# Presentation: 285239

## Implications and Challenges 

- Vincent H.L. Lee
- OPS/CDER/FDA

## Innovation

- A continuous process designed to ensure high quality products

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## Long-term Goal

**To increase the return on investment by fostering innovation**
**To improve the quality of life**
**To lower the health care costs**

## Scenario in 2010

- Development costs reduced by 30%, development time shortened by 50%, success rate increased by a factor of 3 (=30%)
- More drugs will be launched in a controlled delivery platform (no longer used solely as a line-extension tool)
- Sponsors of compounds in a new drug class will join a knowledge-sharing consortium. There will be built in incentives for pioneers.
- Sponsors will subject their science to peer review for open access to the global community.

## Scenario in 2010

- Sunset of the blockbuster era?
- Dawn of a new era of narrower indications in population clustered around genotype
- A growing number of nano-sized assemblies with specialized functionalities (e.g., a satellite in inner space to detect and report biochemical changes characteristic of a disease state)

## Possible Business Models

- Innovators  generics (incentives to improve qualify at a lower cost)
- Consolidation of generic business
- Partnership between innovator and generic sectors
- Expansion in the roster of OTC drugs

## Lessons Learned from Recent Effort 

- Combinatorial chemistry
- High throughput screening
- Choice of animal model
- Reliability of animal data
- Design of clinical trials: Subject selection
- Diagnostic tests for monitoring progression of disease

## The Critical Path for Medical Product Development

## Slide 11

## Possible Solutions

- Drug design = drug action target + drug delivery target
- Leverage on molecular and user diversity
- Mechanism-based, holistic approach
- Incentives for innovation: Brand  Generics
- Knowledge: Information management and sharing within as well as outside the organization

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## Implications

**Individuals: 360****◦ scan**
**Infrastructure **(to respond to emerging opportunities on short notice.)
**Incentives **(innovation and teamwork)
**Interrelationships **(academia, industry, other agencies, organizations)

## What’s Next?

**Root cause?**** Case studies?**
**Benchmark?**** More effective, safer, cheaper drugs?**
**Which sector? ****Pharma, generics, biotech, start-ups**
**Which drug class?**

## Slide 19

## Challenges

**Communication**: broad understanding and shared goal  community-wide
**Inspiration**: to create new paradigms
**Motivation**: to adopt a new approach to decision making (willingness and capacity to unlearn, relearn, and learn)

## Implications

- Be proactive
- Be interactive
- Unlearn, relearn, and learn
- Commitment to innovation
- Reward (incentive) for innovation
- Transforming the culture

## Industrial vs. Knowledge Society

**Means of production**
**Pace of change**
**Degree of complexity**

## Industrial Knowledge 

- Machinery, assembly line, robots
- Single entity
- Individual
- Snail pace
- Static
- Proprietary info
- Institution-centered

- Explicit (head) and tacit (hand) knowledge
- An ensemble
- Network
- Fast pace
- Dynamic
- Shared info
- Consumer-centered

## Beware!

- Knowledge becomes obsolete as soon as it is created. Therefore, new knowledge has to be created constantly and ubiquitously.

## FDA’s Role: Some Examples 

- To champion the cause of modernizing drug development
- To catalyze the creation of new paradigms for increasing the probability of success in drug development
- To demonstrate by action its commitment in innovation
- To influence the education of regulatory scientists for the future

## Road Map

- Create incentives for industry and academia to formulate and test alternate drug development schemes
- Coordinates data mining worldwide for forecasting hurdles to drug action, delivery, formulation and manufacture
- Develops computational tools for integrating discrete events in a system context that allows for forecasting the behavior of the system when perturbed

## Drivers for Change

## Rise in Consumerism

- High price of prescription drugs
- Skepticism of justification for price
- Prescription price control
- New business models

## Life Cycle Management

- 	a. Cost: generics
- 	b. Therapeutic value:
- 		controlled release products
- 	c. New indications

## Globalization 

- Information
- Life style
- Commerce: Emergence of pharmaceutical corridor in south east Asia

## Drivers for Action

- Rapid, Significant Advances in Biology and Engineering
- New Research Tools
- Better Understanding of Disease
- Increased Life Span
- More Sophisticated Consumers
- Cost Containment

## Outreach

- To sponsor retrospective studies on the value of sharing knowledge in accelerating drug development and rendering it more precise.
- To convene a summit with industrial and academic scientific leaders, identify the pros and cons of sharing knowledge and discuss mechanisms to conduct data mining without putting the innovator at a competitive disadvantage (A strategic plan for drug development??)

## Process

- To examine the current review practices with respect to fostering innovation and propose necessary changes.
- To develop mechanisms for facilitating continuous improvement in the quality of approved products
- To be proactive in identifying cutting edge research of pharmaceutical relevance that would fuel innovation.

## Human Resource

- To shape the education of future regulatory scientists
- To evaluate current practice of scientist recruitment and retention as well as leadership development

## Evolution or Revolution?