Additional file 1: Table S1 Compassionate use online petition and social media use campaigns

Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) Approved (Merck, Entered into trial Sept 2014); through normal Change.or pembrolizumab channels, but soon g Facebook (cause page), Stage IV Bristol-Myers (trade Patient A 2013 disqualified due to (525,738 Twitter account, and Anti-PD-1 melanoma Squibb and Merck name Keytruda); complications. Did signatures personal website metastatic non- not receive a) small cell lung expanded access cancer Patient B 2013 Lung cancer Unable to verify Change.or N/A Anti-PD-1 Bristol-Myers Approved (Merck,

(Pancoast tumor) g (448 Squibb and Merck Sept 2014);

signatures pembrolizumab 2 Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) (trade

name Keytruda);

a) metastatic non-

small cell lung

cancer Patient C 2013 Stage IV Did not receive Change.or N/A Anti-PD-1 Merck Approved (Merck,

melanoma access g (30,311 Sept 2014);

signatures pembrolizumab

a) (trade

name Keytruda);

metastatic non-

small cell lung Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) cancer

The U.S. Not approved

White

House Facebook (community Anaplastic Did not receive online Request directly to Patient D Dec 2013 page), Twitter account, and ANP medulloblastoma access petition FDA YouTube video (102,215

signatures

) Patient E 2013 Diffuse intrinsic Received access Change.or N/A ANP FDA approved Not approved

pontine glioma g (19,992 under single

(DIPG) brainstem signatures patient protocol 4 Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s)

tumor a)

Approved (Eli

Lilly, Apr 2014);

Change.or ramucirumab

g (trade

Patient F 2013 Gastric cancer Unknown (103,179 N/A Ramucirumab Eli Lilly name Cyramza);

signatures advanced gastric or

a) gastro-esophageal

junction

adenocarcinoma Patient G 2011 Stage IV Did not receive Change.or Facebook (community Anti-PD-1 Bristol-Myers Not approvedb

melanoma access g page), Twitter account, (BMS-936558) Squibb Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) (200,701 YouTube video, and blog signatures (WordPress) a) Care2.co Facebook (community Not approved Did not receive Care2.com created MPS II Hunter m (62,041 page), YouTube video, and Enzyme Patient H access because of Shire Oct 2014 syndrome signatures blog (WordPress, replacement exclusion criteria ) CaringBridge) Patient I Diagnosed Oct Breast cancer Received treatment N/A Facebook (public figure Pertuzumab Genentech Approved

2005 of experimental page), YouTube video, (Genentech

drug, but switched personal website, and [acquired by

to other treatment affiliated non-profit website Roche], Jun 2012);

Pertuzumab (trade

name Perjeta); 6 Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) HER2-positive

metastatic breast

cancer Change.or Not approved

g (1,465 Patient J 2013 Coxsackievirus Unknown N/A Pleconaril Merck signatures

a) Treatment of Not approved

malignant rhabdoid Change.or Facebook (community tumor of the g (19,786 Brincidofovir Patient K 2013 Received treatment page), Twitter hashtag, Chimerix kidneys that signatures (CMX001) Tumblr, and CaringBridge resulted in a)

adenovirus Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) Approved (Merck,

Sept 2014); Change.or pembrolizumab g Facebook (removed), Did not receive Anti-PD-1 (trade Patient L 2014 Kidney cancer (481,795 Twitter account, and blog Merck access (MK-3475) name Keytruda); signatures (WordPress) metastatic non- ) small cell lung

cancer Change.or Not approved Diffuse intrinsic FDA g (68,714 Facebook (community Patient M 2013 pontine glioma Received treatment ANP compassionate use signatures page) and YouTube video (DIPG) petition a) 8 Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) Patient N 2013 Mantle cell Unknown Change.or N/A Ibrutinib (PCI- Pharmacyclics Approved

lymphoma g (1,303 32765) (Pharmacyclics,

signatures Nov 2013);

a) ibrutinib (trade

name Imbruvica);

mantle cell

lymphoma (2013),

chronic

lymphocytic

leukemia (2014),

and Waldenstrom

macroglobulinemia Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) (2015)

Approved (Merck,

Sept 2014);

Change.or pembrolizumab Anti-PD-1 Stage IV g (1,171 (trade Patient O 2013 Unknown N/A (widen MK3475 Merck melanoma signatures name Keytruda); expanded access) a) metastatic non-

small cell lung

cancer Patient P Blog created 2010 Brain tumor Received treatment N/A YouTube video and blog GDC-0449 Genentech Approved

(suspected, but not (WordPress) (Genentech

proven to be [acquired by 10 Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) Roche], Jan 2012);

vismodegib (trade

name Erivedge);

medulloblastoma) metastatic or

recurrent locally

advanced basal cell

carcinoma Patient Q 2012 Teratoma (brain Received treatment Change.or Facebook (community Pfizer Approved (Pfizer,

tumor) g (3,765 page) and CaringBridge CDK/patient’s Feb 2015);

signatures family self- palbociclib (trade

a) reported the drug name Ibrance);

as palbociclib ER-positive and Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) HER2-negative

metastatic breast

cancer Began ISIS- Change.or Not approved ISIS Spinal muscular SMNRx g Facebook (community Patient R 2012 ISIS-SMNRx Pharmaceuticals atrophy randomized trial 9 (13,421sig page) Inc. Nov 2015 naturesa) Change.or Not approved

Received treatment g BioMarin Stage IIIC ovarian Facebook (community PARP inhibitor Patient S 2013 from competitor (234,837 Pharmaceutical cancer page) and Twitter account BMN 673 company signatures Inc.

a) 12 Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) Change.or Not approved Amyotrophic NurOwn™ Did not receive g (4,519 BrainStorm Cell Patient T 2015 lateral sclerosis N/A (autologous, adult access signatures Therapeutics (ALS) stem cell therapy) ) Approved (Merck,

Sept 2014); Facebook (community pembrolizumab Stage IV alveolar page), Twitter account, Genentech, Bristol- Diagnosed Apr Did not receive (trade Patient U soft part sarcoma N/A YouTube video, blog Anti-PD-1 Myers Squibb, and 2012 access name Keytruda); (ASPS) (WordPress, CaringBridge), Merck metastatic non- and website small cell lung

cancer Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) Approved (Merck,

Sept 2014); Bristol-Myers Received Change.or pembrolizumab Squibb, Merck, treatment, but g (17,125 (trade Patient V 2013 Kidney cancer Blog (blogspot) Anti-PD-1/L1 MedImmune, discontinued due to signatures name Keytruda); CureTech, and side effects a) metastatic non- Genentech small cell lung

cancer

Patient W 2013 Metastatic breast Did not receive Change.or N/A Xofigo (radium- Bayer HealthCare Approved (Bayer,

cancer access (requested g (1,275 223) Pharmaceuticals May 2013);

for off-label use) signatures radium-223

a); dichloride (trade 14 Campaign type Drug characteristics Online FDA market

petition authorization;

website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications

signature

s) name Xofigo);

patients with

MoveOn. castration-resistant

org (81 prostate cancer,

signatures symptomatic bone

) metastases, and no

known visceral

metastatic disease

Data updated as of 30 November 2015. Online petitions reviewed: avaaz.org; care2.com; causes.com; change.org; ipetitions.com; front.moveon.org; thepetitionsite.com; and the U.S. White

House’s online petition site. Social media platforms reviewed: Facebook; Twitter; YouTube; Tumblr; CaringBridge; blog sites (e.g. WordPress); and other linked websites. aClosed/completed petition; bthe Bristol-Myers Squibb candidate did not receive approval, but a similar anti-PD-1 drug pembrolizumab (trade name Keytruda), developed by Merck, was approved by the FDA in

September 2014. ANP, antineoplaston; N/A, not available