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:

SymptomReported byFrequencySeverity
Vomiting86 (37.9%)29.164.7
Nausea165 (72.7%)84.363.5
Abdominal bloating91 (40.1)84.466.5
Heartburn45 (19.8%)83.866.8
Lack of appetite50 (22.0%)83.868.5
Weight loss46 (20.3%)78.961.0
Feeling of fullness72 (31.7%)81.665.3
Retching34 (15.0%)75.764.1
Abdominal pain143 (63.0%)87.973.2
Weight gain32 (14.1%)87.567.5
Other17 (7.5%)87.071.8

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:TypicalWorst
Sleep140 (61.7%)175 (77.1%)
Concentrate106 (46.7%)139 (61.2%)
Perform everyday activities127 (55.9%)179 (78.9%)
Participate in physical activities169 (74.4%)181 (79.7%)
Attend work or school119 (52.4%)155 (68.3%)
Eat185 (81.5%)197 (86.8%)
Socialize147 (64.8%)174 (76.7%)
Be intimate114 (50.2%)156 (68.7%)
Perform activities at home127 (55.9%)175 (77.1%)
Leave home at will116 (51.1%)167 (73.6%)

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

TreatmentUsed by
Changes to diet190 (83.7%)
Parenteral or enteral nutrition43 (18.9%)
Gastroparesis surgery71 (31.3%)
Prescription pain medication72 (31.7%)
Prescription prokinetic / promotility agents95 (41.9%)
Prescription anti-emetics100 (44.1%)
Botox injections16 (7.0%)
Other prescription medication108 (47.6%)
OTC pain medication52 (22.9%)
Other OTC medication53 (23.3%)
Home remedies57 (25.1%)
Emotional therapy67 (29.5%)
Other therapy18 (7.9%)
Alternative treatment33 (14.5%)
Other19 (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 EffectReported by
Pain106 (46.7%)
Fatigue150 (66.1%)
Loss of appetite82 (36.1%)
Nausea108 (47.6%)
Shortness of breath32 (14.1%)
Dizziness61 (26.9%)
Insomnia72 (31.7%)
Altered mood83 (36.6%)
Other17 (7.5%)
Constipation68 (30.0%)
Diarrhea31 (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:

ProblemReported by
Weekly visits to the clinic19 (8.4%)
Frequent blood tests47 (20.7%)
Cost107 (47.1%)
Lack of effectiveness142 (62.6%)
Other25 (11.0%)
None of the above29 (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):

SymptomIdentified for improvement by
Vomiting92 (40.5%)
Nausea142 (62.6%)
Abdominal bloating77 (33.9%)
Heartburn30 (13.2%)
Lack of appetite47 (20.7%)
Weight loss33 (14.5%)
Feeling of fullness73 (32.2%)
Retching23 (10.1%)
Abdominal pain132 (58.1%)
Weight gain22 (9.7%)
Other9 (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 participateIndicated by
Condition is currently well managed60 (26.4%)
Condition does not severely affect life currently35 (15.4%)
Would have to travel too far111 (48.9%)
Would have to make too many clinic visits48 (21.1%)
Financial burden would be too high170 (74.9%)
Would require short-term hospital stays35 (15.4%)
Would require long-term hospital stays101 (44.5%)
Possibility treatment might not be effective22 (9.7%)
Possibility of minor side effects18 (7.9%)
Possibility of serious side effects153 (67.4%)
Possibility that current symptoms might worsen116 (51.1%)
Possibility of permanent disability161 (70.9%)
Possibility of death180 (79.3%)
Other5 (2.2%)

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

ActionWould encourage
Being matched with another participant94 (41.4%)
Transportation is provided120 (52.8%)
Clear explanation of the value of participation139 (61.2%)
Provided a summary of the outcome of the study154 (67.8%)
Frequent communication with researchers about study milestones143 (63.0%)
Financial compensation126 (55.5%)
Other5 (2.2%)
None of the above – no interest11 (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:

SymptomReported byFrequencySeverity
Heartburn77 (60.2%)72.551.2
Chest pain49 (38.3%)66.549.9
Swallowing35 (27.3%)72.357.8
Cough12 (9.4%)78.644.8
Sore throat11 (8.6%)76.238.1
Reflux74 (57.8%)74.152.3
Belching34 (26.6%)77.953.2
Saliva6 (4.7%)67.259.0
Food sticking37 (28.9%)75.558.3
Laryngitis4 (3.1%)76.359.8
Gums2 (1.6%)79.066.0
Teeth13 (10.2%)84.150.0
Chronic irritation throat16 (12.5%)84.352.0
Hoarseness8 (6.3%)81.543.9
Sour taste14 (10.9%)76.841.6
Bad breath12 (9.4%)82.656.7
Other4 (3.1%)71.045.0

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:TypicalWorst
Sleep63 (49.2%)83 (64.8%)
Concentrate17 (13.3%)37 (28.9%)
Perform everyday activities23 (18.0%)45 (35.2%)
Participate in physical activities39 (30.5%)52 (40.6%)
Attend work or school14 (10.9%)34 (26.6%)
Eat81 (63.3%)43 (33.6%)
Socialize34 (26.6%)87 (68.0%)
Be intimate24 (18.8%)59 (46.1%)
Perform activities at home20 (15.6%)43 (33.6%)
Leave home at will19 (14.8%)39 (30.5%)

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

TreatmentUsed by
Changes to diet76 (59.4%)
Prescription pain medication9 (7.0%)
Antacids49 (38.3%)
Prescription prokinetic / promotility agents26 (20.3%)
Proton pump inhibitors65 (50.0%)
H2 blockers14 (10.9%)
Other prescription medication17 (13.3%)
OTC pain medication3 (2.3%)
Other OTC medication12 (9.4%)
Surgery5 (3.9%)
Home remedies13 (10.2%)
Emotional therapy7 (5.5%)
Other therapy1 (0.8%)
Alternative treatment4 (3.1%)
Other7 (5.5%)

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

Side EffectReported by
Pain27 (21.1%)
Fatigue30 (23.4%)
Loss of appetite23 (18.8%)
Nausea38 (29.7%)
Shortness of breath12 (9.4%)
Dizziness11 (8.6%)
Insomnia18 (14.1%)
Altered mood16 (12.5%)
Other19 (14.8%)
Constipation31 (24.2%)
Diarrhea15 (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:

ProblemReported by
Weekly visits to the clinic2 (1.6%)
Frequent blood tests5 (3.9%)
Cost32 (25.0%)
Lack of effectiveness42 (32.8%)
Other1 (0.8%)
None of the above44 (34.4%)

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

SymptomIdentified for improvement by
Heartburn62 (48.4%)
Chest pain35 (27.3%)
Swallowing32 (25.0%)
Cough4 (3.1%)
Sore throat7 (5.5%)
Reflux73 (57.0%)
Belching22 (17.2%)
Saliva4 (3.1%)
Food sticking33 (35.8%)
Laryngitis6 (4.7%)
Gums6 (4.7%)
Teeth16 (12.5%)
Chronic irritation throat18 (14.1%)
Hoarseness5 (3.9%)
Sour taste10 (7.8%)
Bad breath5 (3.9%)
Other0 (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 participateIndicated by
Condition is currently well managed46 (35.9%)
Condition does not severely affect life currently34 (26.6%)
Would have to travel too far57 (44.5%)
Would have to make too many clinic visits23 (18.0%)
Financial burden would be too high77 (60.2%)
Would require short-term hospital stays28 (21.9%)
Would require long-term hospital stays58 (45.3%)
Possibility treatment might not be effective22 (17.2%)
Possibility of minor side effects14 (10.9%)
Possibility of serious side effects77 (60.2%)
Possibility that current symptoms might worsen60 (46.9%)
Possibility of permanent disability78 (60.9%)
Possibility of death86 (67.2%)
Other6 (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 participant42 (32.8%)
Transportation is provided52 (40.6%)
Clear explanation of the value of participation64 (50.0%)
Provided a summary of the outcome of the study63 (49.2%)
Frequent communication with researchers about study milestones59 (46.1%)
Financial compensation55 (43.0%)
Other3 (2.3%)
None of the above – no interest16 (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:

SymptomReported byFrequencySeverity
Abdominal pain54 (88.5%)70.565.1
Bloating41 (67.2%)81.766.2
Flatulence14 (23.0%)85.555.3
Constipation45 (73.7%)77.963.7
Diarrhea32 (52.5%)66.562.9
Other3 (4.9%)90.362.0

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:TypicalWorst
Sleep25 (41.0%)32 (52.5%)
Concentrate0 (0.0%)26 (42.6%)
Perform everyday activities22 (36.1%)37 (60.7%)
Participate in physical activities36 (59.0%)42 (68.9%)
Attend work or school0 (0%)32 (52.5%)
Eat0 (0%)43 (70.5%)
Socialize35 (57.4%)43 (70.5%)
Be intimate33 (54.1%)35 (57.4%)
Perform activities at home21 (34.4%)37 (60.7%)
Leave home at will33 (54.1%)45 (73.8%)

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

TreatmentUsed by
Changes to diet43 (70.5%)
Fiber supplements8 (13.1%)
Prescription pain medication9 (14.8%)
Antibiotics3 (4.9%)
Probiotics26 (42.6%)
Laxatives25 (40.9%)
Anti-diarrheal medication5 (8.2%)
Prescription medication for IBS with constipation9 (14.8%)
Prescription medication for IBS with diarrhea3 (4.9%)
Prescription antispasmodic medication10 (16.4%)
Other prescription medication10 (16.4%)
Over the counter pain medication12 (19.7%)
Other over the counter medications8 (13.1%)
Home remedies5 (8.2%)
Relaxation therapy13 (21.3%)
Hypnosis1 (1.6%)
Cognitive behavioral therapy6 (9.8%)
Emotional or mental health counseling or therapy10 (16.4%)
Other therapies1 (1.6%)
Alternative treatment(s)5 (8.2%)
Other9 (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 EffectReported by
Pain31 (50.8%)
Fatigue32 (52.5%)
Loss of appetite23 (37.7%)
Nausea35 (57.4%)
Shortness of breath5 (8.2%)
Dizziness12 (19.7%)
Insomnia13 (21.3%)
Altered mood18 (29.5%)
Other5 (8.2%)
Constipation29 (47.5%)
Diarrhea22 (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:

ProblemReported by
Weekly visits to the clinic3 (4.9%)
Frequent blood tests5 (8.2%)
Cost17 (27.9%)
Lack of effectiveness35 (57.4%)
Other1 (1.6%)
None of the above18 (29.5%)

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

SymptomIdentified for improvement by
Abdominal pain50 (82.0%)
Bloating38 (62.3%)
Flatulence9 (14.8%)
Constipation38 (62.3%)
Diarrhea25 (40.9%)
Other3 (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 participateIndicated by
Condition is currently well managed20 (32.8%)
Condition does not severely affect life currently13 (21.3%)
Would have to travel too far36 (59.0%)
Would have to make too many clinic visits22 (36.1%)
Financial burden would be too high42 (68.9%)
Would require short-term hospital stays15 (24.6%)
Would require long-term hospital stays30 (49.2%)
Possibility treatment might not be effective8 (13.1%)
Possibility of minor side effects7 (11.5%)
Possibility of serious side effects40 (65.6%)
Possibility that current symptoms might worsen29 (47.5%)
Possibility of permanent disability37 (60.7%)
Possibility of death44 (72.1%)
Other2 (3.3%)

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

ActionWould encourage
Being matched with another participant20 (32.8%)
Transportation is provided22 (36.1%)
Clear explanation of the value of participation33 (54.1%)
Provided a summary of the outcome of the study33 (54.1%)
Frequent communication with researchers about study milestones25 (41.0%)
Financial compensation24 (39.3%)
Other1 (1.6%)
None of the above – no interest13 (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:

SymptomReported byFrequencySeverity
Straining9 (40.9%)86.671.0
Incomplete movements3 (13.6%)83.782.3
Feeling of incomplete5 (22.7%)85.266.3
Hard stools7 (31.8%)82.853.8
Interval between movements7 (31.8%)89.265.8
Abdominal pain11 (50.0%)84.168.5
Abdominal bloating8 (36.4%)94.381.5
Excessive flatulence4 (18.2%)92.069.8
Other1 (4.5%)N/AN/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:TypicalWorst
Sleep5 (22.7%)8 (36.4%)
Concentrate4 (18.2%)6 (27.3%)
Perform everyday activities3 (13.6%)7 (31.8%)
Participate in physical activities7 (31.8%)9 (40.9%)
Attend work or school2 (9.1%)6 (27.3%)
Eat10 (45.5%)11 (50.0%)
Socialize6 (27.3%)10 (45.5%)
Be intimate9 (40.9%)11 (50.0%)
Perform activities at home3 (13.6%)9 (40.9%)
Leave home at will4 (18.2%)7 (31.8%)

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

TreatmentUsed by
Changes to diet8 (36.4%)
Fiber supplements3 (13.6%)
Prescription pain medication0 (0.0%)
Antibiotics0 (0.0%)
Probiotics4 (18.2%)
Laxatives7 (31.8%)
Anti-diarrheal medication1 (4.5%)
Prescription medication for constipation9 (40.9%)
Prescription antispasmodic medication1 (4.5%)
Other prescription medication0 (0.0%)
OTC pain medication1 (4.5%)
Other OTC medications2 (9.1%)
Home remedies2 (9.1%)
Relaxation therapy0 (0.0%)
Hypnosis0 (0.0%)
Cognitive behavioral therapy1 (4.5%)
Emotional therapy0 (0.0%)
Other therapies0 (0.0%)
Alternative treatments0 (0.0%)
Other0 (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 EffectReported by
Pain7 (31.8%)
Fatigue4 (18.2%)
Loss of appetite7 (31.8%)
Nausea8 (36.4%)
Shortness of breath1 (4.5%)
Dizziness4 (18.2%)
Insomnia2 (9.1%)
Altered mood3 (13.6%)
Other0 (0.0%)
Constipation9 (40.9%)
Diarrhea6 (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:

ProblemReported by
Weekly visits to the clinic0 (0.0%)
Frequent blood tests0 (0.0%)
Cost4 (18.2%)
Lack of effectiveness9 (40.9%)
Other1 (4.5%)
None of the above3 (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:

SymptomIdentified for improvement by
Straining8 (36.4%)
Incomplete movements4 (18.2%)
Feeling of incomplete5 (22.7%)
Hard stools5 (22.7%)
Interval between movements8 (36.4%)
Abdominal pain10 (45.5%)
Abdominal bloating6 (27.3%)
Excessive flatulence3 (13.6%)
Other0 (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 participateIndicated by
Condition is currently well managed6 (27.3%)
Condition does not severely affect life currently5 (22.7%)
Would have to travel too far8 (36.4%)
Would have to make too many clinic visits2 (9.0%)
Financial burden would be too high12 (54.5%)
Would require short-term hospital stays4 (18.2%)
Would require long-term hospital stays6 (27.3%)
Possibility treatment might not be effective1 (4.5%)
Possibility of minor side effects1 (4.5%)
Possibility of serious side effects10 (45.5%)
Possibility that current symptoms might worsen6 (27.3%)
Possibility of permanent disability10 (45.5%)
Possibility of death12 (54.5%)
Other0 (0.0%)

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

ActionWould encourage
Being matched with another participant7 (31.8%)
Transportation is provided8 (36.4%)
Clear explanation of the value of participation11 (50.0%)
Provided a summary of the outcome of the study13 (59.1%)
Frequent communication with researchers about study milestones11 (50.0%)
Financial compensation9 (40.9%)
Other0 (0.0%)
None of the above – no interest0 (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.