ABSTRACT
Background:
Many established products (EPs - marketed for eight years or more) are widely used off-label despite little evidence on benefit-
risk ratio. This exposes
patients to
risks related to
safety and lack of
efficacy, and
healthcare providers to liability. Introducing new indications for EPs may represent a high societal value; however, manufacturers rarely invest in R&D for EPs. The objective of this
research was to describe
incentives and
disincentives for developing new indications for EPs in
Europe and to investigate consequences of current
policies.
Methods:
Targeted
literature search and expert panel meetings.
Results:
Within the current European-level and national-level regulatory framework there are limited
incentives for development of new indications with EPs. Extension of indication normally does not allow the
price to be increased or maintained, the market
protection period to be extended, or exclusion from a reference
price system. New indication frequently triggers re-evaluation, resulting in
price erosion, regardless of the level of added value with the new indication. In consequence, manufacturers are more prone to undertake R&D efforts at early to mid-stage of product
life cycle rather than with EPs, or to invest in new chemical entities, even in
therapeutic areas with broad
off-label use. This represents a potentially missed opportunity as developing new indications for EPs offers an alternative to
off-label use or lengthy and expensive R&D for new
therapies, opens new opportunities for potentially
cost-effective
treatment alternatives, as well as greater
equity in
patients'
access to treatment options.
Conclusion:
There are potential benefits from the development of new indications for EPs that are currently not being realized due to a lack of regulatory and
pricing incentives in
Europe.
Incentives for
orphan or paediatric
drugs have proven to be effective in promoting R&D. Similarly,
incentives to promote R&D in EPs should be developed, for the benefit of
patients and healthcare systems.