The Importance of Data Collection Immune Deficiency ... - Ipopi

The Importance of Data CollectionImmune Deficiency Foundation, USAPresentation to IPOPIOctober 8, 2010Marcia BoylePresident & FounderImmune Deficiency Foundation

Mission StatementFounded in 1980, the ImmuneDeficiency Foundation (IDF) isthe national patient organizationin the United States dedicated toimproving the diagnosis,treatment and quality of life ofpersons with primaryimmunodeficiency diseasesthrough advocacy, education andresearch.www.primaryimmune.org

Patient AdvocacyGrassroots AdvocacyPatient EducationSurvey ResearchVolunteer NetworkPatient & Healthcare Publications and CommunicationMedical Outreach

Approach Running a Nonprofit PatientOrganization as a Business• Who are your clients?• Patients and Families• Medical Professionals: physicians, nurses, researchers• Donors, Industry, Government• What do you know about your clients?• Basic contact information• For patients, specific diagnosis, treatment• How do you collect this information?• Database (Relational, Spreadsheet)• How do you track the needs of your clients, and your activitiesin meeting those needs?• How do you communicate with your clients?• Website, Newsletters, email, meetings, etc.

What are Typical Questions Requiring Data?• How many patients and families do you represent?• What are their ages?• What are the diagnoses you represent, and how manypatients do you have with those diagnoses?• How many patients in your country have beendiagnosed with a Primary Immunodeficiency?• What is the demand for Immunoglobulin replacementfor PIDD in your country?• How may patients have disorders that require bonemarrow transplantation?

What Other Types of DataDo You Need to Track?• How many newsletters and other publications do you send ayear?• How many patient meetings and/or medical meetings do youhold?• How many people attend these meetings?• How successful are these meetings?• How many calls and requests for information do you receive?• What kind of requests do you receive?• If you have a website, how many unique visitors do you have?

Why is This Information Important?• Credibility and Impact• Stewardship• Planning• Influencing Government and Regulators

What Is The Basic Information You Need?(Paper)

What Is The Basic Information You Need?(Online)

Patient Contact/Intake Form

Patient Contact/Intake Form

Patient Contact/Intake Form

Patient Contact/Intake Form – Excel

Excel ExampleID Num Q1. Q1_opn. Q2a_fname. Q2a_lname. Q2a_add1. Q2a_add2. Q2a_city. Q2a_state.1000 1 Sally Smith 123 Forest Drive Apt. 2b Annapolis MD1001 2 Robert Jones 1582 Oak Street Indianapolis IN1002 3 Sister1003 1 Phillip Obolo 852 Sassafras Road New Orleans LA1004 1 Wendy VanScoy 462 Main Street Westminster KY1005 2 Brandon Arzabe 18 Music Master Court Boca Raton FL1006 2 Christine Asero 1685 Cha Cha Road Bowie MD1007 3 Friend1008 1 Cherie Mueller 56 Niamoa Street St. Louis MO1009 2 Lance Thoet 999 Mockingbird Lane Sea Cliff NY

Medical Professional Contact Form(Paper)

Medical Professional Contact Form(Online)

Medical Professional Contact Form

Medical Professional Contact Form

Medical Professional Contact Form

Tracking Numbers: Useful DataAt every opportunity, IDF tracks our activities andinteractions• Number of patients, families and medicalprofessionals at IDF meetings• Number and type of phone calls/e-mails received• Number and type of information mailed• Services requests from patientso Education, insurance, diagnosis, peer support,treatment, etc• Volunteer activity

Tracking Interactions: Useful DataAll interactions with patients recorded• Type of Interaction (phone or e-mail)• 16 different patient service categories• Date• Details• Ability to upload documents and files relevantto the interaction

Tracking Meetings• We track the number of attendees at each IDFfunction• Contact forms are taken to EVERY event• If person is new to IDF they are added tothe database• If person is known to IDF they are given anopportunity to update their contactinformation

Tracking Service InteractionsPerson calls/e-mails for materials or service• Number & type of materials IDF sends aretracked• Track 16 different categories of service and alsolog into patient record

Data from SurveysHaving all of this patient data helps IDF perform ourpatient surveys•Know how many patients are in our organization (14,000patients and/or parents of adolescent patients)•How many are children and adults•How many by each diagnosis (direct counts as well asaccurate estimation from surveys)•Know how many have what kinds of therapies and/ortreatments

IDF Data Collection ProgressionNo matter where your NMO may be in relationto IDF’s progression line above, collecting dataproperly, and doing it NOW is of the utmostimportance to your organization and the peopleyou serve.

Reach & Impact of IDF Programs• 2 million hits per month on the IDF Website• Over 16,000 unique visitors monthly on IDF Websites• 21,000 copies of the Advocate Newsletter mailed three times ayear, thousands more handed out at medical and patientmeetings• 12,000 E-Newsletters sent monthly• Approximately 11,555 patients and healthcare professionalsattended more than 116 educational presentations in 83 cities(33 states)• 8,054 patient inquires annually

Patient Services Inquiries: January–June (2008-2010)Service Type 2010 2009 2008Denying IVIG When Medically Necessary 220 257 155Diagnosis 203 185 88Disability 60 184 69Employment Discrimination 10 8 2General Insurance Issues 258 295 227Medicare's Reimbursement for IVIG 148 173 186Patient Education 685 797 1422Peer Support 165 142 158Physician Location 288 270 265Private Health Insurance - Cost Shifting/Sharing 285 334 254School Issues 30 31 33Supply 0 7 120Treatment 263 167 79Uninsured 228 337 126Unrelated to PIDD/Other 197 249 179TOTAL 3,040 3,436 3,363

Meeting Evaluation Form

IDF Retreat – Rye Brook, NYThe evaluations were overwhelmingly positive!• 100% agreed that they would recommend the IDF Retreat to others• 97% agree that there were many opportunities to interact with others• 91 – 100% agreed that the sessions increased their knowledge of thesubject matter• 88 -100% agreed that the information helped them better understand ormanage PIDD• 97% thought the exhibitors were a valuable addition to the retreat• 94% agreed that the materials and resources they received were useful• 100% of the parents with children in the Youth Program said that their childenjoyed the childcare/Kids Club/Tween/Teen Escape

IDF Retreat – Rye Brook, NY• “This was a great learning experience for our family. Our daughtergot to know other teens with PIDD.”• “This event was important to learning how to live better, and bemore proactive in our own treatment and options.”• “Conference was wonderful and energizing. We’re leaving with newinformation and new supports/friends! Location and center wereperfect. Our kids really had a great time and learned lots!”• “This conference is an unexpected joy and reward! I don’t feel soalone anymore. Thank You!”• “Thank you for the amazing ability to network and interact withthose who treat and research in the field. Thank you for all thegreat food and activities. Always educational!”• “Best One Ever---Thank You!!”

IDF & USIDNET 2010LeBien Visiting Professor ProgramProvides teaching hospitalswith an expert clinicalimmunologist to lead grandrounds and other educationalactivities.2010 SitesAllergy, Asthma and Immunology Center of AlaskaChildren’s Hospital of PittsburghDoernbecher Children’s Hospital, PortlandMercy St. Charles HospitalUniversity of Buffalo, Dept. of Family MedicineUniversity of IL, College of Medicine at RockfordUniversity of Tennessee, College of MedicineUniversity of VT, Vermont Children’s HospitalWake Forest UniversityWeill Cornell Medical CollegeWestern Montana Clinic, PC

LeBien Visiting Professor ProgramResidency Program Directors rated the VisitingProfessor Program as “excellent” in all categories•Speaker•Value of the topic•Relevance of the topic•Quality of the presentation•Quality of accompanying materials

IDF Friends1/1/10 – 8/31/10• 1,658 members• 54,731 visits• 11,857 uniquevisitors• 638,279 pageviewsthroughout thesite• 55,406homepageviews

IDF WebsiteMonth Page Views Unique Visitors Avg. Time on SiteJanuary 25,985 10,675 02:17February 24,864 10,329 02:20March 36,824 14,593 02:31April 28,154 10,982 02:25May 24,572 9,676 02:33June 24,573 9,738 02:30July 22,836 9,534 02:20August 24,854 10,497 02:32Total 212,662 86,024 02.26

Immune Deficiency Foundation SurveysBetter Information for Better Outcomes• First National Survey of Physicians(1995)• First National Patient Survey(1996)• First National Treatment Survey(1997)• IVIG Shortage Survey ofPhysicians (1998)• IVIG Use Among Patients: 5 YearUpdate (2001)• Second National Survey ofPatients (2002)• Second National Treatment Survey(2002)• Second National Survey ofPhysicians (2003)• Self-Infusing Patient Survey (2003)• Prevalence Survey (2005)• Access to Care Surveys• (2006) Patients, Physicians,Hospital Pharmacist Directors• Hospital Pharmacist Directors• (2 nd & 4 th Qtrs. 2007)• Third National Patient Survey(2007)• AAP Survey (2007)• Medicare Patient Survey (2008)• Third National Treatment Survey(2008)• National Internet Treatment Surveyof PIDD in the U.S. (2010)

Critical Issues Addressed by IDF Data• IVIG Shortage: 1997-2000• Medicare Reimbursement of IVIG: 2005-Present• Prevalence of PIDD: 2005 Household Survey• Standards of Care for PIDD: AAAAI and ESID SpecialistSurveys• Standards of Care for PIDD: Pediatrics and Family Practiceprimary care physician surveys• Treatment Patterns and Outcomes: Patient Surveys 1996-2008• SCID Survey Testimony for Newborn Screening, 2009• Under-Treatment: Web Survey of Treatment Experiences,2010

PublicationsJournal of Clinical Immunology – 2007• Population Prevalence of Diagnosed Primary Immunodeficiency Diseases inthe United States• J.M. Boyle, R.H. BuckleyClinical Immunology – 2009• Use of Intravenous Immunoglobulin and Adjunctive Therapies in theTreatment of Primary Immunodeficiencies• Pierre L. Yong, John Boyle, Mark Ballow, Marcia Boyle, Jordan S. Orange,et al.Clinical Immunology – 2010• Early vs. Delayed Diagnosis of Severe Combined Immunodeficiency: A FamilyPerspective• Alice Chan, Christopher Scalchunes, Marcia Boyle, Jennifer M. Puck• Manuscript Accepted for Clinical Immunology 2010

2002 & 2007 IDF National Patient SurveysAverage Time to Diagnosis From Symptom Onset141212.4109.2Years to Diagnosis864202002 2007Sources: 2002 IDF National Patient Survey, 2007 IDF National Patient Survey

2002 & 2007 National Patient Surveys Patients by Age40%35%2002 200735%30%29%25%20%21%17%15%10%5%9%7%13%11%9%7%14%14%5%10%0%0 to 6 7 to 12 13 to 17 18 to 29 30 to 44 45 to 64 65+Q5. What is the date of birth of the (adult patient/oldest child) in the household with a primaryimmunodeficiency diseases? (Base 1996 N= 908, Base 2002 N = 1,526 Base 2007 N = 1,351)Sources: 2002 IDF National Patient Survey, 2007 IDF National Patient Survey

Health Status Improves after IG TherapyPrior to diagnosis only 16% good or betterAfter Diagnosis 66% good or betterQ10. Would you describe his/her health in the 12 months prior to diagnosis…..? BASE: Those who are currently using IVIG or SCIG Therapy N=955Q61. Would you describe his/her health in the past 12 months as…..? BASE: Those who are currently using IVIG orSCIG Therapy N=955Source: 2008 IDF National Patient Treatment Survey

Permanent Impairments Or LossesPrior To Initial Diagnosis: 200854% of patients reported a permanentfunctional impairmentQ9. By the time of initial diagnosis as immune deficient, had he/she suffered any permanent impairment or lossof________? (net of cases) N=1,030Source: 2008 IDF National Patient Treatment Survey

IDF Surveys of Specialists and Primary Care Physicians:American Academy of Allergy, Asthma and Immunology(AAAAI), Pediatricians, Family PracticeSurvey Year Mode Incentive Responses*AAAAI 2006 Internet No 405**Pediatricians 2008 Mail Yes 560Family Practice 2009 Mail Yes 528* Article based on this survey published in Clinical Immunology** Manuscript based on survey in final stages to be submitted to Pediatrics

Aware of Professional Guidelines for PIDD:Pediatrician and Family Practice ComparisonN= 560 N= 528Q. Are you aware of any professional guidelines for the diagnosis and management ofprimary immunodeficiency diseases?Sources: IDF 2008 Survey of Pediatricians, IDF 2009 Survey of Family Physicians

PIDD Covered in Medical SchoolPediatrician and Family Practice ComparisonN= 560 N= 526Q. How fully were primary immunodeficiency diseases covered in medical school? Very well,Adequately, Only a little, not at allSources: IDF 2008 Survey of Pediatricians, IDF 2009 Survey of Family Physicians

Level of Comfort with PIDDPediatrician and Family Practice ComparisonPediatricianFamily Practice*N= 560 N= 524Q. What is your level of comfort with the recognition and diagnosis of primaryimmunodeficiency diseases? Completely comfortable, Very comfortable, Somewhatcomfortable, Not At All comfortable* 1% “missing”Sources: IDF 2008 Survey of Pediatricians, IDF 2009 Survey of Family Physicians

Effectiveness of IgG Therapy:Pediatrician and Family Practice ComparisonN= 395 N= 206Q. How effective is immunoglobulin replacement therapy in the treatment of antibodydisorders? Very effective, Somewhat effective, Not too effective, Not effective at all Base:Those answeringSources: IDF 2008 Survey of Pediatricians, IDF 2009 Survey of Family Physicians

Recommend IgG Therapy for All or Most Patients byDiagnosis:Immunologist, Pediatrician & Family Practice ComparisonsQ. Would you recommend IVIG/IgG Replacement therapy for all, most, some or few to nopatients with…Sources: IDF‐AAAAI 2006 Survey of the AAAAI membership; IDF 2008 Survey of Pediatricians; IDF 2009 Survey of FamilyPhysicians

Perceived Treatment Risk of IgG TherapyImmunologist, Pediatrician & Family Practice Comparisons“Moderate to Relatively High Risk”Q. In counseling a patient with a PIDD considering IVIG/IgG replacement therapy, how would youpresent the risk of contracting the following diseases as a result of treatment? No real risk, a smallbut measurable risk, a moderate risk over time, a relatively high riskSources: IDF‐AAAAI 2006 Survey of the AAAAI membership; IDF 2008 Survey of Pediatricians; IDF 2009 Survey of FamilyPhysicians

Use of Survey Results• Share data with medical societies to improve physician awareness• Exhibit at AAAAI, AAP, AAFP• AAP National Meeting: Dinner symposium 2008, 2009, 2010• AAFP National Meeting: IDF and PIDD will be part of the 2010plenary session• Develop articles based on data for the key journals of each medicalsociety• Article based on results of AAAAI specialist survey published inClinical Immunology, online, 12/09; print 6/10• Article based on IDF SCID Survey has been accepted forpublication in Clinical Immunology.• Article based on results of Pediatrician survey being finalized forsubmission

Patients in the General Community:A National Probability Survey of the Prevalence ofPatients diagnosed with PIDDIDF Prevalence Survey• National telephone survey of 10,000households• 1 in 1,200 persons diagnosed with PIDD• Estimate of 250,000 PIDD in the U.S.• 95% confidence level around theestimate suggests 152,000 to 361,000diagnosed PIDD in the U.S.• *66% of the patients in the survey had adiagnosis for which IG therapy isindicated.• Only 22% of antibody deficient patientscurrently treated with IVIG* Originally IgG Subclass Deficiency was left out of this calculation. If IgG Subclass is removed this frequencywould be 57%. IDF decided to revise this number to include IgG Subclass as we feel it is more accurate, andfacilitates direct comparison to the IDF 2010 National Internet Treatment Survey of Primary ImmunodeficiencyDiseases

National Internet Treatment Survey of PrimaryImmune Deficiency Diseases, 2010Study Objectives• In order to characterize the treatment of PIDD patients withimmunoglobulin replacement therapy• We wanted to generate a sample of at least 100 diagnosed patientsto provide more stable estimates of immunoglobulin use• To do so, we used a large web panel of the general public to screenfor a nationally representative community sample of personsdiagnosed with primary immune deficiency diseases (more than850,000 invitations sent)• This survey was nationally representative, although not a probabilitysurvey

Utilization of IgG Therapy:Under-treatment is a Serious ConcernPIDD Indicated as TreatmentBased on DiagnosisIgG Therapy Use inAntibody Deficient PatientsQ5a/b. What specific types of primary immune deficiency hasthe (AGE) been diagnosed with?Base: All living eligible PID diagnosis N=174Q8c. Is the (AGE) currently being treated with any of the following?Base: All living eligible PIDD for which IgG therapy is indicated N=133Source: IDF 2010 National Internet Treatment Survey of Primary Immunodeficiency Diseases

Current IgG Use:1996, 2002 & 2007 IDF Patient Surveys andCommunity SurveysQ. Is the patient currently being treated with IVIG or SCIG?Base: All patientsSources: IDF Primary Immune Deficiency Diseases in America: 1996, 2002 & 2007,2006 IDF National Prevalence Survey, IDF 2010 National Internet Treatment Survey of Primary ImmunodeficiencyDiseases

Perception of the Effectiveness ofIgG Therapy in Treating Antibody DisordersN= 147 N= 395 N= 206Q32. Based on what you know or have heard, how effective is immunoglobulin therapy in treating antibody disorders? Q14. How effective isIgG replacement therapy in the treatment of antibody deficiency disorders? Very effective, Somewhat effective, Not too effective, noteffective at allSources: IDF 2010 National Internet Treatment Survey of Primary Immunodeficiency Diseases,2008 & 2009 IDF Survey of Physician Attitudes and Practices Related to the Diagnosis and Management of PrimaryImmunodeficiency Diseases

Safety of Immunoglobulin for Personswith Primary Immunodeficiency DiseasesQ35. Based on what you know or have heard, how safe is immunoglobulin therapy for persons withprimary immunodeficiency diseases? N=147Source: IDF 2010 National Internet Treatment Survey of Primary Immunodeficiency Diseases

Reason Never Used ImmunoglobulinQ8b. Is there a reason why the patient has never been treated with immunoglobulinreplacement therapy? Base: Antibody deficient patients, Never used IM, IV OR SQN=26Source: IDF 2010 National Internet Treatment Survey of Primary Immunodeficiency Diseases

Immunologist LocationQ16. Is the immunologist seen by the (AGE) located in …..? N=147Source: IDF 2010 National Internet Treatment Survey of Primary Immunodeficiency Diseases

Current Health Status:IDF 2007 Patient vs. IDF 2010 Internet Surveys“Good or Better”61%48%N= 1,351N= 147Q. Would you describe the (AGE)’s current health status as Excellent, very good, good, fair,poor, very poorSources: IDF 2010 National Internet Treatment Survey of Primary Immunodeficiency Diseases, IDF 2007 National PatientSurvey

Ever and Current Treatment withImmunoglobulin by Heard of IDFQ8a. Has the (AGE) ever been treated for a month or longer with the following?Q8c. Is the (AGE) currently being treated with any of the following? All living eligible PIDD,N=174Source: IDF 2010 National Internet Treatment Survey of Primary Immunodeficiency Diseases

Conclusions• In the Prevalence telephone survey, current treatment with immunoglobulinwas 22%, with an expected confidence interval of + 16.9 percentage points• In the Internet survey, current treatment with immunoglobulin is 46%. Ifthis were a probability sample the confidence interval would be + 7.5percentage points• In IDF surveys of its members, current treatment with immunoglobulin hasbeen much higher, e.g., 70%• These findings, coupled with those from two surveys of primary carephysicians, suggest that PIDD patients outside major medical centers maynot be receiving treatment consistent with the guidelines• We all agree that Early Diagnosis is critical to patient health status• This research suggests that even with diagnosis, a significant number ofpatients are likely under treated, as physicians don’t think Ig Therapy is aseffective or safe as specialists believe

Conclusions• Only 47% of patients with PIDD who have not heard of IDFare currently taking immunoglobulin therapy.• 61% of patients with PIDD who have heard of the IDF arecurrently using immunoglobulin therapy for their condition.• These findings confirm that patients with PIDD whoare connected to IDF are more likely to be receivingimmunoglobulin therapy, than those in the generalcommunity.

Thank You!