To celebrate its 50th Anniversary, OHE held a conference this week, The Challenges and Economics of Drug Development in 2022, which explored the likely evolution of the key aspects of drug development over the next ten years. The conference was attended by over 100 participants from the public and private sectors; speakers and panellists included leaders from drug regulatory agencies, health technology assessment (HTA) groups, payers, the pharmaceutical industry, private foundations, and academia.
The conference identified two core themes that will define the challenges for drug development in the next ten years: new, evolving technologies and more difficult disease targets.
Dramatic advances in technology are having a critical impact on all aspects of drug development, from identification of areas for research through improving the value of a new product once on the market. At every step in the process, the ability to collect and analyse data of increasing complexity and sophistication continues to grow and change. The new technologies are producing results already; for example, clinical trials in some therapeutic areas are becoming more efficient through the use of diagnostic and monitoring tools that help select the most appropriate patients and better capture their responses to treatment. The same approach is beginning to have practical application after approval as well, as personalised medicine (also called “stratified” or “precision” medicine) enhances the prospects for treating patients more effectively and safely. At the same time, however, many diseases with great unmet medical need today are very difficult to understand and to treat; for example, cancers, autoimmune diseases and neurological diseases such as Alzheimer’s.
The speakers and panellists generally agreed that the most important advances in treatment over the next decade will be through the development and use of biopharmaceuticals, often with companion diagnostics and/or other monitoring technologies. Indeed, biologics already account for about a third of the drugs now in R&D in the biopharmaceutical industry, with that share expected to grow. Such drugs tend to be riskier to develop and more expensive to produce than “traditional” drugs. To encourage continued innovation and better spread risk, new ways of collaborating across stakeholders will be crucial. This will include a broad range of interests: those involved in R&D — companies, research centres and, in some cases, private foundations and other funders — as well as drug regulatory agencies, HTA bodies, payers and patient groups.
Specific observations and recommendations included the following.
- Partnerships early in R&D can be vital in identifying which targets to select and how to demonstrate effectiveness most efficiently
- New approaches to R&D that spread risks, and share benefits, are important to incentives for innovation and potentially involve a wide range of public and private parties
- Encouraging innovation includes finding approaches to creating “clusters” of life sciences experts that can produce synergistic interactions, advancing science more quickly; the UK is exploring options for increasing acvtivity within and across its clusters
- Decisions about clinical trials often can benefit from timely discussions involving companies, regulators, HTA agencies and patient groups that provide guidance – on end-points, for example – potentially enhancing efficiency and minimising cost
- Efforts to harmonise the benefit-risk standards of different regulatory agencies should continue because these affect clinical trial design and, so, both development times and costs
- Earlier licensing — or a different, “adaptive”, approach to licensing — should be considered as a way to allow new products to reach the market sooner, with provisions for the collection of specific Phase IV data essential for fuller efficacy and safety profiles
- Better, more open communication among all stakeholders is critical to ensuring that each is asking the right questions — based on a more accurate understanding of both challenges and capabilities than is prevalent today
- New approaches to determining pricing and reimbursement should be developed that, for example, provide incentives for stratified medicine and for developing efficacy and safety data for more than one indication per product
- Use of electronic health records, registries and other patient databases to link diagnosis, treatment and outcomes are key to improving knowledge of relative effectiveness and safety
The ultimate goal over the next decade, then, is to reduce uncertainty and cost in drug development to encourage innovation for the toughest diseases and at an affordable price.
Editor's note: The published proceedings may be downloaded by clicking here.
Posted in Drug Development/R&D, Innovation, Precision Medicine, Pricing and Reimbursement, Risk-sharing Arrangements | Tagged 50th Anniversary, Events