An Improved Approach To Measuring Drug Innovation Finds Steady Rates Of First-In-Class Pharmaceuticals, 1987–2011

Lanthier, Michael; Miller, Kathleen L.; Nardinelli, Clark; Woodcock, Janet

doi:10.1377/hlthaff.2012.0541

Cited by 118 publications

(120 citation statements)

References 15 publications

(17 reference statements)

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“…Compounds newly launched in the European Union are then classi…ed following what is done by Lanthier et al (2013), which covers new active ingredients approved by Food Drug Administration (FDA), for marketing in the United States 10 . The classi…cation includes three categories in descending 8 In the …nal sample we have 10 members of the Euro area (Austria, Belgium, Germany, Spain, Finland, France, Greece, Ireland, Italy, Portugal) and 5 countries that still adopt their own currencies (Czech Republic, United Kingdom, Hungary, Poland, Slovak Republic).…”

Section: Data Descriptionmentioning

confidence: 99%

“…As we want to check for the therapeutic relevance of newly marketed molecules, we borrow from Lanthier et al (2013) a classi…cation that includes three categories in descending order of innovative content: i) …rst-in-class; ii) advance-in-class; iii) addition-to-class.…”

Section: Launch Of Molecules and Country Diffusionmentioning

confidence: 99%

“…Finally, we have a subset of drugs that do not match with the list by Lanthier et al (2013). In this case, we checked manually from reports of the European Medicine Agency that these are actually marketed only in EU sample countries.…”

Section: Launch Of Molecules and Country Diffusionmentioning

confidence: 99%

“…In this case, we checked manually from reports of the European Medicine Agency that these are actually marketed only in EU sample countries. As they didn't submit to evaluation by FDA, we cannot assess their therapeutic value consistently with what done by Lanthier et al (2013) and we will treat them separately in our following analyses.…”

Section: Launch Of Molecules and Country Diffusionmentioning

confidence: 99%

“…They provide a …rst snapshot of the survival heterogeneity by country. indicating whether the molecule is considered …rst-in-class or advance-in-class, following Lanthier et al (2013); iii) the Purchasing Power Parity (PPP) of each country c towards the rest of the EU (EU27=100), sourced from Eurostat; iv) levels of country c real income (GDP) per capita; v) expectations on prices and on volumes, proxied (in logs) by average prices and volumes of already marketed molecules within the same k therapeutic submarket (see also with Danzon and Epstein, 2012); vi) company sales in the kth therapeutic reference market, as a proxy of the company's bargaining power against regulatory authorities in country c.…”

Section: Market Integration Income Innovative Content and Country mentioning

confidence: 99%

See 4 more Smart Citations

Access to Medicines and European Market Integration

Pammolli

Rungi

2016

SSRN Journal

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In this paper we document a process of price convergence in the European market for pharmaceuticals and relate it to access to innovative medicines in individual countries. The EU is a peculiar case study, where free circulation of goods exists, but pricing policies are designed and implemented by Member States. Thanks to a unique census database on product sales and launches for …fteen EU countries, we detect a process of price convergence, both in nominal and in real terms. Therefore, we …nd that a faster rate of price convergence and a lower income per capita are associated with stronger delays in launches of new medicines. Moreover, country delays tend to be higher for innovative and …rst in class chemical compounds. Our results suggest that ine¢cien-cies arise from drugs regulation, when countries widely di¤er in income per capita, public …nance sustainability conditions, and regulatory frameworks. Policies of external reference pricing tend to exacerbate welfare losses. A policy of di¤erential pricing is suggested, in order to take into account both therapeutic value and willingness to pay.JEL classi…cation: F15; I11; L65; L11; L51

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Section: Data Descriptionmentioning

confidence: 99%

Section: Launch Of Molecules and Country Diffusionmentioning

confidence: 99%

Section: Launch Of Molecules and Country Diffusionmentioning

confidence: 99%

Section: Launch Of Molecules and Country Diffusionmentioning

confidence: 99%

Section: Market Integration Income Innovative Content and Country mentioning

confidence: 99%

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Access to Medicines and European Market Integration

Pammolli

Rungi

2016

SSRN Journal

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Sharing Clinical Trial Data

2015

JAMA

145

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All patients and their relatives want the best information possible regarding the effectiveness and safety of therapies. Responsible sharing of clinical trial data serves this public interest by strengthening the science that is the foundation of safe and effective clinical care. Sharing also fosters sound regulatory decisions, generates new research hypotheses, and increases the scientific knowledge gained from the contributions of clinical trial participants, the efforts of clinical trial investigators, and the resources of clinical trial funders. However, results from about onethird of clinical trials remain unpublished 4 years after trial completion, 1 and much data from trials are never analyzed. Several large pharmaceutical companies 2 and some academic investigators are already sharing clinical trial data, and the European Medicines Agency will do so beginning in 2015. 3 There are compelling reasons to share clinical trial data. The issue now is no longer whether to share clinical trial data but instead what specific data to share, when the data should be shared, and with what controls and

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Postmarket Safety Events Among Novel Therapeutics Approved by the US Food and Drug Administration Between 2001 and 2010

Downing

Shah

Aminawung

et al. 2017

JAMA

242

233

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IMPORTANCE Postmarket safety events of novel pharmaceuticals and biologics occur when new safety risks are identified after initial regulatory approval of these therapeutics. These safety events can change how novel therapeutics are used in clinical practice and inform patient and clinician decision making.OBJECTIVES To characterize the frequency of postmarket safety events among novel therapeutics approved by the US Food and Drug Administration (FDA), and to examine whether any novel therapeutic characteristics known at the time of FDA approval were associated with increased risk. EXPOSURES Novel therapeutic characteristics known at the time of FDA approval, including drug class, therapeutic area, priority review, accelerated approval, orphan status, near-regulatory deadline approval, and regulatory review time. MAIN OUTCOMES AND MEASURESA composite of (1) withdrawals due to safety concerns, (2) FDA issuance of incremental boxed warnings added in the postmarket period, and (3) FDA issuance of safety communications.RESULTS From 2001 through 2010, the FDA approved 222 novel therapeutics (183 pharmaceuticals and 39 biologics). There were 123 new postmarket safety events (3 withdrawals, 61 boxed warnings, and 59 safety communications) during a median follow-up period of 11.7 years (interquartile range [IQR], 8.7-13.8 years), affecting 71 (32.0%) of the novel therapeutics. The median time from approval to first postmarket safety event was 4.2 years (IQR, 2.5-6.0 years), and the proportion of novel therapeutics affected by a postmarket safety event at 10 years was 30.8% (95% CI, 25.1%-37.5%). In multivariable analysis, postmarket safety events were statistically significantly more frequent among biologics (incidence rate ratio [IRR] = 1.93; 95% CI, 1.06-3.52; P = .03), therapeutics indicated for the treatment of psychiatric disease (IRR = 3.78; 95% CI, 1.77-8.06; P < .001), those receiving accelerated approval (IRR = 2.20; 95% CI, 1.15-4.21; P = .02), and those with near-regulatory deadline approval (IRR = 1.90; 95% CI, 1.19-3.05; P = .008); events were statistically significantly less frequent among those with regulatory review times less than 200 days (IRR = 0.46; 95% CI, 0.24-0.87; P = .02).CONCLUSIONS AND RELEVANCE Among 222 novel therapeutics approved by the FDA from 2001 through 2010, 32% were affected by a postmarket safety event. Biologics, psychiatric therapeutics, and accelerated and near-regulatory deadline approval were statistically significantly associated with higher rates of events, highlighting the need for continuous monitoring of the safety of novel therapeutics throughout their life cycle.

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An Improved Approach To Measuring Drug Innovation Finds Steady Rates Of First-In-Class Pharmaceuticals, 1987–2011

Cited by 118 publications

References 15 publications

Access to Medicines and European Market Integration

Access to Medicines and European Market Integration

Sharing Clinical Trial Data

Postmarket Safety Events Among Novel Therapeutics Approved by the US Food and Drug Administration Between 2001 and 2010

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