DIA Meaningful Change WG
Survey results
2024-03-11
Results
Cohort description & summary statistics
Overall summary of those completing survey.
| I am completing this survey as a: | N = 2481 |
|---|---|
| stakeholder | |
| Caregiver | 7 (2.8%) |
| Employee of a drug/device manufacturer | 155 (63%) |
| Healthcare professional | 52 (21%) |
| Patient | 20 (8.1%) |
| Payer/HTA representative | 5 (2.0%) |
| Regulator | 9 (3.6%) |
| 1 n (%) | |
Sex
Note: analysis will be for those who responded (not missing) for sex
and I am completing this survey as a (caregiver).
| Overall, N = 1301 | Caregiver, N = 41 | Employee of a drug/device manufacturer, N = 901 | Healthcare professional, N = 211 | Patient, N = 121 | Regulator, N = 31 | p-value2 | |
|---|---|---|---|---|---|---|---|
| sex | 0.4 | ||||||
| Female | 88 (68%) | 3 (75%) | 61 (68%) | 14 (67%) | 9 (75%) | 1 (33%) | |
| Male | 37 (28%) | 0 (0%) | 25 (28%) | 7 (33%) | 3 (25%) | 2 (67%) | |
| Prefer not to say | 5 (3.8%) | 1 (25%) | 4 (4.4%) | 0 (0%) | 0 (0%) | 0 (0%) | |
| 1 n (%) | |||||||
| 2 Fisher’s exact test | |||||||
Race
Note: analysis will be for those who responded (not missing) for race
and I am completing this survey as a (caregiver).
| Overall, N = 1301 | Caregiver, N = 41 | Employee of a drug/device manufacturer, N = 901 | Healthcare professional, N = 211 | Patient, N = 121 | Regulator, N = 31 | p-value2 | |
|---|---|---|---|---|---|---|---|
| race | 0.047 | ||||||
| Asian or Asian American | 18 (14%) | 1 (25%) | 17 (19%) | 0 (0%) | 0 (0%) | 0 (0%) | |
| Black or African American | 2 (1.5%) | 0 (0%) | 1 (1.1%) | 1 (4.8%) | 0 (0%) | 0 (0%) | |
| Hispanic or Latino | 1 (0.8%) | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) | 1 (33%) | |
| Other | 5 (3.8%) | 0 (0%) | 4 (4.4%) | 0 (0%) | 1 (8.3%) | 0 (0%) | |
| White or Caucasian | 104 (80%) | 3 (75%) | 68 (76%) | 20 (95%) | 11 (92%) | 2 (67%) | |
| 1 n (%) | |||||||
| 2 Fisher’s exact test | |||||||
Region
Note: analysis will be for those who responded (not missing) for
region and I am completing this survey as a
(caregiver).
| Overall, N = 1291 | Caregiver, N = 41 | Employee of a drug/device manufacturer, N = 901 | Healthcare professional, N = 201 | Patient, N = 121 | Regulator, N = 31 | p-value2 | |
|---|---|---|---|---|---|---|---|
| region | 0.011 | ||||||
| Asia/South Asia/SE Asia | 5 (3.9%) | 0 (0%) | 5 (5.6%) | 0 (0%) | 0 (0%) | 0 (0%) | |
| Australia/NZ | 1 (0.8%) | 0 (0%) | 0 (0%) | 1 (5.0%) | 0 (0%) | 0 (0%) | |
| EU | 36 (28%) | 1 (25%) | 25 (28%) | 3 (15%) | 7 (58%) | 0 (0%) | |
| Latin America/Caribbean | 3 (2.3%) | 1 (25%) | 0 (0%) | 1 (5.0%) | 0 (0%) | 1 (33%) | |
| US/North America | 84 (65%) | 2 (50%) | 60 (67%) | 15 (75%) | 5 (42%) | 2 (67%) | |
| 1 n (%) | |||||||
| 2 Fisher’s exact test | |||||||
Country
Note: analysis will be for those who responded (not missing) for
Country and I am completing this survey as a
(caregiver).
| What country are you based in? | Overall, N = 1231 | Caregiver, N = 41 | Employee of a drug/device manufacturer, N = 831 | Healthcare professional, N = 211 | Patient, N = 121 | Regulator, N = 31 |
|---|---|---|---|---|---|---|
| country | ||||||
| australia | 1 (0.8%) | 0 (0%) | 0 (0%) | 1 (4.8%) | 0 (0%) | 0 (0%) |
| austria | 1 (0.8%) | 0 (0%) | 0 (0%) | 1 (4.8%) | 0 (0%) | 0 (0%) |
| canada | 1 (0.8%) | 0 (0%) | 0 (0%) | 1 (4.8%) | 0 (0%) | 0 (0%) |
| china | 2 (1.6%) | 0 (0%) | 2 (2.4%) | 0 (0%) | 0 (0%) | 0 (0%) |
| france | 6 (4.9%) | 0 (0%) | 6 (7.2%) | 0 (0%) | 0 (0%) | 0 (0%) |
| germany | 6 (4.9%) | 0 (0%) | 2 (2.4%) | 2 (9.5%) | 2 (17%) | 0 (0%) |
| greece | 1 (0.8%) | 0 (0%) | 0 (0%) | 0 (0%) | 1 (8.3%) | 0 (0%) |
| india | 1 (0.8%) | 0 (0%) | 1 (1.2%) | 0 (0%) | 0 (0%) | 0 (0%) |
| netherlands | 3 (2.4%) | 0 (0%) | 2 (2.4%) | 0 (0%) | 1 (8.3%) | 0 (0%) |
| north macedonia | 1 (0.8%) | 1 (25%) | 0 (0%) | 0 (0%) | 0 (0%) | 0 (0%) |
| singapore | 1 (0.8%) | 0 (0%) | 1 (1.2%) | 0 (0%) | 0 (0%) | 0 (0%) |
| spain | 1 (0.8%) | 0 (0%) | 1 (1.2%) | 0 (0%) | 0 (0%) | 0 (0%) |
| sweden | 3 (2.4%) | 0 (0%) | 3 (3.6%) | 0 (0%) | 0 (0%) | 0 (0%) |
| switzerland | 3 (2.4%) | 0 (0%) | 3 (3.6%) | 0 (0%) | 0 (0%) | 0 (0%) |
| the bahamas | 1 (0.8%) | 0 (0%) | 0 (0%) | 1 (4.8%) | 0 (0%) | 0 (0%) |
| u.k. | 9 (7.3%) | 0 (0%) | 6 (7.2%) | 0 (0%) | 3 (25%) | 0 (0%) |
| u.s. | 81 (66%) | 3 (75%) | 56 (67%) | 14 (67%) | 5 (42%) | 3 (100%) |
| ukraine | 1 (0.8%) | 0 (0%) | 0 (0%) | 1 (4.8%) | 0 (0%) | 0 (0%) |
| 1 n (%) | ||||||
Age
Note: analysis will be for those who responded (not missing) for age
and I am completing this survey as a (caregiver).
| Overall, N = 1301 | Caregiver, N = 41 | Employee of a drug/device manufacturer, N = 901 | Healthcare professional, N = 211 | Patient, N = 121 | Regulator, N = 31 | |
|---|---|---|---|---|---|---|
| age | ||||||
| 18-30 | 4 (3.1%) | 0 (0%) | 3 (3.3%) | 0 (0%) | 1 (8.3%) | 0 (0%) |
| 31-40 | 27 (21%) | 0 (0%) | 22 (24%) | 3 (14%) | 1 (8.3%) | 1 (33%) |
| 41-50 | 39 (30%) | 1 (25%) | 32 (36%) | 3 (14%) | 3 (25%) | 0 (0%) |
| 51-60 | 31 (24%) | 3 (75%) | 19 (21%) | 3 (14%) | 5 (42%) | 1 (33%) |
| 61-70 | 19 (15%) | 0 (0%) | 7 (7.8%) | 9 (43%) | 2 (17%) | 1 (33%) |
| 71-80 | 4 (3.1%) | 0 (0%) | 1 (1.1%) | 3 (14%) | 0 (0%) | 0 (0%) |
| Prefer not to answer | 6 (4.6%) | 0 (0%) | 6 (6.7%) | 0 (0%) | 0 (0%) | 0 (0%) |
| 1 n (%) | ||||||
(Simplified) Age
Take age midpoint to get (weighted) averages
## # A tibble: 5 × 2
## stakeholder weighted_avg
## <chr> <dbl>
## 1 Caregiver 53
## 2 Employee of a drug/device manufacturer 46.4
## 3 Healthcare professional 58.4
## 4 Patient 50.4
## 5 Regulator 52.2
Highest Education
Note: analysis will be for those who responded (not missing) for
highest education and I am completing this survey as a
(caregiver).
| Overall, N = 1301 | Caregiver, N = 41 | Employee of a drug/device manufacturer, N = 901 | Healthcare professional, N = 211 | Patient, N = 121 | Regulator, N = 31 | p-value2 | |
|---|---|---|---|---|---|---|---|
| education | 0.6 | ||||||
| Completed an undergraduate degree program at college/university | 15 (12%) | 0 (0%) | 11 (12%) | 1 (4.8%) | 3 (25%) | 0 (0%) | |
| Completed post-graduate education (after undergraduate degree) | 114 (88%) | 4 (100%) | 78 (87%) | 20 (95%) | 9 (75%) | 3 (100%) | |
| Prefer Not to Answer | 1 (0.8%) | 0 (0%) | 1 (1.1%) | 0 (0%) | 0 (0%) | 0 (0%) | |
| 1 n (%) | |||||||
| 2 Fisher’s exact test | |||||||
What is your degree?
Note: in data: variables x118 and x119
| N = 211 | |
|---|---|
| degree | |
| MD | 5 (24%) |
| MD, PhD, MSc | 1 (4.8%) |
| PA | 1 (4.8%) |
| Ph.D,MD | 1 (4.8%) |
| PharmD | 2 (9.5%) |
| PhD | 8 (38%) |
| Physical Therapist | 1 (4.8%) |
| PsyD | 1 (4.8%) |
| RN | 1 (4.8%) |
| 1 n (%) | |
What is your therapeutic area of expertise?
Note: in data: variable x120
All HCP responders
| therapeutic area of expertise | N = 211 |
|---|---|
| TA | |
| clinical pharmacology, internist | 1 (4.8%) |
| clinical psychology, psychopharmachology | 1 (4.8%) |
| generalist | 4 (19%) |
| geriatrics/ palliative care | 1 (4.8%) |
| gynecology | 1 (4.8%) |
| hiv | 1 (4.8%) |
| hospitalist pharmacist | 1 (4.8%) |
| infectiology | 1 (4.8%) |
| infectious diseases | 1 (4.8%) |
| neuromuscle | 1 (4.8%) |
| neuropsychology and clinical psychology | 1 (4.8%) |
| not relevant. i am not a clinician | 1 (4.8%) |
| pediatric hemato-oncology/stem cell transplantation | 1 (4.8%) |
| pharmacist | 1 (4.8%) |
| psychiatry | 1 (4.8%) |
| psychology | 1 (4.8%) |
| rheumatology/immunology | 1 (4.8%) |
| symptom researcher | 1 (4.8%) |
| 1 n (%) | |
In which therapeutic area do you provide support?
Note: in data: variables x167 and x221. And x221 is empty.
All are Regulators.
| In which therapeutic area do you provide support? | N = 31 |
|---|---|
| area | |
| Medicaid administration, Population Health Management Program | 1 (33%) |
| Regulatory antiviral drug development | 1 (33%) |
| Tobacco products | 1 (33%) |
| 1 n (%) | |
For which regulator/HTA do you provide advice?
Note: in data: variables x168 and x222. And x222 is empty.
All are Regulators.
| For which regulator/HTA do you provide advice? | N = 31 |
|---|---|
| provide advice | |
| CA Department of Health Care Services, CA Department of Public Health, CA State Legislature | 1 (33%) |
| FDA | 1 (33%) |
| US Food and Drug Administration | 1 (33%) |
| 1 n (%) | |
Do you provide care to adults, children/adolescents, or both?
All HCP
| Do you provide care to adults, children/adolescents, or both? | N = 211 |
|---|---|
| care for | |
| adults | 5 (24%) |
| both | 7 (33%) |
| children/adolescents | 1 (4.8%) |
| i did back when i was doing clinical work, now working in research | 1 (4.8%) |
| i do not provide care but work in pro research | 1 (4.8%) |
| my current work focuses on research, develompment, diagnostics and teaching rather than patient care | 1 (4.8%) |
| no | 4 (19%) |
| research only at this point | 1 (4.8%) |
| 1 n (%) | |
What is your relationship to the person you care for?
Note: in data: variables x48 and x49.
All caregivers.
| What is your relationship to the person you care for? | N = 41 |
|---|---|
| relationship | |
| Child | 2 (50%) |
| I work at a patient advocacy organization and provide support for patients | 1 (25%) |
| Parent | 1 (25%) |
| 1 n (%) | |
What disease/condition do you/the person you care for have?
Note: in data: variables x18 and x50.
| What disease/condition do you/the person you care for have? | Overall, N = 151 | Caregiver, N = 41 | Patient, N = 111 | p-value2 |
|---|---|---|---|---|
| disease/condition | >0.9 | |||
| Asthma | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Cancers | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Eosinophilic esophagitis (EoE) | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Erectile dysfunction. | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Ewing Sarcoma | 1 (6.7%) | 1 (25%) | 0 (0%) | |
| excema | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Fracture / TBI | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| hepatitis B | 1 (6.7%) | 1 (25%) | 0 (0%) | |
| HIV | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Liver disease | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Neurofibromatosis | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| Obesity | 1 (6.7%) | 1 (25%) | 0 (0%) | |
| primary sclerosing cholangitis | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| pyrophosphate arthrosis / arthritis | 1 (6.7%) | 0 (0%) | 1 (9.1%) | |
| STXBP1-related disorder | 1 (6.7%) | 1 (25%) | 0 (0%) | |
| 1 n (%) | ||||
| 2 Fisher’s exact test | ||||
How long have you/they had this disease/condition?
Note: in data: variables x19 and x51.
| How long have you/they had this disease/condition? | Overall, N = 141 | Caregiver, N = 31 | Patient, N = 111 | p-value2 |
|---|---|---|---|---|
| disease_condition_duration | 0.8 | |||
| 1.5 years | 1 (7.1%) | 1 (33%) | 0 (0%) | |
| 10 years | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| 10-30 years | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| 17 years | 2 (14%) | 0 (0%) | 2 (18%) | |
| 2 weeks/ 25 years | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| 34 years | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| 5 years | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| 50 years | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| 70 years | 1 (7.1%) | 1 (33%) | 0 (0%) | |
| for 30 years | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| since birth | 3 (21%) | 1 (33%) | 2 (18%) | |
| 1 n (%) | ||||
| 2 Fisher’s exact test | ||||
How old is the person you care for?
Note: in data: variable x52.
All caregivers.
| How old is the person you care for? | N = 31 |
|---|---|
| age | |
| 19 | 1 (33%) |
| 79 | 1 (33%) |
| 9 years | 1 (33%) |
| 1 n (%) | |
How long have you been caring for the patient?
Note: in data: variable x53.
All caregivers.
| How long have you been caring for the patient? | N = 41 |
|---|---|
| long | |
| 1-5 years | 2 (50%) |
| Less than a year | 1 (25%) |
| More than 5 years | 1 (25%) |
| 1 n (%) | |
Overall, how would you rate the severity of your/their disease/condition?
Note: in data: variables x20 and x54.
| Overall, how would you rate the severity of your/their disease/condition? | Overall, N = 141 | Caregiver, N = 31 | Patient, N = 111 | p-value2 |
|---|---|---|---|---|
| disease_condition_severity | 0.3 | |||
| Mild | 2 (14%) | 0 (0%) | 2 (18%) | |
| Moderate | 5 (36%) | 0 (0%) | 5 (45%) | |
| Severe | 4 (29%) | 2 (67%) | 2 (18%) | |
| Very mild | 1 (7.1%) | 0 (0%) | 1 (9.1%) | |
| Very severe | 2 (14%) | 1 (33%) | 1 (9.1%) | |
| 1 n (%) | ||||
| 2 Fisher’s exact test | ||||
Do you consider yourself to be an advocate?
Note: in data: variables x21 and x55.
| Do you consider yourself to be an advocate? | advocate | Total | p-value1 | |
|---|---|---|---|---|
| No | Yes | |||
| stakeholder | 0.5 | |||
| Caregiver | 1 (25%) | 3 (75%) | 4 (100%) | |
| Employee of a drug/device manufacturer | 1 (100%) | 0 (0%) | 1 (100%) | |
| Patient | 3 (27%) | 8 (73%) | 11 (100%) | |
| Total | 5 (31%) | 11 (69%) | 16 (100%) | |
| 1 Fisher’s exact test | ||||
Are you a member of any communities, or medical charities?
Note: in data: variables x22 and x56.
| Are you a member of any communities, or medical charities? | communities_charity | Total | p-value1 | |
|---|---|---|---|---|
| No | Yes | |||
| stakeholder | 0.5 | |||
| Caregiver | 1 (25%) | 3 (75%) | 4 (100%) | |
| Employee of a drug/device manufacturer | 1 (100%) | 0 (0%) | 1 (100%) | |
| Patient | 4 (36%) | 7 (64%) | 11 (100%) | |
| Total | 6 (38%) | 10 (63%) | 16 (100%) | |
| 1 Fisher’s exact test | ||||
Have you/the person you care for ever taken part in a clinical trial?
Note: in data: variables x23 and x57.
| Have you/the person you care for ever taken part in a clinical trial? | clinical_trial | Total | p-value1 | |
|---|---|---|---|---|
| No | Yes | |||
| stakeholder | 0.13 | |||
| Caregiver | 3 (100%) | 0 (0%) | 3 (100%) | |
| Employee of a drug/device manufacturer | 1 (100%) | 0 (0%) | 1 (100%) | |
| Patient | 4 (36%) | 7 (64%) | 11 (100%) | |
| Total | 8 (53%) | 7 (47%) | 15 (100%) | |
| 1 Fisher’s exact test | ||||
What type of company do you work for?
Note: in data: variable x82.
Employee of a drug/device manufacturer responders
only.
| What type of company do you work for? | N = 891 |
|---|---|
| company | |
| Biotech | 9 (10%) |
| Consulting | 13 (15%) |
| CRO | 9 (10%) |
| Diagnostic/devices | 1 (1.1%) |
| Other (please specify) | 2 (2.2%) |
| Pharmaceutical | 55 (62%) |
| 1 n (%) | |
Where do you work?
Note: in data: variable x123.
HCP responders only.
| Where do you work? | N = 211 |
|---|---|
| work | |
| Center for Excellence | 3 (14%) |
| Private Practice | 2 (9.5%) |
| Public hospital/facility | 4 (19%) |
| University/Academic Center | 12 (57%) |
| 1 n (%) | |
opinion of meaningful improvement (free text)
Note: analysis will be for those who responded (not missing) for
opinion of meaningful improvement and
I am completing this survey as a (caregiver).
| stakeholder | meaningful_improvement |
|---|---|
| Caregiver | Social support. Mental health |
| Caregiver | Examples: (1) she is nonverbal, so having 10-20 spoken words would be meaningful. (2) she has behavior issues, so stopping or significant decrease in pulling hair, (3) decrease in tremor so she can draw with a pencil |
| Caregiver | Access to Clinical Trials within my country North Macedonia. There was NOTHING available and we could not afford to go outside the country for clinical trials wihout financial support. My daughter passed away 10mo after initial diagnosis |
| Healthcare professional | Positive changes in diagnostic tests, signs and symptoms. |
| Healthcare professional | HIV viral suppression in individuals who live in very challenging situations where it’s difficult for them to take their HIV medications every day. Reduction of eGFR by at least 0.4 from a baseline of 1.4 or higher |
| Healthcare professional | increase in function, reduction of disease-related symptoms |
| Healthcare professional | highly subjective |
| Healthcare professional | Early arrestation of disease progression or dramatic slowing of disease progression |
| Healthcare professional | Improved quality of life with potential lengthening of lifespan |
| Healthcare professional | attainment of low disease activity or remission |
| Healthcare professional | The smallest amount of positive change that would be detectable/measurable and viewed by patients as having a positive impact on their lives. |
| Healthcare professional | Clearance of HBsAg for patients with chronic HBV |
| Healthcare professional | Improved ability to accomplish necessary day-to-day activities and reduced emotional distress from/about the symptoms |
| Healthcare professional | More energy and time spent doing things they enjoy |
| Healthcare professional | Optimized diagnostics as a basis for timely and appropriate therapy |
| Healthcare professional | For them not to have the condition in the first place |
| Healthcare professional | remove the costs, profits and complexities of health insurance corporations from health care |
| Healthcare professional | a more comprehensive bio psych social model of health care evaluation and delivery |
| Healthcare professional | Meaningful improvement is a noticeable improvement as perceived by the patient her-/himself which she/he feels has a meaningful positive impact on her/his quality of life |
| Healthcare professional | living in the community, quality of life |
| Healthcare professional | disease prevention |
| Healthcare professional | stabilization or remission |
| Patient | Slowed or diminished progression of the number of tumors grown and/or size of the tumors grown and/or reduction in the size or number of tumors. |
| Patient | A change that I or my caregivers would recognise |
| Patient | An improvement to mild would be meaningful and an improvement to ‘none’ would be even more meaningful. For mild, this would mean no difficulty breathing and infrequent stridor/wheezing. For none, it would mean none of these. |
| Patient | drop the “race” item in clinical trials / research stop exclusion of people with HIV from non-HIV trials (e.g. cancer) |
| Patient | Complete disappearance of eczema patches |
| Patient | Something that improves survival (helps me live longer and reduces cancer risk)) and improves how I feel and function on a daily basis. |
| Patient | Better quality of life, that I can fullfill my dreams |
| Patient | No longer feeling that food is attached to my esophagus; being able to eat without drinking lots of water; being able to eat known trigger foods in moderation |
| Patient | Increased overall survival that also improves quality of life and functioning for people |
| Patient | Slow down joint and spine deterioration, exceptional need to take painkillers, move easier, no need for further surgeries |
| Patient | Being able to smash at will. |
# Combine responses by stakeholder
grouped_data <- meaningful_improvement %>%
group_by(stakeholder) %>%
summarise(meaningful_improvement = paste(meaningful_improvement, collapse = " "))# Create a corpus from the combined text
corpus <- VCorpus(VectorSource(grouped_data$meaningful_improvement))
# Text preprocessing
corpus <- tm_map(corpus, content_transformer(tolower))
corpus <- tm_map(corpus, removePunctuation)
corpus <- tm_map(corpus, removeNumbers)
corpus <- tm_map(corpus, removeWords, stopwords("english"))
corpus <- tm_map(corpus, stripWhitespace)
corpus <- tm_map(corpus, removeWords, "andor")
#corpus <- tm_map(corpus, stemDocument)
dtm1 <- DocumentTermMatrix(corpus, control = list(weighting = weightTfIdf))
inspect(dtm1)## <<DocumentTermMatrix (documents: 3, terms: 213)>>
## Non-/sparse entries: 228/411
## Sparsity : 64%
## Maximal term length: 20
## Weighting : term frequency - inverse document frequency (normalized) (tf-idf)
## Sample :
## Terms
## Docs able country decrease disease improves none positive
## 1 0.00000000 0.07371919 0.07371919 0.0000000 0.00000000 0.00000000 0.0000000
## 2 0.00000000 0.00000000 0.00000000 0.0483958 0.00000000 0.00000000 0.0483958
## 3 0.04322625 0.00000000 0.00000000 0.0000000 0.04322625 0.04322625 0.0000000
## Terms
## Docs support trials tumors
## 1 0.07371919 0.02720756 0.00000000
## 2 0.00000000 0.00000000 0.00000000
## 3 0.00000000 0.01063568 0.04322625dtm2 <- as.data.frame(t(as.matrix(dtm1)))df <- tibble(word = rownames(dtm2), freq = dtm2[,3] )pal <- brewer.pal(9,"BuGn")
pal2 <- brewer.pal(7,"Blues")
wordcloud(words = df$word, freq = df$freq, min.freq = 0.02,
max.words = 10, random.order = FALSE, rot.per = 0.1,
colors = pal2)
opinion of meaningful worsening (free text)
Question: In your opinion, what would a meaningful worsening be in your disease/condition / in the disease/condition of the person you care for?
| stakeholder | meaningful_worsening |
|---|---|
| Caregiver | Increased weight. Worsening diabetes and hypertension |
| Caregiver | Examples: increased tremor, increased fatigue, if she developed seizures, having more difficulty standing/walking |
| Caregiver | She was not able to breathe as it spread to her lungs, she became very tired easily and out of breath. |
| Healthcare professional | Negative changes in diagnostic tests with worsening of signs and symptoms. That is no improvements being show. |
| Healthcare professional | HIV viral load of >200 in those who were previously suppressed Increase in eGFR of 0.2 from a baseline of 1.1 or higher |
| Healthcare professional | decrease in function, increase in symptoms |
| Healthcare professional | highly subjective |
| Healthcare professional | Acceleration of disease progression |
| Healthcare professional | Decreased quality of life, loss of independent mobility, loss of life |
| Healthcare professional | any deterioration in PROs as well as disease activity measures |
| Healthcare professional | The smallest amount of negative change that would be detectable/measurable and viewed by patients as having a negative impact on their lives. |
| Healthcare professional | Development of HCC, advanced cirrhosis or liver transplant |
| Healthcare professional | Reduced functioning that threatens desired/typical standard of living |
| Healthcare professional | Loss of job, inability to complete ADLs, not engaging with friends/family |
| Healthcare professional | Inability to develop and market effective drugs |
| Healthcare professional | Not to receive needed care |
| Healthcare professional | any reduction in oversight or increase in monopoly power of pharmaceutical, health insurance or hospital corporations |
| Healthcare professional | continue lack of national health insurance /access/ and competent medical interpretation |
| Healthcare professional | Meaningful worsening is a noticeable deterioration as perceived by the patient her-/himself which she/he feels has a meaningful negative impact on her/his quality of life |
| Healthcare professional | Unable to care for self Hospitalization |
| Healthcare professional | resistance to prescribed drugs |
| Healthcare professional | disease progression impacting ADL and life expectancy |
| Patient | Continued progression or growth in the number and/or size of tumors that have grown. |
| Patient | A change that I or my partner caregivers would recognise |
| Patient | Increase in stridor, wheezing, difficulty breathing, coughing with breathing. |
| Patient | weakening the patients’ position |
| Patient | double the number of patches |
| Patient | Getting more complications, more unplanned hospital admissions/ visits, fibrosis progressing to cirrhosis |
| Patient | Honebound |
| Patient | Feeling esophageal spasms or chest pain; being afraid to eat because of increased fear of impactions |
| Patient | Less quality of life for people going through clinical trials |
| Patient | Decreased mobility, need for joint replacement and/or further spine stabilisation, increased and/or longer-lasting pain |
| Patient | Additional shrinkage |
# Combine responses by stakeholder
grouped_data <- meaningful_worsening %>%
group_by(stakeholder) %>%
summarise(meaningful_worsening = paste(meaningful_worsening, collapse = " "))# Create a corpus from the combined text
corpus <- VCorpus(VectorSource(grouped_data$meaningful_worsening))
# Text preprocessing
corpus <- tm_map(corpus, content_transformer(tolower))
corpus <- tm_map(corpus, removePunctuation)
corpus <- tm_map(corpus, removeNumbers)
corpus <- tm_map(corpus, removeWords, stopwords("english"))
corpus <- tm_map(corpus, stripWhitespace)
corpus <- tm_map(corpus, removeWords, "andor")
#corpus <- tm_map(corpus, stemDocument)
dtm1 <- DocumentTermMatrix(corpus, control = list(weighting = weightTfIdf))
inspect(dtm1)## <<DocumentTermMatrix (documents: 3, terms: 167)>>
## Non-/sparse entries: 180/321
## Sparsity : 64%
## Maximal term length: 20
## Weighting : term frequency - inverse document frequency (normalized) (tf-idf)
## Sample :
## Terms
## Docs able became breath breathe developed diabetes
## 1 0.07204375 0.07204375 0.07204375 0.07204375 0.07204375 0.07204375
## 2 0.00000000 0.00000000 0.00000000 0.00000000 0.00000000 0.00000000
## 3 0.00000000 0.00000000 0.00000000 0.00000000 0.00000000 0.00000000
## Terms
## Docs easily examples fatigue increased
## 1 0.07204375 0.07204375 0.07204375 0.07976761
## 2 0.00000000 0.00000000 0.00000000 0.00000000
## 3 0.00000000 0.00000000 0.00000000 0.01857024dtm2 <- as.data.frame(t(as.matrix(dtm1)))df <- tibble(word = rownames(dtm2), freq = dtm2[,3] )pal <- brewer.pal(9,"BuGn")
pal2 <- brewer.pal(3,"OrRd")
wordcloud(words = df$word, freq = df$freq, min.freq = 0.02,
max.words = 15, random.order = FALSE, rot.per = 0.25,
colors = pal2)
wordcloud2(df)define meaningful
(1)
Question: Do you think that how you define meaningful (in terms of improvement or worsening of your disease/condition) is the same as how your healthcare provider(s) defines meaningful?
| Overall, N = 141 | No, N = 91 | Yes, N = 51 | p-value2 | |
|---|---|---|---|---|
| stakeholder | >0.9 | |||
| Caregiver | 3 (21%) | 2 (22%) | 1 (20%) | |
| Patient | 11 (79%) | 7 (78%) | 4 (80%) | |
| 1 n (%) | ||||
| 2 Fisher’s exact test | ||||
(2)
Question: Do you think that how you define meaningful (in terms of improvement or worsening of your disease/condition) is the same as how your loved ones / the person you care for define meaningful?
| Overall, N = 141 | No, N = 31 | Yes, N = 111 | p-value2 | |
|---|---|---|---|---|
| stakeholder | >0.9 | |||
| Caregiver | 3 (21%) | 0 (0%) | 3 (27%) | |
| Patient | 11 (79%) | 3 (100%) | 8 (73%) | |
| 1 n (%) | ||||
| 2 Fisher’s exact test | ||||
meaningful improvement on a COA/PRO (free text)
Question: How do you define describe meaningful improvement on a COA/PRO endpoint when evaluating a new treatment? (free text)
| stakeholder | meaningful_improvement_COA_PRO |
|---|---|
| Employee of a drug/device manufacturer | Improvement that exceeds the meaningful change threshold for improvement that has been defined in a prior study |
| Employee of a drug/device manufacturer | As described by the patient |
| Employee of a drug/device manufacturer | Whether the patient experiences an obviously noticeable and consistent improvement in feel or function that positively impacts their quality of life. |
| Employee of a drug/device manufacturer | The range of values reflective of a meaningful improvement in a specific score over time - as perceived by a respondent (patient, observer, clinician) or assessed on a performance task or through digital health technologies. |
| Employee of a drug/device manufacturer | If the change protects the subjects and meets regulatory requirements |
| Employee of a drug/device manufacturer | Meaningful improvement would be a tool(s) that help to inform regulators, payors, healthcare providers and patients on the use of a new medicine. |
| Employee of a drug/device manufacturer | A change in a score that is felt by the patient and translates to improvements in their lived experience |
| Employee of a drug/device manufacturer | the amount of improvement a patient feels is worthwhile to take a treatment for |
| Employee of a drug/device manufacturer | meaningful improvement refers to an improvement that patients define meaningful for themselves and not defined, razed or judge by others in the system. Patients perspective on this this might vary to other stakeholders that have still loader voices like HCPs. Meaningful improvement defined by patients might change over time as care and treatment landscape evolves. So improvements defined 10 years ago might not be meaningful today or in the future. Meaningful improvement can also be very different for patients in different geographies, HCS, socio economic communities etc. |
| Employee of a drug/device manufacturer | A modification in subject’s health status in the direction towards a matched healthy control. |
| Employee of a drug/device manufacturer | Meaningful improvement is ideally at the individual patient level and signifies an improvement in how a patient feels or functions in their lives. |
| Employee of a drug/device manufacturer | Whether the improvement on a COA/PRO endpoint is meaningful from an individual patient perspective. |
| Employee of a drug/device manufacturer | That this is meaningful to patients on clinical outcomes and quality of life. |
| Employee of a drug/device manufacturer | The improvement in perceived concept that is meaningful to patients in the target patient population, which preferably also understood and accepted by regulators, payers and HCPs. The supporting evidence is of such high quality that the reaching the meaningful change can be a critical part of decision making. |
| Employee of a drug/device manufacturer | The improvment that is clinically meaningful to patients/caregivers, clinicians and other key stakeholders. |
| Employee of a drug/device manufacturer | A threshold on a patient-reported outcome measure is a way for interpreting the magnitude of within-patient change score over two separate times that an individual patient would consider a meaningful improvement. |
| Employee of a drug/device manufacturer | An improvement that is more than just noticeable but that also has a substantial impact on one’s day-to-day life |
| Employee of a drug/device manufacturer | For our purpose a change is meaningful if it meets a threshold that patients consider meaningful. |
| Employee of a drug/device manufacturer | Anchor-based, e.g., using global items which are well-understood by patients and where patients have described what a meaningful improvement would look like to them according to the response scale(s) |
| Employee of a drug/device manufacturer | The score change that indicates a within-patient meaningful response to treatment. |
| Employee of a drug/device manufacturer | I generally cite the definition from Jaeschke et al. (1989): “The smallest difference [change] in score in the domain of interest which patients perceive as beneficial and which would mandate, in the absence of troublesome side effects & excessive cost, change in a patient’s management.” |
| Employee of a drug/device manufacturer | meaningful improvement would be an improvement that is considered significant by patients themselves to live better / to improve their quality of life. |
| Employee of a drug/device manufacturer | Did the treatment resolve symptoms and Does the patient feel better? |
| Employee of a drug/device manufacturer | Change from baseline on a health or QoL aspect that is relevant and important to the patient and aligns with their primary motivators to initiate a treatment or trial. |
| Employee of a drug/device manufacturer | A change that the patient considers meaningful |
| Employee of a drug/device manufacturer | Meaningful improvement as defined by patients that note the change matters to them. Number of points change in scale, ability to do tasks, etc |
| Employee of a drug/device manufacturer | This question gets at the heart of the issue. Meaningfulness is a decision, not a method. This decision can be made at the group level, as in randomized controlled trials, but also at the individual level. This means that there are multiple ways to evaluate and even define meaningful. |
| Employee of a drug/device manufacturer | Meaningful improvement goes beyond statistical significance and is the real-world, experienced value of the improvement. The definition of what is considered meaningful improvement should be grounded in the lived experience and perspectives of people living with a certain disease, in terms of what level of improvement in specific symptoms/important life areas that would be considered impactful. Meaningful improvement should also be supported by clinical evidence in terms of the natural history of the specific disease. |
| Employee of a drug/device manufacturer | that I can noticeable tell the difference from before or after and it outweighs the risks |
| Employee of a drug/device manufacturer | a thresold (or a range of thresolds) that has been evaluated via several methods to show an improvement on patient signs, symptoms and/or functioning that is clinically meaningful and important for the patient |
| Employee of a drug/device manufacturer | Meaningful improvement is when the patient considers the amount of change (score change) on the aspect of their condition captured by the COA as important to them. It’s the point when their internal interpretation of the specific aspect moves to “better” |
| Employee of a drug/device manufacturer | Minimum change in the COA/PRO score/endpoint that corresponds to patients observing/experience a beneficial change in their health/functional status |
| Employee of a drug/device manufacturer | What is the improvement that an individual patient would consider meaningful. |
| Employee of a drug/device manufacturer | Improvement in how a patient feels or functions that the patient is both aware of (i.e. can detect) and considers to be non-trivial. |
| Employee of a drug/device manufacturer | if it significantly improves patients’ lives compared to standard of care -sometimes it may not be statistically significant in the clinical trials, but identified during post marketing period via real world evidence |
| Employee of a drug/device manufacturer | An improvement in the concept of interest that makes a difference to the person’s daily life/ HRQoL |
| Employee of a drug/device manufacturer | Change in score that allows stating that there has been a real improvement on patient specific concept of interest assesed with this COA/PRO |
| Employee of a drug/device manufacturer | I would describe it as the minimum change captured by an instrument that translates in a meaningful improvement in the patient’s life. |
| Employee of a drug/device manufacturer | a change in score that can be appreciated by a patient as having a true beneficial impact of their daily life |
| Employee of a drug/device manufacturer | Meaningful improvement on a COA/PRO should correlate with a change in the patient and/or caregiver experience of the disease that they find meaningful. |
| Employee of a drug/device manufacturer | when a change in signs/symptoms or impacts due to treatment is noticeably better–as observed by the patient–and results in a positive change in the response scale on a COA that was selected a priori for the study |
| Employee of a drug/device manufacturer | Ideally, meaningful improvement is the amount of change on a COA/PRO that is meaningful for patients. In reality, the meaningful amount of change varies across individuals and contexts. |
| Employee of a drug/device manufacturer | The minimum amount of change on the scale/instrument that is perceived as meaningful (beneficial) for an individual |
| Employee of a drug/device manufacturer | a change in measurement noticed by the individual |
| Employee of a drug/device manufacturer | threshold at which subject-level improvement is determined. |
| Employee of a drug/device manufacturer | an improvement that improves how a patient feels, functions (or survives) in a way that is noticeable and has a meaningful impact on daily life. It could also be a meaningful extension of time that a patient is in better health, compared to SOC (time to meaningful degradation, deterioration). (PerfOs may indirectly reflect a change that is indicative or predictive of a clinically meaningful improvement). |
| Employee of a drug/device manufacturer | Effectively measure the outcome of interest (or effects of the therapeutics) and clinically relevant to patients and providers. |
| Employee of a drug/device manufacturer | COA that can be used across disease states |
| Employee of a drug/device manufacturer | The improvement should reflect a noticeable improvement in symptoms, functioning, or quality of life compared with current status or expected future decline. This should not merely be a percentage, since the percentage is arbitrary and does not necessarily equate to something meaningful for patients. Because the MC could vary based on disease characteristics, the MC should be identified for individual disease areas and severity levels, using accepted statistical methodology and qualitative research. |
| Employee of a drug/device manufacturer | A meaningful improvement is an improvement in a symptom or function that is important to the patient or caregiver and that the patient perceives is large enough to indicate a improved quality of life or functional status. |
| Employee of a drug/device manufacturer | Whether 1) the patient considers it to be a meaningful/important change in symptoms, signs, or impacts, and then 2) if a clinician considers a change similarly. |
| Employee of a drug/device manufacturer | Meaningful improvement is the amount of change where patients notice a difference and they feel better and that amount of difference is important to them / makes a difference in their lives |
| Employee of a drug/device manufacturer | The amount a patient would have to improve on the COA/PRO during a period that could be interpreted as a meaningful benefit. |
| Employee of a drug/device manufacturer | A change that makes a notable, important, or significant improvement in a person’s life or experience. This can be either a functional change, where someone is able to do something they weren’t before, or an emotional change, where someone experiences improved emotional health or temperament due to changes in their condition. |
| Employee of a drug/device manufacturer | A meaningful improvement is one that reaches a target response and is observed consistently in patients who are adequately treated. |
| Employee of a drug/device manufacturer | I consider an improvement as meaningful when it is reported as such by patients, when patients describe that it made a difference to them in their lives |
| Employee of a drug/device manufacturer | Several requirements: - it is a relevant improvement to the patient - the difference can actually be measured using the PRO endpoint (scale) - it is larger than the random variation alone |
| Employee of a drug/device manufacturer | An improvement that matters to patients and/or caregivers, and is considered by clinicians as clinically relevant in reference to more established outcomes. |
| Employee of a drug/device manufacturer | Smallest improvement in symptoms which are meaningful to patients |
| Employee of a drug/device manufacturer | A meaningful improvement provides a quantifiable and detectable benefit to the patient when compared to alternatives. |
| Employee of a drug/device manufacturer | When it is definitively linked to patient benefit i.e., it shows clinical significance in improving the overall patient’s health |
| Employee of a drug/device manufacturer | i would look at the validation work for a COA or published studies using that instrument to see if the change reflects what was found to be relevant in other work. Does a change correlate with overall global assessment? etc |
| Employee of a drug/device manufacturer | Is the change interpretable and meaningful to a patient, caregiver or person who treats them |
| Employee of a drug/device manufacturer | Result that change the management of an individual in a way that is meaningful to them. |
| Employee of a drug/device manufacturer | If the change make different as per patient and caregiver evaluation. |
| Employee of a drug/device manufacturer | Clinically meaningful and relevant COA/PRO endpoints require informative natural history studies and patient input (for example from qualitative interviews). |
| Employee of a drug/device manufacturer | The patient has significantly improved based on their own perception as well as physical function and social interactions. Side effects of medication should also be taken into account. |
| Employee of a drug/device manufacturer | increase or decrease (depending on the item(s) referred to on the scale) of a measurable score on a scale |
| Employee of a drug/device manufacturer | Regulatory acceptance, past experience across studies, acceptance from physicians |
| Employee of a drug/device manufacturer | When it has demonstrable benefit to a patient |
| Employee of a drug/device manufacturer | Using appropriate standards for the disease |
| Employee of a drug/device manufacturer | It should be a clinical meaningful improvement which has impact on patient’s clinical symptom and health status. |
| Employee of a drug/device manufacturer | Patient engagement in completion for accurate and timely data |
| Employee of a drug/device manufacturer | It should be from patient, care giver and clinician |
| Employee of a drug/device manufacturer | Improved quality of life. |
| Employee of a drug/device manufacturer | Must be both Clinically and statistically significant. Needs to make a difference for the patient |
| Regulator | clinical outcomes data from health plans and key informant qualitative data in semi-structured surveys and stakeholder interviews indicate outcomes in line with known measures based on peer reviewed research findings. Community based services rendered improved for populations where health disparities have been identified. Measurement tools for risk stratification are identifying health access gaps in specific segmented populations of focus to inform future intervention. |
| Regulator | A statistically significant and clinically meaningful improvement in the endpoint being measured. |
meaningful worsening on a COA/PRO (free text)
Question: How do you define describe meaningful worsening on a COA/PRO endpoint when evaluating a new treatment? (free text)
| stakeholder | meaningful_worsening_COA_PRO |
|---|---|
| Employee of a drug/device manufacturer | Worsening that exceeds the meaningful change threshold for worsening that has been defined in a prior study |
| Employee of a drug/device manufacturer | As described by the patient |
| Employee of a drug/device manufacturer | Whether the patient experiences an obviously noticeable and consistent worsening in feel or function that negatively impacts their quality of life |
| Employee of a drug/device manufacturer | The range of values reflective of a meaningful worsening in a specific score over time - as perceived by a respondent (patient, observer, clinician) or assessed on a performance task or through digital health technologies. |
| Employee of a drug/device manufacturer | If these is increased risk to subjects or deviation from regulations. |
| Employee of a drug/device manufacturer | Meaningful worsening on a COA/PRO endpoint would show that the new medicine does not provide benefit over existing SOC. |
| Employee of a drug/device manufacturer | A change in a score that is felt by the patient and translates to a worsening in their lived experience |
| Employee of a drug/device manufacturer | the about of worsening that a patient can detect |
| Employee of a drug/device manufacturer | Same as questions 14; patients are needed to define a meaningful worsening. |
| Employee of a drug/device manufacturer | A modification in subject’s health status in the direction away from a matched healthy control. |
| Employee of a drug/device manufacturer | Meaningful worsening is ideally at the individual patient level and signifies a worsening in how a patient feels or functions in their lives. |
| Employee of a drug/device manufacturer | Whether the deterioration on a COA/PRO endpoint is meaningful from an individual patient perspective. |
| Employee of a drug/device manufacturer | That, with all measures, there is a negative overall benefit to patients (i.e. one might get worse, but others may improve). |
| Employee of a drug/device manufacturer | The perceived worsening of a concept in a target patient population as measured and understood from patients and with the additions from above. |
| Employee of a drug/device manufacturer | The worsening that is clinically meaningful to patients/caregivers, clinicians and other key stakeholders. |
| Employee of a drug/device manufacturer | A threshold on a patient-reported outcome measure is a way for interpreting the magnitude of within-patient change score over two separate times that an individual patient would consider a meaningful deterioration. |
| Employee of a drug/device manufacturer | A worsening that is more than just noticeable but that has a substantial impact on one’s day-to-day life |
| Employee of a drug/device manufacturer | For our purpose a change is meaningful if it meets a threshold that patients consider meaningful. |
| Employee of a drug/device manufacturer | Anchor-based, e.g., using global items which are well-understood by patients and where patients have described what a meaningful worseningwould look like to them according to the response scale(s) |
| Employee of a drug/device manufacturer | The score change that means the patient doesn’t think the treatment is ‘wroth it’. |
| Employee of a drug/device manufacturer | The same as above for improvement from Jaeschke et al., but replacing “beneficial” with “harmful” |
| Employee of a drug/device manufacturer | meaningful worsening would be a worsening that is considered by patients themselves to have a significant impact on their life/ to decrease their quality of life. |
| Employee of a drug/device manufacturer | Did the treatment make the symptoms worse? does the patient feel worse, did the treatment create another problem with side effects or AE? |
| Employee of a drug/device manufacturer | Change from baseline on a health or QoL aspect that is a relevant and important concern to the patient. |
| Employee of a drug/device manufacturer | A change that the patient considers worse |
| Employee of a drug/device manufacturer | Change in number of points in scale, reduction or loss of ability |
| Employee of a drug/device manufacturer | Similarly to improvement, although it is probable that worsening and improvements are simply opposite directions of the same measurement–e.g., a small worsening could be, to a given patient, much worse than a larger improvement on the measurement scale. |
| Employee of a drug/device manufacturer | This is a topic I have not yet explored in my role as I am new (less than one year of experience). I would think that worsening should be defined in the context of the specific disease, it’s natural history, prognosis, whether there are stages/levels of severity, and again based on the lived-experience of patients in terms of how they experience deterioration of their functioning. |
| Employee of a drug/device manufacturer | that I can noticeable tell the difference from before or after and the benefit does not outweighs the risks |
| Employee of a drug/device manufacturer | a thresold (or a range of thresolds) that has been evaluated via several methods to show a worsening n patient signs, symptoms and/or functioning that is clinically meaningful and important for the patient |
| Employee of a drug/device manufacturer | This is usually any movement below the COA score that the patient entered the trial with. |
| Employee of a drug/device manufacturer | Minimum change in the COA/PRO score/endpoint that corresponds to patients observing/experience a harmful or burdensome change in their health/functional status |
| Employee of a drug/device manufacturer | What is the worsening that an individual patient would consider meaningful. |
| Employee of a drug/device manufacturer | A worsening in feeling or function (i.e. symptoms or disease impact) that the patient themselves considers substantial or significant (in the lay sense, not statistical). |
| Employee of a drug/device manufacturer | if it significantly worsens patients’ lives compared to standard of care -sometimes it may not be statistically significant in the clinical trials, but identified during post marketing period via real world evidence |
| Employee of a drug/device manufacturer | A worsening in the concept that makes a difference to the person’s daily life/ HRQoL |
| Employee of a drug/device manufacturer | Change in score that allows stating that there has been a real deterioration on patient specific concept of interest assesed with this COA/PRO |
| Employee of a drug/device manufacturer | I would describe it as the minimum change captured by an instrument that translates in a noticeable and meaningful deterioration in the patient’s life. |
| Employee of a drug/device manufacturer | a change in score that can be appreciated by a patient as having a negative impact of their daily life |
| Employee of a drug/device manufacturer | Meaningful worsening on a COA/PRO should correlate with a worsening of the disease experience that creates a meaningful extra burden on the patient and/or caregiver. |
| Employee of a drug/device manufacturer | when a change in signs/symptoms or impacts due to treatment is noticeably worse–as observed by the patient–and results in a negative change in the response scale on a COA that was selected a priori for the study |
| Employee of a drug/device manufacturer | Ideally, meaningful worsening is the amount of change on a COA/PRO that is meaningful for patients. In reality, the meaningful amount of change varies across individuals and contexts. |
| Employee of a drug/device manufacturer | The minimum amount of change on the scale/instrument that is perceived as detrimental for an individual |
| Employee of a drug/device manufacturer | a change in measurement noticed by the individual |
| Employee of a drug/device manufacturer | threshold at which subject-level deterioration is determined. |
| Employee of a drug/device manufacturer | Deterioration in how a patient feels or functions in a way that substantially impacts daily life; in a way that would warrant a change in treatment. |
| Employee of a drug/device manufacturer | The outcomes and patient experience is worse than standard of care. |
| Employee of a drug/device manufacturer | Relative to QALY score |
| Employee of a drug/device manufacturer | Meaningful worsening would be a noticeable worsening of symptoms, functioning, or quality of life compared with current state or expectations based on diagnosis. Note that in diseases where decline is expected, maintenance of the status quo or even less worsening than expected could indicate a positive outcome. |
| Employee of a drug/device manufacturer | A meaningful worsening is an worsening in a symptom or a decline in function that is important to the patient or caregiver and that the patient perceives is large enough to indicate a significant decrease in their quality of life or ability to function. |
| Employee of a drug/device manufacturer | Hopefully, we would’ve established what amount of change in a COA score constituted an important worsening in the patient’s (or patients’) condition that would then be relayed as a worsening of the COA-supported endpoint. |
| Employee of a drug/device manufacturer | Meaningful worsening is the amount where patients are not feel better and are feeling worse and it makes a difference in how they feel and can function (in a negative way) |
| Employee of a drug/device manufacturer | The amount a patient would have to worsen on the COA/PRO during a period that could be interpreted as a meaningful worsening. |
| Employee of a drug/device manufacturer | A change that makes a notable, important, or significant worsening in a person’s life or experience. This can be either a functional change, where someone is no longer able to do something they were able to do before, or an emotional change, where someone experiences worsening emotional health or temperament due to changes in their condition. |
| Employee of a drug/device manufacturer | A meaningful worsening is one that indicates treatment failure and is related to early discontinuation. |
| Employee of a drug/device manufacturer | Same as above, but going in the opposite direction, when patients describe that the change had a negative impact on their lives |
| Employee of a drug/device manufacturer | Several requirements: - the patient and/or clinicians consider it a worsening that is relevant in practice - the difference can actually be measured using the PRO endpoint (scale) - it is larger than the random variation alone |
| Employee of a drug/device manufacturer | Worsening that matters to patients and/or caregivers, and is considered by clinicians as clinically relevant in reference to more established outcomes. |
| Employee of a drug/device manufacturer | Smallest decline (worsening) in symptoms which are meaningful (impact their daily life) to patients |
| Employee of a drug/device manufacturer | Quantifiable / detectable worsening in disease, as determined by the patient. |
| Employee of a drug/device manufacturer | When it is shown to have little to no patient benefit |
| Employee of a drug/device manufacturer | same process |
| Employee of a drug/device manufacturer | Has an endpoint that was measured changed /worsened over time and would a patient, caregiver or treating physician say that it is due to the new treatment |
| Employee of a drug/device manufacturer | Results that worsen individual outcomes and make management more challenging based on individual feedback. |
| Employee of a drug/device manufacturer | If per patient and caregiver opinion the effect is not improvement but worsening |
| Employee of a drug/device manufacturer | My suggestion would be to use qualitative interview data (from patients/caregivers), along with quantitative evidence, to determine what constitutes a meaningful improvement, stability, or worsening in COA scores. |
| Employee of a drug/device manufacturer | same as 14: increase or decrease of a measurable score on a scale |
| Employee of a drug/device manufacturer | Regulatory acceptance, past experience across studies, acceptance from physicians |
| Employee of a drug/device manufacturer | When it has demonstrable worsening of the patients condition |
| Employee of a drug/device manufacturer | Noe having established standards for the disease to interpret the data |
| Employee of a drug/device manufacturer | It should correspond to worsening of clinical symptom and health status. |
| Employee of a drug/device manufacturer | Less control of data without specifying the reduction of control. |
| Employee of a drug/device manufacturer | when the condition is worsening in all aspects including clinically, QOL, care giver prospective |
| Employee of a drug/device manufacturer | Decreased quality of life. |
| Employee of a drug/device manufacturer | Trending negative from Clinically normal; especially concerning when clinically or statistically significant. |
| Regulator | Gaps in preventative service utilization indicating disparities in access to services and treatments and culturally congruent providers. Data suggesting measurement tools not designed to collect meaningful data for key performance indicator measurement. |
| Regulator | A statistically significant and clinically meaningful worsening in the endpoint being measured or an association with another treatment-associated adverse event(s). |
How long should a clinical change be maintained
Question: How long should a clinical change be maintained to be truly meaningful?
| Overall, N = 231 | Healthcare professional, N = 211 | Regulator, N = 21 | p-value2 | |
|---|---|---|---|---|
| how_long_meaningful_change | >0.9 | |||
| At least 3 months | 2 (8.7%) | 2 (9.5%) | 0 (0%) | |
| At least 6 months | 2 (8.7%) | 2 (9.5%) | 0 (0%) | |
| Depends on condition/population | 17 (74%) | 15 (71%) | 2 (100%) | |
| More than 6 months | 1 (4.3%) | 1 (4.8%) | 0 (0%) | |
| Not sure | 1 (4.3%) | 1 (4.8%) | 0 (0%) | |
| 1 n (%) | ||||
| 2 Fisher’s exact test | ||||
consider the duration
Question: Do you consider the duration of maintaining clinically meaningful change as something that should be included in the definition?
| Overall, N = 231 | No, N = 21 | Not sure, N = 91 | Yes, N = 121 | p-value2 | |
|---|---|---|---|---|---|
| stakeholder | 0.2 | ||||
| Healthcare professional | 21 (91%) | 1 (50%) | 9 (100%) | 11 (92%) | |
| Regulator | 2 (8.7%) | 1 (50%) | 0 (0%) | 1 (8.3%) | |
| 1 n (%) | |||||
| 2 Fisher’s exact test | |||||
Meaningful improvement experimental (1: PCA)
Looking at text clustering in freetext response
In your opinion, what would be a meaningful improvement by
stakeholder
Meaningful improvement experimental (2: Network graphs)
Group 1: negatively correlated
- x92 : A certain percentage change in patients symptoms/behaviour
- x93 : A change that matters to patients
Group 2: positively correlated
- x91 : A change that I consider meaningful based on qualitative judgement
- x94 : Based on a threshold defined by the scale developer / in the literature
- x95 : Based on a threshold defined in a trial by the trial sponsor