Wednesday, December 17, 2014 3:00pm-4:30pm EST National Coalition for Cancer Survivorship Post-Training Webinar ©2014 National Coalition for Cancer Survivorship

Wednesday, December 17, 20143:00pm-4:30pm EST

National Coalition for Cancer Survivorship

Post-Training Webinar

©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

How FDA Review Activities Affect Cancer Patients

Elizabeth Goss


FDA and Cancer Patients• FDA undertakes four main activities that affect

cancer patients:oDetermines if protocols submitted under an

investigational new drug (IND) application are safe to proceed

oApproves cancer drugs and biological products for commercial marketing

oWorks with pharmaceutical companies to manage production and to address safety issues that arise

oPermits access to investigational drugs or biological products on a compassionate use basis


Standards for Approval

• To obtain approval of a new drug or biological product, a pharmaceutical manufacturer must:oDemonstrate effectiveness with acceptable safety in

adequate and well-controlled clinical trialsoGenerate information for a product label that: • Defines the patient population for treatment with

the drug• Provides adequate information to enable safe and

effective use of the product


Expedited Review• FDA will expedite the review of new products when that is

appropriateo May grant “breakthrough therapy” status to promising drugso May give priority review status to certain therapieso May approve cancer drugs according to accelerated approval

standards• The investigational cancer therapy must affect a surrogate

endpoint other than mortality or irreversible morbidity• The surrogate endpoint must be reasonably like to predict

clinical benefit• Additional studies must be completed to confirm the

clinical benefit of the drug


FDA and Diagnostic Tests• FDA has proposed a plan for regulatory review of tests that

are used to diagnose disease or guide treatment• Plan is a reversal of long-standing FDA practice of

forgoing review of certain diagnostic tests• FDA is acting to provide greater assurances about the

reliability and utility of tests that diagnose cancer and other diseases and that guide treatment• New plan is risk-based; some tests would be subject to

minimal review and others to more aggressive review• Regulatory plan will be phased in over years• Plan is not final but is open for public comment


Expanded Access and Compassionate Use

• Expanded Access and Compassionate Use:• Means of providing patients access to investigational, or

unapproved, therapies• Require the engagement of treating physician, FDA, and

the company developing the new drug• Manufacturers may decline to provide compassionate

access for a number of reasons• Decisions related to expanded access and

compassionate use require a difficult balancing of the needs of the individual and the interests of the community for completion of clinical trials and approval of new drugs


Discussion Questions•Are changes in the compassionate use and expanded access standards necessary to improve responsible access to unapproved therapies for cancer patients?

•Should incentives be provided to or requirements imposed on drug manufacturers to enhance patient access to unapproved therapies?

Opportunities for Reforming Cancer

Care Delivery Shelley Fuld Nasso


IOM: Cancer Care in Crisis• IOM concluded in 2013 that cancer care is not

adequately:oPatient-centeredoAccessibleoCoordinatedo Evidence-based

• The strains in the system are exacerbated by:oAging populationoRising cost of cancer careoComplexity of cancer careoReliance on family caregivers


Current State of Cancer Care• Buy and Bill system and the “business” of medicine

oManaging inventoryoUsing profit margin to fund other services to patients

• Silos that systematically prevent coordination of care• Payment reform models will allow practices to

innovate without reliance on volume• Patients/survivors/advocates need to be at the

table when discussing care delivery improvements



Delivery & payment reforms essential to quality improvement• NCCS considers changing the conversation

between doctor and patient as the essential foundation of delivery reform• Payment reforms must be aligned to improve

communication, treatment decision-making, symptom management, and coordination of care


NCCS Principles for Patient-Centered Payment Reform• Reward quality of services, not just

quantity• Require shared decision-making and a

cancer care planning process, including advance care planning• Promote evidence-based care by

encouraging adherence to guidelines, quality measures, and standards of care• Include consideration of clinical trial

options, where appropriate, in the care planning discussion


• Communicate patient cost-sharing responsibilities as part of the care planning process• Include protections against underutilization or

overutilization • Incorporate outcome, process, and patient-

reported outcome measures, developed with input from patients• Ensure that therapeutic innovations will be

rapidly incorporated into the standard of care• Reimburse for coordination of care, from

screening through survivorship

NCCS Principles for Patient-Centered Payment Reform


Discussion Questions• How would you change the way the cancer care

team interacts with patients? • Can you provide advice about how office visits

might be changed? • How chemotherapy administration might be

changed? • How cancer treatment side effects are

communicated?


Current Experiments in New Payment

ModelsElizabeth Goss

Efforts to Improve Cancer Care• General movement away from volume-based care and

payment• Emphasis on care planning and coordination• Reliance on practice guidelines• Changes in the processes of care to enhance patient-

centeredness (shared decision-making, 24-hour clinical response, prevention of emergency department visits and inpatient admissions)• Performance-based payments for providers• Quality measures to assess care delivery and patient

outcomes


Incentives and Pressures for Change• Practices undertaking reform on their own

oPatient-centered oncology medical home• Professional societies identifying strategies for reform

oEpisodes of care• CMS Innovation Center has designed a Medicare episode-of-care plan that retains fee-for-service

and incorporates payments for practice transformationoVoluntary for private payersoVoluntary for physicians


Discussion Questions• Does the movement away from volume-based care result in

less care? Does this change affect quality of care?• Are new payment reform systems consistent with the effort

to provide personalized cancer care?• What level of transparency is necessary in payment reform

efforts? oDisclosure of the payment reform effort? Disclosure of

performance-based payments?oDisclosure of how the new payment system affects

patient costs?


Measurement of Quality to Protect

PatientsShelley Fuld Nasso


Measurement of Quality to Protect Patients

• Quality is the right care, at the right time, for the right reason• Quality varies and is inadequate within our own system, by provider,

state and region• Measurement is a way to evaluate and improve care provided by

hospitals, doctors, health plans and other health care entities based on accepted national guidelines or evidence

• Types of measures: process, outcome, patient experience, structural• Measures for cancer should be

o Understandable, meaningful, and usable by consumerso Outcomes-focusedo Based on patient and/or family/caregiver-generated health data


• Symptoms, quality of life and functional status• Choose based on value/meaning to patient• Standardize methodology, demonstrate

reproducibility, gather benchmark standards• Examples-

oPainoNauseaoSatisfaction with healthcare providers

Patient Reported Outcomes


Discussion Questions• Are current quality assessment measures adequate to

assess care and protect patients?• What information is meaningful to patients in assessing the

quality of cancer care delivered?oProcesses of careo Experience of careoOutcomes – survival, quality of life

• What information should be measured and publicly reported to help patients in choosing providers and hospitals for their cancer care?


Next Steps

Thank You

Documents

Wednesday, December 17, 2014 3:00pm-4:30pm EST National Coalition for Cancer Survivorship Post-Training Webinar ©2014 National Coalition for Cancer Survivorship