What Lies Ahead for 2013?
DIA's first annual What Lies Ahead? report, provides expert insight into the year ahead for pharmaceuticals, biotechnology, and the product development of medical devices.
With thought leaders from industry and academia asked to choose the trends they foresee shaping the world of medical product development in 2013 most agreed that the number one trend for this year will be “new models of innovation.” Based on the report, it is highly anticipated that industry will focus on collaboration and partnerships which are becoming the new norm for therapeutic research and development as companies look to capitalize on innovations outside their own walls. The report points to shrinking industry R&D budgets as being a motivator for such partnerships. The desire to reduce the time to market for urgently needed medicines and to avoid needlessly exposing patients to previously failed approaches were highlighted as strong motivators as well.
Susan Cantrell, Director, North America at DIA said, “We are excited to provide insights into where the industry is heading each year and what may continue to grow in importance over the next few years. These projections of the future landscape can be valuable to companies as they strategize to successfully meet the ever-changing needs of the marketplace.”
The study shows expectations of 2013 are that of the continued strengthening of consumer/patient empowerment (2nd most expected trend), a focus on unmet medical needs as the driver for innovations, and growing regulatory cooperation and convergence across multiple regions.
“While this study is specific to North America, and the United States in particular, as the global forum for therapeutic innovation and regulatory science we anticipate it being useful on a worldwide scale,” said Cantrell.
Therapeutic Innovation & Regulatory Science in 2013
1. New Models of Innovation. Collaboration and partnerships are becoming the new norm for therapeutic research and development as companies look to capitalize on innovations outside their own walls. Collaboration in precompetitive spaces, once taboo, is now sought after with the formation of such groups as TransCelerate and public-private partnerships like the Target Validation Consortium between NIH and industry. Industry-academic partnerships are increasingly important as industry leverages the innovations created in universities and academic medical centers while contributing its expertise in development and commercialization. Shrinking industry R&D budgets are a motivator for such partnerships, but the desire to reduce the time to market for urgently needed medicines and to avoid exposing patients needlessly to previously failed approaches are strong motivators as well.
2. The Continued Rise of Patient/Consumer Empowerment: the Importance of Patient/Consumer Engagement. The goal of therapies is to improve patient health and healthcare outcomes, and the patient/consumer has been recognized and accepted as an important stakeholder in the development of therapeutic products. Patient input has a growing influence on the design of clinical trials, the endpoints/outcomes sought, the weighing of benefit vs risk, the products that will be developed, and whether they will stay on the market. Industry and regulatory agencies continue to look for the best ways to engage patients in meaningful dialog and optimize their input.
3. Learning How to Utilize Big Data. The vast amount of data available is spurring innovations in medical research, such as uncovering the genetic basis for disease and response to treatment. It is now possible to combine multiple data sets to conduct meta-analyses to gain insight into complex medical issues in days rather than years. It is becoming possible to utilize a variety of different data sets to understand how therapeutic products are performing in the real world. And under healthcare reform, accountable care organizations (ACOs) will reimburse healthcare providers based on quality outcomes and measures. All of these applications of big data will require collaboration, new ways of thinking, and new skill development for industry professionals.
4. Achieving Market Access is the New Goal, and Value Is a Key Driver. Companies must now demonstrate the value of their products in meeting medical needs effectively, safely, and affordably. Connecting with regulatory agencies and patients/consumers is only part of the task; companies must develop early dialogs with payers to understand their expectations as well. Biopharmaceutical and device companies can no longer function solely as manufacturers of products; they must integrate into the healthcare ecosystem partner with providers and payers as a provider of therapeutic options. They will do this by investing in comparative effectiveness research and by designing trials to meet the needs of both regulators and payers.
5. Regulatory Cooperation and Convergence. The alignment of regulatory requirements across countries or regions is not new but is becoming more common for multiple reasons. With a growing global market and the need to use resources efficiently at both industry and regulatory levels, regulatory agencies may share their workloads through consortia of agencies, such as the International Regulators Consortium (TGA of Australia, Health Canada, Swissmedic of Switzerland, and the Health Sciences Authority of Singapore). Cooperative efforts among regulatory agencies may include anything from supply chain inspections to clinical site monitoring to review of submissions. The most compelling reason for convergence, however, is to share expertise to improve the quality of reviews and to promote consistent approaches to the protection of patient health in a scientifically complex and global healthcare market. Other examples of strong convergence initiatives include the Asia-Pacific Economic Cooperation (APEC); the China/Korea/Japan Tripartite Cooperation; the International Serious Adverse Events Consortium (iSAE); the increasing cooperation between the US FDA and the EMA; and even cooperation through parallel review of products by regulatory and medical coverage agencies.
6. Personalized Medicine/Tailored Therapies and Companion Diagnostics. Scientific advances have begun to improve our understanding of disease states, mechanisms of action of new and existing treatments, and some reasons for variable responses to therapies among individuals. Industry has recognized that there is potential for success of therapies in smaller, more appropriately identified patient groups. Increasing flexibility of regulatory agencies in trial designs is helping sponsors to identify sensitive sub-populations for whom therapies at appropriate doses will be successful. Companies, with the cooperation of regulatory agencies, are optimizing processes for developing companion diagnostics for new and existing therapeutic products. Successful patient outcomes on first prescribed therapies will benefit overall costs of care, making this an important trend for payers, as well.
7. An Explosion of Mobile Health Applications. The possibilities are almost endless as mobile technologies become more reliable, sophisticated, and interoperable. Companies are utilizing the technology for patient reported data, monitoring, and simple communication. Context-based applications can collect data about the patient’s environment and prompt reminders or medical team alerts. Regulators are looking for ways to leverage the technology to communicate risk information to patients and consumers. Patient recruitment can be facilitated through mobile communications, and one company has even experimented with a virtual clinical trial, enabled by multiple mobile applications. Mobile applications have also proven valuable in linking remote workers and multiple worksites to central data and support services to maximize the efficiency of professional teams. This is just the beginning of the innovations that mobile health technology will support, but advances in data security will be essential to sustaining growth, and regulatory agencies will be challenged to establish rules and guidelines to protect patients and their privacy.
8. Continued Importance of Global Markets.* Due to changing demographics and the influence of emerging markets, companies must work within a global market to achieve the greatest success. There are significant benefits to expanding globally, such as increased revenues and lower costs, but also new risks. Growth can be sporadic in different regions, and health needs, healthcare systems, health payment structures, business concerns and cultural concerns are unique within each country. Successful companies conduct early stage analysis and planning to account for varying global factors when selecting countries for expansion. They also create partnerships to address these varying needs and to build infrastructure, including training of the workforce. BRIC countries are still important as emerging markets, and companies are looking at “pharmerging” countries in Indonesia, Asia, Africa, and Latin America. Higher growth rates for therapeutic product sales in regions like Latin America, India, and China are having an impact in the US and Europe, too, by prompting a shift in sponsors’ development efforts away from the mature and toward the growing regions. This affects not only the product pipeline but also marketing, sales, reimbursement, and medical affairs efforts, which must be culturally compatible and meet local needs. The development of appropriate teams abroad is expected to continue to accelerate.
9. Clinical Trial Data Transparency.* Though clinical trial data may be viewed as proprietary by some, there is a growing movement to share clinical trial data, within the bounds of protection of individual patient privacy. In November 2012, the EMA expressed its intention to create an implementation plan for the release of clinical trial data, and that effort is set to begin in April 2013. FDA has discussed in various forums the benefits of the prudent release of clinical trial data, and though there is no specific plan to do so, at least one US-based company (GSK) has announced that it will share its clinical trial data publicly. TransCelerate and similar groups are planning to share data within their own boundaries for now, and further developments can be expected in 2013.
10. Growing Ability to Make Meaningful Benefit–Risk Assessments.* Many different approaches to assessing the balance of benefit versus the risk of using a therapeutic product are beginning to converge around a set of common elements that are accepted as integral to a meaningful analysis. The industry and regulatory agencies are also growing in their understanding of the variable importance of different elements at different stages of the product development. The use of a common framework for decision-making will enable the process to be more transparent, but methods and best practices for successful communication of the assessment to the patient (or caregiver) will continue to evolve.
11. Laser Focus on Unmet Medical Needs.* In both mature and developing markets, the search for innovations is focusing on unmet medical needs. Products that duplicate available treatments are no longer valued unless they are meaningfully superior or lower in cost. In developing markets, needs may be country-specific, and products must fill a need at a competitive price in order to be successful. In mature markets, as well as in some developing markets, there is a huge chronic disease burden and unmet medical need in diseases such as Alzheimer’s Disease, cancers, and diabetes. Emerging trends such as a focus on wellness, health, and prevention of chronic disease are driving research on obesity therapies. Rare diseases also represent a large collective disease burden and will continue to be a focus of therapeutic development.
*Trends 8-11 each received the same number of votes for inclusion in the “Top 10” trends.
The next trend received almost as many votes as the four-way tie for the last spots in the top “10” and deserves an honorable mention – it will certainly be important in the next few years:
12. The Era of Data Standardization Is Here. Driven by regulatory agencies’ needs to meet the new review timelines in PDUFA and other user fee agreements and to do the best, most thorough reviews, data standardization is being given high priority this year. Industry and regulators alike need better tools and readily available data to make better informed decisions more quickly. Data standardization efforts in therapeutic disease areas will enable sharing of data across platforms to leverage multiple data sets for real world assessment of therapeutic efficacy and safety.
To obtain a copy of the study or to interview a respondent, please contact Eric Rosen at Eric.Rosen@diahome.org or at +1.215.290-9153.