposed and final rulemakings, includ-ing several noteworthy proposals related to clinical trials.

The agency’s plans for the remainder of 2019 include a proposed rule on responsi-bilities for initiating and conducting clinical trials; a proposed rule harmonizing several provisions of FDA regulations on human subject protection; proposed new reporting requirements under the Right to Try Act; and a proposed rule adding development safety update reports to IND application annual reporting requirements.

Additionally, a proposed rule is being con-sidered that would require any U.S. institution participating in multi-site cooperative research to rely on approval by a single IRB for research conducted in the U.S. “with some exceptions.”

Read the Unified Agenda here: https://bit.ly/2whya8S.

Feds Dole Out $9 Million for Alzheimer’s Candidate TrialFederal officials have awarded $9 million to a North Carolina drugmaker to help bring an anti-Alzheimer’s drug through a clinical trial.

Pharma firm T3D Therapeutics says the money will be doled out over the next four years to support its T3D PIONEER study, a Phase II trial designed to test the safety and efficacy of its candidate pill, T3D-959.

PIONEER hopes to recruit 252 adults with mild-to-moderate Alzheimer’s disease and give them either one of three doses of T3D-959 or a placebo for 24 weeks. The trial is supposed to start next year.

The money comes from a grant through the National Institute on Aging.

Drug Giants Partner with Data Company to Ramp Up Clinical TrialsVerily, a subsidiary of Google parent company Alphabet, is partnering with drug

giants Novartis, Otsuka, Pfizer and Sanofi to explore novel methods for generating real-world data.

Verily’s evidence generation platform, Project Baseline, will help the partners collect health information from electronic health records, sensors and other digital sources.

The companies plan to use the data to inform future trials in various therapeutic areas, including cardiovascular disease, oncology, mental health, dermatology and diabetes.

The collaboration aims to make clinical trials more accessible to patients and accel-erate the clinical research process.

Drugmakers Comment on FDA’s Guidance on Natural Histories for Rare DiseasesIonis, Merck, Lundbeck and six other compa-nies offered feedback on the FDA’s guidance for developing natural history studies for rare disease drug development, praising some parts of the guidance but calling for more details.

Responding to the agency’s call for comments on the guidance, which closed on May 24, Ionis suggested that the agency add three additional uses for natural history studies to the guidance — establishing the burden of disease, characterizing genetic mutations and biological consequences and reducing trial burden in interventional studies.

The guidance should strongly encour-age sponsors to share with the public and

community “not only methods, practical aspects and results, but also subject-level data in a timely fashion,” the drugmaker said. It also called for greater emphasis on interim analyses.

Lundbeck noted that many rare diseases have established disease registries made available by clinicians and institutions, call-ing on the agency to acknowledge that it may not always be feasible for sponsors to influence registry development.

Establishing a free-standing prospective natural history study can be challenging in terms of recruitment as well, the drugmaker added.

The company also suggested electronic health records as a potential source for creating a control group, and requested a separate section on methods to deal with bias. The agency should also look into how the guidance’s principles could apply to other vulnerable populations and pediatric development, it said.

Merck asked the agency to clarify its expectations for data submissions. The company said it’s unclear if sponsors should submit only external control data or the whole natural history study.

It also stressed the importance of outside organizations and programs running natural history studies to improve the characteriza-tion of rare diseases, and urged the agency to emphasize that industry collaboration with them could be beneficial.

Read all nine comments here: https://bit.ly/2HOQOdM.

continues on next page »

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continues on next page »

WCG | CWWeekly May 27, 2019 3 of 9

Industry Briefs

Genentech Added to Corrona Multiple Sclerosis RegistryRoche’s Genentech has become the first pharmaceutical company to have its data be included in a national registry of multiple sclerosis patients.The Multiple Sclerosis Registry is one of six efforts created by Corrona to collect data on diseases ranging from rheumatoid arthritis to inflammatory bowel disease. The regis-tries collaborate with more than 400 partici-

pating investigator sites and the data have been used in more than 140 peer-reviewed manuscripts. More than 700 patients have enrolled in the multiple sclerosis registry alone.

MAGI, ACRP Call for Proposals and SpeakersClinical research professionals looking for speaking opportunities can look to two upcoming national conferences.

MAGI has issued a call for proposals for sessions at its October 2019 conference in Las Vegas. The conference will feature more than 100 sessions and workshops in six tracks.

ACRP will open a call for proposals on June 3 for its ACRP 2020 conference to be held in Seattle next May.

Learn more about MAGI speaking oppor-tunities here: https://bit.ly/2VZmwix.

Get information on ACRP’s 2020 confer-ence here: https://bit.ly/30IrvCC.

The case for patient engagement to improve clinical trialsBy John W. Mitchell, MS

I n the quest to achieve better out-comes, control costs, and improve health under a slew of government

policies and laws in recent years (i.e., the -

able Care Act [ACA]), something un-precedented happened: Clinicians of all stripes began asking: What do patients want?

“Patient engagement is the blockbuster drug of the century,” Farzad Mostashari,

-tional Coordinator of Health Informa-

Phar-maceutical Outsourcing, patient involve-

engagement, which has alternatively been known by many names, including patient centricity and patient activation, refers to the process of involving and empowering patients to improve their healthcare. … Even as patient engage-

ment in clinical trials takes multiple shapes, the end goals are nearly always

The issue of proper documentation in IRB liabilityBy Sue Coons, MA

T he guidance Minutes of Institutional Review Board (IRB) Meetings in

Federal Register describes the requirements for IRB meet-ing minutes and provides recommenda-tions for meeting applicable regulatory

Protections (OHRP) and Food and Drug Administration (FDA). It is intended for institutions and IRBs responsible for oversight of human subject research under HHS and FDA regulations.

However, a court case from the early -

ing IRB meeting activity not only puts IRBs in violation of regulatory standards, but also could put them in legal cross-hairs if research participants are harmed or perceive harm in a clinical trial.

Inadequate documentation in IRB

during FDA inspections, the guidance

FDA’s website for bioresearch monitor-ing (www.fda.gov/ScienceResearch/SpecialTopics/RunningClinicalTrials/default.htm -cies found during IRB inspections to be: inadequate initial and/or continuing review; inadequate written procedures; inadequate meeting minutes and mem-

bership rosters; quorum issues; prompt reporting of noncompliance, suspension/termination; Subpart D issues; and lack of or incorrect risk determination.

© 2018 CenterWatch. Duplication or sharing of this publication is strictly prohibited. Volume 19, Number 1

Learning Objectives:

1. List some of the documentation deficiencies FDA is finding in its IRB inspections.

2. Describe the claims plaintiffs in Robertson v McGee made against IRB members.

3. Explain the importance of proper documentation of IRB activities.

4. Discuss the concept of individual liability in IRB decisions.

Learning Objectives:

1. Explain the state of patient engagement in clinical trials.

2. Describe the barriers to and case for patient engagement strategies in clinical trials.

3. List examples of marketing and sales strategies in patient engagement.

4. Discuss the future of patient engagement in clinical trials.

2 CE program information

3 Regulatory update

6 IRB meeting minutes

16 CE post-test

In this issue:

see IRB liability on page 4

see Patient engagement on page 10

se for patient engagement to improve clinical trialschell, l, l MS MS M

to achieve better out-trol costs, and improve er a slew of government ws in recent years (i.e., the

-[ACA]), something un-appened: Clinicians of all sking: What do patients

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sue of proper documentation in IRB liability, MAMAM

ance Minutes of Institutional Board (I(I( RIRI B)B)B Meetingsgsg in

Federal RegRegRe isterrequirements fofof r IRB meet-

and provides recommenda-ting applicable regulatory

OHRP) and Food and Drug on (FDA). It is intended ns and IRBs responsible of human subject research nd FDA regulations. court case frfrf om the early

-

ing IRB meeting activity not only puts IRBs in violation of regulatory standards, but also could put them in legal cross-hairs if research participants are harmed or perceive harm in a clinical trial.

Inadequate documentation in IRB

during FDA inspections, the guidance

FDA’A’As website fofof r bioresearch monitor-ing (wwwwww..ffddffdff aa..ggoovv//vv/vv ScSciieenncceeRReesseeaarrcchh//SSpepecciiaallTTooTToTT ppiiccss//RRuunnnniinnggCClliinniiccaallTTrriiaallss//ddeeffaaffaff uulltt..hhttmm -cies fofof und during IRB inspections to be: inadequate initial and/or continuing review; inadequate written procedures; inadequate meeting minutes and mem-

bership rosters; quorum issues; prompt reporting of noncompliance, suspension/termination; Subpart D issues; and lack of or incorrect risk determination.

Learning Objectives:

1. List some of the documentation deficiencies FDA is finding in its IRB inspections.

2. Describe the claims plaintiffs in Robertson v McGee made against IRB members.

3. Explain the importance of proper documentation of IRB activities.

4. Discuss the concept of individual liability in IRB decisions.

Learning Objectives:

1. Explain the state of patient engagement in clinical trials.

2. Describe the barriers to and case for patient engagement strategies in clinical trials.

3. List examples of marketing and sales strategies in patient engagement.

4. Discuss the future of patient engagement in clinical trials.

see IRB liability on page 4

16 Research Practitioner | January–February 2018

Exam for Continuing Education

Research Practitioner 19.1, 3 Contact Hours

IRB liability and meeting minutes

1. Who did the clinical trial plaintiffs not name in the lawsuit?a. Principal investigatorb. IRB chairc. Nurse coordinatord. IRB members

2. What did the plaintiffs mention in their complaint about the IRB in this clinical trial?a. The IRB failed to examine the

design of the protocolb. The IRB failed to examine the

qualifications of the principal investigator

c. The IRB failed to assure the protection of the participants

d. Both A and Ce. A, B, and C

3. Author Remigius N. Nwabueze argued that:a. IRB members may have

individual liability in the case of harm of a research subject

b. Research institutions protect IRB members from liability

c. IRB meeting documentation has no bearing on potential liability of IRB members

d. None of the above

4. Critical self-examination and internal audits are some of the best ways to catch errors, says attorney Daniel L. Icenogle.a. True b. False

5. Which of the following commonly is observed during inspections of IRB operations?a. Missing minutesb. Inaccurate account of meeting

attendancec. Insufficient or incomplete detail

in minutesd. All of the above

6. Requirements for IRB meeting minutes are found in 21 CFR Part ___:a. 50b. 56c. 312d. 812

7. Although guidance documents are not regulations, when a regulatory authority publishes its current thinking on IRB meeting minutes in a guidance document, it is prudent to use that current thinking to guide your operations. a. Trueb. False

8. Which of the following is useful to guide the preparation of IRB meeting minutes?a. 21 CFR 56.115b. Standard operating procedure

on minutes’ preparation and maintenance

c. A standardized minutes’ template

d. All of the above

9. An IRB is comprised of seven members. At the meeting, four members are present (57%) including a non-scientist and an unaffiliated member. For one hour during the meeting, the non-scientist leaves the room for an hour to take a conference call. For that hour, a quorum technically is still present because the requisite four members were at the start of the meeting. Therefore, any approval vote on a study made during that hour is valid.a. Trueb. False

10. For studies involving children, the IRB would document (in meeting minutes or other IRB record), its discussion of 21 CFR 50 subpart D conditions and approval determination. a. Trueb. False

11. For medical device clinical trials, the IRB would document its device significant risk or nonsignificant risk determination about the study in the minutes.a. Trueb. False

Please note: The website for Research Practitioner exams and evaluations has changed. Please visit the following website which will navigate you to each exam for completion:http://store.centerwatch.com/t-exams.aspx If you have any questions, feel free to email customerservice@centerwatch.com or call 866-219-3440.

Requirements for Successful CompletionTo receive contact hours, participants must register, read the full journal and then go to: http://store.centerwatch.com/t-exams.aspx

Read the instructions on this page to register, select an active exam, successfully complete the post-test with a minimum score of 70%, complete the evaluation, and view and print your certificate.

T

Keep your certifi cation up to date Earn up to 18 contact hours accepted by ACRP, SoCRA and CCIP organizations

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WCG | CWWeekly May 27, 2019 4 of 9

Up and Coming

This feature highlights changes in clinical research companies’ personnel.

EisaiLuca Dezzani has been appointed as vice president, U.S. medical affairs, of Esai’s Oncol-ogy Business Group. He will also oversee Eisai’s health economics and outcomes research, medical science liaison and medical information and education teams. Dezzani previously held senior medical roles in Europe and the U.S. at Novartis Oncology.

Arch OncologyKirk Christoffersen has been named chief business officer at California-based Arch Oncology. Christoffersen was previously head of corporate and business development at Compugen.

Protalix BioTherapeuticsDror Bashan has been selected to be the new president and chief executive officer at Protalix BioTherapeutics, Inc. Bashan previously held a number of senior positions at Teva Pharmaceuti-cal Industries Ltd., most recently as Teva’s senior vice president of global business development.

ArcutisArcutis, Inc., a privately held immuno- dermatology drug development company,

has named John W. Smither as chief financial officer. Prior to this appointment, Smither held CFO positions with Sienna Biopharmaceuticals and Kite Pharma.

Eiger BioPharmaceuticalsEiger BioPharmaceuticals, Inc., has announced that David Apelian will step down as the company’s chief operating officer and executive medical officer, effective June 14, to become the chief executive officer at a private biotech company. Eiger plans to announce a new senior executive medical lead in the near future.

CSL BehringPaul McKenzie has been named as the new chief operating officer at CSL Behring, the drug and plasma unit of CSL Ltd. McKenzie was previ-ously executive vice president of pharmaceuti-cal operations and technology at Biogen.

Voyager TherapeuticsVoyager Therapeutics, Inc., has announced the retirement of Dinah Sah as chief scientific officer, to be replaced by Omar Khwaja, the current chief medical officer, in an expanded role that includes head of research and development.

RedHill BiopharmaRedHill Biopharma Ltd. has named Rob Jackson as vice president of marketing, Robert

Gilkin as vice president of market access and Steven Thomasian as vice president of supply chain. Jackson most recently served as the na-tional director of market access for the surgical business unit at Bioventus. Gilkin was previously vice president of market access at Synergy Pharmaceuticals. Thomasian most recently held the position of president of supply chain at Kala Pharmaceuticals.

Akrevia TherapeuticsAkrevia Therapeutics, a privately held bio-pharmaceutical company, has announced the appointment of René Russo as chief executive officer. Russo most recently served as president and chief executive officer of Arsanis, Inc.

Seattle GeneticsSeattle Genetics has named Robin Taylor as the company’s first chief commercial officer. Taylor spent the past seven months as the franchise head for immune-oncology at AstraZeneca. Prior to his stint with AstraZen-eca, Taylor led the cancer immunotherapy franchise at Genentech.

LocanaLocana, Inc., a start-up gene therapy company, has named Jeffrey Ostrove as CEO. Ostrove was formerly the chief executive officer of AbVi-tro and Ceregene.

Clinical study leads and business opportunities

Research Center Profile9:45 AMiPad3

Clinical Research of WestFloriday, Inc.

Therapeutic AreasTherapeutic Areas

Cardiology/Vascular DiseasesDermatologyDevicesEndocrinologyGastroenterologyHealthy VolunteersImmunologyInfections and Infectious DiseasesInternal MedicineMusculoskeletalNeurologyObstetrics/Gynecology (Women’s Health)OphthalmologyOrthopedics/Orthopedic SurgeryPharmacology/ToxicologyPsychiatry/PsychologyPulmonary/Respiratory DiseasesRheumatologyUrology

Aydin KeskinerPresidentClinical Research of West Florida, Inc.2147 NE Coachman RoadClearwater, FL 33765-2616USA727-466-0078727-461-7793 (fax)research@crwf.comwww.crwf.comView Map

Contact Center

Currently Enrolling Trials

• Are you a current or former smoker? • Are you one of 5 million living with Lupus? • Crohn's Study • Cutaneous Lupus Study[Show More]

Clinical Research of WestFloriday, Inc.

Therapeutic AreasAydin KeskinerPresidentClinical Research of West Florida, Inc.2147 NE Coachman RoadClearwater, FL 33765-2616USA727-466-0078727-461-7793 (fax)research@crwf.comwww.crwf.comView Map

Contact Center

Clinical Research of West Florida, Inc.

Center Information

Aydin KeskinerPresidentClinical Research of West Florida, Inc.2147 NE Coachman RoadClearwater, FL 33765-2616USA727-466-0078727-461-7793 (fax)research@crwf.comwww.crwf.comView Map

Contact Center

Cardiology/Vascular DiseasesDermatologyDevicesEndocrinologyGastroenterologyHealthy VolunteersImmunologyInfections and Infectious DiseasesInternal MedicineMusculoskeletalNeurologyObstetrics/Gynecology (Women’s Health)OphthalmologyOrthopedics/Orthopedic SurgeryPharmacology/ToxicologyPsychiatry/PsychologyPulmonary/Respiratory DiseasesRheumatologyUrology

�erapeutic Areas

A Research Center Profile highlights your site’s expertise to patients and industry experts alike4When you create a Research Center Profile page, you can 4list an unlimited number of clinical trials on the Clinical Trials 4Listing Service™4Profiles are selected for a monthly feature in our CWWeekly 4publication to showcase your site to Sponsors and CROs

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WCG | CWWeekly May 27, 2019 5 of 9

Features Market Value continued from page 1

”It’s important for sites to build up a dossier or

database of their own on how much time they spend on certain

services that are not subject-related, such as administrative

time-related costs.”

—Russell John, global director, grants management, Clintrax Global

says Vulcano. But if 70 percent to 80 percent of sites accept the lower price, he says, then the average could drop to $90 the next time a sponsor checks the price. And the cycle continues from there.

“Sometimes, sponsors call us and tell us that their budget is non-negotiable and that they’re only paying as high as they can without exceeding fair market value,” explains Vulcano. “But that’s their fair market value for research-naïve sites,” he says. “And about six months later, we often get a call from these sponsors and they tell us that they’re not recruiting very well and they’re looking for new sites and they’ve increased their budget.”

To determine value, sites generally must look at various aspects of the study, includ-ing payments toward nurses, physicians and administrative staff, says Russell John, global director of grants management at Clintrax Global. “Sites that do less research don’t build a granular financial budget and don’t consider how much time it’s going to take,” John adds. “I think it’s important for sites to build up a dossier or database of their own on how much time they spend on certain services that are not subject-related, such as administrative time-related costs.”

Sites need evidence to support what they ask for, agrees Wendy Tate, director of analytics at Forte Research. “Many research sites don’t have empirical data on how long it takes to do something,” Tate says. “One of the things I highly recommend from an analytics perspective is to determine how long it takes to do something, like obtaining consent of participants or how long it takes to prepare a sample. That way, sites have a little more justification for their line items and aren’t just pulling a number out of the air.”

“It can be very valuable to discuss what considerations go into determining a dollar amount,” explains Laura Hilty, vice president of product management & strategy at Forte Research. “Documenting this and sharing

these details with the sponsor can go a long way in helping them understand the real reason and justification for costs,” Hilty says.

In addition to the challenges of establish-ing a fair market value that ensures a well-run clinical trial, both payers and payees are responsible for navigating the regulatory framework that governs clinical trial fund-ing. “Sponsors, sites, investigators and cer-tain downstream contractors may be wise to have policies and procedures to ensure that payments made in the context of clinical trials are in each case fair market value for legitimate, reasonable and necessary items or services,” says Andrea Ferrari, a partner at HealthCare Appraisers in Florida.

Ferrari suggests a “toolkit” of policies that include a summary of and/or access to previ-ously determined fair market value ranges for services that are most common in clinical trials of a similar nature. This toolkit might do well to include general guidance for how to apply the fair market value ranges in the context of applicable laws and regulations, she says. “Guidance should focus on how to deconstruct a budget to match budget line items to actual items/costs, caveats to applying the fair market value ranges to certain specific circumstances and triggers for higher-level or more specific review than can be accomplished through the tools in the general toolkit.”

Learn more about this webinar at store.centerwatch.com. REGISTER TODAY!

Using KPIs to Improve Site PerformanceA CenterWatch Webinar presented by Kelly Smith, CCRP, Sr. Solutions Consultant for Bio-Optronics

Tuesday, June 11, 2019 · 1:30–3:00 pm EDTLocation: Your office or conference room (no need to travel!)

Transform your raw and unstructured data into meaningful information. This presentation will illustrate how technology can assist in streamlining your operations and improve your

business decisions based on intelligence derived from the clinical trial data.

JUNE

11

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WCG | CWWeekly May 27, 2019 6 of 9

Features

Lack of Trainingcontinued from page 1 “Training has been viewed

as a checkbox activity or something to do to please

regulators, but not something that has a direct impact on our ability to achieve our scientific

objectives in clinical trials.”

—Nathaniel Katz, founder and chief science officer, WCG Analgesic Solutions

This is especially clear when it comes to training trial participants to report accurate-ly on their reactions and experiences.

“Although pharma, device companies, CROs and regulatory agencies invest heavily in internal training, the concept of training clinical trial participants to perform their tasks better has scarcely filtered into the clinical trials managed by these organiza-tions,” Katz said.

He cited one example in which partici-pants had not been appropriately trained on how to use electronic diaries the trial used to gather patient data. It resulted in skewed and flawed findings on drug effectiveness.

“It’s rare that the skill we are asking people to do (in a trial) has been defined, yet that’s what’s necessary if you want (them) to do it,” he explained.

Katz laid out a best practice training model for clinical trial staff and subjects based on adult learning principles used widely in other industries.

First, it’s vital to understand the difference between education and training. Education is about what you know, he said, and training is

about what you do. Giving someone a manual to read is education. Showing them how to per-form tasks discussed in the manual and allowing them to practice is training. Education doesn’t necessarily change behavior, but training does.

Practice should progress from simple to more advanced tasks and be followed by constructive and diagnostic feedback, Katz said. To facilitate the transfer of new skills to action, training and practice conditions should be increasingly dif-ficult, trainer support should gradually decrease and practice conditions should increasingly resemble real-world conditions.

Katz also made a strong case for validat-ing that any training done has achieved verifiable process improvement. It is the responsibility of leadership to demonstrate that there is a return on investment through more reliable clinical trial outcomes, he said.

Listen to the full webinar here: https://bit.ly/2wr21Mx.

Are your operations measuring up? How can you best position yourselves for sustainability and growth?

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.

I N C R E A S E YO U R S P E E D TO M A R K E TUnderstanding your metrics can help you optimize for speed, cost and quality of results.

The report consists of three data sets:

■ Benchmark data for small sites

■ Benchmark data for large sites

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

BUY NOW

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%

Protocol design • Study design • Patient engagementGenetic testing & counseling

Together, we’re helping our partners deliver on the promise of precision medicine.

Genetics-oriented solutions to support clinical trials

The Center for Genetics and Precision Medicine in Clinical Trials

www.wcgclinical.com

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WCG | CWWeekly May 27, 2019 8 of 9

Drug & Device Pipeline News

Company Drug/Device Medical Condition Status Sponsor ContactInovio and CEPI INO-4500 Lassa virus Phase I trial initiated

enrolling 60 volunteersinovio.comCEPI.net

Compugen, Ltd. COM701 in combination with Opdivo (nivolumab)

advanced solid tumors Phase I trial initiated enrolling 140 subjects at ten sites in the U.S.

cgen.com

Atriva Therapeutics GmbH

ATR-002 influenza Phase I trial initiated enrolling 60 healthy volunteers in Belgium

atriva-therapeutics.com

Axovant Gene Therapies, Ltd.

AXO-AAV-GM1 (also known as AAV9-GLB1)

GM1 gangliosidosis Phase I trial initiated axovant.com

Dicerna Pharmaceuticals, Inc.

DCR-HBVS chronic hepatitis B virus (HBV) infection in adults

Phase I trial initiated enrolling healthy volunteers (HVs) and subjects with non-cirrhotic chronic HBV infection

dicerna.com

NeuClone Pharmaceuticals, Ltd.

Stelara (ustekinumab)

moderate to severe plaque psoriasis in adults and children 12 years or older, active psoriatic arthritis and moderate to severe Crohn's disease in adults

Phase I trial initiated enrolling subjects in Australia

neuclone.com

ImaginAb, Inc. 89Zr-Df-IAB22M2C cancer Phase II trial initiated enrolling metastatic cancer subjects

imaginab.com

IO Biotech IO102 in combination with KEYTRUDA (pembrolizumab)

metastatic non-small cell lung cancer (NSCLC)

Phase II trial initiated enrolling 96 subjects at more than 20 sites across the U.S. and Europe

iobiotech.com

Respivant Sciences RVT-1601 idiopathic pulmonary fibrosis (IPF) Phase IIb trial initiated enrolling 180 subjects in the U.S., Canada and Europe

respivant.com

Rigel Pharmaceuticals, Inc.

fostamatinib disodium hexahydrate (fostamatinib)

warm antibody autoimmune hemolytic anemia (AIHA)

Phase III trial initiated enrolling 80 subjects in the U.S.

rigel.com

Perrigo, Co. Voltaren Gel, 1% (diclofenac sodium topical gel, 1%)

osteoarthritis ANDA approval granted by the FDA

perrigo.com

Mayne Pharma Group, Ltd.

SORILUX (calcipotriene) Foam, 0.005%

plaque psoriasis of the scalp and body in patients aged 12 years and older

Approval granted by the FDA maynepharma.com

Pfizer, Inc. Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis)

cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults

Approval granted by the FDA pfizer.com

UCB NAYZILAM acute treatment of intermittent, stereotypic episodes of frequent seizure activity that are distinct from a patient’s usual seizure pattern in patients with epilepsy 12 years of age and older

Approval granted by the FDA ucb.com

Takeda GATEX (teduglutide) for injection

pediatric patients one year of age and older with Short Bowel Syndrome (SBS)

Approval granted by the FDA takeda.com

Pfizer, Inc. Fragmin (dalteparin sodium) injection

symptomatic venous thromboembolism (VTE) in pediatric patients one month of age and older

Approval granted by the FDA pfizer.com

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

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WCG | CWWeekly May 27, 2019 9 of 9

CWMarketPlace

CWMarketPlace is a monthly section featuring a range of clinical research service providers who have Industry Provider Profile pages posted on CenterWatch.com. Included in their annual subscriptions, company profiles are randomly selected to appear in this section, providing added exposure for their products and services. To learn more about becoming an Industry Provider Profile page subscriber, contact Sales at (617) 948-5100 or sales@centerwatch.com.Click on any provider to view the company’s complete online profile or click here to search more profiles.

Pivotal S.L. Madrid, Spain 0034 679 488 022bd@pivotal.es

Pivotal was founded in 2001 by Dr. Ibrahim Farr on the principle that strategic medical advice and support should be the backbone of all clinical trials. Pivotal currently employs 200 cross-functional professionals.

Medpace Cincinnati, OH(513) 579-9911info@medpace.com

Medpace employs approximately 2,500 people across 35 countries and provides Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries.

CROMSOURCE Waltham, MA(617) 871-1128april.mccall@cromsource.com

CROMSOURCE was among the first CROs to become active in Central & Eastern Europe and Russia. Their successful growth over has been built on stability, integrity, high levels of customer satisfaction and repeated business.

Accell Clinical Research, LLC Culpeper, VA+7 981 844 10 48 svetlana.kazanskaya@accellclinical.com

ACCELL has been providing clinical CRO services to pharmaceutical and biotechnology companies since 2007. They are a full-service CRO specializing in Phase I-IV clinical trials in Eastern Europe, Russia and the CIS.

Confidence Pharmaceutical Research Burlington, CA(401) 965-3377 anna.ravdel@confidenceresearch.com

Confidence Pharmaceutical Research is a CRO and a vendor management organization that helps clinical trial sponsors run better trials. Our focus is on transparency, reducing waste and producing rigorous metrics.

Palm Beach Research Center West Palm Beach, FL (561) 689-0606 david@palmbeachresearch.com

Palm Beach Research Center is a fully dedicated, modern, spacious, multi-specialty research facility for the purpose of conducting Phase I-IV clinical trials. They are committed to bringing state of the art medical treatment to patients.

Virtrial Scottsdale, AZ(480) 462-2222kimkundert@virtrial.com

Virtrial offers a patient management program that combines live video, text and email for clinical trial sites to easily, efficiently and conveniently address specific patient and/or study needs.

Complion Cleveland, OH(800) 615-9077contact@complion.com

Leading sites, hospitals, academic medical centers, health systems and cancer centers around the country use Complion to go paperless, improve compliance and streamline operations.

LabConnect, LLC Seattle, WA(206) 322-4680 info@labconnectllc.com

LabConnect, with more than 4,000 validated tests across their network, has an extensive test menu that includes specialized oncology assays, biomarker analysis, pharmacokinetic analysis and method development services.

Consolidated Clinical Trials, Inc. Monroeville, PA (412) 273-9100 smh@consolidatedclinicaltrials.com

Consolidated Clinical Trials, Inc., established in 2000, has focused on independent physician practices located throughout the Pittsburgh region and surrounding counties.

CONTRACT RESEARCH ORGANIZATION LABORATORY SERVICES

SITE MANAGEMENT ORGANIZATION

TECHNOLOGY SOLUTIONS

TRIAL MANAGEMENT ORGANIZATION