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

2. How FDA Review Activities Affect Cancer Patients Elizabeth Goss ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

3. FDA and Cancer Patients FDA undertakes four main activities that affect cancer patients: o Determines if protocols submitted under an investigational new drug (IND) application are safe to proceed o Approves cancer drugs and biological products for commercial marketing o Works with pharmaceutical companies to manage production and to address safety issues that arise o Permits access to investigational drugs or biological products on a compassionate use basis ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

4. Standards for Approval To obtain approval of a new drug or biological product, a pharmaceutical manufacturer must: o Demonstrate effectiveness with acceptable safety in adequate and well-controlled clinical trials o Generate 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 ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

5. Expedited Review FDA will expedite the review of new products when that is appropriate o May grant “breakthrough therapy” status to promising drugs o May give priority review status to certain therapies o 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 ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

6. 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 ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

7. 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 ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

8. 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?

9. Opportunities for Reforming Cancer Care Delivery Shelley Fuld Nasso ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

10. IOM: Cancer Care in Crisis IOM concluded in 2013 that cancer care is not adequately: o Patient-centered o Accessible o Coordinated o Evidence-based The strains in the system are exacerbated by: o Aging population o Rising cost of cancer care o Complexity of cancer care o Reliance on family caregivers ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

11. Current State of Cancer Care Buy and Bill system and the “business” of medicine o Managing inventory o Using 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 ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

15. 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?

16. ©2014 National Coalition for Cancer Survivorship. All Rights Reserved. Current Experiments in New Payment Models Elizabeth Goss

17. 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 ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

18. Incentives and Pressures for Change Practices undertaking reform on their own o Patient-centered oncology medical home Professional societies identifying strategies for reform o Episodes of care CMS Innovation Center has designed a Medicare episode-of-care plan that retains fee-for- service and incorporates payments for practice transformation o Voluntary for private payers o Voluntary for physicians ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

19. 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? o Disclosure of the payment reform effort? Disclosure of performance-based payments? o Disclosure of how the new payment system affects patient costs? ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

20. Measurement of Quality to Protect Patients Shelley Fuld Nasso ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

21. 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 consumers o Outcomes-focused o Based on patient and/or family/caregiver-generated health data ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

22. Symptoms, quality of life and functional status Choose based on value/meaning to patient Standardize methodology, demonstrate reproducibility, gather benchmark standards Examples- o Pain o Nausea o Satisfaction with healthcare providers Patient Reported Outcomes ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

23. 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? o Processes of care o Experience of care o Outcomes – survival, quality of life What information should be measured and publicly reported to help patients in choosing providers and hospitals for their cancer care? ©2014 National Coalition for Cancer Survivorship. All Rights Reserved.

24. Next Steps

25. Thank You