Over the past several years the biopharmaceutical industry has made incredible leaps in many disease areas bringing new drugs to market that deliver greater patient benefits, in part by integrating the patient perspective in clinical research decision-making. At the same time, the European Medicines Agency (EMA) has adopted new strategies to speed the approval process for some of these drugs. Conditional approvals and adaptive pathways are specifically designed to accelerate access to potentially life-changing products for patients in dire need of new treatment options. But this speed and innovation on the drug development and approval side, has led to some gaps between the regulatory and payer environments. In many cases Health Technology Assessment (HTA) bodies and regulators are struggling to evaluate fast-tracked products that come with limited or immature data sets, and how to incorporate patient important end-points in their valuation criteria.

These gaps -- and how we as an industry can work together to close them -- will be key issues covered at ISPOR’s 19th Annual European Congress which takes place October 29-November 2 in Vienna. The theme of this year’s event, “Managing Access to Medical Innovation: Strengthening the Methodology-Policy Nexus,” touches on the challenges the industry faces to harmonize payer and regulatory processes with efforts to shorten time-to-market and incorporate the patient voice in the development conversation.

Collaboration on the adaptive pathway

Several of this year’s panels and plenary sessions will explore the need for greater collaboration among stakeholders to address these issues, including the first plenary session on how synergies could be created between regulatory and health technology assessments. The speakers will discuss the need for more formal interactions between regulators and HTA bodies as they determine which patient groups should have access to new therapies and at what point in time; and the roadblocks in the EU health sector ecosystem that need to be removed in order to realize synergies between these groups, while respecting their different roles.

And on Tuesday, there will be a break-out sessions entitled: adaptive pathways and patient access – pushing payer boundaries or facilitating new payment models where panelists will discuss the challenges HTA bodies face in assessing adaptive pathway products, and potential solutions for making decisions on pricing and coverage while dealing with uncertainties regarding the product therapeutic and economic value.

The patient perspective

The influence of the patient’s voice in HTA decision-making is another topic that will be covered extensively at this event. Precision medicine allows us to target specific conditions, to treat diseases without many of the side effects of previous generations of drug, and also to substantially improve a patient’s quality of life. Key elements of the value proposition for many of these treatments are patient-important endpoints and patient reported outcomes. Whilst HTA bodies increasingly welcome and request such information, there are on-going challenges interpreting these data in a consistent manner and providing clarity as to how to factor them into assessments. Several sessions at ISPOR will touch on this trend, including two issue panels on Monday. The first will explore patient-centric decision-making in HTAs, where the panel will debate the role of the patient voice in these assessments; and the second will examine how HTA bodies can consider the voice of rare disease patients when granting access to orphan drugs.

There will also be several sessions throughout the event on the use of real world data in HTAs, and how these data can support efforts to safely provide patients with earlier access to innovative treatments.

Economic impact of innovation

Another interesting topic tied to recent innovations in precision medicine are the economic implications of paying for drugs that prolong life. Many HTA guidelines recommend that “unrelated future costs” are excluded from an appraisal, but some are reconsidering this decisions. This topic will be explored at length in a session on Wednesday morning on the pros and cons of factoring future medical costs into HTA assessments, and how those costs can be estimated. Tuesday’s plenary session, Differential pricing of medicines in Europe: implications for access, innovation, and affordability, also explores this topic, with speakers looking at options for a systematic approach to differential pricing across Europe and how it could improve dynamic efficiency for all of the countries in the region. These are just a few of the many presentations I’m looking forward to at ISPOR.

ISPOR Europe is an opportunity for stakeholders across the industry to work together to address the challenges we face in providing safe and efficient access to medical innovation for patients around the world. It’s an opportunity to have conversations and foster collaborations that help us align the needs of payers, regulators and developers so that we eliminate friction in bringing innovative drugs to market, while ensuring everyone’s needs are met.

Topics in this blog post: Biopharma, EMA, Evidence, EU, HTA, Value