Orphan drug development is not taking off.

PubWeight™: 2.27‹?› | Rank: Top 2%

🔗 View Article (PMC 2686065)

Published in Br J Clin Pharmacol on January 21, 2009

Authors

Roberta Joppi, Vittorio Bertele, Silvio Garattini

Articles citing this

Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat Rev Drug Discov (2010) 4.02

Estimating the budget impact of orphan medicines in Europe: 2010 - 2020. Orphanet J Rare Dis (2011) 1.95

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol (2012) 1.39

New treatments for mitochondrial disease-no time to drop our standards. Nat Rev Neurol (2013) 1.26

Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol (2011) 1.15

Forecasting drug utilization and expenditure in a metropolitan health region. BMC Health Serv Res (2010) 1.13

An empirical review of major legislation affecting drug development: past experiences, effects, and unintended consequences. Milbank Q (2011) 1.12

Reconciling uncertainty of costs and outcomes with the need for access to orphan medicinal products: a comparative study of managed entry agreements across seven European countries. Orphanet J Rare Dis (2013) 1.08

Delays in the submission of new drugs in Canada. CMAJ (2014) 1.05

Developing and paying for medicines for orphan indications in oncology: utilitarian regulation vs equitable care? Br J Cancer (2012) 0.92

[Off-label therapy: current problems from the perspective of the Pharmaceutical Commission of the German Medical Profession]. Z Rheumatol (2012) 0.84

Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe? Orphanet J Rare Dis (2011) 0.84

Time to revisit the orphan drug law. Eur J Clin Pharmacol (2011) 0.78

Articles cited by this

Why rare diseases are an important medical and social issue. Lancet (2008) 4.20

Rare diseases: what's next? Lancet (2008) 3.06

Rare diseases and orphan drugs. Br J Clin Pharmacol (2006) 1.83

Orphan drug development is progressing too slowly. Br J Clin Pharmacol (2006) 1.79

Clinical trials of orphan medicines. Lancet (2008) 1.34

Predictors of orphan drug approval in the European Union. Eur J Clin Pharmacol (2008) 1.16

Articles by these authors

Rare diseases: what's next? Lancet (2008) 3.06

Smoking in Italy 2005-2006: effects of a comprehensive National Tobacco Regulation. Prev Med (2007) 2.61

Efficacy, safety, and cost of new anticancer drugs. BMJ (2002) 2.53

Risk sharing arrangements for pharmaceuticals: potential considerations and recommendations for European payers. BMC Health Serv Res (2010) 2.21

Systemic safety of bevacizumab versus ranibizumab for neovascular age-related macular degeneration. Cochrane Database Syst Rev (2014) 1.95

Orphan drug development is progressing too slowly. Br J Clin Pharmacol (2006) 1.79

Disappointing biotech. BMJ (2005) 1.66

Laparoscopic approach to acute abdomen from the Consensus Development Conference of the Società Italiana di Chirurgia Endoscopica e nuove tecnologie (SICE), Associazione Chirurghi Ospedalieri Italiani (ACOI), Società Italiana di Chirurgia (SIC), Società Italiana di Chirurgia d'Urgenza e del Trauma (SICUT), Società Italiana di Chirurgia nell'Ospedalità Privata (SICOP), and the European Association for Endoscopic Surgery (EAES). Surg Endosc (2012) 1.55

Errors in the approval process and post-marketing evaluation of drotrecogin alfa (activated) for the treatment of severe sepsis. Lancet Infect Dis (2009) 1.55

Anything new in EU pharmacovigilance? Eur J Clin Pharmacol (2011) 1.53

Mild depression in general practice: is the automatism of antidepressant prescribing an evidence-based approach? Acta Psychiatr Scand (2006) 1.42

Varying and "atypical" indications for atypical antipsychotics. Psychopharmacology (Berl) (2003) 1.40

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol (2012) 1.39

Italy must invest more in science and technology. Nature (2008) 1.38

Comparing policies to enhance prescribing efficiency in Europe through increasing generic utilization: changes seen and global implications. Expert Rev Pharmacoecon Outcomes Res (2010) 1.31

How can research ethics committees protect patients better? BMJ (2003) 1.29

Policies to enhance prescribing efficiency in europe: findings and future implications. Front Pharmacol (2011) 1.19

Recent national and regional drug reforms in Sweden: implications for pharmaceutical companies in Europe. Pharmacoeconomics (2008) 0.99

Enhancing the rational use of new medicines across European health care systems. Eur J Clin Pharmacol (2008) 0.99

Reconsidering the Declaration of Helsinki. Lancet (2013) 0.98

Dabigatran - a case history demonstrating the need for comprehensive approaches to optimize the use of new drugs. Front Pharmacol (2013) 0.95

Fish oil and mental health: the role of n-3 long-chain polyunsaturated fatty acids in cognitive development and neurological disorders. Int Clin Psychopharmacol (2006) 0.95

Are new models needed to optimize the utilization of new medicines to sustain healthcare systems? Expert Rev Clin Pharmacol (2015) 0.91

Fluoxetine dose and outcome in antidepressant drug trials. Eur J Clin Pharmacol (2002) 0.90

A double-blind, placebo-controlled, randomized trial of bupropion for smoking cessation in primary care. Arch Intern Med (2007) 0.89

Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders. J Comp Eff Res (2014) 0.87

Withdrawal of 'Xigris' from the market: old and new lessons. J Epidemiol Community Health (2012) 0.86

Pre-market clinical evaluations of innovative high-risk medical devices in Europe. Int J Technol Assess Health Care (2012) 0.86

Drug sales in four European countries still differ. BMJ (2003) 0.85

Flaws in animal studies exploring statins and impact on meta-analysis. Eur J Clin Invest (2014) 0.84

Thrombolysis or nothing for acute myocardial infarction? It's all the same! Br J Clin Pharmacol (2008) 0.84

Regulatory policies on medicines for psychiatric disorders: is Europe on target? Br J Psychiatry (2007) 0.84

Independent clinical research in Europe. Lancet (2004) 0.83

Role of presynaptic alpha2-adrenoceptors in antidepressant action: recent findings from microdialysis studies. Prog Neuropsychopharmacol Biol Psychiatry (2004) 0.82

Haematological anticancer drugs in Europe: any added value at the time of approval? Eur J Clin Pharmacol (2007) 0.82

New antirheumatic drugs: any real added value? A critical overview of regulatory criteria for their marketing approval. Eur J Clin Pharmacol (2007) 0.82

Patient organizations' funding from pharmaceutical companies: is disclosure clear, complete and accessible to the public? An Italian survey. PLoS One (2012) 0.82

Having your cake and eating it: office of fair trading proposal for funding new drugs to benefit patients and innovative companies. Pharmacoeconomics (2008) 0.81

Most appropriate animal models to study the efficacy of statins: a systematic review. Eur J Clin Invest (2014) 0.81

European regulatory policies on medicines and public health needs. Eur J Public Health (2003) 0.80

Europe also needs agency for postmarketing surveillance. BMJ (2005) 0.80

[A cross-sectional survey to evaluate the cardiovascular risk profile in subjects referred to the Verona-ASL 20 general practitioners]. Ital Heart J Suppl (2005) 0.80

Predicting the clinical relevance of drug interactions from pre-approval studies. Drug Saf (2009) 0.79

New drugs for thromboprophylaxis in atrial fibrillation. Eur J Intern Med (2011) 0.79

EMA's reflection on placebo does not reflect patients' interests. Eur J Clin Pharmacol (2011) 0.78

Time to revisit the orphan drug law. Eur J Clin Pharmacol (2011) 0.78

CNS drugs approved by the centralised European procedure: true innovation or dangerous stagnation? Psychopharmacology (Berl) (2006) 0.78

A new anti-cancer drug in the market: Good news for investors or for patients? Eur J Cancer (2008) 0.77

Confidentiality. Lancet (2003) 0.77

Bevacizumab and ranibizumab. A matter of public interest. BMJ (2010) 0.77

Controlling cervical cancer. Pharmacoeconomics (2009) 0.75

Informed consent: meet patients' needs. Nature (2012) 0.75

Is there discrimination in access to therapy for HCV patients? J Hepatol (2008) 0.75

Exenatide for type 2 diabetes. Lancet (2009) 0.75

Effects of physical inactivity on non-communicable diseases. Lancet (2012) 0.75

Ethics of testing drugs with readily available alternatives. Lancet (2002) 0.75

Discontinuation of Vioxx. Lancet (2005) 0.75

Antibiotics versus surgery for appendicitis. Lancet (2011) 0.75

Cetuximab for metastatic colorectal cancer. N Engl J Med (2009) 0.75

Dutasteride and prostate cancer. N Engl J Med (2010) 0.75

Regulatory evaluation of efficacy and safety of new drugs in China. Chin Med J (Engl) (2004) 0.75

Clinical trial registries: from an omen to a common and disclosed practice. Eur J Clin Pharmacol (2013) 0.75

Efficacy and safety of immunosuppressive drugs approved in EU through the centralised procedure. Eur J Clin Pharmacol (2007) 0.75

[A model for integrating biomedical research information]. Recenti Prog Med (2004) 0.75

[Considerations on clinical drug research in Italy]. G Ital Cardiol (Rome) (2006) 0.75

The European Commission should require better medicines, not just faster reimbursements. Eur J Intern Med (2012) 0.75

EMEA: for patients or for industry? Pharmacoeconomics (2005) 0.75

Ethics committees in Italy: a survey on a sample of oncologists. Tumori (2003) 0.75

Clinical databases of patients receiving antidepressants. The missing link between research and practice? J Affect Disord (2002) 0.75

[Effects of new smoking regulations in Italy]. Salud Publica Mex (2006) 0.75

Targeted kinase inhibitors in lung cancer: from EGFR to patients. Eur J Cancer (2005) 0.75

The scientific community should lobby to be able to apply for drug licences. BMJ (2012) 0.75

Sparing animals and progressing in science. Trends Pharmacol Sci (2003) 0.75

Italy: court's expert panel condemns tobacco. Tob Control (2002) 0.75

[Great discoveries: from the painstaking efforts of researchers to the contribution of accidental findings and the dissemination of study results]. Recenti Prog Med (2015) 0.75

Limits of add-on trials: antirheumatic drugs. Eur J Clin Pharmacol (2008) 0.75

Use of cigarette vending machines by minors in Italy. Int J Epidemiol (2004) 0.75