Clinical Trials in the Age of Personalized Cancer Medicine: The Evolution of a More Efficient,...

Clinical Trials in the Age of Personalized Cancer Medicine: The Evolution of a More Efficient, Patient Focused Clinical Research System

Session Chair: Eric LynamVice President Business DevelopmentPharmatech Oncology Inc.

46th Annual MeetingWashington, DC - 2010

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Session Update

• Program Substitution – Eric Lynam DH for• Matthew B. Wiener, Pharm.D.• Pharmatech Oncology Founder and COO• Unable to participate today due to injury


Session Learning Objectives

• Define rare cancers by histological and molecular definitions

• List three challenges in conducting oncology clinical trials in rare cancers

• Discuss modifications in workflow that enable greater research efficiency through patient-focused project management

• Discuss regulatory solutions which permit patient focused research while providing proper oversight and patient protection

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Clinical Trials in the Age of Personalized Cancer Medicine

• ASCO 2009: ‘Personalizing Cancer Care’• ASCO 2010: ‘Advancing Quality Through

Innovation’

• “Oncology is no longer a one-size-fits-all medicine" – Richard Schilsky, MD

• "We are increasingly able to tailor treatments to an individual's or to a tumor's unique biology, which ultimately helps us to improve outcomes for patients by matching the right treatment to the right patient at the right time.“


Challenges to Oncology Clinical Trials

• US cancer patients are treated in thousands of independent practices

• Participation is clinical trials remains low (<5%)• How to connect the right investigational treatments

to the right patients to develop targeted therapies?• How to deliver investigational treatment options to

more patients within context of patient care?• How to accelerate research and ensure protection of

patients, in compliance with GCP and CFR 21?


Dr. Jeffrey Vacirca

• Clinical Trials in Oncology: Re-examining the Site-based Research Paradigm


The Evolution of a More Efficient, Patient Focused Clinical Research System

• Migration from a site centered system to a patient and treatment focus

• Patient first system requirements – Network of qualified, research ready sites– Identify candidate patients where they present– Connect patients to appropriate clinical trial treatment

opportunities– Rapid trial entry – Need to treat in weeks not months– Logistical Alignment – Site supply, initiation, training– Full compliance with CFR 21, GCP and all study

oversight measures


Just In Time Research Model

Protocol Review

Wait for Patients (weeks, months, or more)

Site Based Research

Protocol Review

Look for Patients

Patient Based JIT Research

Cost & Risk

Avg. 10 Days

IRB SiteApproval

SIVTraining

Pre-IdentifyPatient

Qualify,Consent, &

Enroll

Diminishing Study Awareness

Pre-IdentifyPatient

IRB SiteApproval

SQV/SIVTraining

Qualify, Consent, &

Enroll

SQV


What Makes Just In Time a Potential Solution?

• Higher throughput patient ID and enrollment rate– Broader detection by many sites– Immediate enrollment increases trial enrollment rate

• Alignment of administrative activities to immediate enrollment opportunities– No lag between site initiation and first patient consent

• Few or zero non-enrolling sites– Efficient use of sponsor resources, materials, start up

costs• Secondary enrollment

– X Factor. High study awareness


JIT Research and Protection of Human Subjects

• Can research administration be accelerated and still comply with CFR 21 and GCP?

• Are patient rights properly protected in a JIT research model?


Matt Baker

• Protecting Patient Rights and Regulatory Compliance in a Patient-Directed Clinical Research System


Feasibility Of JIT Research ModelCollaboration Between Sponsor and CRO

Pros

Eliminate rate limiting steps to activate sites

Use community-based MD practices where study subjects are first diagnosed

Decrease time from site initiation to enrollment

Cost-effective (i.e. sites with no subjects will not be initiated and monitored)

Central IRB, speeds approval of subsequent protocol/ICF changes

Cons

High level of coordination needed to streamline/execute critical steps across cross-

functional groups

Sites potentially require more oversight and training

Resources at sponsor and SMO must be adequate to execute intense pre-initiation

activities

Potential impact of cost may be difficult to measure, large# of sites may be qualified but

not initiated

Sites with pre-identified subjects per SoC may have high screen failure rate, still not enroll

Adapted from S. Gevorkian, F. Patterson PRCT Annual Meeting March 2010


Case Studies

Trial Sites Initiated Months Enrollment Non-Enrolling Sites

Rate (Pt/Site-mo)

CML Traditional 14 23 17 Several 0.052

JIT 14 17 18 0 0.076 Pancreatic

Traditional 16 20 42 2 0.13 JIT 8 7 20 0 0.36

Lymphoma Traditional 24 11 4 20 0.015

JIT 8 9 8 0 0.11


Sponsor Conclusions from CML Trial • More JIT sites that were opened actually enrolled subjects

• More than half of total US subjects enrolled came from JIT sites with few screen failures

• Streamlined processes decreased time to execute steps to qualify, initiate, and enroll subjects

• Model is not ‘one size fits all’: Conduct realistic feasibility assessment whether right for your therapeutic area, study protocol, and organization

– Ensure adequate resources are identified and part of study budget

– Buy-in from different groups within the organization is key and should be sought early in the decision-making process

– Partner with SMO to establish a clear and comprehensive work flow

• Ensure commitment and planning on both sites to execute deliverables in a time sensitive and intense environment

S. Gevorkian, F. Patterson PRCT Annual Meeting March 2010


Future Directions: Patient Focused Oncology Research

• Further R&D – – Solutions like Just In Time– Commercial and NIH supported

• Expanded Connectivity– Trial registries (e.g. ClinicalTrials.gov)– Patient Matching (e.g. advocacy, enrollment support

organization)

• Systematic Integration – National healthcare (e.g. NCI, caBIG)– Patient care - Any patient to any research site


Conclusions - Discussion• Development of personalized cancer therapies requires much greater patient participation in US (5% 20%)

– Greater availability of trials across research practices– Greater access for individual patients to appropriate cancer trials– Meet all regulatory, documentation, and patient protection commitments – Alignment of research activities with patient care needs– One solution currently in development through Just In Time method

• Thank you for your participation!

Clinical Trials in the Age of Personalized Cancer Medicine: The Evolution of a More Efficient,...

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