Rare disease patients in the United Kingdom face unusual barriers to accessing medicines because of the multiple review and reimbursement paths for therapies across jurisdictions, according to a new white paper commission by Roche Products.

The white paper from the life sciences data and analytics company IQVIA and the policy advisory Public Policy Projects characterizes the existing system of as a “postcode lottery,” suggesting that rare disease patients access to medicines can depend on where they live because of inconsistencies in evaluation by different agencies to determine approval and reimbursement decisions.

“Sometimes there are four separate decisions being made on the same treatment—not all of them reaching the same conclusion,” the paper said as it charged the system is failing to keep pace with the scale of medicines being developed.

Already patients in the United Kingdom face a harder time of getting access to orphan drugs than their counterparts in nearby European countries. The paper notes patients in Germany and France are seven times more likely to get a newly launched medicine. In comparison to the key European Union markets, the United Kingdom takes an additional six months to approve an orphan drug, with average time to reimbursement of around two years for orphan medicines from the point of an approval.

The authors also suggest that pharmaceutical companies and clinicians have difficulty navigating the evaluation process and said that a lack of transparency and guidance prevents many rare disease patients from gaining access to new medicines.

The situation is expected to grow worse as the number of orphan drugs approved by the European Medicines Agency is expects to increase four-fold between 2017 and 2020. The United Kingdom’s withdrawal from the European Union could further complicate matters for rare disease patients, the paper warns.

The paper says the current system needs to be redesigned and recommends a series of steps the United Kingdom should take to improve rare disease patient access to orphan drugs and make the evaluation process of these medicines more efficient and access more equitable.

Among the recommendations the authors make is a call for is a strengthening of the patient voice in assessing the impact a treatment has and its cost effectiveness to ensure that what is important to the patient is part of the evaluation.

It calls for better coordination between agencies to address confusion about reimbursement routes and to reduce duplication and conflicting evaluations and harmonizing the process across the United Kingdom.

And it calls for a broader concept of value in the analysis of therapies by moving beyond quality adjusted life years as the major criterion in the appraisal of rare disease therapies to include such considerations as the improvement on patients’ lives and those of their families.

That last point is an issue that payers around the world will increasingly grapple with, particularly as gene therapies and other potentially curative therapies make their way to market with the promise of delivering dramatic results at a steep price.

Though it is not surprising to see industry make an argument from a broad concept of value, this will be an important policy debate in the years to come for how to measure the worth of a therapy and whether its affects beyond the patient’s physical health, to include a patient’s psychological wellbeing, impact on a patient’s family, and greater societal benefits should matter as well.

May 3, 2018