A Source of Hard- to- Find Pa3ents and Caregivers For Researchers. Peter Ziedins

Transcription

1 A Source of Hard- to- Find Pa3ents and Caregivers For Researchers Peter Ziedins

2 About RPV Rare Pa3ent Voice, LLC was formed to provide pa;ents and caregivers with rare diseases an opportunity to share their insights through interviews and surveys to improve medical products and services. Pa;ent advocacy and support groups have benefited from encouraging pa;ents to sign up. In just over two years, over 7,000 pa;ents in the US have signed up. 2

3 So far this year. 2,600 pa;ents have shared their experiences Illustra;ng how they are affected by rare disorders Evalua;ng poten;al new products being developed by research ins;tu;ons and the industry Discussing which treatment benefits are most important to pa;ents Describing how side effects of chemotherapy impacted their lives 270 research projects conducted Internet surveys Telephone interviews 3

4 What s in it for advocacy groups? Rare Pa;ent Voice partners with advocacy groups in several ways: You can invite pa;ents and caregivers to contact us via , your newslexer, or other means; if they decide to sign up, they receive a $5 gi[ card and your organiza;on receives a dona;on of $5 per pa;ent. Rare Pa;ent Voice can sponsor one of your group s events and invite members to sign up at the event. We can do a free survey of your members via the Internet and provide you the results. 4

5 What s in it for pa3ents? Help others by sharing their experiences, insights and opinions Help biopharma companies focus on your rare condi;on $5 gi[ card just for signing up If they refer a pa;ent who signs up, they get another $5 gi[ card $25 for a 15 minute survey to $100 or more for an hour- long telephone interview 5

6 How can we make it work here? Can we adapt the US model or do we need to take an en;rely different approach? How do we get advocacy groups in Canada to partner with us? Concerns from pa;ents? Should we offer an incen;ve to sign up? Could it backfire? Is $5 enough? For which merchant(s)? Tim Horton s? Starbucks? Amazon? 6

7 Patient Counts (7/10/15) Condi3on Pa3ents/ Caregivers Colum n1 Condi3on2 Pa3ents/ Caregivers3 Acromegaly 8 Cys;c Fibrosis 1,122 Addison s 21 Duchenne Muscular Dystrophy 25 Allergies 201 Ehrlers Danlos Syndrome 307 Alpha 1 An;trypsin Deficiency 16 Eosinophilia Esophagi;s 64 ALS 24 Epilepsy 108 Ankylosing Spondyli;s 151 Factor V Leiden 17 Anthrogryposis 25 Factor VII Deficiency 18 Aplas;c Anemia 70 Fibromyalgia 201 Asthma 84 Gaucher Disease 39 CDKL5 19 Hemophilia A 466 Celiac Disease 47 Hemophilia B 143 Cerebral Palsy 55 Hepi;;s C 81 Chiari Malforma;on 67 Hereditary Angioedema 11 Crohn s 344 HTC Nurses and PAs 47 Cushing s 32 Human Growth Hormone Deficiency 132 7

8 Patient Counts, continued (7/10/15) Condi3on Pa3ents/ Caregivers Colu mn1 Condi3on2 Pa3ents/ Caregivers2 Hun;ngton s Disease 102 Psoriasis, Psoria;c Arthri;s 52 Idiopathic Thrombocytopenia Purpura 32 Pulmonary Arterial Hypertension 235 Juvenile Idiopathic Arthri;s 42 Pulmonary Fibrosis 32 Lennox- Gastaut Syndrome 159 ReX Syndrome 49 Lupus 356 Rheumatoid Arthri;s 43 Meniere s 15 Sarcoidosis 53 Mucopolysaccharidosis (MPS) 35 Scleroderma 120 Mul;ple Sclerosis 1,823 Sickle Cell Disease 188 Muscular Dystrophy 60 Sjogren s Syndrome 56 Myasthenia Gravis 13 Spinal Muscular Atrophy 158 Organ Transplant 44 Tuberous Sclerosis 110 Osteogensis Imperfecta 84 Turner Syndrome 22 Parkinson s 28 Ulcera;ve Coli;s 36 Polycythemia Vera 22 Von Willebrand Disease 127 ü Pa;ent profiles include age, gender, date of diagnosis, severity, and medica;ons 8

9 9 Cancer Patient Counts Cancers Cancer Type Patients Total 19,437 Anal Cancer 203 Bladder Cancer 195 Bone Cancer 166 Brain/Spinal Cord 396 Breast Cancer 7,451 Cervical Cancer 476 Colorectal Cancer 1,362 Endometrial Cancer 698 Esophageal Cancer 154 Head/Neck/Throat 711 Hodgkin Disease 392 Kidney Cancer 283 Leukemia 582 Liver Cancer 143 Lung Cancer 1,194 Multiple Myeloma 251 Non- Hodgkin Lymphoma 898 Ovarian Cancer 989 Pancreatic Cancer 290 Prostate Cancer 510 Sarcoma soft tissue 208 Skin Cancer Melanoma 360 Skin Cancer non- Melanoma 109 Stomach Cancer 139 Testicular Cancer 95 Thyroid Cancer 428 Waldenstrom s Macroglobulinemia 107 Cancer pa;ents iden;fied by: ü tumor type ü sub- type ü stage of cancer ü muta;on ü stage of treatment ü age ü loca;on By Stage Patients Stage 1 2,597 Stage 2 3,289 Stage 3 2,670 Stage 4 2,353 Recurrent 405 Stage of Treatment Patients Newly diagnosed 2,778 Currently in active treatment 5,991 Finished active treatment less than 5 years ago 4,594 Finished active treatment more than 5 years ago 1,207 Living with cancer as a chronic illness 1,279 Receiving hospice or palliative care 73