Proceedings: DIA Europe 2019

Evolution of Science and Policy – Where Are We Heading?

Adam Istas
Science Writer

DIA Europe 2019

ith numerous advances in science, data analytics and regulatory innovation, the future of biopharmaceutical medicine presents enormous opportunities to address unmet medical needs. From harnessing cell- and gene-based therapies to quantifying patient preferences, these new approaches and techniques will require collaboration amongst all stakeholders to align various initiatives and incentives for maximal benefit.

Key Takeaways

  • Cell- and gene-based therapeutic approaches are advancing rapidly and hold potential to personalized treatment, yet existing regulatory guidelines for quality control and efficacy evaluations must be further developed.
  • Patient preferences—and new ways of capturing and analyzing data—are being incorporated into clinical development to support regulatory and health technology assessment (HTA) decision making.
  • The democratization of digital innovation is here, requiring regulators to develop partners and ecosystems to take full advantage of these ubiquitous tools.
  • Regulatory science in the EU continues to become more collaborative, as demonstrated through numerous initiatives aimed at reducing regulatory and reimbursement uncertainty for innovative therapies.

Why this is important: With so many advances in so many areas of medicine, the EU legislative framework is in need of transformative legislative change.

“We have built our legislation bottom-up, in different bits, but maybe now we need to apply that same organizational IT architectural approach to look at it top-down,” said Esteban Herrero-Martinez, Director Regulatory Policy and Intelligence at AbbVie. “We need to look at the interfaces, where we have challenges and build a list of things we need to do to help build a long-term strategy for that legislative framework that we want down the line.”

Advancing Science, Advancing Analytics

Few innovations hold more potential to deliver precision medicine than genomic editing. As CRISPR/Cas9 technologies enable better precision in genome editing, modifying and using cells to treat cancer, blood disorders and infectious diseases will soon become common practice. Further, population scale genomics will be used to identify individual disease risks and suitability of treatment, even before the onset of disease. For benefit-risk assessment in precision medicine, individualized approaches such as pairwise comparison of patient outcomes will be critical.

Generalized pairwise comparisons (GPC) is a method to help analyze multiple populations in pairs to determine whether they are significantly different from one another. In precision medicine, this helps determine the proportion of patients who might respond to one therapy or another (or none at all). Additionally, GPC allows for several outcomes to be assessed in order of priority, allows for the introduction of thresholds of benefit, and computes the net benefit.

Quantifying Patient Preferences

The PREFER consortium—a public-private collaborative research project under the Innovative Medicines Initiative—is leading the charge to incorporate patient perspectives on benefit and risk. By developing expert and evidence-based recommendations, PREFER aims to guide industry, regulatory authorities, HTA bodies and reimbursement agencies on how patient preferences can be assessed and used to inform medical product decision making. The consortium is currently building a repository of case studies to answer methodological and operational questions, to demonstrate the comparative value of different methods, and to guide stakeholders on when and how to elicit patient preferences.

Improving Regulatory Systems

The European Commission has long cooperated on various HTA projects on both the scientific/technical level and the policy/strategic level. Its support for the European Network for Health Technology Assessment (EUnetHTA) includes piloting joint clinical assessments, facilitating early dialogues and scientific advice to technology developers, and providing input to methodologies and tools. Similarly, the Commission has contributed numerous strategy and reflection papers to the HTA Network.

The Commission recently introduced a proposal for a regulation on HTA that would include joint scientific consultations with HTA bodies (with input by patient/clinical experts) to technology developers on clinical study design. Occurring during the clinical study planning stage—and potentially offering parallel consultation with the European Medicines Agency (EMA)—the goal is to pool HTA resources and expertise to build a better evidence base for HTA across the EU.

Also working to reduce uncertainty is the European Innovation Network (EIN), a UK-based organization that matches potential innovators with potential funders. The EIN also promotes knowledge sharing between the various national innovation offices and the EMA, flags regulatory issues to be discussed at the EMA level, and works to improve the capabilities of national competent authorities help innovators advance their health technologies from the national level to the EU level.

Creative, Collaborative & Transparent

The proliferation of scientific advances and the myriad new ways to collect and analyze data has resulted in a number of new initiatives that will collectively help to move innovation forward. There is unlimited potential to develop new treatments and to use new forms of data—and creative, collaborative, and transparent efforts are needed across the system to take full advantage in this era of rapid scientific progress.