Selection and Use of Patient-Reported Outcome Measures The Role of Outside Consultants Janice Hogan, Partner, Hogan Lovells LLP

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1 Selection and Use of Patient-Reported Outcome Measures The Role of Outside Consultants Janice Hogan, Partner, Hogan Lovells LLP Tuesday, November 27, 2012

2 Key Topics for Consultants Selection of Instruments Adequacy of Validation Establishing Thresholds for Success Pre-IDE Communications with FDA Balancing FDA vs. Reimbursement Requirements 2

3 Selection of Instruments Key criteria for selection Extent of validation Adequate sensitivity to change for target population Acceptance by FDA Clinician familiarity Ease of completion/robustness Acceptance by insurers Often, all of the above factors do not point to selection of the same instrument 3

Clinician familiarity Ease of completion/robustness Acceptance by insurers

4 Potential Problems Extent of validation Validation exists, but not in the same patient population/disease severity Endpoint is validated at one time point, but not the primary time point of interest (e.g., short-term versus long-term) Degree of validation is not considered sufficiently robust/acceptable, by either insurers, FDA, or the clinical community PRO is validated in some but not all necessary languages Although a sponsor can conduct its own validation, this does not address all concerns Lack of familiarity to clinicians May not be accepted by insurers 4

, short-term versus long-term) Degree of validation is not considered sufficiently robust/acceptable, by either insurers, FDA, or the

5 Instrument Sensitivity and Establishing Threshold for Success Threshold for clinically meaningful degree of change may be difficult to establish, especially for very innovative treatments -Did not previously exist on the continuum of care -Much lower risk or higher risk than other existing alternatives Not clear whether and how the threshold for success should be adjusted on the basis of these considerations e.g.: Threshold for significant change with respect to a PRO established for noninvasive treatments; what standard should be applied for an invasive treatment? or Threshold for significance is established for surgical interventions; should same threshold be applied to less invasive treatments? 5

success should be adjusted on the basis of these considerations e.g.

6 Instrument Sensitivity and Establishing Threshold for Success Adjunctive treatments or combinations, where multiple constituents of treatment (e.g., surgical and non-surgical, drug and device, etc.), contribute to the outcome -MCID on an acceptable PRO may be defined, but degree of change required for each component may not be established e.g., if MCID for a PRO is 20 points, and treatment involves surgery that achieves a 20-point average reduction, what threshold should be required for an adjunct to surgery? 6

), contribute to the outcome -MCID on an acceptable PRO may be defined, but degree of change required for each component may not

7 Acceptance by All Audiences FDA, insurers, and clinicians all must ultimately accept the same primary outcome instrument to meet the sponsor s needs Not all audiences may agree on which instrument is best Even if an MCID has been established in the literature or prior studies, not all audiences may agree on what constitutes a clinically meaningful change Although multiple instruments can be used to try to address this issue, respondent fatigue and inconsistent data may result 7

in the literature or prior studies, not all audiences may agree on what constitutes a clinically meaningful change

8 Balancing Reimbursement Needs with FDA Requirements Different standards apply to the PRO data FDA determines the safety and effectiveness of the product CMS assesses whether the product is reasonable and necessary to make coverage determinations Private insurers often may follow CMS determinations 8

product CMS assesses whether the product is reasonable and necessary to

9 Examples of PRO Challenges Zurich Claudication Questionnaire (ZCQ), a validated outcomes instrument specific to lumbar spinal stenosis MCID has been defined in the literature Accepted by FDA in multiple studies However, limited initial familiarity to clinicians and insurers Although recognized as validated, initially viewed as validated only at 6 months, not for longer-term endpoints required by FDA. Degree of change that represents the MCID was initially challenged in Advisory Panel deliberations: Does the minimal detectable difference equate to clinically meaningful improvement? 9

recognized as validated, initially viewed as validated only at 6 months, not for longer-term endpoints required by FDA.

10 Other Considerations Should use of the same PRO be required for all products in the same class/for the same target population, even if multiple instruments are validated? Facilitates the ability to compare products on similar endpoints However, limits the ability of sponsors to design studies best tailored to their products and research objectives 10

Facilitates the ability to compare products on similar endpoints However, limits the

11 Pre-IDE Process Many issues related to PROs can be addressed through pre-ide interaction Instrument selection Validation MCID/threshold for success Issues that cannot be as readily resolved through the pre- IDE process include differences between FDA, clinicians, and insurers on: what instruments are most meaningful what constitutes an appropriate threshold for success 11

resolved through the pre- IDE process include differences between FDA, clinicians, and

12 Key Challenges for Sponsors Validation of new instruments or of existing instruments in additional populations Establishing thresholds for success that match the treatment and population Meeting the needs of all audiences 12

populations Establishing thresholds for success that

13 Questions? Janice Hogan, Partner Hogan Lovells US LLP Hogan Lovells has offices in: Abu Dhabi Alicante Amsterdam Baltimore Beijing Berlin Brussels Budapest* Caracas Colorado Springs Denver Dubai Dusseldorf Frankfurt Hamburg Hanoi Ho Chi Minh City Hong Kong Houston Jeddah* London Los Angeles Madrid Miami Milan Moscow Munich New York Northern Virginia Paris Philadelphia Prague Riyadh* Rome San Francisco Shanghai Silicon Valley Singapore Tokyo Ulaanbaatar* Warsaw Washington DC Zagreb* "Hogan Lovells" or the "firm" refers to the international legal practice comprising Hogan Lovells International LLP, Hogan Lovells US LLP, Hogan Lovells Worldwide Group (a Swiss Verein), and their affiliated businesses, each of which is a separate legal entity. Hogan Lovells International LLP is a limited liability partnership registered in England and Wales with registered number OC Registered office and principal place of business: Atlantic House, Holborn Viaduct, London EC1A 2FG. Hogan Lovells US LLP is a limited liability partnership registered in the District of Columbia. The word "partner" is used to refer to a member of Hogan Lovells International LLP or a partner of Hogan Lovells US LLP, or an employee or consultant with equivalent standing and qualifications, and to a partner, member, employee or consultant in any of their affiliated businesses who has equivalent standing. Rankings and quotes from legal directories and other sources may refer to the former firms of Hogan & Hartson LLP and Lovells LLP. Where case studies are included, results achieved do not guarantee similar outcomes for other clients. New York State Notice: Attorney Advertising. Hogan Lovells All rights reserved. * Associated offices

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