Involving Patients in Reducing Decision Uncertainties Around Orphan and Ultra-Orphan Drugs: A Rare Opportunity?

[1] Meekings KN(2012)Orphan drug development: an economically viable strategy for biopharma R&D Drug Discov Today 17 660-664

[2] Williams CS(2012)Time to revisit the orphan drug law Eur J Clin Pharmacol. 68 113-2326

[3] Arrowsmith JE(2011)Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer JAMA 305 2320-702

[4] Garattini S(2012)Disease-branding and drug-mongering: could pharmaceutical industry promotional practices result in tort liability? Seton Hall Law Rev 42 643-190

[5] Kesselheim AS(2006)Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy CMAJ 174 189-279

[6] Myers JA(2011)Estimating the budget impact of orphan medicines in Europe: 2010–2020 Orphanet J Rare Dis 6 62-836

[7] Avorn J(2011)Drugs for rare diseases: influence of orphan designation status on price Appl Health Econ Health Policy 9 275-181

[8] Cetel JS(2005)Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 98 829-142

[9] Clarke JT(2002)Evidence-based policy: in search of a method Evaluation 8 157-S125

[10] Schey C(2010)Funding the unfundable: mechanisms for managing uncertainty in decisions on the introduction of new and innovative technologies into healthcare systems Pharmacoeconomics 28 113-357

[1] Meekings KN(2012)Orphan drug development: an economically viable strategy for biopharma R&D Drug Discov Today 17 660-664

[2] Williams CS(2012)Time to revisit the orphan drug law Eur J Clin Pharmacol. 68 113-2326

[3] Arrowsmith JE(2011)Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer JAMA 305 2320-702

[4] Garattini S(2012)Disease-branding and drug-mongering: could pharmaceutical industry promotional practices result in tort liability? Seton Hall Law Rev 42 643-190

[5] Kesselheim AS(2006)Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy CMAJ 174 189-279

[6] Myers JA(2011)Estimating the budget impact of orphan medicines in Europe: 2010–2020 Orphanet J Rare Dis 6 62-836

[7] Avorn J(2011)Drugs for rare diseases: influence of orphan designation status on price Appl Health Econ Health Policy 9 275-181

[8] Cetel JS(2005)Drugs for exceptionally rare diseases: do they deserve special status for funding? QJM 98 829-142

[9] Clarke JT(2002)Evidence-based policy: in search of a method Evaluation 8 157-S125

[10] Schey C(2010)Funding the unfundable: mechanisms for managing uncertainty in decisions on the introduction of new and innovative technologies into healthcare systems Pharmacoeconomics 28 113-357