The new reality of access to orphan medicines in Europe
As many market access professionals experience today, a negative correlation exists between the vastness of (often revolutionary) innovations becoming available to the healthcare systems and payer’s willingness to pay for them. In Europe alone, according to the most recent EFPIA W.A.I.T. Indicator survey (IQVIA, 2021), the average delay between market authorization and patients access to orphan medicines, varies between countries from 55 to 1470 days. Despite a small improvement in rate of availability in some eastern European countries as compared to the 2019 survey, the overall picture is saddening as in most countries the time to availability has increased, even in the countries that previously were performing very well with fast patient access (such as Germany for example).
The Orphanizing of Healthcare brings HTA challenges
Health Technology Assessment (HTA) procedures are based on the willingness to pay that translates as the ratio between benefits and price, relative to the burden of the disease and existing treatment options (relative effectiveness). With the increasing introductions of rare disease and ultra-rare diseases treatment options, precision medicine and personalized healthcare approaches, often referred to as the Orphanizing of Healthcare, it is no longer the costs per QALY gained, but plainly the budget impact that has become a predominating factor in decision making. Appropriate use of these treatments should contribute to the (cost-) effectiveness of these drugs, and help to lower budget impact, but is difficult to manage and to monitor. Orphan medicines are often registered with surrogate endpoints and often, treatment decisions about these drugs depend on new diagnostic techniques which results in complex health technologies that cannot be assessed as single pharmaceutical products. A recent survey by Hogervorst et al., (2021) among European HTA organizations indicated that challenges in HTAs of complex health technologies mainly root in data insufficiencies rather than in the complexity of the technology itself. There is a need for additional evidence generation and synthesis to be able to solve the data insufficiencies and enable the development of pricing and reimbursement schemes that mitigate the risks of the uncertainty on the relative effectiveness. Thus, HTA agencies cannot fully address the payer concerns and due to budget constraints, national health authorities not only have to decide whether these new therapies are worth the price, but they may also have to face hard questions about who will get access and who will not. This will cause decisions to take longer, which in the end contribute to a longer lasting unmet need despite available treatment options. And as some drugs may pass the scrutiny of budgetary impact measures in one country, while in others they are rejected, inequity of access to drugs between countries (but even among citizens within one country) occur. But the data insufficiencies are not easy to solve. Not only is the clinical evidence base limited at registration, but the supporting diagnostic techniques are often not developed by the drug producing companies themselves and may not even have been included in the pivotal studies. The big question is who will need to generate the needed evidence as the data insufficiencies follow from a multi-factorial problem that cannot not be solved by one single actor.
The daunting reality is that ultimately patients are suffering from the HTA complexities that have been introduced with these promising innovative treatment options. Solutions for data generation may contribute to mitigate the risks of uncertainty about the relative effectiveness, but as pricing and reimbursement is a national decision-making process which differs from country to country, data generation by itself will not solve the existing solidarity conflict.
Background sources (hyperlinked) used for this article:
EFPIA Patients W.A.I.T. Indicator 2020 Survey. IQVIA © April 2021.
Hogervorst et al., Reported Challenges in Health Technology Assessment of Complex Health Technologies. Value in Health, 22 December 2021, in press.