When it comes to less profitable markets such as rare diseases, paediatric diseases or antimicrobials, medicines in the EU could be hard to come by, both in terms of price and availability. On Thursday 19 October, Senior Assistant Professor Simona Gamba (University of Milan, Italy) presented the study “Improving public access to medicines while promoting pharmaceutical innovation” at the live webstreamed meeting of the European Parliament’s Panel for the Future of Science and Technology (STOA).
The study will be published on the STOA website once finalised.
What was the goal of the study? And why?
Simona Gamba: We started from the premise that health is a fundamental right and pharmaceuticals play a vital role within the broader strategy to improve patient outcomes. Although over time many patients have had the opportunity to benefit from some outstanding innovations, some issues remain unresolved.
For example, with healthcare budgets under pressure in several countries, access to costly innovative medicines cannot always be granted to all patients. Moreover, the current innovation system in the pharmaceutical sector, mainly based on patents and regulatory exclusivities, makes less profitable markets, such as those of medicines treating rare diseases, paediatric diseases or the market of antimicrobials, scarcely attractive.
The big challenge is combining wide and equitable access to medicines with a framework that sustains sufficient investment in research and development (R&D) that will lead to more new products of high quality in the future.
In this context, the study explores which frameworks may be adopted to achieve a proper balance between:
- the need to grant accessibility, both in terms of prices (affordability) and availability, and
- the need to reward R&D efforts to stimulate research for the development of new therapies.
To examine the impact of regulatory mechanisms on public health, this study compares various incentive models adopted to stimulate private R&D in the pharmaceutical sector. The study also presents alternative frameworks based on a wider involvement of the public sector in drug innovation.
According to the study, it is currently very hard to achieve the balance between incentives for innovation and more access to medicines. Why is that?
Simona Gamba: The current framework is mainly based on patents and regulatory exclusivities, which limit competition. On the one hand, the monopoly rights granted by these tools provide an incentive to innovate (at least in markets that are sufficiently large to be attractive for the industry). On the other hand, the reduction in competition leads to higher prices.
What does the study suggest to tackle these challenges?
Simona Gamba: Given our starting point, and considering the results from the literature review and from interviews with stakeholders, we propose five policy options. The most ambitious one relies on a comprehensive approach. This involves strengthening EU coordination of intellectual property rights management and procurement of pharmaceuticals.
Furthermore, it includes the revision of existing incentives, with a reduction in exclusivities’ lengths granted to products. This should be accompanied by the introduction of specific incentives (subscription models), which are delinked from market size, for specific unmet medical needs (antimicrobials and ultra-rare diseases).
In addition, this policy option encompasses the creation of a public R&D infrastructure which is active throughout the whole R&D and production process, to address areas that remain insufficiently attractive for private investors.
What more can we in Europe do to improve access to medicines?
Simona Gamba: Many of the interviewed stakeholders mentioned that the management of procedures related to reimbursement and pricing at the national level is a major hurdle to ensure timely and equitable access to innovation for patients. Interestingly, this view was shared by patients, the industry and experts with diverse backgrounds.
One way to solve this issue may be to increase the coordination among Member States both in the protection of intellectual property rights and in joint procurement – the increase should be in coordination, not in the protection of intellectual property rights itself. For example, in 2023 the European Patent Office (EPO) created a unitary patent. This unitary patent grants protection in the 17 Member States that ratified the agreement with a single application at the EPO.
Strictly related to patents, the Supplementary Protection Certificates extend the patent length to compensate for the time spent during the clinical trials and the authorisation procedures. These are currently managed at the national level, but the creation of a Supplementary Protection Certificate managed at EU level is ongoing. The combination between unitary patent and unitary SPC would reduce heterogeneity in the timing of exclusivity expiry among countries.
The recent pandemic showed that joint procurement of pharmaceuticals is certainly possible; the study proposes to establish an EU procurement authority alongside an EU pharmaceutical fund. This should lead to a centralised price negotiation and the definition of a transparent “EU price”, while prices paid by the Member States to the EU fund can take into account the ability to pay (proxied by appropriate measures to be agreed upon).
The possibility should be given to countries to opt out of joint procurement. An experimental phase could be planned, during which joint procurement is restricted to selected products/areas.
This increase in coordination will be beneficial for:
- patients, who would benefit from earlier access to new products and reduced cross-country disparities in availability;
- the industry, which could improve efficiency by reducing the costs associated with national market access procedures, and which could enter the markets earlier;
- national regulators/payers, via a reduction in the transaction costs related to pricing and reimbursement decisions, and greater transparency on prices.
Would these require expanding EU’s health competence?
Simona Gamba: The study does not analyse the possibility of expanding the EU’s health competence in general . The proposal is restricted to coordination of intellectual property rights management and procurement of pharmaceuticals, as part of a package of interventions.
Countries that opt for EU procurement would still be free to define their national health policies, including decisions related to the use of that specific medicine within the country.
Do you think the proposed EU’s revision of its pharmaceutical rules that is currently being discussed, is a step in the right direction?
Simona Gamba: The proposal shows attention toward the issue of affordability and access, recognises that high unmet medical needs require special attention, and introduces specific provisions to address antimicrobial resistance, which is a great challenge that urgently needs action.
However, we suggest that alternative tools could be considered to stimulate innovation to address unmet medical needs. We also believe that the impact of the reform could be further improved by making it part of a broader strategy that could include greater EU coordination and a more active role of the public sector throughout the whole R&D and production process in specific areas.