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see Precision Medicine Trials on page 6 »

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March 9, 2020

Industry Briefs…2

Up and Coming…4

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

Research Center Spotlight…9

CenterWatchWeekly

By Suz Redfearn

Designers of precision medicine trials need access to more data sources than traditional trials to

refine the parameters of their studies and boost enrollment.

Integrating information from a variety of sources — electronic medical records (EMR), lab results, imaging and molecular and claims data — can help trial designers make decisions about inclusion/exclusion criteria, geographic availability of patients, use of technology, statistical analysis methods and trial cessation.

Of 323 pharma executives respond-ing to an NTT DATA and Informa Pharma Intelligence survey, 81 percent cited understanding the patient population as

the most important factor in developing a precision medicine program. To achieve understanding, trials need to integrate information from patient records, other trials’ data and eligible investigators, said Mark Philhower, senior director of NTT DATA’s healthcare and life science industry vertical.

“Evaluating solutions that integrate information across multiple data sources” was the second most important factor, with 11 percent of respondents.

Geographic location was the most sig-nificant factor in defining inclusion/exclu-sion criteria for trial recruitment, according to 66 percent of survey respondents. “Too often, study participants are narrowly

Precision Medicine Trials Rely on Data Integration, Survey Shows

By Leslie Ramsey

A new, detailed template for protocol development is available to sponsors of medical device trials that rely on

real-world evidence (RWE).The 38-page template sets out a 12-part

framework for building a protocol and provides a set of guiding principles for medical device trial designers, including set-ting study objectives, identifying the target population and describing trial procedures, the monitoring plan and statistical analysis of results.

The template was developed by the Na-tional Evaluation System for Health Technol-ogy Coordinating Center (NESTcc), a consor-tium of device manufacturers, health systems, academic institutions and the FDA.

In the section on Study Specific Objec-tives, NESTcc recommends explaining how the specific objectives were chosen in line with the SMART principle (specific, measur-able, attainable, relevant and time-framed). It also wants to know how statistical significance will be defined and whether the product being assessed is a device system or a specific component.

The general principles to follow in the section on Target Population, Patient Selec-tion and Source for Patient Recruitment include the use of objective criteria for defining inclusion and exclusion features. It also asks devicemakers to provide a high-level description of steps taken to assess data quality as described in the NESTcc Data

New Template Guides Protocol Development for Medical Device Trials Using RWE

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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-

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Industry Briefs

Study Finds Average Cost of $1.3 Billion for Drug R&DDrugmakers spend an average of $1.3 billion in research and development costs to get a new medicine to market, according to a new study reported in JAMA last week, a number considerably lower than a 2016 Tufts University report citing development costs of $2.8 billion.

The research team from the London School of Economics and Political Sci-ence, the London School of Hygiene and Topical Medicine, and the Leuven Institute of Healthcare Policy looked at 63 FDA-approved drugs developed by 47 pharma companies between 2009 and 2018 and estimated the R&D costs for each one based on data available to the public, finding an average cost of $1.3 billion. The 2016 Tufts report was based on 106 randomly selected new drugs from just 10 companies.

Per product, the median R&D cost was $985 million, including the expenses from failed trials, the researchers said. Investments ranged from $143 million for Jaguar Health’s Mytesi (crofelemer), an anti-diarrheal drug for HIV patients, to $6.4 billion for Sanofi and Re-generon’s blockbuster Dupixent (dupilumab), an eczema biologic.

Development costs differed widely depend-ing on the drug’s therapeutic area. The median cost for nervous system drugs, for example, was $765.9 million, while the most expensive area, cancer, had a median cost of $2.7 billion.

Access the JAMA study here: https://bit.ly/ 2x6i3yF.

Complex Trial Design Pilot Has Seen 50 Percent Acceptance Rate, FDA SaysFour of eight complex trial designs submitted in the FDA’s complex innovative design (CID) pilot program have been accepted for review, the agency said Monday.

The five-year pilot has accepted CIDs in several therapeutic areas, including treat-ments for Duchenne muscular dystrophy, pediatric multiple sclerosis, systemic lupus

erythematosus and multiple interventions across multiple pain conditions by sponsors Eli Lilly, Amgen and others.

The agency denied half of the submissions based on a lack of clarity on endpoints and a low level of innovation, said Dionne Price, director of the FDA’s Division of Biometrics IV, during a two-day Drug Information As-sociation meeting held in Silver Spring, Md. Although the designs were rejected for the pilot program, sponsors may continue using other regulatory routes, she said.

Sponsors whose designs are accepted for review in the pilot that was launched in August 2018 get increased access to the agency, including two primary meetings and two alternate meeting requests per quarter. Prior to the meetings, the FDA and partici-pants work out a disclosure agreement on what information the agency may include in public case studies.

Price hailed the program’s disclosure ele-ment as one of its greatest benefits because it allows for discussions to happen while pre-INDs are being investigated. “The disclosure piece allows us to talk about pre-INDs while the drugs are still under investigation, in an effort to advance science. That has been a key success from my perspective.”

Test Topical Skin Products for Risk of Contact Dermatitis, FDA Guidance SaysClinical trials of drug products intended for topical application should assess the risk of contact dermatitis, a new FDA draft guid-ance says.

The guidance advises researchers to use static scales, such as current state and noncomparative methods, to evaluate signs of cutaneous issues such as erythema, edema and erosion.

Patient-reported outcome measures should be used to assess symptoms such as pruritus or burning.

Trials should plan the timing and frequency of assessments to identify anticipated reactions and characterize suspected adverse reactions using patch or photopatch testing with the active and excipient ingredients as well as the finished product.

The deadline for comments on the draft guidance is May 8.

Read the guidance here: https://bit.ly/3a uJ7Gc.

Increase Representation of the Elderly in Cancer Trials, FDA Guidance SaysThe FDA is encouraging cancer trials to pay greater attention to patients age 75 and older, according to a draft guidance issued last week.

Adults age 65 and older, especially those over 75, are underrepresented in clinical re-search on cancer despite comprising a grow-ing portion of the cancer patient population, the agency said.

Sponsors need to discuss their enroll-ment goals with sites and investigators to achieve greater participation of elderly patients. The guidance also recommends

continues on next page »

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WCG | CWWeekly March 9, 2020 3 of 9

Industry Briefs (continued from page 2)

recruiting more physicians specializing in geriatric oncology as investigators.

Because outcomes of cancer patients age 65 and older may vary by age, the guidance encourages sponsors to evaluate data by subgroups, for example, patients age 65 to 74 vs. patients age 75 or older.

Comments on the draft guidance are due May 6.

Read the guidance here: https://bit.ly/2w w9MDZ.

Pharma Industry Outpaces NIH, Academia in Sponsoring Gene Therapy TrialsPharma companies outstrip all other spon-sors of gene therapy trials, running 40 per-cent compared to 25 percent by hospitals, 25 percent by universities and 10 percent by the NIH.

Sponsorship varied by trial type and phase, according to a new study published in JAMA, which tracked 341 gene therapy trials posted to clinicaltrials.gov.

Industry sponsored 61 percent of in vivo trials, 61 percent of trials for therapies correcting genetic defects, 63 percent of noncancer trials, 27 percent of ex vivo trials, 30 percent of trials for therapies not cor-recting genetic defects and 27 percent of cancer trials. Industry sponsored 22 percent of phase 1 trials, 55 percent of phase 2 trials and 100 percent of phase 3 trials.

The study, which came from authors at the University of Pittsburgh School of Medi-cine and Brigham and Women’s Hospital in Boston, also looked at the funding of gene therapy trials.

Pharma companies were the sole funder for 36 percent of gene therapy trials com-pared to 50 percent for NIH and academia, with the remaining 14 percent coming from collaborations among the three fund-ing groups.

To read the study, click here: https://bit.ly/ 2TpGxf2.

FDA Releases Request for Applications for Funding of Rare Disease TrialsThe FDA will begin accepting applications for grants for 2020 beginning this August for trials of drugs that address unserved needs in rare diseases.

The agency’s Office of Orphan Prod-ucts Development awards $15 million a year — $10 million to ongoing trials and $5 million to new trials. In 2019, the FDA awarded grants to 12 applicants out of 89 received.

The FDA encourages applicants to apply early to allow adequate time to make cor-rections. The deadline for 2020 applications is Oct. 6, and the deadline for next year is Oct. 5, 2021.

To read the FDA’s request for applications, click here: https://bit.ly/2POBliw.

Study Identifies Disparities in Clinical Trial Access in U.S.A lack of geographically convenient trial sites hinders study participation, especially for rural residents.

A JAMA study published last week con-cluded that unequal distribution of trial sites relative to population may impede patients’ participation in trials and contribute to the well-documented low representation of minorities in many trials.

Data on population, median household income, racial demographics, funding and number of U.S. inpatient hospital beds used in trials was collected.

Factors related to trial recruitment and retention, not the location of trial sites, are most likely responsible for racial and socioeconomic inequities in trial access and participation, the study concluded.

The study was conducted by authors from Pharmagellan, Boston University School of Public Health and Columbia University.

To read the study, click here: https://bit.ly/ 2xeEPVj.

New Illinois Law Ensures All Patients with Cancer Can Participate in Clinical TrialsA new law in Illinois will allow low-income patients with cancer from underserved com-munities to be reimbursed for clinical trials within 30 days.

The Cancer Clinical Trial Participation Act will reimburse patients for out-of-pocket expenses, including costs associated with traveling to sites. Only patients who meet certain criteria on a sliding scale of income will receive financial assistance.

Laws similar to the Illinois law are in effect in California, Pennsylvania and Texas. Similar legislation is being consid-ered in Florida, Massachusetts, New Mexico and Wisconsin.

Financing Round Secures $14 Million for Inato’s Clinical Trials Matching PlatformFrench company Inato has secured $14 million for its platform that matches clinical trials with sponsors and sites. The new funds will help the organization improve the predictive capa-bilities of its platform and also help develop a global clinician network.

The funding comes from Obvious Ventures, Cathay Innovation, Serena and Fly Ventures. Prior to this financing round, Inato has already used its platform for 50 clinical trials across 2,000 global research sites.

Owkin and University of Pittsburgh to Launch Pilot on AIStartup Owkin is collaborating with the Uni-versity of Pittsburgh to launch a pilot pro-gram that marries its artificial intelligence and Pitt’s trial datasets and research findings to come up with better trial designs, more effective drug development strategies and biomarker discovery.

Owkin’s federated network produces research from data in clinical trials at U.S. and European academic medical centers.

© 2020 CenterWatch CWW2410

This feature highlights changes in clinical trial organizations’ personnel.

Altavant SciencesHoward Lazarus is now chief medical officer of Altavant Sciences, joining the company from Boehringer Ingelheim Pharmaceuticals as the medical expert/executive director of clinical development and medical affairs.

AstraZenecaAlicyn Campbell has been appointed to head of digital health oncology R&D at Astra-Zeneca. Campbell recently led an oncology patient-centered outcomes research group at Roche/Genentech.

BlueRock TherapeuticsSeth Ettenberg is now the chief scientific officer for BlueRock Therapeutics, joining the firm after previously serving as chief scientific officer of Unum Therapeutics.

CureVacAlan Kimura has been appointed to head the rare disease department at CureVac. Recently, Kimura was the chief medical officer at Enzyvant.

Eloxx PharmaceuticalsEloxx Pharmaceuticals has named Gregory Williams the firm’s new CEO. Williams most recently served as the company’s chief operating officer. Eloxx has also announced the appointment of Neil Bel-loff to the role of chief operating officer.

Previously, Belloff was the senior corporate counsel at Celgene.

GlaxoSmithKline IndiaSridhar Venkatesh has been appointed to the role of managing director and vice presi-dent of GlaxoSmithKline India. Venkatesh has held multiple roles at Avalara GlaxoSmithKline India prior to this appointment.

Global GenesSusan Brisendine has been named vice president of engagement programs at Global Genes, joining the company after serving as the chief architect of the global cell and gene medicine educational pro-gram at the ARM Foundation for Cell & Gene Medicine. Global Genes has also appointed Christian Rubio as vice president of strate-gic advancement. Rubio previously served as vice president of marketing at PlatformQ Health. Pam Rattananont has joined Global Genes as the vice president of development and marketing.

KBI BiopharmaKBI Biopharma has promoted Dirk Lange to the position of CEO. Prior, Lange was the company’s chief operating officer.

NouscomImmuno-oncology company Nouscom has named Marina Udier the company’s CEO. Previously, Udier was chief operating officer at Nouscom. Additionally, Nouscom announced the appointment of Patricia Delaite to the role of chief medical officer. Prior to this ap-

pointment, Delaite was chief medical officer at AMAL Therapeutics.

OculisOculis has appointed Marcia de Souza Lima to the role of chief medical officer. Most re-cently, de Souza Lima served as the vice presi-dent and head of global medical affairs for the global ophthalmology franchise at Shire.

Rafael PharmaceuticalsSanjay Sehgal has been named chief of regulatory affairs and quality assurance officer at Rafael Pharmaceuticals, a newly created position within the organization. Previously, Sehgal was senior vice president of regulatory affairs and conformance at Celularity.

TheramexRobert Stewart has been appointed CEO of Theramex, replacing Anish Mehta who has stepped down from the position. Stewart was most recently the CEO of Amneal.

TheratechnologiesPaul Lévesque has been appointed president and CEO of Theratechnologies, replacing newly retired Luc Tanguay. Lévesque will hold his current position of global president of Pfizer’s rare disease division until he assumes his new position at Theratechnologies.

UNITY BiotechnologyAnirvan Ghosh will now act as UNITY Biotech-nology’s CEO, having been tapped from his pre-vious position as senior vice president and head of research and early development at Biogen.

WCG | CWWeekly March 9, 2020 4 of 9

Up and Coming

CONTACT SALES4 sales@centerwatch.com 617.948.5100

Your leading resource for trends, analysis, compliance guidance, expert insights and more. With original content and exclusive interviews in each issue, The CenterWatch Monthly is like having your own full-time research team helping you understand how and why the industry is changing.

By John W Mitchell

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

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

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

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

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

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

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

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

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

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

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

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

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

September 2018 A CenterWatch Publication Volume 25, Issue 09

see Site Viability on page 7

By John W Mitchell

C linical trial site finances are a problemthat keeps getting bigger. While there’sagreement improvements are on the

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

gish payments, contract viability, preferredsite status, interconnectivity and somethingless tangible — a lack of consideration in therelationship.

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

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

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

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

see Start-up Costs on page 6

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WCG | CWWeekly March 9, 2020 5 of 9

Features

New Template Guidescontinued from page 1

The 38-page template sets out a 12-part framework for building a protocol and provides a set of guiding principles for medical

device trial designers.

—The National Evaluation System for Health Technology Coordinating Center

Quality Framework, which was released last week and focuses primarily on the use of EHR data in the clinical care setting.

In the section on Outcomes, NESTcc says that sponsors of medical device trials must provide clear definitions of primary and secondary endpoints and method of outcomes assessment. Sponsors must also list specific procedure outcomes, such as procedure time, physiological and biological data captured as part of the procedure, and procedure-specific data. On device out-comes, sponsors must clearly identify which features of the device will be measured. On control outcomes in observational studies, sponsors need to describe why the outcome is highly unlikely to be causally related to the device or comparator. And the outcome schedule must include timing of patient evaluation, a rationale for both short-term and late outcomes, and pre-specification of a list of potential adverse effects.

NESTcc recommends in the section on Patient Exposure to the Device that expo-sure definitions should be as specific and detailed as possible. General principles to follow include describing the units for expo-sure measurement, the precision with which exposure will be measured, the approach to confirming exposure to the device, and the training and experience of the device opera-tor/surgical team.

In the section on Study Design, NESTcc says fundamental features required include the number and type of comparison groups,

blinding and outcomes. General principles include justifying the choice of design as precisely as possible; defining the primary study objective; and if a randomized trial, a description and justification of the treat-ment allocation.

On the subject of blinding, NESTcc says that to the extent possible, whether a randomized or observational study, proper blinding/masking is encouraged. The Study Design section has guidance on the units of randomization, observation and analysis.

NESTcc also addresses the issue of mecha-nism of treatment assignment. NESTcc says in randomized trials, the treatment assignment mechanism is described as known because the investigators have control of the process. In observational studies, the treatment assign-ment mechanism is characterized as unknown and must be estimated.

In the section on Study Procedures, NESTcc says a clear description of how the study will be conducted should be included in the pro-

tocol. Information regarding how patients are approached and consented, how randomiza-tion will be conducted, how data will be col-lected, definitions of protocol deviations and how those will be treated, what constitutes subject withdrawal or discontinuation, and what stopping rules will be used, if applicable, should also be included.

On the subject of Required Sample Size, NESTcc recommends indicating the type of study design, the approach to evaluation and justifying additional features of the trial that impact sample size, such as the adjustment for multiplicity or clustering, the approach to controlling for confounding variables, prevalence/incidence rates and accounting for missing data.

NESTcc says that trials should be regis-tered on the clinicaltrials.gov website prior to enrolling the first patient, with no exceptions. Observational trials could also be registered at the NESTcc website or at the Center for Open Science, https://bit.ly/3avUVIj.

The consortium’s recommendations also emphasize the importance of data safety monitoring boards/committees. Finally, NESTcc includes the general principles to follow on the Statistical Analysis Plan that includes the treatment of missing data, specifying the use of statistical software and providing a plan for adjustment for multiplicity of all endpoints. In conclusion, the framework points out that additional considerations will be required when assess-ing imaging modalities and diagnostics.

To read the framework, click here: https://bit.ly/32SoS2V.

SOP Highlights include:nn New requirements established in the

2016 revision of ICH E6nn New FDA guidances, including new

data integrity expectationsnn The impact of risk-based monitoring

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Standard Operating Procedures for Good Clinical Practice by Sponsors of Clinical Trials: Drugs and Biologics 33 SOPs and 82 attachments that can be customized to meet your needs

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Features

Precision Medicine Trialscontinued from page 1 “The challenge will be to translate

and apply these discoveries to affect precision diagnosis and

precision therapy for improved patient outcomes.”

—Mark Philhower, senior director of NTT DATA’s healthcare and life science

industry vertical

defined, but geographically diffused,” Philhower said.

The second most important factor to consider in recruiting was medical/dis-ease condition or progression, cited by 24 percent of respondents. Genetic composi-tion has the biggest impact on progres-sion, said 61 percent, followed by disease pathogenesis (19 percent), patient behav-ioral characteristics (12 percent) and social circumstances (8 percent).

The most valuable data sources for measuring disease progression are radiology data, clinical data (lab data, EMRs, genomic data) and pathology data, respondents said. Information on responsiveness to therapy and the number of lines of therapy used also is important, they said.

EMRs are the key source for identify-ing clinical trial populations, 58 percent of respondents said. Physician referrals, labora-tory diagnostics and healthcare claims scored more modestly.

Retaining patients in precision medicine trials relies most heavily on patient behavior, according to half of respondents. Efforts to influence behavior positively include patient

counseling, community and family coordina-tion, and provision of resources, such as lodg-ing, travel and meals.

Questioned on specific technologies they use to improve patient retention, 83 percent of respondents referred to such traditional communication channels as email, text or voicemail messages. Survey responses showed some evidence of emerging digital technolo-gies coming into play, however, with virtual digital assistants, such as Amazon’s Alexa and Google Assistant, scoring 6 percent, and social robots like Jibo, Pepper and Aibo 1 percent.

The survey also shed light on the potential for using machine learning in precision medi-cine trials and therapeutic programs.

“The new [artificial intelligence (AI)] mod-els have potential to offer preventive, diagnos-tic and treatment options and alternatives to healthcare practitioners, supplementing their expertise and insights at a resolution and scale unimaginable just a few years ago,” said Philhower. “The challenge will be to translate and apply these discoveries to affect precision diagnosis and precision therapy for improved patient outcomes.”

Among the emerging technology drivers for precision medicine, statistical analytics was ranked as the top priority for 70 percent of survey participants. Statistical packages, such as cluster or regression analysis tools, were favored by 71 percent of respondents. Excel-based modeling tools followed with 17 percent and AI/machine learning at 9 percent.

Forty-one percent of respondents in data management and analytics roles preferred empirical and system-progression models for quantifying disease progression, followed by semi-mechanistic models at 16 percent. Clini-cal operations specialists, however, favored clinical (33 percent), laboratory (27 percent), pathology (20 percent) and radiographic methods (19 percent).

To read the survey, click here: https://bit.ly/2vzGNiG.

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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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Drug & Device Pipeline News

Company Drug/Device Medical Condition Status Sponsor ContactMiNa Therapeutics MTL-CEBPA in

combination with Keytruda (pembrolizumab)

advanced solid tumors initiation and first patient treated in phase 1/1b trial

minatx.com

TargoVax SA ONCOS-102 in combination with Keytruda (pembrolizumab)

advanced anti-PD1 checkpoint inhibitor (CPI) refractory melanoma

patient enrollment completed in phase 1 trial

targovax.com/en/

HUYA Bioscience International

HBI-3000 cardioversion of atrial fibrillation completion of phase 1 trial huyabio.com

Dynacure DYN101 myotubular and centronuclear myopathies

first patient dosed in phase 1/2 trial

dynacure.com

Allergan plc

Editas Medicine

AGN-151587 (EDIT-101) Leber congenital amaurosis 10 (LCA10)

first patient dosed in phase 1/2 trial

allergan.com

editasmedicine.com

Arrowhead Pharma ARO-HSD patients with NASH or suspected NASH

first patients dosed in phase 1/2 trial

arrowheadpharma.com

DiscGenics IDCT, allogeneic, injectable discogenic cell therapy

degenerative disc disease completed enrollment in phase 1/2 trial

discgenics.com

Novus Therapeutics OP0201 pediatrics with acute otitis media completed enrollment in phase 2a trial

novustherapeutics.com

VBI Vaccines VBI-1901 in combination with either GM-CSF or AS01B, (GlaxoSmithKline)

recurrent GBM first patient dosed in the second study arm of phase 2a trial

vbivaccines.com

Innovent Biologics IBI375 (pemigatinib) advanced cholangiocarcinoma with FGFR2 fusions or rearrangements

first patients dosed in phase 2 trial

innoventbio.com/en

Neuraly NLY01 Parkinson’s disease first patient dosed in phase 2 trial

neuralymed.com

Moderna mRNA-1647 cytomegalovirus completed enrollment in phase 2 trial

modernatx.com

American BriVision ABV-1505 adult attention-deficit hyperactivity disorder

initiation of phase 2 trial ambrivis.com

EpicentRx AIM-001 tumors IND approved by FDA epicentrx.com

Prevail Therapeutics PR006 frontotemporal dementia patients with GRN mutation

IND approved by FDA prevailtherapeutics.com

Ayala Pharmaceuticals

AL101 recurrent or metastatic adenoid cystic carcinoma

Fast Track designation granted by FDA

ayalapharma.com

Genentech Esbriet (pirfenidone) adults with unclassifiable interstitial lung disease

Breakthrough Therapy Designation granted by FDA

gene.com

Sanofi Sarclisa (isatuximab-irfc) in combination with pomalidomide and dexamethasone (pom-dex)

adults with multiple myeloma who have had two previous therapies including lenalidomide and a protease inhibitor

approved by the FDA sanofi.com

© 2020 CenterWatch CWW2410

WCG | CWWeekly March 9, 2020 9 of 9

Research Center Spotlight

Research Center Spotlight is a monthly selection of clinical research centers who have Research Center 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 expertise and services in conducting and managing clinical studies.

To learn more about becoming a Research Center Profile page subscriber, contact Sales at 617.948.5100 or sales@centerwatch.com.

Miami, FL 305.225.0400 strinidad@intldermresearch.com

International Dermatology Research is a privately owned practice specializing in dermatology. Established in 1993, it has success-fully completed more than 500 clinical trials. It specializes in phase 2, 3 and 4 trials in dermatology, dermatology devices and cosmetics.

Maitland, FL 407.636.6961 lgoggin@cfendocrine.com

Central Florida Endocrine & Diabetes Consultants is a private practice with a staff of six endocrinologists, a podiatrist, dietitian, diabetic educa-tor, certified clinical research nurse and a large supportive medical staff.

Los Angeles, CA 310.966.9022 sharon@calallergy.com

California Allergy and Asthma Research is an independent, free-standing clinical research facility serving the greater Los Angeles area.

Methuen, MA 978.655.7155 tstubbs@activmedresearch.com

ActivMed Practices & Research is a registered woman-owned company operating in Massachusetts and New Hampshire since 1994. It offers a multi-specialty free-standing research facility with experienced research physicians, psychiatrists and highly trained coordinators.

Palmdale, CA 661.266.8944 sharon@calallergy.com

Antelope Valley Clinical Trials is an independent, free-standing clinical research facility located in Southern California and serving the Antelope Valley and northern Los Angeles County.

Raleigh, NC 919.781.2514 pifs@wakeresearch.com

Wake Research is a unique and innovative resource for phase 1-4 clinical trials. Our site is known for effectively combining strategic patient re-cruitment and retention with high-quality clinical research procedures.

Bingham Farms, MI 248.644.7770 dolly@questri.com

QUEST Research Institute has successfully completed 350 phase 1-4 clinical protocols in the past 20 years. It is the first dedicated research center of its kind to offer southeastern Michigan access to promising medications under strict safety guidelines.

Indian Harbour Beach, FL 321.773.7119 ConciseReport@earthlink.NET

OsNET is a multi-specialty informal alliance of 10 sites located throughout nine states. Its members’ studies focus on osteoporosis, arthritis, obesity, COPD, ACS, diabetes, vaccines, sleep disorders, OC and women’s health.

New Windsor, NY 844.337.8839 wchrvala@mhmresearch.com

Established in 2007, Mid Hudson Medical Research is a dedicated research site/private group practice.

Miami, FL 305.226.3933 stevenh@nhorizonresearch.com

New Horizon Research Center is a dedicated, multi-specialty clinical research site serving the Miami community. Founded in 2009, it has conducted more than 100 clinical trials.

Mid Hudson Medical Research

New Horizon Research Center

ActivMed Practices & Research

Antelope Valley Clinical Trials

California Allergy & Asthma

Wake Research Associates International Dermatology Research

OsNET

Central Florida Endocrine and Diabetes Quest Research Institute