Polls and Research Studies

At times, unscientific polls will be developed by the Research Department. Polls will be posted on G-PACT social media sites. Once data is tabulated, results will be shared on the same social media sites.

G-PACT also works with physicians, medical companies, and researchers to stay current on research studies and describe the needs that patients have in order to direct studies in the appropriate way. As research results are available, they will be disseminated to members via the social media groups.

Articles

Spore Wars Help Fend Off Life-Threatening Bacterial Infections - article on C-Diff:  http://www.alaskapublic.org/NPRstory/?404221880

 

Polls and Research Studies

Take part in the largest research study for gastroparesis in the world!  https://www.g-pact.org/registry

 

­

Patient Powered Drug Development: Platform For Engaging Everyone Responsibly Interim Report

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.

 

 

*********************************************************************

Efficacy of Gastric Stimulator (Pacemaker), Survey Conducted August, 2013

This survey is for people who have had the gastric stimulator (pacemaker) implanted.  109 people participated in the survey.

1. How is your gastroparesis classified?

Diabetic  12%

Idiopathic  75%

Post-Viral   5%

Post-Surgical   2%

Other   6%

2. How long have you had gastroparesis?

Less than one year   5%

1-3 years   27%

3-10 years   49%

More than 10 years   19%

3. Did you have a trial with the temporary pacemaker before you had the permanent one implanted?

Yes  13.1%

No   86.9%

4. If you had the temporary pacemaker, did you notice improvement?

Yes  15.2%

No   5.4%

Not Applicable   79.3%

5. How long have you had the permanent pacemaker?

Less than 1 year   35%

1-3 years   39%

4-5 years   11%

More than 5 years  15%

6. How long was it before you had to change your battery?

Less than 1 year                  2%

1 year   0%

2 years   3%

3 years   3%

4 years   4%

5 years   4%

More than 5 years  3%

Have not replaced it     81%

7. What symptoms have improved with the pacemaker?

Nausea   67.4%

Vomiting   62%

Pain  21.7%

Other (please specify)  31.5%

Comments under “Other”:

Showing 31 text responses

*early satiety; weight loss

*Stomach pressure

*None. It's been unsuccessful so far.

*I haven't had much improvement unfortunately

*BM acids.

*I don't vomit as much

*Able to eat real food - no TPN and no tube feeds

*It did not improve anything

*none

*never had pain

*Variety of foods

*bloating. And now my stomach will growl!!!

*Bloating and general bloat feeling and early siation (spelled wrong) is gone

*I think it has helped with constipation, but my dr said that it wasn't suppose to.

*Bloat & early satiety

*Distention

*The temporary stimulator helped with nausea, vomiting while the permanent did not help. The temporary also helped me tolerate my nighttime g-tube feedings better so that I could actually finish them. The permanent one I had before the temporary one did not help me, so all the positive answers in these questions refer to the temporary stimulator that I have now and have had for a little more than half a year.

*none

*None. No improvement.

*Did not help had it removed after several years

*I've been able to increase amount of what I eat, therefore calories have increased

*improvement was most significant the 1st year .After that symptoms returned

*bloating

*None

*None

*No improvement

*obstructed bowels

*Reflux

*None as of yet

*none m pacemaker got turned off 3 weeks ago.

*I never had vomiting but I had dry heaves which have gone away.

8. How significant has the improvement been?

No improvement   18.2%

10-25% improvement   15.2%

26-50% improvement   19.2%

51-75% improvement   25.3%

75-100% improvement  22.1%

9. Do you have issues with intestinal motility?

Yes  76%

No   24%

10. If you had to do it over again, would you choose to have the pacemaker implanted?

Yes   80%                             

No   20%