@ddwn @ArmyneNice little run GLJust a report I found from May 20...

@ddwn@Armyne

Nice little run

GL

Just a report I found from May 20 so a little dated & obviously not include latest Covid developments etc but discusses Ryoncil GVHD & provides some Key Stakeholders perspectives in summary. Just pasted some background info and a couple of poss more pertinent sections. GL

Original report HERE

PCORI Health Care Horizon Scanning System Volume 2 Issue 1
High Potential Disruption Report
May 2020
Prepared for:
Patient-Centered Outcomes Research Institute
1828 L St., NW, Suite 900
Washington, DC 20036

Report Methods

The purpose of the stakeholder survey process is to help determine which topics and trends
have the highest potential to significantly disrupt patient care in some manner, such as patient
outcomes, access to care, health disparities, care delivery, staffing, and costs. Twice annually,
the horizon scanning team reviews all stakeholder comments and ratings (for currently
included topics and trends) received in the past 12 months. This review begins a process
culminating in the production and delivery of the High Potential Disruption Report, which
highlights topics and trends with high potential to be significantly disruptive to patient
care in the United States within the next 3 years.

Selecting Topics and Trends for the High Potential Disruption Report

To be considered for inclusion in the High Potential Disruption Report, topics and trends
must be active (ie, not archived) and must have received a minimum of 5 stakeholder surveys
within the past 12 months. Topics and trends selected for inclusion are those for which,
based on review of stakeholder ratings, stakeholders generally agreed have high potential
to be significantly disruptive to health care in the United States. Topics and trends
selected for inclusion are assigned to analysts for report drafting.
Analysis of stakeholder comments must generally support conclusions suggested by ratings.
Topics with borderline ratings, high variance, or questionable comments are scheduled for
discussion at the High Potential Disruption Report topic selection meeting (see below). Each
scheduled topic is reviewed by the analyst assigned to the applicable clinical priority area. The
analyst rereads the topic profile and reviews each survey received for the topic, paying
particular attention to stakeholder comments. The analyst prepares a summary of stakeholder
comments to present at the topic selection meeting.
Trends with borderline ratings, high variance, or questionable comments are reviewed by a 3-
member panel of senior horizon scanning team members to determine inclusion or exclusion.
A majority affirmative vote by this panel selects the trend for inclusion.

Table 2.1. Included Topics for Priority Area: Cancer
Topic title

Remestemcel-L (Ryoncil) to treat pediatric steroid-refractory acute graft-versus-host disease
(**and a number of others in original Table)

Key Stakeholder Perspectives

Nine stakeholders, reflecting clinical, patient advocate, research, and health systems
perspectives, provided comments and ratings on the topic of remestemcel-L for
treating pediatric steroid-refractory acute GVHD.

The list below provides a summary of key stakeholder
perspectives.

• Remestemcel-L has moderate to large potential to improve health outcomes for children
with acute GVHD, given the lack of FDA-approved therapies for this potentially fatal
condition.
• Similar improvement was not observed in the overall larger trial populations that
included adults through 70 years of age.
• Remestemcel-L could give clinicians a standardized, off-the-shelf, and more effective
option for treating children with acute GVHD instead of a cocktail of
immunosuppressants used to manage the condition.
• As a stem cell–based therapy, remestemcel-L is likely to cost much more than the
standard of care for acute GVHD; such high costs could hinder adoption, limit patient
access, and increase disparities in care.
• Although delivering remestemcel-L is more labor intensive than oral immunosuppressant
therapy for acute GVHD, the disruption to the health care delivery system is likely to be
low relative to the overall management of this patient population.


Also 29/7 from Evaluate Vantage :

Go or no go? Valuable drugs set for FDA decisions

https://www.evaluate.com/vantage/articles/events/company-events/go-or-no-go-valuable-drugs-set-fda-decisions

You obviously get a mention with others due for "Notable first-time US regulatory verdicts due in August"

	Project	Company	Pdufa date	Sales by indication ($m)	Note
1	Ryoncil (remestemcel-L)	Mesoblast	Sep	274	Ad com Aug 13; steroid-refractory acute GvHD in paediatric patients