Limited data about safety risks tend to be available at the time of the marketing authorisation by the EMA. Therefore, possible concerns about adverse events after long treatment periods have to be addressed through pharmacovigilance studies after authorisation and, if appropriate, through post-authorisation safety studies. A detailed risk-management plan must be submitted to the EMA. For instance, to minimise the risk of infections following administration of natalizumab for relapsing multiple sclerosis (MS), the risk-management plan imposes many restrictions. These include a clear-cut definition of the target population, the requirement for established MS, an escape rule for non-responders, the administration in specialised centres and only by experienced physicians, clear contraindications, a patient alert card, and an educational programme for physicians [4]. In short, only a tightly selected group of patients may be given this treatment.

Innovative mechanisms have been proposed for the reimbursement of biopharmaceuticals. Risk-sharing arrangements are schemes in which the pharmaceutical company shares the risk with the third-party payer that the product may not be effective for a particular patient. If the product does not have the expected effect, the company may lose some or all product revenue, or may have to provide a replacement product [5].

Such arrangements are instituted at the level of a defined patient population, rather than a group of patients cared for by an individual institution or healthcare provider. They may require physicians to be trained in the appropriate use of the biopharmaceutical and may also necessitate the implementation of a tracking system to follow-up its use. With respect to biopharmaceuticals, for instance, the National Institute for Clinical Excellence denied reimbursement for a number of drug classes treating MS (including beta interferons) in the UK in 2002 as they were expensive and not cost effective. Subsequently a risk sharing scheme was set up so that these drugs can be funded for use in selected people with MS. In this scheme, pharmaceutical companies reduce prices if their medicines do not attain a target level of effectiveness in the patient population [6, 7].

Related articles

Market access for biopharmaceuticals and biosimilars: a case study

The case for health economic studies on biopharmaceuticals

Health economic challenges for biosimilars

References

1. Simoens S. Health economics of market access for biopharmaceuticals and biosimilars. J Med Econ 2009;12(3):211–8.

2. European Medicines Agency. European Public Assessment Report (EPAR) for Herceptin (Trastuzumab). London: European Medicines Agency, 2010.

3. Schneider CK, Schaffner-Dallmann G. Typical pitfalls in applications for marketing authorization of biotechnological products in Europe. Nat Rev Drug Discov 2008;7(11):893–9.

4. European Medicines Agency. European Public Assessment Report (EPAR) for Tysabri (Natalizumab). London: European Medicines Agency, 2010.

5. Cook JP,Vernon JA, Manning R. Pharmaceutical risk-sharing agreements. Pharmacoeconomics 2008;26(7):551–6.

6. Barham L. Multiple sclerosis risk-sharing: four years on. Pharma Pricing Reimbursement 2006;11(4):100–1.

7. National Institute for Clinical Excellence (NICE). Clinical Guideline 8: Multiple sclerosis. Management of multiple sclerosis in primary and secondary care. November 2003.