It is expected that companies will participate, as part of an adaptive approach, in a joint scientific consultation process involving hTA regulators and regulators, which has already been done for some of the impilote products. This process should lead to an integrated data generator plan, in which regulatory applications are supplemented by hTA applications. These applications would be implemented in relevant national instruments, such as cash entry agreements, in accordance with national procedures and guidelines, as pricing and reimbursement fall within national jurisdiction. The evidence in practice gathered after the first authorization would be used to supplement the initial marketing authorization requirements – without substituting – and to allow a better understanding of the optimal use of the product (for example. B understanding of subgroups of patients and responders) to maximize effects and minimize risk. Health authorities and pharmaceutical companies have sought alternative funding formulas to share the risks and uncertainties arising from public coverage of new drugs whose clinical effectiveness and budgetary impact are still unknown. In Finland, one-fifth of all patented drugs administered in hospitals are consulted through managed entry agreements. Several examples of difficulties related to results-based agreements are available in the literature. A Dutch evaluation of a stage III-managed oxaliplatin accession agreement for the treatment of colon cancer based on the results, which collected additional data on a patient registry, showed that patient heterogeneity made it difficult to estimate the incremental cost-effectiveness of treatment based on the data collected (Mohseninejad et al., 2015). A Dutch registry of hematological malignancy indicated that the three main problems were confused due to the indication, lack of data and insufficient number of comparable patients (Blommestein et al., 2015). In Italy, an analysis of 2006-2012 data showed that 67% of expected repayments were actually recovered through certain agreements (Navarria et al., 2015). Among the reasons identified were difficulties related to the required follow-up period, problems with reimbursement notification and insufficient (financial) incentive from the prescribing centre to participate in a program (Garattini et al., 2015); Navarria et al., 2015). These examples underline the crucial importance of robust methods and the clear agreement of upstream stakeholders on the expectations associated with the use of the evidence to be collected.
Note: This taxonomy is based solely on the structure of the agreements. All types of agreements mentioned above may exist not only between companies and health organizations, but also between companies and other types of institutions that constitute a health system, including government agencies or national authorities responsible for making coverage or pricing decisions and/or evaluating health technologies (HTA), regional health authorities, health care providers, etc. In particular, for products used in hospitals, MEAs may be available between companies and hospitals. Source: authors of the study based on Carlson (2010), Ferrario and Kanavos (2013) and Gerkens et al. (2017). Despite the lack of evidence, experience to date with results-based agreements indicates a number of good practices. They cover four main themes: the questionnaire responses were used to identify payers and HTA agencies that had experience with management input agreements for these products. In addition, the manufacturers who marketed these products were identified and invited to participate in semi-structured interviews that were used to answer the same questions as interviews with payers and HTA agencies. The results were presented during a one-day workshop in July 2016.