Orphan Drug Price: Is Eurordis Call To Reduce Prices By 3-5 Times The Answer to Unequitable Access?

MARYLEBONE, U.K. - Jan. 24, 2018 - PRLog -- Earlier this month Eurordis published its position paper on orphan drug access. Whilst the report is commendable in pushing to drive equitable access for patients; their call to reduce orphan drug prices by 3 to 5 times by 2025 is no guarantee of full patient access. Affordability is the issue at hand and reducing drug prices will not, by itself, alleviate that.

The OECD report from 2016 confirms "between 2009 and 2014, expenditure on pharmaceuticals dropped by 1.1% in real terms on average in the European Union" while overall health expenditure increased. Eurordis itself highlights that "The extent of the actual contribution of pharmaceutical products to the worsening of the sustainability of healthcare systems is, however, debatable." Why then call for a specific drug price reduction to manage the issue?

In line with the EU Regulation for orphan medicines, rare disease patients should receive the same quality of care as all patients. However, the chances of a sufficient return on investments for treatment of rare and ultra-rare diseases is different due to smaller sales numbers. This is why the European Regulation and the US Orphan Drug Act exists; this is why there are benefits available for orphan drug manufacturers. Certainly, there are extreme cases with a number of excessive prices, but it has to be recognised that recent gene therapy approvals in Europe and the US, where prices were anticipated around USD 1 million stayed well below expectations (e.g. Kymriah and CAR-T therapies in the US, Strimvelis in the UK).

The role of budget impact

Current HTA and regulatory systems focus strongly on budget impact, which as Eurordis rightly points out prioritises the financial aspect over the patient and access dimension. They suggest finding "collectively new ways and agree new solutions to ensure that the prices of future orphan medicines are determined in a decisively more fair manner" making patient outcomes the "single most important factor in the decision" and finding a "acceptable balance between the conflicting demands of a scientific uncertainty that is here to stay, and of a financial sustainability that more than ever needs to be restored."

At Partners4Access, we believe in striking an equilibrium in the rare disease world. That means helping drive access to the most appropriate treatments for patients and physicians, a fair return on investment for manufacturers at an affordable proposition for payers. We do not believe that such an equilibrium will be reached by making products 3 to 5 times cheaper. Consider that expenditure on pharmaceuticals did not change in the last years while at the same time more and better products became available for all, including rare disease patients.

We question a few aspects of Eurordis call to action. Firstly, the number itself: is 3 to 5 times cheaper an arbitrary number? Are Eurordis suggesting all orphan drugs to be targeted, or just the more expensive ones? Importantly, will such an approach lead to a price no longer attractive to invest for industry? Rather ironically, closing down the incentive that the Orphan Drug Act was intended to serve.

A need for collaboration to achieve a system change

We fundamentally believe that a system change can only take place if all involved stakeholders are involved in such a process and this clearly includes the manufacturer. It was disappointing to see that Eurordis did not involve industry as part of the stakeholders actively collaborating in its recent position paper.

There are more and highly innovative therapies in late stage clinical trials approaching the market in the future, according to the Alliance for Regenerative Medicines there were 946 clinical trials ongoing in regenerative medicines including cell and gene therapy in 2017, several in a late stage setting with expected key events in 2018. For a successful future with an equilibrium between the key stakeholders, all parties need to work and collaborate together. As Eurordis suggests, we must explore alternatives: how can we work together to ensure that the healthcare system as a whole can become more efficient?

Pharmaceuticals and orphan drugs are just a minor part of the current spending. Partners4Access suggests exploring alternative collaboration models in search of increasing affordability. For example, mandatory efficiency targets in healthcare provision across primary and secondary care settings, greater investment into disease prevention and public education programs, consolidation of private and public healthcare systems, alternative and innovative payment models, incentivising generics and perhaps more provocatively, only partially reimbursing treatment for lifestyle diseases, which could be prevented.

Working collaboratively is the future. Together we can reshape the healthcare system for the better. Contact us at http://partners4access.com/  to find out more and get involved.