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This paper investigates the impact of the introduction of new orphan drugs on premature mortality from rare diseases using longitudinal, disease-level data obtained from a number of major databases. The analysis is performed using data from two countries: the United States (during the period 1999-2006) and France (during the period 2000-2007). For both countries, we estimate models using two alternative definitions of premature mortality, several alternative criteria for inclusion in the set of rare diseases, and several values of the potential lag between new drug approvals and premature mortality reduction. Both the United States and French estimates indicate that, overall, premature mortality from rare diseases is unrelated to the cumulative number of drugs approved 0-2 years earlier but is significantly inversely related to the cumulative number of drugs approved 3-4 years earlier. This delay is not surprising, since most patients probably do not have access to a drug until several years after it has been launched. Although the estimates for the two countries are qualitatively similar, the estimated magnitudes of the US coefficients are about four times as large as the magnitudes of the French coefficients. This may be partly due to greater errors in measuring dates of drug introduction in France. Also, access to new drugs may be more restricted in France than it is in the United States. Our estimates indicate that, in the United States, potential years of life lost to rare diseases before age 65 (PYLL65) declined at an average annual rate of 3.3% and that, in the absence of lagged new drug approvals, PYLL65 would have increased at a rate of 0.9%. Since the US population aged 0-64 was increasing at the rate of 1.0% per year, this means that PYLL65 per person under 65 would have remained approximately constant. The reduction in the US growth rate of PYLL65 attributable to lagged new drug approvals was 4.2%. In France, PYLL65 declined at an average annual rate of 1.8%. The estimates imply that, in the absence of lagged new drug approvals, it would have declined at a rate of 0.6%. The reduction in the French growth rate of PYLL65 attributable to lagged new drug approvals was 1.1%. Earlier access to orphan drugs could result in earlier reductions in premature mortality from rare diseases.

Objectives: While a French language version of the EQ-5D exists, to date, there has been no French value set to accompany it. The objective of our study was then to derive the French TTO value set of the EQ-5D., Methods: A total of 452 respondents aged over 18 were recruited who were representative of the French population with regard to age, gender, and socio-professional group. The direct valuation of 24 health states was first obtained by Time Trade-Off (TTO), and the negative TTO values were bounded using the monotonic transformation. Several alternative model specifications were investigated to estimate the values for all 243 states in the EQ-5D descriptive system. Only the best fitting model is presented in this paper. The analysis was conducted at an individual level to make the maximum use of the available data, and we estimated mixed models with random intercept. Models were compared through the Akaike information criterion (AIC), the mean absolute error (MAE), and the Pearson correlation coefficient between the observed and the predicted values of each model., Results: After exclusion, 443 respondents took part in the study. The best fitting model included the same variables as the N3-model used in UK., Conclusion: This study provides the French value set of the EQ-5D based on the stated preferences of the French general public facilitating cost-effectiveness analysis.

Objectives: Choices between formal and informal care for disabled elderly people living at home are a key component of the long-term care provision issues faced by an ageing population. This paper aims to identify factors associated with the type of care (informal, formal, mixed or no care at all) received by the French disabled elderly and to assess the care's relative costs., Methods: This paper uses data from a French survey on disability; the 3,500 respondents of interest lived at home, were aged 60 and over, had severe disability and needed help with activities of daily living. We use a multinomial probit model to determine factors associated with type of care. We also assess the cost of care with the help of the proxy good method., Results: One-third of disabled elderly people receive no care. Among those who are helped, 55% receive informal, 25% formal, and 20% mixed care. Low socioeconomic status increases difficulties in accessing formal care. The estimated economic value of informal care is 6.6 billion euro [95% CI = 5.9-7.2] and represents about two-thirds of the total cost of care., Conclusion: Public policies should pay more attention to inequalities in access to community care. They also should better support informal care, through respite care or workplace accommodations (working hours rescheduling or reduction for instance) not detrimental for the career of working caregivers.

This paper investigates whether a satisfactory work environment can promote employee health even after controlling for socioeconomic status and life style factors. A dynamic panel model of health is estimated from worker samples from Denmark, France and Spain, employing both self-assessed general health and the presence of a functional limitation. In all three countries and for both types of health measures, a good perceived work environment is found to be a highly significant determinant of worker health even after controlling for unobserved heterogeneity and minimizing reverse causality. The marginal effect is, however, larger in France and Denmark than in Spain. Several potential explanations for this finding are discussed. Further, a satisfactory working environment is found to be at least as important for employee health as socioeconomic status. Thus, investing in giving workers a satisfying work environment could be a low-cost way of improving employee health.