Forum 1 on Wednesday afternoon posed the (optimistic) question “How Good Are Our Medicines?” in a lively debate involving two panels. Among many ‘hot topics’ being discussed at European Health Forum Gastein, this could be one of the hottest. It comes at a time of ongoing policy developments in Europe, where we are considering evidentiary requirements (are we, the public, asking the right questions of pharmaceutical companies?), collaboration in Health Technology Assessment at the European level, and the orphan drug regulatory framework.
The first panel debated how we can evaluate quality of innovation, and how current regulatory systems incentivise this? In a panel of two halves, initial discussion focussed on oncology therapies. Wolf-Dieter Ludwig lamented that of the many new therapeutic agents, not all are really necessary for treating our patients. Many are approved on limited evidence, such as a single pivotal trial or a surrogate endpoint of progression free survival (PFS). Ameet Sarpatwari cited a meta-analysis hot off the press in JAMA Internal Medicine, which provides further evidence that prolonging survival does not necessarily improve health-related quality of life. We are seeing more medicines coming to market, at a faster rate, with an increasing number approved via expedited pathways. This means less evidence at the point of approval and the increasing importance of post-approval evidence generation.
This can put governments and payers in the difficult position of having to disinvest in therapies, which is against the expectations of stakeholders such as patients and healthcare professionals. Branding a medicine as “good” (or better than the current standard of care) puts the imperative on payers to provide it. Natasha Azzopardi-Muscat questioned whether the quality, safety and efficacy paradigm is still fit for purpose and proposed that the public good perspective also needs to be considered. The panel wrapped up an energetic question and answer session with the message that we can’t just complain about pharmaceutical companies behaving badly, but rather we should look at the regulatory system and structure the rules to get the outcomes we want. For example, we may need to rebalance the system of incentives in orphan drug regulations so that they only apply to drugs that otherwise would not come to market.
The second panel elaborated on the first, focusing on key cases where regulatory incentives have been exploited, deviating from the letter and/or spirit of the legislation. The first illustrative example was the case of Truvada, a combination medicine used as first line treatment for HIV/AIDS and for HIV pre-exposure prophylaxis or PrEP. Some European countries granted a supplementary protection certificate on the basis of one ingredient (tenofovir) to extend protection for the combination product. The Court of Justice of the European Union ruled against the manufacturer of Truvada, however some individual countries may still recognise the SPC which will prevent generic version entering the market. This could give rise to a two-tier system of access to PrEP across Europe. The current SPC regulations prevents European generics and biosimilar companies from producing medicines for export to countries where there is no SPC, and also forces them to move manufacturing outside of Euro
pe to have generic and biosimilar products ready for day one of the SPC expiring.
We wrapped up with an optimistic message on people power, with the potential for consumer organisations to affect change and empowering citizens and governments with transparency on pharmaceutical prices and R&D costs. These, along with small tweaks to the regulatory system, could improve access to innovative, effective medicines that serve the public good.
This Blog was written by the Young Gasteiner Frank Moriarty