Skip to main content
. 2017 Feb;23(2):10.18553/jmcp.2017.23.2.125. doi: 10.18553/jmcp.2017.23.2.125
Question Response Option Numbera
1. What is your role in the treatment chain for oncology? There are several roles that may apply. Please check all that apply.
  • Make prescribing decisions for individual patients (e.g., treating physician)

  • Make decisions regarding which of 2 or more comparable treatments is recommended at a higher level (e.g., for an entire health plan or a large geographic region) level

  • Negotiate treatment prices for an organization

  • Advise decision makers regarding appropriate use of a treatment based on clinical factors only (e.g., in the form of clinical treatment guidelines for a health plan or geographic area)

  • Advise decision makers regarding whether a treatment is a good value for money (e.g., conduct or publish health technology assessments)

  • Other (please specify, especially as it pertains to making prescribing or formulary decisions or advising those decision makers)

26
2. Does PRO data impact decision making when considering one therapy over another? Yes/no; if yes, rank the following 1-6:
  • CNS: Neurology and Neurodegenerative

  • CNS: Psychiatry

  • Oncology

  • Diabetes/Metabolic Disease

  • Autoimmune Disorders

  • “Lifestyle” (e.g., obesity, smoking)

26b
3. How is the evaluation process different for PRO data included in postmarketing trials vs. registration trials? Select the best response:
  • Same for both types of trials

  • Higher regards/more useful information from registration trials

  • Higher regards/more useful information from postmarketing trials

  • Other (please specify)

24
4. How frequently do you use PRO study data for risk-sharing agreements/risk-sharing schemes? Please respond using a scale of 1 to 7 where 1 means “never” and 7 means “always” 24
5. What is the level of importance of PRO data for market access for new oncology treatments in your health care system? Please respond using a scale of 1 to 7 where 1 means “not important” and 7 means “extremely important” 23
6. To what extent do PRO label claims increase your likelihood of paying for a treatment? Please respond using a scale of 1 to 7 where 1 means “never” and 7 means “always” 23
7. Which has more impact on decision making for a new treatment, PRO data in the label or PRO data in a peer-reviewed publication? Indicate label or publication 23
8. How important is full validation of the PRO measures in your use of PRO information in decision making? Please respond using a scale of 1 to 7 where 1 means “not important” and 7 means “extremely important” 23
9. To what extent do data collected through PROs that have been newly developed and may not have full validation influence your decision making? Please respond using a scale of 1 to 7 where 1 means “not important” and 7 means “extremely important” 23
10. What characteristics should a PRO measure for a treatment in oncology have in order to support market access and HTA? Select all that apply and rate by level of importance:
  • Developed per FDA PRO guidance document

  • Frequently used in oncology or for a specific type of cancer

  • Validated in target disease population

  • Validated in country-specific populations

  • Adopted by key opinion leaders

  • Data from measure published in peer-reviewed journals

  • Results supported by minimally clinically important difference estimates

  • Results in alignment with objective measure results (e.g., laboratory value, clinician rating)

  • Other (please specify)

23
11 a. Are you aware of oncology products that received favorable reimbursement decisions because of PRO data? Yes/no; if yes, please indicate 21
b. Conversely, are you aware of examples of oncology products that did not receive a favorable reimbursement decision because they did not include a PRO?
12. a. How useful are PROs collected postprogression for an oncology therapeutic? Please rate on a scale of 1 to 7 where 1 means “not at all useful” and 7 means “extremely useful” 21
b. How long should postprogression data be collected?
13. In what types of cancers would it be most useful to collect PRO data while the cancer is progressing?
  • Please rate the cancer indications on a scale of 1 to 7 where 1 means “not at all useful” and 7 means “extremely useful”:

  • Lung (non-small cell lung cancer)

  • Breast

  • Bladder

  • Hematological

  • Other (please specify)

20
14. What type of data (PRO or other types of data) would be convincing for supporting reimbursement of oncology treatments that have stopped preventing the cancer from progressing (postprogression)? Please respond using a scale of 1 to 7 where 1 means “not important” and 7 means “extremely important”:
  • Stability of disease

  • Improvement of HRQOL

  • Improvement in symptom severity or frequency (e.g., cough, fatigue)

  • Improvement in functional status (e.g., physical, social, emotional)

  • Slower rate of functional deterioration compared with control/comparator

  • Other (please specify)

20
15. How influential are the following sources of PRO data for market access decision making? Please respond using a scale of 1 to 7 where 1 means “no impact at all” and 7 means a “very high impact” to market access decision making
  • Peer-reviewed publication

  • PRO data source from registration trials

  • PRO data source from postmarketing trials

  • Professional conference presentation (e.g., ASCO)

  • Treatment guideline from clinician organization

  • Additional documentation provided by the manufacturer

  • Manufacturer’s website for the product

  • Patient advocacy organization

  • Social media

  • Other

20
16. If you impart one piece of advice for pharmaceutical manufacturers with respect to communicating PRO evidence to payer decision makers, what would it be?   20

aNumber of respondents who answered the question.

bTwelve participants responded “yes” and provided a ranking.

ASCO = American Society of Clinical Oncology; CNS =central nervous system; FDA = U.S. Food and Drug Administration; HRQOL = health-related quality of life; HTA = health technology assessment; PRO = patient-reported outcome.