Final Report to the FDA

Patient Powered Drug Development: Platform For Engaging Everyone Responsibly

Gastroparesis-Patient Association for Cures and Treatments (G-PACT)

Advocacy Organization Engagement

September 9, 2015

For Delivery To:

The Food and Drug Administration (FDA)

Prepared By:

Cyan James, Genetic Alliance

Sharon F. Terry, Genetic Alliance

Rex Edwards

Jim Sliney Jr., G-PACT

Contact:

Genetic Alliance

4301 Connecticut Ave NW Suite 404

Washington DC 20008-2369

202.966.8533 ext. 217

cjames@geneticalliance.org

EXECUTIVE SUMMARY

This report provides an overview of data collected in collaboration with the Gastroparesis Patient Association for Cures and Treatments, Inc. (G-PACT), and includes views from patients with gastroesophageal reflux disease and irritable bowel syndrome. Data collection was guided by questions posed by the Patient Focused Drug Development (PFDD) initiative. Individual-level data will be sent to the FDA in separate files to facilitate independent data discovery, analysis, sharing, and reporting. This report, a basic summary of results to date, has been compiled with data received between April 9th and June 27th of 2015, and is being both entered into the Federal Register Docket and sent to the FDA. The portals for collecting this information remain open at the discretion of G-PACT.

BACKGROUND

As per its Prescription Drug User Fee Act V (PDUFA V) commitment, the Food and Drug Administration (FDA) developed the Patient Focused Drug Development (PFDD) initiative to gain a better understanding of patients’ perspectives on the severity of specific diseases and applicable therapies. The FDA holds that drug development and the FDA’s review process could benefit from a more systematic and expansive approach to obtaining patient perspectives on disease severity and currently available therapeutic options. Using a community consultation process, the FDA chose multiple disease areas to inform developing policy; the FDA considers each disease in a one-day hearing and open comment docket process of several months.

This report focuses on views obtained from patients with gastroparesis and/or gastroesophageal reflux disease and irritable bowel syndrome, and is intended to inform  ongoing decision-making.

In support of the PFDD and of FDA concerns, the Pharmaceutical Research and Manufacturers of America (PhRMA) offered Genetic Alliance an unrestricted educational grant to develop information-gathering methods suitable for informing FDA questions about patient preferences. Genetic Alliance has been analyzing whether or not the Platform for Engaging Everyone Responsibly (PEER) technology it developed together with the company Private Access can indeed successfully solicit patient perspectives through collaboration with disease advocacy organizations (DAOs). PEER uses granular, dynamic sharing and privacy settings so those affected can share their medical information with researchers and other advocacy organizations as they wish; PEER also enables cross-disease comparison, which can pin-point promising research opportunities and achieve deeper understanding of patient perspectives.

Genetic Alliance’s goal during this process has been to collect a robust dataset of patient needs, views, and preferences for FDA use and for potential DAO collaboration.

Using a Request for Proposals process targeting the diseases chosen by FDA as part of PFDD, Genetic Alliance teamed with DAOs that provide support, advocacy, or both, to 1) gastroparesis, 2) gastroesophageal reflux disease (GERD), and 3) irritable bowel syndrome. Each organization and its members were invited to help customize PEER for suitable information gathering before surveys were developed and circulated.

Protocol #gpact00001 received Institutional Review Board approval on March 4, 2015 from Genetic Alliance’s Institutional Review Board, where it was approved under Expedited Review category 7. The protocol remains under IRB approved status through March 4, 2016.

REPORT

Data collection occurred under Private Access’s consent process, which is built into PEER and permits granular, dynamic consent for each portion of participant data—this permits a great deal of customization tailored to individual preferences, congruent with ethical principles of respect and non-maleficence.

Overall Sample

We report here on survey data from 309 respondents.

Population Description

While 65 (21.0%) respondents did not indicate gender, those who did were predominantly female, representing 228 of 244 (93.4%) of those who provided a response.

Of 244 respondents who provided an age, the mean and median age was 40, with a range from 1 year to 79 years old.

Over half (57.6%) of respondents live in a city or urban environment, with 27.5% residing in a suburban area, and 14.9% indicating they live in a rural area.

The educational attainment of respondents matches closely with US census data.  Of 193 respondents providing answers to educational level:

  • 85 (44.0%) achieved a high school diploma, GED, or less
  • 55 (28.5%) attended some college, without a degree
  • 32 (16.6%) earned a bachelor’s degree, and
  • 21 (10.9) achieved a post-graduate degree

Diagnosis

When asked to indicate their disease diagnosis, 77 (24.9%) respondents did not provide an answer.  Of those who responded:

  • 227 (98.7%) indicated gastroparesis,
  • 128 (55.7%) indicated gastroesophageal reflux disease (GERD),
  • 61 (26.5%) indicated irritable bowel syndrome (IBS), and
  • 59 indicated chronic idiopathic constipation (CIC)

There was considerable overlap among diagnoses.  Of all respondents, 170 (55.0%) indicated they had been diagnosed with more than one of these diseases. There were 107 (34.6%) respondents who had received two diagnoses and 53 (17.2%) who had been diagnosed with three conditions. Ten (3.2%) patients indicated that they had received all four diagnoses.

Of the 170 respondents indicating multiple diagnoses, by far the most common diagnostic pairing was gastroparesis and GERD, indicated by 125 (86.4%) respondents.  Pairings of gastroparesis with IBS and CIC were much lower, (19.4% and 18.1%, respectively.)

Additional information on respondents indicating each condition is presented in the following sections.

Gastroparesis

Of 309 respondents, 227 (73.5%) indicated that they had been diagnosed with gastroparesis.

Demographics

Gastroparesis patients were predominantly female and white.  Females represented 214 out of 227 (94.3%) respondents, and 211 (93.0%) were white.

The mean age of gastroparesis patients was 41 years (median age 39 years).

Symptoms and Treatments

Patients reported both the frequency and severity (on a scale of 1-99) of a range of common symptoms.  The table below shows the results:

Symptom Reported by Frequency Severity
Vomiting 86 (37.9%) 29.1 64.7
Nausea 165 (72.7%) 84.3 63.5
Abdominal bloating 91 (40.1) 84.4 66.5
Heartburn 45 (19.8%) 83.8 66.8
Lack of appetite 50 (22.0%) 83.8 68.5
Weight loss 46 (20.3%) 78.9 61.0
Feeling of fullness 72 (31.7%) 81.6 65.3
Retching 34 (15.0%) 75.7 64.1
Abdominal pain 143 (63.0%) 87.9 73.2
Weight gain 32 (14.1%) 87.5 67.5
Other 17 (7.5%) 87.0 71.8

Patients also provided information about how these symptoms affect their lives. They were asked whether symptoms made things difficult: a) on a typical day and b) on their worst day.  This information is summarized below:

Hard to: Typical Worst
Sleep 140 (61.7%) 175 (77.1%)
Concentrate 106 (46.7%) 139 (61.2%)
Perform everyday activities 127 (55.9%) 179 (78.9%)
Participate in physical activities 169 (74.4%) 181 (79.7%)
Attend work or school 119 (52.4%) 155 (68.3%)
Eat 185 (81.5%) 197 (86.8%)
Socialize 147 (64.8%) 174 (76.7%)
Be intimate 114 (50.2%) 156 (68.7%)
Perform activities at home 127 (55.9%) 175 (77.1%)
Leave home at will 116 (51.1%) 167 (73.6%)

Gastroparesis patients can select a range of treatments. The table below summarizes participant answers to treatment-related questions:

Treatment Used by
Changes to diet 190 (83.7%)
Parenteral or enteral nutrition 43 (18.9%)
Gastroparesis surgery 71 (31.3%)
Prescription pain medication 72 (31.7%)
Prescription prokinetic / promotility agents 95 (41.9%)
Prescription anti-emetics 100 (44.1%)
Botox injections 16 (7.0%)
Other prescription medication 108 (47.6%)
OTC pain medication 52 (22.9%)
Other OTC medication 53 (23.3%)
Home remedies 57 (25.1%)
Emotional therapy 67 (29.5%)
Other therapy 18 (7.9%)
Alternative treatment 33 (14.5%)
Other 19 (8.4%)

Patients were asked about what negative side effects were associated with their treatment plans and which most impacted their daily lives.  Responses are shown below:

Side Effect Reported by
Pain 106 (46.7%)
Fatigue 150 (66.1%)
Loss of appetite 82 (36.1%)
Nausea 108 (47.6%)
Shortness of breath 32 (14.1%)
Dizziness 61 (26.9%)
Insomnia 72 (31.7%)
Altered mood 83 (36.6%)
Other 17 (7.5%)
Constipation 68 (30.0%)
Diarrhea 31 (13.7%)

Patients were also asked to indicate other problems they identified with their treatment plan.  Lack of efficacy was the leading response, indicated by over half (62.6%) of patients.  A full tabulation includes:

Problem Reported by
Weekly visits to the clinic 19 (8.4%)
Frequent blood tests 47 (20.7%)
Cost 107 (47.1%)
Lack of effectiveness 142 (62.6%)
Other 25 (11.0%)
None of the above 29 (12.8%)

Patients were asked which three symptoms they felt most needed better treatments.  This table must still be populated by data (still undergoing analysis, and to be updated soon):

Symptom Identified for improvement by
Vomiting 92 (40.5%)
Nausea 142 (62.6%)
Abdominal bloating 77 (33.9%)
Heartburn 30 (13.2%)
Lack of appetite 47 (20.7%)
Weight loss 33 (14.5%)
Feeling of fullness 73 (32.2%)
Retching 23 (10.1%)
Abdominal pain 132 (58.1%)
Weight gain 22 (9.7%)
Other 9 (4.0%)

Clinical Trial Participation

Patients were asked several questions about their willingness to participate in gastroparesis clinical trials as well as what would prevent or encourage their participation.

2.6% of patients would consider participating in trials in which they would not gain direct health benefits, while 11.5% indicated they would participate even if it meant having to give up their current treatment.

Patients indicated reasons why they would not participate in a clinical trial, with 180 patients (79%) indicating possibility of death as the most likely reason they would not.

Reason would not participate Indicated by
Condition is currently well managed 60 (26.4%)
Condition does not severely affect life currently 35 (15.4%)
Would have to travel too far 111 (48.9%)
Would have to make too many clinic visits 48 (21.1%)
Financial burden would be too high 170 (74.9%)
Would require short-term hospital stays 35 (15.4%)
Would require long-term hospital stays 101 (44.5%)
Possibility treatment might not be effective 22 (9.7%)
Possibility of minor side effects 18 (7.9%)
Possibility of serious side effects 153 (67.4%)
Possibility that current symptoms might worsen 116 (51.1%)
Possibility of permanent disability 161 (70.9%)
Possibility of death 180 (79.3%)
Other 5 (2.2%)

When asked what would encourage them to participate in a clinical trial, the following reasons were indicated:

Action Would encourage
Being matched with another participant 94 (41.4%)
Transportation is provided 120 (52.8%)
Clear explanation of the value of participation 139 (61.2%)
Provided a summary of the outcome of the study 154 (67.8%)
Frequent communication with researchers about study milestones 143 (63.0%)
Financial compensation 126 (55.5%)
Other 5 (2.2%)
None of the above – no interest 11 (4.8%)

Gastroesophageal Reflux Disease (GERD)

Of 309 respondents, 128 (41.4%) indicated that they had been diagnosed with GERD.

Demographics

GERD patients were predominantly female and white.  117 or 128 (91.4%) respondents identified as female and 120 (93.8%) identified as white.

The mean age of GERD patients was 42 years (median age 39 years).

Symptoms and Treatments

Patients reported both the frequency and severity (on a scale of 1-99) of a range of common symptoms.  The table below shows the results:

Symptom Reported by Frequency Severity
Heartburn 77 (60.2%) 72.5 51.2
Chest pain 49 (38.3%) 66.5 49.9
Swallowing 35 (27.3%) 72.3 57.8
Cough 12 (9.4%) 78.6 44.8
Sore throat 11 (8.6%) 76.2 38.1
Reflux 74 (57.8%) 74.1 52.3
Belching 34 (26.6%) 77.9 53.2
Saliva 6 (4.7%) 67.2 59.0
Food sticking 37 (28.9%) 75.5 58.3
Laryngitis 4 (3.1%) 76.3 59.8
Gums 2 (1.6%) 79.0 66.0
Teeth 13 (10.2%) 84.1 50.0
Chronic irritation throat 16 (12.5%) 84.3 52.0
Hoarseness 8 (6.3%) 81.5 43.9
Sour taste 14 (10.9%) 76.8 41.6
Bad breath 12 (9.4%) 82.6 56.7
Other 4 (3.1%) 71.0 45.0

Patients also provided information about how these symptoms affect their lives.  They were asked whether symptoms made things difficult: a) on a typical day and b) on their worst day.   This information is shown below:

Hard to: Typical Worst
Sleep 63 (49.2%) 83 (64.8%)
Concentrate 17 (13.3%) 37 (28.9%)
Perform everyday activities 23 (18.0%) 45 (35.2%)
Participate in physical activities 39 (30.5%) 52 (40.6%)
Attend work or school 14 (10.9%) 34 (26.6%)
Eat 81 (63.3%) 43 (33.6%)
Socialize 34 (26.6%) 87 (68.0%)
Be intimate 24 (18.8%) 59 (46.1%)
Perform activities at home 20 (15.6%) 43 (33.6%)
Leave home at will 19 (14.8%) 39 (30.5%)

Questions asked about treatments used to address GERD symptoms are presented below:

Treatment Used by
Changes to diet 76 (59.4%)
Prescription pain medication 9 (7.0%)
Antacids 49 (38.3%)
Prescription prokinetic / promotility agents 26 (20.3%)
Proton pump inhibitors 65 (50.0%)
H2 blockers 14 (10.9%)
Other prescription medication 17 (13.3%)
OTC pain medication 3 (2.3%)
Other OTC medication 12 (9.4%)
Surgery 5 (3.9%)
Home remedies 13 (10.2%)
Emotional therapy 7 (5.5%)
Other therapy 1 (0.8%)
Alternative treatment 4 (3.1%)
Other 7 (5.5%)

Patients were asked which negative side effects associated with their treatment plans most impacted their daily lives.  Responses are shown below:

Side Effect Reported by
Pain 27 (21.1%)
Fatigue 30 (23.4%)
Loss of appetite 23 (18.8%)
Nausea 38 (29.7%)
Shortness of breath 12 (9.4%)
Dizziness 11 (8.6%)
Insomnia 18 (14.1%)
Altered mood 16 (12.5%)
Other 19 (14.8%)
Constipation 31 (24.2%)
Diarrhea 15 (11.7%)

Patients were also asked to indicate other problems they identified with their treatment plan.  None of the above was the leading response, indicated by over a third (34.4%) of patients.  The full range of responses is tabulated below:

Problem Reported by
Weekly visits to the clinic 2 (1.6%)
Frequent blood tests 5 (3.9%)
Cost 32 (25.0%)
Lack of effectiveness 42 (32.8%)
Other 1 (0.8%)
None of the above 44 (34.4%)

Patients were asked which three symptoms they felt most needed better treatments; this information will be updated soon.

Symptom Identified for improvement by
Heartburn 62 (48.4%)
Chest pain 35 (27.3%)
Swallowing 32 (25.0%)
Cough 4 (3.1%)
Sore throat 7 (5.5%)
Reflux 73 (57.0%)
Belching 22 (17.2%)
Saliva 4 (3.1%)
Food sticking 33 (35.8%)
Laryngitis 6 (4.7%)
Gums 6 (4.7%)
Teeth 16 (12.5%)
Chronic irritation throat 18 (14.1%)
Hoarseness 5 (3.9%)
Sour taste 10 (7.8%)
Bad breath 5 (3.9%)
Other 0 (0.0%)

Clinical Trial Participation

Patients were asked several questions about their willingness to participate in GERD clinical trials, as well as what would prevent or encourage their participation.

11.7% of patients would consider participating in trials where they would not gain direct health benefits, while only 7.8% indicated they would participate if it meant having to give up their current treatment.

Patients indicated reasons why they would not participate in a clinical trial, with 86 patients (67%) indicating possibility of death as the most likely reason they would not.

Reason would not participate Indicated by
Condition is currently well managed 46 (35.9%)
Condition does not severely affect life currently 34 (26.6%)
Would have to travel too far 57 (44.5%)
Would have to make too many clinic visits 23 (18.0%)
Financial burden would be too high 77 (60.2%)
Would require short-term hospital stays 28 (21.9%)
Would require long-term hospital stays 58 (45.3%)
Possibility treatment might not be effective 22 (17.2%)
Possibility of minor side effects 14 (10.9%)
Possibility of serious side effects 77 (60.2%)
Possibility that current symptoms might worsen 60 (46.9%)
Possibility of permanent disability 78 (60.9%)
Possibility of death 86 (67.2%)
Other 6 (4.7%)

When asked what would encourage them to participate in a clinical trial, the following reasons were indicated:

Would encourage
Being matched with another participant 42 (32.8%)
Transportation is provided 52 (40.6%)
Clear explanation of the value of participation 64 (50.0%)
Provided a summary of the outcome of the study 63 (49.2%)
Frequent communication with researchers about study milestones 59 (46.1%)
Financial compensation 55 (43.0%)
Other 3 (2.3%)
None of the above – no interest 16 (12.5%)

Irritable Bowel Syndrome

Of 309 respondents, 61 (26.5%) indicated that they had been diagnosed with irritable bowel syndrome (IBS).

Demographics

IBS patients were predominantly female and white.  Both represented 57 out of 61 (93.4%) respondents.

The mean age of IBS patients was 43 years (median age 41 years).

Symptoms and Treatments

Patients reported both the frequency and severity (on a scale of 1-99) of a range of common symptoms.  The table below shows the results:

Symptom Reported by Frequency Severity
Abdominal pain 54 (88.5%) 70.5 65.1
Bloating 41 (67.2%) 81.7 66.2
Flatulence 14 (23.0%) 85.5 55.3
Constipation 45 (73.7%) 77.9 63.7
Diarrhea 32 (52.5%) 66.5 62.9
Other 3 (4.9%) 90.3 62.0

Patients also provided information about how these symptoms affect their lives.  They were asked whether symptoms made things difficult: a) on a typical day and b) on their worst day.   This information is shown below:

Hard to: Typical Worst
Sleep 25 (41.0%) 32 (52.5%)
Concentrate 0 (0.0%) 26 (42.6%)
Perform everyday activities 22 (36.1%) 37 (60.7%)
Participate in physical activities 36 (59.0%) 42 (68.9%)
Attend work or school 0 (0%) 32 (52.5%)
Eat 0 (0%) 43 (70.5%)
Socialize 35 (57.4%) 43 (70.5%)
Be intimate 33 (54.1%) 35 (57.4%)
Perform activities at home 21 (34.4%) 37 (60.7%)
Leave home at will 33 (54.1%) 45 (73.8%)

The survey asked respondents to list which IBS treatments they have tried. Results are tabulated below:

Treatment Used by
Changes to diet 43 (70.5%)
Fiber supplements 8 (13.1%)
Prescription pain medication 9 (14.8%)
Antibiotics 3 (4.9%)
Probiotics 26 (42.6%)
Laxatives 25 (40.9%)
Anti-diarrheal medication 5 (8.2%)
Prescription medication for IBS with constipation 9 (14.8%)
Prescription medication for IBS with diarrhea 3 (4.9%)
Prescription antispasmodic medication 10 (16.4%)
Other prescription medication 10 (16.4%)
Over the counter pain medication 12 (19.7%)
Other over the counter medications 8 (13.1%)
Home remedies 5 (8.2%)
Relaxation therapy 13 (21.3%)
Hypnosis 1 (1.6%)
Cognitive behavioral therapy 6 (9.8%)
Emotional or mental health counseling or therapy 10 (16.4%)
Other therapies 1 (1.6%)
Alternative treatment(s) 5 (8.2%)
Other 9 (14.8%)

Patients were also asked about what negative side effects that most impacted their daily lives were associated with their treatment plans. Responses included:

Side Effect Reported by
Pain 31 (50.8%)
Fatigue 32 (52.5%)
Loss of appetite 23 (37.7%)
Nausea 35 (57.4%)
Shortness of breath 5 (8.2%)
Dizziness 12 (19.7%)
Insomnia 13 (21.3%)
Altered mood 18 (29.5%)
Other 5 (8.2%)
Constipation 29 (47.5%)
Diarrhea 22 (36.1%)

Patients were furthermore asked to indicate other problems they identified with their treatment plan.  Lack of efficacy was the leading response, indicated by over half (57.4%) of patients.  The full listing includes:

Problem Reported by
Weekly visits to the clinic 3 (4.9%)
Frequent blood tests 5 (8.2%)
Cost 17 (27.9%)
Lack of effectiveness 35 (57.4%)
Other 1 (1.6%)
None of the above 18 (29.5%)

Patients were asked what symptoms most pressingly need better treatments—this information will be completed soon.

Symptom Identified for improvement by
Abdominal pain 50 (82.0%)
Bloating 38 (62.3%)
Flatulence 9 (14.8%)
Constipation 38 (62.3%)
Diarrhea 25 (40.9%)
Other 3 (4.9%)

Clinical Trial Participation

Patients were asked about their willingness to participate in IBS clinical trials as well as what factors would prevent or encourage their participation.

11.5% of patients would consider participating in trials in which they would not gain direct health benefits, while only 6.5% indicated they would participate if it meant having to give up their current treatment.

Patients indicated reasons why they would not participate in a clinical trial, with 44 patients (72%) indicating possibility of death as the most likely reason they would not.

Reason would not participate Indicated by
Condition is currently well managed 20 (32.8%)
Condition does not severely affect life currently 13 (21.3%)
Would have to travel too far 36 (59.0%)
Would have to make too many clinic visits 22 (36.1%)
Financial burden would be too high 42 (68.9%)
Would require short-term hospital stays 15 (24.6%)
Would require long-term hospital stays 30 (49.2%)
Possibility treatment might not be effective 8 (13.1%)
Possibility of minor side effects 7 (11.5%)
Possibility of serious side effects 40 (65.6%)
Possibility that current symptoms might worsen 29 (47.5%)
Possibility of permanent disability 37 (60.7%)
Possibility of death 44 (72.1%)
Other 2 (3.3%)

When asked what would encourage them to participate in a clinical trial, the following reasons were indicated:

Action Would encourage
Being matched with another participant 20 (32.8%)
Transportation is provided 22 (36.1%)
Clear explanation of the value of participation 33 (54.1%)
Provided a summary of the outcome of the study 33 (54.1%)
Frequent communication with researchers about study milestones 25 (41.0%)
Financial compensation 24 (39.3%)
Other 1 (1.6%)
None of the above – no interest 13 (21.3%)

Chronic Idiopathic Constipation (CIC)

Of 309 respondents, 22 (7.1%) indicated that they had been diagnosed with CIC.

Demographics

CIC patients were predominantly female and white.  Overall, 21 of 22 (95.5%) respondents identified as female and 21 of 22 (95.5%) identified as white.

The mean age of CIC patients was 40.6 years (median age 38.5 years).

The mean time that CIC patients have been experiencing symptoms is approximately 7.5 years, while diagnosis occurred on average 3.2 years ago.

Symptoms and Treatments

Patients reported both the frequency and severity (on a scale of 1-99) of a range of common CIC symptoms.  The table below shows the results:

Symptom Reported by Frequency Severity
Straining 9 (40.9%) 86.6 71.0
Incomplete movements 3 (13.6%) 83.7 82.3
Feeling of incomplete 5 (22.7%) 85.2 66.3
Hard stools 7 (31.8%) 82.8 53.8
Interval between movements 7 (31.8%) 89.2 65.8
Abdominal pain 11 (50.0%) 84.1 68.5
Abdominal bloating 8 (36.4%) 94.3 81.5
Excessive flatulence 4 (18.2%) 92.0 69.8
Other 1 (4.5%) N/A N/A

Patients also provided information about how these symptoms affect their lives.  They were asked whether symptoms made things difficult on a typical day and on their worst day.  They reported the following:

Hard to: Typical Worst
Sleep 5 (22.7%) 8 (36.4%)
Concentrate 4 (18.2%) 6 (27.3%)
Perform everyday activities 3 (13.6%) 7 (31.8%)
Participate in physical activities 7 (31.8%) 9 (40.9%)
Attend work or school 2 (9.1%) 6 (27.3%)
Eat 10 (45.5%) 11 (50.0%)
Socialize 6 (27.3%) 10 (45.5%)
Be intimate 9 (40.9%) 11 (50.0%)
Perform activities at home 3 (13.6%) 9 (40.9%)
Leave home at will 4 (18.2%) 7 (31.8%)

Responses about treatments being used to address CIC symptoms are presented below:

Treatment Used by
Changes to diet 8 (36.4%)
Fiber supplements 3 (13.6%)
Prescription pain medication 0 (0.0%)
Antibiotics 0 (0.0%)
Probiotics 4 (18.2%)
Laxatives 7 (31.8%)
Anti-diarrheal medication 1 (4.5%)
Prescription medication for constipation 9 (40.9%)
Prescription antispasmodic medication 1 (4.5%)
Other prescription medication 0 (0.0%)
OTC pain medication 1 (4.5%)
Other OTC medications 2 (9.1%)
Home remedies 2 (9.1%)
Relaxation therapy 0 (0.0%)
Hypnosis 0 (0.0%)
Cognitive behavioral therapy 1 (4.5%)
Emotional therapy 0 (0.0%)
Other therapies 0 (0.0%)
Alternative treatments 0 (0.0%)
Other 0 (0.0%)

Patients were asked to provide information about what negative side effects were associated with their treatment plan that most impacted their daily life.  Responses shown below:

Side Effect Reported by
Pain 7 (31.8%)
Fatigue 4 (18.2%)
Loss of appetite 7 (31.8%)
Nausea 8 (36.4%)
Shortness of breath 1 (4.5%)
Dizziness 4 (18.2%)
Insomnia 2 (9.1%)
Altered mood 3 (13.6%)
Other 0 (0.0%)
Constipation 9 (40.9%)
Diarrhea 6 (27.3%)

Patients were also asked to indicate other problems that they identified with their treatment plan.  Lack of effectiveness was the leading response, indicated by over a third (40.9%) of patients.  Full listing here:

Problem Reported by
Weekly visits to the clinic 0 (0.0%)
Frequent blood tests 0 (0.0%)
Cost 4 (18.2%)
Lack of effectiveness 9 (40.9%)
Other 1 (4.5%)
None of the above 3 (13.6%)

Patients were asked which three symptoms they felt most needed better treatments.  Those most mentioned were abdominal pain (46%), straining during movements (36%), and interval between movements (36%).  Full presentation below:

Symptom Identified for improvement by
Straining 8 (36.4%)
Incomplete movements 4 (18.2%)
Feeling of incomplete 5 (22.7%)
Hard stools 5 (22.7%)
Interval between movements 8 (36.4%)
Abdominal pain 10 (45.5%)
Abdominal bloating 6 (27.3%)
Excessive flatulence 3 (13.6%)
Other 0 (0.0%)

Clinical Trial Participation

Patients were asked several questions about their willingness to participate in CIC clinical trials as well as what would prevent or encourage their participation.

4.5% of patients would consider participating in trials where they would not gain direct health benefits, while 18.2% indicated they would participate if it meant having to give up their current treatment.

Patients indicated reasons why they would not participate in a clinical trial, with 86 patients (54.5%) indicating possibility of death as the most likely reason they would not.

Reason would not participate Indicated by
Condition is currently well managed 6 (27.3%)
Condition does not severely affect life currently 5 (22.7%)
Would have to travel too far 8 (36.4%)
Would have to make too many clinic visits 2 (9.0%)
Financial burden would be too high 12 (54.5%)
Would require short-term hospital stays 4 (18.2%)
Would require long-term hospital stays 6 (27.3%)
Possibility treatment might not be effective 1 (4.5%)
Possibility of minor side effects 1 (4.5%)
Possibility of serious side effects 10 (45.5%)
Possibility that current symptoms might worsen 6 (27.3%)
Possibility of permanent disability 10 (45.5%)
Possibility of death 12 (54.5%)
Other 0 (0.0%)

When asked what would encourage them to participate in a clinical trial, the following reasons were indicated:

Action Would encourage
Being matched with another participant 7 (31.8%)
Transportation is provided 8 (36.4%)
Clear explanation of the value of participation 11 (50.0%)
Provided a summary of the outcome of the study 13 (59.1%)
Frequent communication with researchers about study milestones 11 (50.0%)
Financial compensation 9 (40.9%)
Other 0 (0.0%)
None of the above – no interest 0 (0.0%)

CONCLUSIONS:

We have demonstrated that patient views can be solicited by partnering with DAOs, and that PEER can collect and store these views, subject to participants’ individual preferences.

While the available data is limited, we still observe a range of views across the conditions under consideration. It is also possible to analyze the data with increased granularity and to consider a variety of confounding effects, and/or examine potential subpopulations. This initial data-collection provides proof of concept and is a foundation for future participant-engagement and data-collection efforts to inform disease research.

Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s