Volume 24, Issue 27. © 2020 CenterWatch

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see Building an RWD “Ecosystem” on page 8 »

see Independent Sites Can Increase on page 7 »

July 20, 2020

COVID-19 Update…2

Industry Briefs …3

Up and Coming…5

Drug & Device Pipeline News…10Thirty-six drugs and devices have entered a new trial phase this week.

JobWatch…12

CenterWatchWeekly

By Elizabeth Tilley Hinkle

Interest in making greater use of real-world data (RWD) is growing among clinical researchers, regulators and

healthcare providers alike, but effective collection and analysis of RWD on a large scale will require a collaborative “ecosystem” in which data collection is standardized for easier analysis.

A critical problem is that RWD is often unstructured and difficult to transfer to clini-cal trial use, noted Laura Esserman, professor of surgery and radiology at the University of California, San Francisco (UCSF) and director of the UCSF Breast Care Clinic. Electronic health records (EHRs) provide a wealth of RWD, but clinical data management is not done through EHRs, going instead through

study-specific case report forms (CRF) and proprietary data management systems.

“The model wasn’t developed to lever-age digital healthcare data because it wasn’t available,” said Stephanie Reisinger, vice president and general manager of Veradigm Life Sciences. “We are trying to plug RWD into a model not designed to accept it.”

Standardizing data collection practices so that “clinical trials look more like care and care like clinical trials” would help create and scale up the RWD ecosystem, Esserman said at a two-day workshop sponsored by the FDA and Duke University’s Margolis Center for Health Policy.

It could be more efficient to take the data as it is collected and move it directly

Building an RWD “Ecosystem” Requires Standardization, Collaboration

By Suz Redfearn

Independent sites without the time, staff or accounting experience to keep a close eye on sponsor billing and payments

can avoid losing thousands of dollars by instituting a few best practices to help keep the cash flowing.

On average, independent sites are failing to collect $3,500 in sponsor/CRO payments per study, according to WCG PharmaSeek. Fifteen percent of those lost payments are the result of sites’ inadequate invoicing practices. In one case, PharmaSeek’s auditors uncovered $56,000 of revenue across 16 studies that the site hadn’t invoiced for or that the sponsor or CRO just hadn’t paid.

The primary culprit is lack of staff dedicated to accounting tasks, Pharma-

Seek’s Ryleigh Mosley told participants in a recent webinar. “While many sites have an accountant or bookkeeper to run monthly reports and manage high-level financials, many sites don’t have an employee dedicated to daily operational-level accounting or managing revenue,” said Mosley, who is associate manager of operational accounting. “And too often, coordinators split their time between trial conduct and administrative work, leaving little time to manage collections and recordkeeping.”

But it doesn’t need to be this way, she said, not if site staff understand how and when to dig in hard on their accounts receiv-able and keep great records.

Independent Sites Can Increase Accounting Efficiency with Best Practices

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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

ruary 2017 CenterWatch article about survey

s of Jan. 25, 2018, research institu-

Earn ANCC contact hoursMaintain nursing certificationSubscriptions at $197

Research billing compliance continues to challenge research practitioners

Investigational drug management requires strict controls, constant documentationBy Elizabeth Tilley Hinkle

W hen it comes to managing in-vestigational drug products, the stakes are high. If even one small

batch of control drugs get mixed in with the test drug supply or if one shipment of medication is stored outside of the proper temperature range or one person admin-isters drugs incorrectly, it can put patients at risk, throw research data into question and even lead to the study’s termination.

are so complex, documentation procedures so rigorous and federal regulations so strict.

While the prinicipal investigator (PI) is generally given full responsibility for inves-tigational drug management, anyone with drug handling or administration authority

— including nurses and other clinicians, on-site pharmacists, researchers and research

investigational drugs are handled properly.

— and federal regulations — about drug shipping and intake, product labeling and storage; authorizations and training; medi-cation dispensing and administration; and returning or destroying unused drugs.

And it means thoroughly, accurately and promptly documenting these proto-cols and actions every step of the way.

Unfortunately, safe investigational drug ordering, handling, dispensing and admin-istering procedures have not been univer-sally standardized, Jamie Brown et al., wrote

of Biology and Medicine. As a result, some areas of the process could pose safety risks during clinical trials that would be less likely when commercially available drugs are used outside a research setting, the authors note.

26 CE program information

27 Regulatory update

39 National coverage determination...

40 CE post-test

March-April 2020

see Investigational drug management

Learner Outcomes:

1. Describe principal investigators’ role in drug management.

2. Explain drug management labeling protocols.

3. Demonstrate knowledge of drug storage requirements.

4. Describe key FDA regulations about investigational drug product handling.

Where can clinical trial professionals turn for practical, useful and easily accessible training?

.Train your entire staff

to ANCC standards with a side-wide subscription to Research Practitioner.

L E A R N M O R E

© 2020 CenterWatch CWW2427

CenterWatch Weekly (ISSN 1528-5731)

Beth Belton Editorial DirectorStephanie Akers ProductionRussell Titsch Business Development Director

© 2020 CenterWatch. 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.

CenterWatch Main and Editorial Offices 300 N. Washington St., Suite 200, Falls Church, VA 22046 Tel: 866.219.3440 • 617.948.5100editorial@centerwatch.com / sales@centerwatch.com

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WCG | CWWeekly July 20, 2020 2 of 13

COVID-19 Update

COVID-19 Drug Research Roundup

COVID-19 Vaccines:Sinopharm has begun a phase 3 trial of

its COVID-19 inactivated vaccine candidate in the United Arab Emirates. The study, which dosed its first group of volunteers last week, is the world’s first phase 3 trial for an inactivated vaccine, according to Abu Dhabi’s health department. The trial is approved to enroll up to 15,000 participants, and at least 5,000 patients between age 18 and 60 are expected to be recruited in its first stage.

Moderna will begin enrolling partici-pants for its phase 3 COVID-19 experimen-tal vaccine trial on July 27. The 30,000-par-ticipant study is supported by phase 1 study results that showed all 45 partici-pants generated neutralizing antibodies following vaccination with mRNA-1273, the company’s candidate. The results showed the vaccine was generally safe and well-tolerated by participants.

Johnson & Johnson has said that it will start the first phase 1 study for its COVID-19 vaccine candidate, Ad26.COV2-S, which uses recombinant DNA technology, in Belgium on Wednesday. The human trial is antici-pated to begin in the U.S. next week and will enroll more than 1,000 healthy adults be-tween age 18 and 55 in addition to patients age 65 and older.

The Chinese government has approved a phase 1 trial of BioNTech’s and Pfizer’s COVID-19 vaccine candidate BNT162b1, BioNTech’s Chinese partner, Fosun Pharma, has announced.

Canada’s Medicago has begun dosing the first participants in a phase 1 trial of its plant-derived COVID-19 vaccine candidate. The trial will evaluate the vaccine in 180 healthy male and female subjects between age 18 and 55 at three different dosage levels of the vaccine, either alone or with ad-juvants from GlaxoSmithKline and Dynavax.

Australia’s University of Queensland has dosed the first volunteers in a phase 1

trial of its COVID-19 vaccine candidate. The study will evaluate about 120 volunteers between age 18 and 55, with a number of patients to be given a placebo. Preliminary results are expected in about three months.

Russia’s Sechenov University has com-pleted the first clinical trial of a COVID-19 vaccine candidate. The vaccine, which was developed by the Gamaleya Institute in Rus-sia, was proven safe in 38 healthy patients, according to the researchers.

COVID-19 Treatments:Indian drugmaker Biocon’s randomized,

controlled, open-label COVID-19 study of itolizumab demonstrated that the monoclo-nal antibody significantly reduced mortality in hospitalized patients. The trial enrolled 30 patients with moderate to severe acute respiratory distress syndrome (ARDS).

Shanghai-based Junshi Biosciences has finished enrolling participants in a phase 1 trial of JS016, its COVID-19 neutralizing antibody. The randomized, double-blind, placebo-controlled study is evaluating the intravenous injection of JS016 in 40 healthy subjects. The candidate is the first COVID-19 neutralizing antibody to enter trials in China.

Keck Medicine of the University of Southern California has begun enrolling patients in an international phase 3 trial evaluating the safety and efficacy of its antiviral candidate, DAS181, as a COVID-19 treatment. More than 30 sites across the U.S., Australia, South Korea and Taiwan are taking part in the trial.

Results published from the University of Oxford’s RECOVERY trial show that the anti-malarial drug hydroxychloroquine increased

participants’ time in hospital and their risk of progressing to mechanical ventilation or death and did not lower 28-day mortal-ity. The results showed that of the 1,561 patients who received the drug, 26.8 percent died within 28 days of starting treatment compared to 25 percent of participants re-ceiving standard care. The study also found that patients on hydroxychloroquine had to wait longer for hospital discharge than patients not given the drug and were less likely to survive after 28 days.

PhaseBio Pharmaceuticals has dosed the first of more than 200 patients in the phase 2 VANGARD trial of its PB1046 treat-ment for hospitalized COVID-19 patients at high risk of clinical deterioration and ARDS. PB1046 is a once-weekly subcutaneously injected anti-inflammatory drug.

Korean firm Celltrion has launched a phase 1 study of its antiviral antibody treatment and plans to complete the trial by the third quarter. In addition, Celltrion plans to conduct phase 1 trials at sites across Europe and in the UK. The company plans to follow that study with global phase 2/3 trials, with preliminary results due by the end of the year.

Tetra Bio-Pharma, a developer of cannabinoid-derived drugs, says the FDA has responded positively to its Pre-Investi-gational New Drug application, which the Canadian company submitted under the agency’s Coronavirus Treatment Accelera-tion Program. Tetra’s candidate, ARDS-003, is designed to dampen the cytokine re-lease syndrome and prevent the develop-ment of ARDS.

© 2020 CenterWatch CWW2427

WCG | CWWeekly July 20, 2020 3 of 13

Industry Briefs

FDA Says It’s Reviewed 230 COVID-19 Treatment Trials to DateThe FDA said last week that it has reviewed more than 230 clinical trials of potential COVID-19 treatments so far through its Coronavirus Treatment Acceleration Pro-gram (CTAP).

More than 180 of the trials are in late-stage testing for safety and efficacy, while more than 40 are in early-stage testing for safety and dosing, the agency said.

More than 510 drug development programs for COVID-19 are currently in the planning stages.

“While we have shortened our timelines, our regulatory review and decision-making processes have not changed,” said FDA Com-missioner Stephen Hahn in a joint statement with Patrizia Cavazzoni, acting director of the Center for Drug Evaluation and Research, and Peter Marks, director of the Center for Biologics Evaluation and Research.

The FDA launched CTAP in late March as an effort to accelerate COVID-19 therapies. The program is not involved in reviewing vaccines.

Russian Hackers Seek to Steal COVID-19 Vaccine ResearchAs research on COVID-19 vaccines advances at an unprecedented pace, Russian hackers have been attempting to steal research in-formation, the UK’s National Cyber Security Centre (NCSC) reported last week.

The hacker group APT29 has been engaging in cyberattacks throughout the year against U.S., UK and Canadian orga-nizations involved in COVID-19 vaccine research and development.

The group’s likely intent is to steal infor-mation and intellectual property related to vaccine development and testing research, the cybersecurity center said, noting that the group has mainly targeted government, diplomatic, think-tank, healthcare and energy-related entities in its attacks.

The group is “almost certainly part of the Russian intelligence services” and leverages malware to gain access to systems, NCSC said, noting that the group has been suc-cessful on four occasions. Once the hackers get access to a system, it is likely that it sets up persistent access by installing further malware or by obtaining login credentials.

Scientists Urge NIH to Consider Human Challenge Trials for COVID-19 VaccineIn an open letter to the National Insti-tutes of Health (NIH), more than 125 of the world’s top scientists have called on the U.S. government to start conduct-ing human challenge trials to accelerate COVID-19 vaccine development.

Citing the urgency of finding a vaccine for the deadly virus, the scientists — among them 15 Nobel laureates as well as the director of Oxford University’s large CO-VID-19 vaccine program — say the ethical objections to infecting healthy volunteers with a disease that has no known cure are outweighed by the common good.

In a similar letter sent to the FDA in April, 35 U.S. lawmakers also encouraged the use of challenge trials, saying “justifi-able risks may be taken” (CenterWatch Weekly, April 27). The agency responded by suggesting animal testing could be used instead.

NIH Director Francis Collins’ previously stated position on the subject has been that challenge trials are open to discus-sion, but that NIH is not yet ready to move forward with a human challenge trial plan.

Earlier this month, members of NIH’s Ac-celerating COVID-19 Therapeutic Interven-tions and Vaccines (ACTIV) working group wrote in an article in the New England Journal of Medicine that deliberately infect-ing healthy individuals without a life-saving treatment on hand would be unethical. ACTIV members expressed support for traditional randomized controlled trials of

SARS-CoV-2 vaccines as the most efficient path to a vaccine.

The July 15 letter to the NIH was pub-lished on the website of 1 Day Sooner, a nonprofit organization that has recruited more than 32,000 healthy volunteers in 140 countries who are willing to participate in challenge trials.

To read the letter, click here: https://bit.ly/2CKSFBD.

Trial Recovery Requires More Technology Support from Sponsors/CROs, Sites Say Site staff believe more telecommunication technology will be needed to get trials back on their feet post-pandemic and say they need help from sponsors and CROs to imple-ment alternative approaches to in-person patient visits, according to a new report.

Respondents to a survey conducted by Clinical SCORE said it will take 5 to 6 months on average to recover from financial losses and return the number of trials being activated to pre-COVID-19 levels. Financial recovery will require scaling up remote and virtual visits to make up for loss of staff dur-ing the pandemic.

Most of the 255 survey respondents expect the number of in-person site visits to decline, and 62 percent said telephone communications will see the greatest increase, followed by virtual visits (e.g., web-based video visits) at 51 percent and remote visits conducted at facilities other than the trial sites at 47 percent. Home visits also will increase, according to 20 percent of respondents.

Three-quarters of respondents said they need more support from sponsors for the protocol changes required to implement virtual and remote visits as well as funding to do so. One-third called for CRO assistance in executing remote visits.

To read the report, click here: https://bit.ly/32s8ZBR.

continues on next page »

© 2020 CenterWatch CWW2427

WCG | CWWeekly July 20, 2020 4 of 13

Industry Briefs (continued from page 3)

U.S. Clinical Trials Taking Longer Despite FDA Efforts to Accelerate Drug DevelopmentThe average time needed to complete clinical development of a new drug in the U.S. is increasing, outpacing the FDA’s efforts to accelerate drug approval and lengthening the overall amount of time needed to bring a new drug to market, according to a new report.

The average combined clinical trial and approval timeline increased by almost five months between 2008 and 2018, an analysis by the Tufts Center for the Study of Drug Development (CSDD) shows. At the same time, the clinical trial timeline grew by nearly seven months, while the approval phase dropped by nearly two months.

Drugs that fell under the FDA’s expe-dited review program, however, were brought to market 11 percent faster than those without expedited review. And the

trial-to-approval timeline for orphan drugs decreased 12 percent.

The duration of clinical trials increased across all phases, the study shows, with the greatest increase (9.2 percent) in phase 2.

To read a summary of the results, click here: https://bit.ly/32t9vj7.

Guidance UpdateThis occasional column provides summaries of newly released FDA guidances of interest to our readers.

Clinical Trials for BPH DevicesA draft guidance released by the FDA last

week contains new recommendations for sponsors conducting clinical trials of devices to treat benign prostatic hyperplasia (BPH), including advice for randomization, end-points, statistical analysis, patient selection and followup.

The provisions in the draft would replace those in the current final guidance on clini-

cal investigations of devices to treat BPH, dated August 2010. Comments on the draft are due by Sept. 14.

To read the draft guidance, click here: https://bit.ly/32sJSPq.

Trials for Prostate Tissue AblationDevicemakers submitting premarket

applications for high-intensity ultrasound prostate tissue ablation systems must first conduct clinical trials of the systems under a final regulation the FDA released last week.

Prostate tissue ablation devices of this type present significant risk, the guid-ance says, and sponsors must comply with Investigational Device Exemption regulations, including IRB review. It also gives advice on study design and dura-tion, inclusion/exclusion criteria, protocols, endpoints and data analysis.

To read the final guidance, click here: https://bit.ly/30oIsTg.

Real World Evidence and Data in Clinical TrialsA Tufts Study of 30 Pharma Companies

Thursday, Aug. 13, 2020 1:30 p.m. - 3:00 p.m. EDT

REGISTER TODAY!

Webinar Webinar Takeaways:

n Types of technology used to access or collect real world data and evidence and partnerships that support usage

n Significant challenges to using RWD as well as strategies and practices that impact return on investment or performance

n The key drivers for change and the adoption of RWE

n The potential of RWE and how it may be used across the clinical development pipeline

n A look into regulators position regarding COVID-19 studies

Learn more: www.centerwatch.com/realworldevidence

© 2020 CenterWatch CWW2427

This feature highlights changes in clinical trial organizations’ personnel.

Abeona Therapeutics Michael Amoroso has been named chief commercial officer of Abeona Therapeu-tics. Prior to this role, Amoroso was senior vice president and head of worldwide commercial cell therapy at Gilead Sci-ences’ Kite.

AbverisTracey Mullen, former chief operating of-ficer of Abveris, has been promoted to CEO of the company. Garren Hilow has moved from his role of CEO at the company to the role of chief business officer.

AimmuneNarinder Singh, who previously served as senior vice president of pharmaceutical sciences and manufacturing at Genocea Biosciences, has been hired by Aimmune to assume the role of executive vice president of technical operations.

Akcea TherapeuticsAkcea Therapeutics has named William Andrews as its new chief medical officer. Andrews was most recently chief medical officer at Acer Therapeutics.

AnaptysBioDennis Mulroy has been appointed chief financial officer of AnaptysBio. Prior to joining AnaptysBio, Mulroy was chief financial officer of La Jolla Pharma-ceutical. AnaptysBio also named Eric Loumeau chief operating officer of the company. Loumeau was most recently general counsel and chief compliance officer of Otonomy.

AntengeneAntengene has named Dirk Hoenemann head of medical affairs for the Asia Pacific region and early clinical development.

Hoenemann most recently served as the lead of development of the early clinical development programs for the Asia Pacific region at Celgene.

Arrakis Therapeutics Erik Spek has been tapped by Arrakis Therapeutics to serve as the company’s vice president of legal and intellectual property. Spek was senior vice president and head of legal affairs and intellectual property at Vedanta Biosciences prior to joining Arrakis.

Cardurion PharmaceuticalsChris Morabito has been appointed chief medical officer of Cardurion Pharmaceu-ticals. Most recently, Morabito was senior vice president and head of research and development and plasma-derived therapies of Takeda Pharmaceuticals.

Citius PharmaceuticalsMyron Czuczman has been named chief medical officer and executive vice presi-dent of Citius Pharmaceuticals. Czuczman previously served as therapeutic area head and vice president of clinical research and development of the global lymphoma/CLL program at Celgene.

COVAXXCOVAXX has named Farshad Guirakhoo as its new chief scientific officer. Guira-khoo was recently the chief scientific officer of GeoVax.

Cytovance Biologics Cytovance Biologics has named John Mott vice president of development. Prior to joining Cytovance, Mott was vice president of laboratory services at Aragen Bioscience. Yuk Chun Chiu has also been enlisted as Cytovance’s vice president of manufactur-ing operations. Chiu recently served as the director of manufacturing and engineering at AstraZeneca.

DuPage Medical GroupDuPage Medical Group’s newest CEO has been found in Steve Nelson, the former CEO of UnitedHealthcare.

EyePoint PharmaceuticalsJay Duker, chair of ophthalmology at Tufts Medical Center and the Tufts University School of Medicine, has been appointed chief strategic scientific officer of EyePoint Pharmaceuticals.

GTX MedicalDave Marver has been named CEO of GTX Medical. Prior to this role, Marver was CEO of VICIS.

ImCheck TherapeuticsImCheck Therapeutics has appointed Claude Knopf to the role of chief business officer. Knopf most recently served as chief business officer at Gemini Therapeutics.

Kiadis PharmaKiadis Pharma has named Ray Barlow as chief business officer and Govert Schouten as head of innovation. Barlow previously served as chief executive officer at e-therapeutics, and Schouten was most recently the founder of Idmon Consulting B.V.

Mateon TherapeuticsBiotech company Mateon Therapeutics has appointed Giancarlo Mennella to lead its licensing activity for a potential treatment for COVID-19. Mennella is managing director at EGAMID.

Neurogene Kenneth Huttner has been appointed to the role of senior vice president of clinical development at Neurogene. Prior to this new appointment, Huttner was senior vice president and head of clinical development at LogicBio.

WCG | CWWeekly July 20, 2020 5 of 13

Up and Coming

continues on next page »

© 2020 CenterWatch CWW2427

WCG | CWWeekly July 20, 2020 6 of 13

Up and Coming (continued from page 5)

NotableJoseph Wagner has been hired as chief scientific officer of Notable. He was previ-ously the executive director of the University of California Drug Discovery Consortium.

OncologiePrecision medicine company Oncologie named Matthew Osborne, former chief financial officer at Unum Therapeutics, as the company’s new chief financial officer. Hagop Youssoufian was also appointed by Oncologie to interim chief medical officer. Youssoufian was previ-ously head of experimental medicine at Bristol-Myers Squibb. Oncologie has also hired Bill McDonald as vice president of chemistry, manufacturing and controls (CMC) and Kerry Culm-Merdek as vice president of clinical development. McDonald and Culm-Merdek previously

held the respective roles of vice presi-dent of CMC at Contrafect and head of clinical pharmacology and quantitative bioanalytics at ImmunoGen.

Scholar Rock Scholar Rock has named Tony Kingsley as president and CEO. Kingsley was formerly the president and CEO of Taris Bio.

Sentien Biotechnologies Allen Nissenson, former emeritus chief medical officer of DaVita Kidney Care, has been named chief medical officer of Sentien Biotechnologies.

Synaptive MedicalMarc Buntaine has been appointed CEO of Synaptive Medical. Buntaine was formerly the executive vice president of sales and marketing for Mobius Imaging.

Turning Point Therapeutics Andrew Partridge has been named execu-tive vice president and chief commercial of-ficer at Turning Point Therapeutics. Partridge most recently served as chief operating officer and chief commercial officer at Cen-trexion Therapeutics.

Vor Biopharma Christopher Slapak has been appointed to the role of chief medical officer of Vor Biopharma. Slapak was formerly the lead of global clinical development for all early-stage oncology compounds at Eli Lilly.

Zikani TherapeuticsVijay Modur will be Zikani Therapeutics’ new chief scientific and medical officer. Mo-dur comes to Zikani from Sanofi-Genzyme, where he was global project head in rare disease clinical development.

By Elizabeth Tilley Hinkle

S CenterWatch survey of how trial sites view their

-

CenterWatch sur-vey.

startup times, improved patient recruit-ment and retention, and overall increased

-ing reiterated the need for the changes that we were in the process of making,” says Victoria DeBiasio, co-leader of SMILE and

strategy.

-eas where it ranked lowest and gave it to

--

● Protocol optimization, focused on reduc-ing burdens for both sites and patients and reducing protocol amendments;

● Finance and contracting, making sure sites are paid quickly on an ongoing basis and ensuring billing transparency

faster;

● Site relationships, especially in patient recruitment;

● Supporting CRAs with training and orientation to reduce turnover; and

● Improved query processes and produc-tive, well-timed investigator meetings.

program to steering committees that in-cluded representatives from sites and

regularly to ensure that all metrics are be-ing met.

-

trials results in real optimization of new product development, so patients also ben-

Some of the greatest improvements

and productivity, as measured by a reduced number of non-enrolling sites — less than

-cent improvement — of patients enrolled per site. DiBiasio touted early patient input as key to good enrollment and retention

From Disappointment to ‘SMILE’: Sano� Revamps Site Support Program

January 2020 A CenterWatch Publication Volume 27, Issue 01

By Colin Stoecker

S ome daunting statistics from the last

--

to calculate since it includes the cost of all

costs several hundred million dollars in di-rect costs to carry through all clinical test-ing phases.”

expensive and resource-intensive waste of time.

One way to avoid late-stage failures is to do a better job planning the early phases,

Mike Rea.

see on page 4

In this issue3-4 In Review

Regulatory Update

5 Action Items

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

Top Considerations for Using Digital Health Devices in Clinical TrialsBy Vicki Gashwiler

Risk-based Quality Management: The New NormalBy Brion Regan

10-11 Pipeline NewsFDA Actions

The CenterWatch MonthlyISSN 1556-3367

Editorial DirectorProduction

© 2018 CenterWatch, LLC. All rights reserved.No part of this publication may be distributed orreproduced 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.

For inquiries on multi-reader and corporate subscription rates and article reprints:Tel: (617) 948-5100Email: sales@centerwatch.com

InReview

Rare, Adrenal Gland Tumors

drug Azedra for rare cancers of the adre-

therapy OK’d for these tumors. -

apy drug that attacks tumors with a high,

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-

percent who received at least one dose and

saw their blood pressure drop enough to cut their hypertension meds in half.

license is held by Progenics Pharmaceuticals.

Japan Greenlights Parkinson’s Trial

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

-ered promising for regenerative research

-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

of its kind to use iPS cells.

Regulatory Update

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Features

It starts, she said, with getting to know the payment details of the clinical trial agreement (CTA) for each of the studies a site has taken on. Sites should ask a few questions when reviewing a CTA:

} Where are the key payment terms? } Are there hidden items we can

invoice for? } Is it clear what items we’ll need to send

an invoice for? } Is it clear how we’ll be receiving pay-

ments? } What exactly needs to be sent along

with the invoice itself?To get reimbursed for all services in

a timely and uncomplicated manner, it’s better if CTAs are negotiated to be very detailed, said Mosley. Make sure the language makes all payment expectations very clear.

“There can be some pretty tricky lan-guage hidden in these contracts that can definitely slow down” your understand-ing of how exactly to get paid and when, she said.

For instance, a clear statement about monthly invoicing would be:

Institution shall invoice CRO monthly for services performed related to study subject costs as set forth in the attached budget; this invoice will be payable after it has been determined whether the study subject has been randomized or is a screen failure. CRO will verify the amount of money owed the

institution based on completed and accepted electronic case report forms.

A less precise statement would be:Payment for study subject visits shall be

made monthly for confirmed, completed visits upon receipt of valid invoice.

In addition to reviewing the CTA, sites should improve their recordkeeping, ideally using a program, software or application to store all study payment information, Mosley said. She suggested opting for a program that allows the site to keep all its financial in-formation in one place so site staff don’t have to click through too many pages to find it.

Mosley also recommended classifying ac-counts receivable into three groups: admin-istrative fees; patient visits; and conditional fees. Some of these categories can blend, she warned.

“There are some fees that can border on administrative or conditional, depend-ing on terms,” said Mosley. “For example, dry ice. Ask yourself: Is it a flat fee for shipment? That’s administrative. Or is it

per subject per visit? That would be more conditional. Or even is it included in the cost of each visit?”

Invoices should be as detailed as possible, containing date of service, description of the item/visit/procedure, protocol number, principal investigator (PI) name and subject number if it’s a patient visit charge. And always include supporting documents like receipts. It’s likely that if you don’t include those the first time around, she said, the sponsor or CRO will just kick it back to you and you’ll have to start all over again, further delaying payment.

Finally, Mosley recommended sites create a master reconciliation docu-ment, listing in columns all the studies they’re working on, who the PI is, the payment terms and due dates, sponsor or CRO contact info, and most impor-tantly, amount outstanding. Then keep it constantly up to date even if it seems like a pain, she said.

“While that may seem like overkill or extra work, I guarantee it’s something you’re going to want to do just to make sure your bases are covered,” she said.

This may all seem like a lot, especially for staff who are already doing double or triple duty. But Mosley said it’s worth it. “Even just carving out 30 minutes to an hour each week on collections can make a big difference, and it will help decrease time spent on this at the end of the month, which is always a crunch.”

Independent Sites Can Increasecontinued from page 1 “Even just carving out 30

minutes to an hour each week on collections can make a big difference.”

—Ryleigh Mosley, associate manager of operational accounting at

WCG PharmaSeek

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Features

from the EHR into the clinical research database in response to the demands of a study protocol, suggested Monica Bert-agnolli, professor of surgical oncology at Harvard Medical School. The Minimal Com-mon Oncology Data Elements (mCODE) could offer a model for future RWD collec-tion efforts, Bertagnolli noted. The mCODE has been successfully used to track disease history and treatment response in clinical trials for low-burden data collection.

Structured data fields can help support the systematic collection of key variables, said Leslie Harrold, chief scientific officer at biotech company Corrona. Those fields could include patient demographics, patient lifestyle information, treatment history, dis-ease characteristics and activity, patient-re-ported outcomes, laboratory measurements and adverse events. The electronic data capture design is also important, she said. It needs to include automatic edit checks, routine site performance checks and routine analytic data checks based on logic. In ad-dition to the data itself, terminology, codes and formatting need to be standardized.

But the real challenge is not in collecting data per se, Harrold said, but in how to col-lect “regulatory-grade” data.

From the FDA’s point of view, the most important concern is that the RWD be fit for purpose, said Jacqueline Corrigan-Curry, director of the Office of Medical Policy in the agency’s Center for Drug Evaluation and Research. It must be relevant, representing

the conditions it is supposed to reflect and must meet key quality standards.

Patient-generated data is considered critical for creating real-world evidence (RWE) that can be used for regulatory decisionmaking. Patient surveys are one way of getting this information, but more and more, researchers and healthcare providers alike are turning to technologies capable of objectively measuring patient statistics and reporting that information electronically in real time.

The FDA provides one model of an effec-tive RWD ecosystem. Amy Abernethy, FDA principal deputy commissioner of food and drugs, discussed the COVID-19 Evidence Accelerator, an initiative by the Reagan-Udall Foundation for the FDA in collaboration with Friends of Cancer Research. The Evidence Ac-celerator provides a venue for the collection and quick turnaround and sharing of data and results related to COVID-19 research.

“At its heart, an RWD ecosystem is an RWD community, with people sharing data and ideas,” Abernethy said. The Evidence Accelerator relies on the following features

to work toward that goal in the COVID-19 research and treatment communities:

} Common data elements;} FDA-provided translation tables be-

tween common data models;} Common protocols, including ideas for

master protocols;} Parallel analysis, including how to

review and use different findings and how to handle lack of convergence;

} Individual accelerator communities focused on certain topics; and

} Frequent meetings and forums for rapid cycle feedback and learning.

The draft principles governing the Evidence Accelerator also reflect priori-ties already identified within the clinical research industry, including respect for patient privacy, transparency about how data is collected and analyzed, traceability and provenance of the data, and prompt dissemination, not only to other research-ers but back to healthcare providers for use in treatment plans.

But merely collecting data is not suf-ficient to create an ecosystem in which all stakeholders have ready access to both the data and its analysis. Work spurred by the COVID-19 pandemic has shown that re-searchers can perform extraordinarily well both in terms of generating and analyzing data at speed and sharing results, said Lesley Curtis, professor and chair of the Department of Population Health Sciences in the Duke Medical Center.

“We need to make sure we keep some of these attributes going forward,” Curtis said.

Building an RWD “Ecosystem”continued from page 1 “We are trying to plug

[real-world data] into a model not designed to accept it.”

—Stephanie Reisinger, vice president and general manager of Veradigm

Life Sciences

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Drug & Device Pipeline News

Company Drug/Device Medical Condition Status Sponsor ContactCOVID-19 Trials and ActionsDynavax

Medicago

Medicago’s Coronavirus Virus-Like Particle (CoVLP) with Dynavax’s CpG 1018

COVID-19 vaccine first patient dosed in phase 1 study

dynavax.com

medicago.com

Junshi Biosciences JS016 SARS-CoV-2 patient enrollment complete in phase 1 trial

junshipharma.com

BioSig Technologies/ViralClear Pharmaceutical

merimepodib advanced COVID-19 enrollment of first patients in phase 2 trial

biosig.com

Bellerophon Therapeutics

INOpulse inhaled nitric oxide (iNO) therapy

COVID-19 patients requiring supplemental oxygen

dosing of first patient in phase 3 trial

bellerophon.com

NeoImmuneTech NT-I7 (efineptakin alfa) adults with mild COVID-19 IND approved by the FDA neoimmunetech.comBioNTechPfizer

BNT162b1 and BNT162b2

COVID-19 vaccine Fast Track designation granted by the FDA

BioNTech.depfizer.com

electroCore gammaCore Sapphire CV noninvasive vagus nerve stimulation

adults with COVID-19 experiencing exacerbation of asthma-related dyspnea and reduced airflow, and for whom approved drug therapies are not tolerated or are insufficient

Emergency Use Authorization (EUA) granted by the FDA

electrocore.com

Other Trials and ActionsESSA Pharma EPI-7386 metastatic castration-resistant

prostate cancer patients who failed standard of care treatments

dosing of first patient in phase 1 trial

essapharma.com

JS InnoPharm and Strategia Pharmaceutical

JSI-1187 relapsed, refractory solid tumors with MAPK mutations

dosing of first patient in phase 1 trial

strategiatx.com

Landos Biopharma NX-13 inflammatory bowel disease dosing of first patient in phase 1 trial

landosbiopharma.com

Oncorus ONCR-177 alone and in combination with Keytruda

advanced and/or refractory cutaneous, subcutaneous or metastatic nodal solid tumors

initiation of phase 1 trial oncorus.com

Ribomic RBM-007 achondroplasia dosing of first patient in phase 1 trial

ribomic.com

Rocket Pharmaceutical

RP-L301, lentiviral vector (LVV)-based gene therapy

pyruvate kinase deficiency dosing of first patient in phase 1 trial

rocketpharma.com

Ziopharm Oncology CD19-specific CAR-T, using its Rapid Personalized Manufacturing (RPM) technology

relapsed CD19+ leukemias and lymphomas

initiation of phase 1 trial ziopharm.com

Calithera Biosciences

CB-280 adults with cystic fibrosis and chronic airway infection

dosing of first patient in phase 1b trial

calithera.com

Galera Therapeutics avasopasem manganese (GC4419) in combination with stereotactic body radiation therapy

locally advanced pancreatic cancer patient enrollment complete in phase 1b/2a trial

galeratx.com

continues on next page »

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Drug & Device Pipeline News (continued from page 10)

Company Drug/Device Medical Condition Status Sponsor ContactHistogen HST 001 androgenic alopecia in men patient enrollment complete

in phase 1b/2a trialhistogen.com

Adverum Biotechnologies

ADVM-022 diabetic macular edema enrollment of first patient in phase 2 trial

adverum.com

Arcutis Biotherapeutics

ARQ-252 chronic hand eczema initiation of phase 2b trial arcutis.com

BridgeBioQED Therapeutics

infigratinib achondroplasia first patient dosed in phase 2 trial

bridgebio.comqedtx.com

Byondis B.V. [vic-]trastuzumab duocarmazine (SYD985)

HER2-expressing recurrent, advanced or metastatic endometrial cancer

initiation of phase 2 trial byondis.com

Valbiotis TOTUM-63 reduction of type 2 diabetes metabolic risk factors

initiation of phase 2/3 trial valbiotis.com

Albireo odevixibat biliary atresia enrollment of first patient in phase 3 trial

albireopharma.com

BiogenEisaiAlzheimer's Clinical Trials Consortium

BAN2401 preclinical Alzheimer's disease initiation of phase 3 trial biogen.comeisai.comactcinfo.org

LintonPharm catumaxomab advanced gastric cancer with peritoneal carcinomatosis

initiation of phase 3 trial lintonpharm.com

Tonix Pharmaceutical

TNX-102 SL (cyclobenzaprine HCl sublingual tablets)

management of fibromyalgia patient enrollment complete in phase 3 trial

tonixpharma.com

Knopp Biosciences KB-3061 KCNQ2 epileptic encephalopathy Rare Pediatric Disease designation granted by the FDA

knoppbio.com

Ascentage Pharma APG-2575 Waldenström macroglobulinemia Orphan Drug designation granted by the FDA

ascentagepharma.com

Inflazome Inzomelid Cryopyrin-Associated Periodic Syndrome

Orphan Drug designation granted by the FDA

inflazome.com

Revamp Medical Doraya device percutaneous device for the treatment of acute heart failure

Breakthrough Device designation granted by the FDA

revampmedical.com

Allergan Botox (onabotulinumtoxinA)

spasticity in pediatric patients 2 years of age and older, including those with lower limb spasticity caused by cerebral palsy

approved by the FDA for expanded indication

allergan.com

Baxter International Altapore Shape Bioactive Bone Graft

enhance bone growth and help achieve fusion in skeletal surgeries

approved by the FDA baxter.com

Edward’s Life Sciences

KONECT RESILIA aortic valve conduit

complex aortic valve surgeries approved by the FDA edwards.com

Ipsen Dysport (abobotulinumtoxinA)

upper and lower limb spasticity in pediatric patients two years of age and older, including spasticity caused by cerebral palsy

approved by the FDA for expanded indication

ipsen.com

Janssen Tremfya (guselkumab) adults with active psoriatic arthritis approved by the FDA for expanded indication

janssen.com

Paragon 28 APEX 3D ankle replacement system approved by the FDA paragon28.com

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Clinical Research CoordinatorPharmaceutical Research & Consulting, Inc. Dallas, TX

Clinical Research Coordinator - Part-TimeAB Clinical Trials Las Vegas, NV

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