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The Future of Orphan Drugs

Orphan Drugs: Future opportunities and challenges  

The orphan drug sector is booming. Record product approvals, robust pipelines and regulatory incentives. But will the good times last? As the market matures expect increasing competition, clinical research pressures and mounting payer scrutiny. It’s time to plan for the future.

The Future of Orphan Drug provides a 360-degree scan of the horizon for rare disease therapies. Through primary research with leading industry experts, the report identifies key strategic and tactical pressure points for companies seeking to consolidate or enter this high-growth space.

“You will see more companies getting into this space. We will see more novel treatments come out for diseases that never had a treatment. There will be more patient involvement in the process as we go forward.”

Tom Croce

Head of Global Patient Advocacy, Shire

Leading Pharma Experts Answer Key Questions:

Market drivers: What’s driving investor and Pharma interest in orphan drugs?

Rising competition: What impact could rising competition in key sectors such as Duchenne’s Muscular Dystrophy have on prices, market access and innovation?

Education is key: How can orphan drug developers help payers and regulators determine the value of drugs for rare disease for which clinical knowledge is limited?

Pricing: Payer scrutiny is expected to increase as orphan drug price continue to rise. How can industry proactively respond to demonstrate value and support pricing?

Clinical trials: As the orphan drug market matures what are the growing challenges for the design of, and recruitment to, clinical trials and why is patient engagement so critical?

Gene therapy: With the first products coming to market, will gene therapy unlock the full potential of the orphan drug market?

Key Topics Explored

The drivers of pharma and investor interest in the potential of orphan drugs are demonstrated, as the sector is expected to achieve total annual sales of US$178 billion by 2020 - a 2014-2020 CAGR of 10.5%.

The importance of KOLs is expected to increase in research, usage and payer/regulator advocacy.

Rising competition means orphan drug marketing will have to evolve; but how and what impact could this have on smaller companies/biotechs who lack commercial infrastructure?

Big Pharma will have to become more flexible if they are to remain competitive in the orphan drugs market in the future

Delivering innovative products that make a meaningful difference to patients, physicians and payers are critical “must haves” for orphan drug commercial success in the future.


The current market for orphan drugs
Key findings
Orphan drug definition
Current market overview
The orphan drugs market is increasing in value
There are a large number of orphan drugs being approved each year
The orphan drugs market is split between blockbuster products and niche therapies
New products will continue to drive growth
Companies involved in the orphan drugs market
The orphan drugs market has benefitted from several positive trends in recent years
The orphan drug marketplace is maturing
The orphan drugs market attracts strong investment
Interest in rare diseases has led to new technologies emerging
Current challenges in the orphan drugs market
Key stakeholders may lack deep knowledge of rare diseases
Certain rare diseases are becoming increasingly competitive
Companies are facing increasing attention from payers
The future for orphan drug development
Key findings
New pathways and technologies are expected to advance orphan drug R&D
Gene therapy is a promising area for rare disease
Academic research will continue to be pivotal for orphan drug development
Clinical trials will continue to be the main challenge in R&D
Defining endpoints will remain challenging for orphan drug trials
The positive direction for regulatory approvals is expected to continue
The FDA has well-established processes for rapid approval of orphan drugs
The EMA is following suit in establishing a pathway for rapid review of drugs
PDUFA VI will also boost the environment for orphan drugs
It is hoped there will be greater dialogue between manufacturers and regulators
Regulatory agency capacity and knowledge is still a challenge
There may be greater scrutiny of products using the orphan drug pathway
The future for orphan drug market access
Key findings
Overall, the orphan drug market access environment is positive
Orphan drug prices have historically been very high
Orphan drugs have generally enjoyed unrestricted reimbursement and access
Increasing scrutiny of orphan drug prices is expected
Innovative pricing is seen as a potential differentiator
Reimbursement and access is expected to become increasingly challenging
Demonstrating value is seen as critical for access in the future
The future for orphan drug commercialisation
Key findings
Traditional sales and marketing strategies are not generally used for orphan drugs
Education and awareness will continue to drive orphan drug marketing
Increasing competition means orphan drug marketing will have to evolve
Stakeholder relationships critically important for commercial success
Patient engagement is central
Engagement with physicians and payers will continue to play an important role
The future outlook for the orphan drugs market
Key findings
The orphan drugs market offers significant opportunity
Specialist companies will continue to lead the orphan drugs market
Big pharma’s future in the orphan drugs market is uncertain
Three areas of focus identified
Knowledge and experience will be critical for future orphan drugs success
Collaboration with all stakeholders should be central to corporate strategy
Innovation will continue to be rewarded

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