<p>Additional file 1: Table S1 Compassionate use online petition and social media use campaigns</p><p>Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> 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, </p><p>(Pancoast tumor) g (448 Squibb and Merck Sept 2014); </p><p> signatures pembrolizumab 2 Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) (trade </p><p> name Keytruda); </p><p> a) metastatic non-</p><p> small cell lung </p><p> cancer Patient C 2013 Stage IV Did not receive Change.or N/A Anti-PD-1 Merck Approved (Merck, </p><p> melanoma access g (30,311 Sept 2014); </p><p> signatures pembrolizumab </p><p> a) (trade </p><p> name Keytruda); </p><p> metastatic non-</p><p> small cell lung Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) cancer </p><p>The U.S. Not approved</p><p>White </p><p>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 </p><p> signatures</p><p>) Patient E 2013 Diffuse intrinsic Received access Change.or N/A ANP FDA approved Not approved</p><p> pontine glioma g (19,992 under single </p><p>(DIPG) brainstem signatures patient protocol 4 Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s)</p><p> tumor a)</p><p>Approved (Eli </p><p>Lilly, Apr 2014); </p><p>Change.or ramucirumab </p><p> g (trade </p><p>Patient F 2013 Gastric cancer Unknown (103,179 N/A Ramucirumab Eli Lilly name Cyramza); </p><p> signatures advanced gastric or</p><p> a) gastro-esophageal </p><p> junction </p><p> adenocarcinoma Patient G 2011 Stage IV Did not receive Change.or Facebook (community Anti-PD-1 Bristol-Myers Not approvedb </p><p> melanoma access g page), Twitter account, (BMS-936558) Squibb Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> 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 </p><p>2005 of experimental page), YouTube video, (Genentech </p><p> drug, but switched personal website, and [acquired by </p><p> to other treatment affiliated non-profit website Roche], Jun 2012);</p><p>Pertuzumab (trade </p><p> name Perjeta); 6 Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) HER2-positive </p><p> metastatic breast </p><p> cancer Change.or Not approved</p><p> g (1,465 Patient J 2013 Coxsackievirus Unknown N/A Pleconaril Merck signatures</p><p> a) Treatment of Not approved</p><p> 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)</p><p> adenovirus Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) Approved (Merck, </p><p>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 </p><p> 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 </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) Patient N 2013 Mantle cell Unknown Change.or N/A Ibrutinib (PCI- Pharmacyclics Approved </p><p> lymphoma g (1,303 32765) (Pharmacyclics, </p><p> signatures Nov 2013); </p><p> a) ibrutinib (trade </p><p> name Imbruvica); </p><p> mantle cell </p><p> lymphoma (2013), </p><p> chronic </p><p> lymphocytic </p><p> leukemia (2014), </p><p> and Waldenstrom </p><p> macroglobulinemia Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) (2015) </p><p>Approved (Merck, </p><p>Sept 2014); </p><p>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-</p><p> small cell lung </p><p> cancer Patient P Blog created 2010 Brain tumor Received treatment N/A YouTube video and blog GDC-0449 Genentech Approved </p><p>(suspected, but not (WordPress) (Genentech </p><p> proven to be [acquired by 10 Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) Roche], Jan 2012); </p><p> vismodegib (trade </p><p> name Erivedge); </p><p> medulloblastoma) metastatic or </p><p> recurrent locally </p><p> advanced basal cell</p><p> carcinoma Patient Q 2012 Teratoma (brain Received treatment Change.or Facebook (community Pfizer Approved (Pfizer, </p><p> tumor) g (3,765 page) and CaringBridge CDK/patient’s Feb 2015); </p><p> signatures family self- palbociclib (trade </p><p> a) reported the drug name Ibrance); </p><p> as palbociclib ER-positive and Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) HER2-negative </p><p> metastatic breast </p><p> 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</p><p>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.</p><p> a) 12 Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> 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, </p><p>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 </p><p> cancer Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) Approved (Merck, </p><p>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 </p><p> cancer </p><p>Patient W 2013 Metastatic breast Did not receive Change.or N/A Xofigo (radium- Bayer HealthCare Approved (Bayer, </p><p> cancer access (requested g (1,275 223) Pharmaceuticals May 2013); </p><p> for off-label use) signatures radium-223 </p><p> a); dichloride (trade 14 Campaign type Drug characteristics Online FDA market </p><p> petition authorization; </p><p> website drug name; Date petition Self-reported Drug candidate Companies Name Disease (number Social media platforms approved created outcome name petitioned of indications</p><p> signature</p><p> s) name Xofigo); </p><p> patients with </p><p>MoveOn. castration-resistant </p><p> org (81 prostate cancer, </p><p> signatures symptomatic bone </p><p>) metastases, and no </p><p> known visceral </p><p> metastatic disease </p><p>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 </p><p>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 </p><p>September 2014. ANP, antineoplaston; N/A, not available</p>