Focus on the Future Orphan Diseases: The Next Chapter

1
Focus on the FutureOrphan DiseasesThe Next
Chapter
2
The Biotechnology Industry

• The Orphan Drug Act along with Bayh-Dole have
  been instrumental in building the biotechnology
  industry.
• The 7 years exclusivity incentivized companies to
  invest in therapies for rare disorders.
• Today, BIO represents over 1,100 members in all
  50 states and 34 countries and growing.

3
Challenges

• Limited public and private funding for basic to
  clinical research, the translational stage, has
  affected the development of better therapeutics
  across the board rare diseases are no
  exception.
• Small patient populations compounded with genetic
  components associated with many rare diseases
  lead to a variability in response rates.
• Need for creativity in trial design.

4
Benefits

• Good investment for Venture Capital low number
  of patients translates to smaller trials, lower
  costs, and less risk for investors should trial
  fail.
• Since a larger percentage of the patient
  population is exposed to the drug/biologic during
  development, orphan drugs tend to be more
  effective.
• Same requirements for safety and efficacy,
  however, FDA more flexible.
• The Orphan Drug Act made it good business sense
  to invest in biologics for rare diseases.

5
Issues

• There are numerous state and federal policy
  issues that can affect the development of the
  biotechnology and pharmaceutical industries.

• I.P. Protection
• Technology Transfer
• Capital Formation

• Regulatory Environment
• Reimbursement
• Bioethics

6
High Cost ofDrugs and Biologics

• Company Stage Private investment per company
• Proof of Concept 25,000 100,000
• Pre-seed 50,000 500,000
• Seed 150,000 2 million
• Early-stage 1 million 5 million
• Expansion-stage Up to 10 million
• Mezzanine Up to 20 million
• Successful Product Launch 10-15 years - 1
  billion

7
Future

• Simplify the United States and European Union
  regulatory processes
• Savings for patients and investigators
• Time-saver
• Money-saver
• Personalized Medicine
• Will everything be a rare disease?
• Will people want to change the ODA?
• Will costs increase or decrease with smaller
  populations?

8
Conclusion

• Too many diseases and disorders remain untreated
  or poorly treated. The Biotechnology Industry
  Organization is dedicated to encouraging
  continued research and development of
  therapeutics for all diseases. We look forward to
  working with you to help find the necessary
  balance of access, availability, and incentives
  for future innovation.

9
Thank You
Tricia Brooks Managing Director, Alliance
Development
Biotechnology Industry Organization
1201 Maryland Avenue, SW - Suite 900
Washington, DC 20024
[phone]
tbrooks_at_bio.org