Access challenges for orphan therapies
It is well known that the clinical data that is necessary to achieve regulatory approval for medicines is often not sufficient to achieve market access for the same medicines. For over 10 years, MME, an Indegene PRMA business, has tracked the time it takes for biopharmaceutical products to gain market access following regulatory approval in the US and EU4/UK6. Based on our periodic data cuts, we have observed differences in both if, and when, therapies reach the market. While some of these differences are a result of how the procedures work in the individual countries, others are more nuanced, and change based on the country and circumstances.
Recently we analyzed similar 4-year cohorts (EC approvals between Jan 2013-Dec 2016 and Jan 2017- Dec 2020) for all therapies, therapies with orphan indications, and those with oncology indications, in order to understand not just the overall statistics, but to look for changes in trends. What we uncovered is that there are distinct differences. Most notable are the following key takeaways (Table 1):
The US remains the quickest in terms of time to market for all products as well as orphan medicines
In Europe, there are some differences based on trends analysis over time. In France, Italy, and Spain, where the procedures require a local filing and negotiation before entering the market, the gap between regulatory and pricing approval is longer, albeit declining in Italy and Spain.
Between the time cohorts, we see declines in time to market for France Italy, and Spain, but this is offset by a fewer percentage of approved drugs being launched in the more recent years. This seems like the decline might be a "false positive" until a number of the remaining products complete the process. For instance, although the average time to market in Spain appears to be shorter at 58 weeks, patients only have access to 41% of all therapies and 29% of orphan therapies.
Table 1: Average number of Weeks to Launch Post Regulatory Approval across EU4/UK and US and percentage of launched therapies. Subsets selected: 2013 to 2016 and 2017 to 2020
*Data collection cut off –July 2021
Further analysis of recent approvals (EC approval: 2017 to 2020) indicates that market access barriers increase for therapies with conditional approval and those approved under exceptional circumstances (Table 2). Only 25% of drugs with conditional approval have completed P&R negotiations in both France and Spain and only 40% in Italy. Orphan drugs approved under exceptional circumstances seem to have a particularly difficult time in Spain as evidenced by none of the recently approved drugs available gaining access.
Table 2: Average number of Weeks to Launch Post Regulatory Approval across EU4/UK and US and percentage of launched therapies across 189 medicinal products from Jan 2017 to Dec 2020
^All drugs approved under exceptional circumstances were designated as orphan therapies. 75% of therapies with conditional approval had orphan designation
Our analysis suggests that despite regulatory incentives and a higher willingness to approve medicines under special conditions (conditional approval and exceptional circumstances), patient access to orphan therapies is suboptimal. This issue has likely been exacerbated as more innovative expensive orphan therapies have been launched and are expected to continue in the future. This further illustrates the point that the clinical data that is necessary to achieve regulatory approval for medicines is often not sufficient to achieve market access for the same medicines.