1. Cystinosis Patient Survey Data Analysis Final Report August 30, 2010

2. Scope of Research 1. Partnership initiated with CRN to reach patients and families with cystinosis. 2. JPA fielded survey via online, mail and phone calls – July 17 through present 3. Total responses to date: 192 US = 162 Ex-US = 21 Unknown = 9 2 * The following analysis is based on US surveys Cystinosis Patient Survey Preliminary Data Analysis

3. Key Takeaways While the vast majority (94%) of cystinosis patients have experienced crystal formation, only 61% currently use the eye drops. For those that aren’t using the drops, it is primarily because they haven’t been recommended or the patient doesn’t experience symptoms; Currently, price is not a major factor in use of the drops. The primary source of the eye drops is NIH Eye Clinic. Most patients start using the drops over 1 year after cystinosis diagnosis. Compliance in terms of daily use and storage varies. The majority (89%) of patients have health insurance, but household income varies widely among patients and families. Cystinosis Patient Survey Preliminary Data Analysis 3

4. Cystinosis Diagnosis 4 3.4 yearsAverage age at cystinosis diagnosis PrenatalMinimum age at cystinosis diagnosis 25 yearsMaximum age at cystinosis diagnosis Cystinosis Patient Survey Preliminary Data Analysis

5. Cystine Crystal Formation 5 Age when crystals were first identifiable: Average age = 4.5 years Min age =.25 years Max age = 28 years Only 16 respondents indicated that they know their crystal score: Min score = 1 Max score = 8 Cystinosis Patient Survey Preliminary Data Analysis

6. Eye Drop Usage 6 Cystinosis Patient Survey Preliminary Data Analysis

7. Eye Drop Usage 7 Other includes quality of life issues: “Stink” “Need to use too often” “Uncomfortable” “Hassle” “Symptoms are minimal” Cystinosis Patient Survey Preliminary Data Analysis Respondents selected multiple responses.

8. Treatment Continuum 8 Cystinosis Patient Survey Preliminary Data Analysis

9. Eye Drop Prescribers 9 Other includes specific locations and doctors: NIH Dr. Gahl Dr. Kaiser Dr. Matalon Cystinosis Patient Survey Preliminary Data Analysis

10. Method of Distribution 10 Other includes: Mayo Clinic S&R Drug Company in Kirbyville, TX Cystinosis Patient Survey Preliminary Data Analysis Respondents selected multiple responses.

11. Eye Drop Compliance 11 Cystinosis Patient Survey Preliminary Data Analysis

12. Eye Drop Storage 12 Cystinosis Patient Survey Preliminary Data Analysis

13. Eye Drop Effectiveness 13 Cystinosis Patient Survey Preliminary Data Analysis

14. Commercially Available Eye Drops Cystinosis Patient Survey Data Analysis: Preliminary14

15. Kidney Transplants 15 Age at transplant Average age = 14 Min age = 5 Max age = 56 Cystinosis Patient Survey Preliminary Data Analysis

16. Health Insurance Status 16 Other primarily includes state-run health programs, e.g., Mass Safety Net, Illinois Public Aid Cystinosis Patient Survey Preliminary Data Analysis Respondents selected multiple responses.

17. Demographic Information 17 Cystinosis Patient Survey Preliminary Data Analysis

18. Patient Location (n=162) 18 DE 1 AZ 6 CA 6 CO 1 CT 2 AL 4 FL 7 GA 7 IA 4 IL 11 IN 4 KS 1KY 5 MA 6 MD 2 ME 1 MI 7 MN 4 MO 1 MS 2 NC 5 NE 2 NH 2 NJ 1 NY 14 OH 7 SC 4 OR 2 PA 7 OK 2 TN 3 TX 8 UT 5 VA 3 WA 4 VT 2 WI 6 WV 2 Cystinosis Patient Survey Preliminary Data Analysis MO 2

19. Cystinosis Information & Resources 19 Cystinosis Patient Survey Preliminary Data Analysis Respondents selected multiple responses.

20. Cystinosis Information & Resources (cont.) 20 Cystinosis Patient Survey Preliminary Data Analysis Respondents selected multiple responses.

21. 21 Cystinosis Information & Resources (cont.) Cystinosis Patient Survey Preliminary Data Analysis Respondents selected multiple responses.

22. Jones Public Affairs Contact Please direct any questions to: Kelly Butterworth 202.591.4052 kelly@jonespa.com Cystinosis Patient Survey Data Analysis: Preliminary 22