CAR-T & Cell Therapy Products: Next generation of oncology products create chance for boom or bust

The potential of Novartis' Kymriah and Gilead's Yescarta fueled blockbuster projections from analysts and a flurry of industry interest in CAR-T development. But large price tags ($373,000 - $475,000), manufacturing challenges, and serious side effects have stymied wide-scale utilization of the therapies.

Why have these promising drugs under-performed, and what is the future of CAR-T?

Best Practices, LLC conducted an extensive research project to examine the genesis, launch and market struggles of these ground-breaking therapies. "CAR-T & Cell Therapy Products: Next generation of oncology products create chance for boom or bust" is a new 78-page case study that examines:

· CAR-T's pathway to the marketplace
· Kymriah's and Yescarta's market entry strategies…and slow launch curves
· The manufacturing and pricing challenges facing CAR-T therapies
· Why payers and pharma need to find reimbursement common ground
· What the future holds for next generation CAR-T therapies and pharma

Novartis and Gilead each took billon-dollar gambles with CAR-T, and now face heavy headwinds from payers that have hit the pause button to figure out how to afford $400,000 products. But the possibilities of CAR-T as a powerful weapon against other cancers has attracted an unprecedented wave of new T-cell therapy companies - as well as Top 10 pharma companies - that are investing heavily in next generation CAR-T products.

This study pulls together multiple sources of information across key issues affecting CAR-T development and launch to give new product development leaders critical insights into the potential and challenges facing CAR-T development and launch.

Study Snapshot

The report was based on extensive secondary research that involved dozens of industry, media, company and government reports and regulatory filings.

Key Findings

Select key insights uncovered from this report are noted below. Detailed findings are available in the full report.

• Problem: Future Competition Coming: CAR-T therapies launched by Gilead and Novartis are just the start of innovative personalised therapies and the near future will see new therapies entering the market that are less labor intensive to make, treat more types of cancer and likely will cost less.

Solution: Launch Multiple Indications and Combinations: Gilead plans to launch 6 new combinations & 6 indications. Similarly, Kymriah has 3 new indications & 6 combination trials on-going. Gilead has invested in Dr. Carl June's Tmunity to leapfrog Novartis, and Novartis acquired Avexis and partnered with Spark Therapeutics.

• Problem: Payer Pressure: Both government and private payers were vocal in their criticism of CAR-T costs. To keep a grip on costs, private payers are reviewing CAR-T utilization on a case-by-case basis rather than setting standard coverage guidance; CMS was slow to set reimbursement levels, causing patient access issues.

Solution: Use Innovative Payment Methods and Negotiate Higher CMS Reimbursement: Novartis used outcome-based & indication based pricing for ALL and DLBCL. Both Novartis and Gilead are eyeing innovative approaches such as installment or annuity payments. While CMS has boosted its inpatient reimbursement level for CAR-T, some say it is still too low. Likewise, CMS is still years away from creating a separate reimbursement category for CAR-T.

• Executive Summary
• Novartis’ & Gilead’s Pathway to CAR-T Marketplace
• Gilead & Novartis Used Different Market Entry Strategies to Capture CAR-T Market
• Slow Launch Curves & Politics Create Head Winds for Both Companies
• Manufacturing CAR-T is a Complicated Bioengineering Challenge
• The Complexity of CAR-T Market is Amplified Due to Logistics & Supply Chain
• First-in-Class CAR-T Therapies Called for Extensive Market Education
• Pricing is Another Delicate Dance
• Government and ICER Weigh-in with their Opinions on Pricing CAR-T Therapies
• Kymriah & Yescarta Also Faced Problems at Global Front
• Competitive Forces Grow
• Future Unclear for CAR-T; Next Gen Products will Overcome Older Cell/Gene Therapies
• What Lies Ahead for CAR-T?

Kymriah and Yescarta Market Entry Timelines 19

Kymriah and Yescarta Target Patient Groups 22

Launch Curves Slow for Kymriah and Yescarta 26

Kymriah and Yescarta Projected Sales and Consensus Estimates 27

Manufacturing Turnaround Challenges for Kymriah and Yescarta 34

Type of Treatment Centers Favored by Novartis and Gilead 23

Kymriah and Yescarta Market Entry Price Tags 49

ICER Analysis of Cost Effectiveness for Kymriah and Yescarta 57