Researchers at Swedens Karolinska Institute ana-lyzed the relation between the availability of new anti-cancer drugs and the associated effect on survival in 3different ways.13 First, according to 1 analysis, 44% of theimprovement in cancer survival rates from 1992 to 2000at 50 US cancer centers could be attributed to newer anti-cancer drugs. Second, 14% to 19% of the intercountrydifferences in 5-year cancer survival rates across 5 majorEuropean Union countries were because of the uptake ofnewer drugs (after 1985). Finally, these researchers mod-eled the effect of newer drug vintages on cancer mortal-ity (adjusted for age and gross domestic product) in 20countries from 1995 to 2003; adjusted mortalitydecreased by approximately 16% during this period, andthe use of newer drugs accounted for 30% of this trend(ie, an absolute reduction of approximately 5%).13
The Karolinska study was criticized for flawed dataand methodology,38,39 with more rapid access to innova-tive drug therapy suggested as a surrogate marker of otherfactors that prolong survival, including improved diagno-sis.38,39 However, although the Karolinska data have limi-tations, they are worth considering.
Substantial disparities exist between European countries inper capita spending on novel anticancer agents. Accordingto 2005 data, France, Switzerland, and Austria were theleading European nations.13 By 2005, the use of trastuzu-mab in France was approximately 50% higher than theEuropean average. Data from 2007 to 2008 suggest thatuptake of new agents remains high in France, moderate inGermany and Italy, and low in the United Kingdom.1
Access to new anticancer drugs in France has beenimproved by several measures. More than 75% of healthcare funding in France is provided by the National SocialSecurity system.40 Cancer care is covered under the Affec-tions de Longue Duree and French patients receive fullreimbursement of their health care costs. In 2004, I per-suaded the French government to set up a new locus bywhich hospitals receive full, unrestricted state reimburse-ment for costly innovative anticancer drugs. This freedclinicians to prescribe novel agents according to clinicalneed, although it was contingent on meeting specific crite-ria with full reimbursement only realized when drugs areprescribed according to the good use contract. Duringthe 5 years after the introduction of this legislation, ex-penditure on cancer drugs increased from 230 million to1 billion, suggesting substantial under use of innovativetherapies before 2004.
Access to an effective drug can be delayed by regula-tory approval even when data support its use. In 2005, Ilobbied the French government to establish the Tempo-rary Treatment Protocol (PTT) to help prevent delays.The PTT convenes a panel of experts to assess data con-cerning new indications for drugs that are alreadyapproved (eg, in 2005, it was demonstrated that trastuzu-mab provides significant benefits as adjuvant treatmentfor breast cancer,41,42 but it approved only for metastaticdisease; therefore, patients could not benefit from thesefindings pending regulatory review). The PTT panelreviews new data, and, if it expects that a drug will begranted a new indication by the European MedicinesAgency, patients are given immediate access in that indi-cation. If the drug gains this indication, then patients con-tinue to be treated. If not, then access is stopped.
In conclusion, the adoption of novel anticancertherapies into clinical practice is vital to reduce cancermortality rates. The potential reduction in indirect cancercosts could exceed the direct costs of therapy as well asproviding patient benefits. In France, where innovativedrugs are fully reimbursed subject to a good use con-tract, such benefits were achieved in 2006 using a rela-tively small proportion of the health care budget. TheFrench health care system differs from that of many othercountries, but the key issue is the cost in proportion to thetotal health care budget, rather than who pays. Crucially,well designed, noninterventional, postapproval studies arerequired to properly assess the costs and benefits of inno-vative anticancer drugs.
FUNDING SOURCESMedical writing assistance from Prism Ideas Ltd. wasfunded by Association pour la Vie, Espoir Contre leCancer.
CONFLICT OF INTEREST DISCLOSURESDavid Khayat has received consultancy fees from Roche, AstraZenecaand Sanofi-Aventis.
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