Adam Barak, Director of PPI Healthcare Consulting Ltd, spoke in the 'Engaging with Pharma' panel at the Cambridge Rare Disease Summit 2015.

1. Engaging with Pharma:
Pricing and Reimbursement Perspectives
The White House, Main Road, Dibden, Hampshire, SO45 5TB, UK
Tel: +44 (0)2380 847891 Email: abarak@ppi.consulting Web: www.pharmaprice.net
Adam Barak, MCIM
Cambridge Rare Disease Summit, 2015

2. OD EMA Approvals (5.6pa) but rising
2
• 16 approvals in 2014
Source: www.orphan.net

3. RD EMA Approvals (4.8pa)
3 Source: www.orphan.net 2014
• 9 approvals in 2013, 3 2014

4. Rare conditions but lots of them!
4
Simple RD / OD population extraction illustration
Approved
ODs 67
Approved
non-­‐ODs 91
Total 158
Ave.
pts/10000 3
Pop
('000) OD
/
RD
Pop
Germany 81,000
3,839,400
France 67,000
3,175,800
UK 64,000
3,033,600
Italy 60,000
2,844,000
NL 17,000
805,800

5. ODs and ODs
5
Molecule Brand Company Condition Comments
imiglucerase Cerezyme® Genzyme Gaucher’s
Disease
Annual sales
$1.2b
zinc acetate Wilzin® Orphan
Europe
Wilson’s
disease
$4.50/day

6. Why Orphan Drugs are Different:
The Challenge
6
§ Very low patient numbers
– Misdiagnosis / under-­reporting
– Few specialist treatment centres
– But lots of ODs (30m Europeans, 25m North Americans)
– 5000-­8000 rare diseases affect 3-­10% of the total EU population
§ Extreme unmet need
– No effective therapies ever, desperation
– Death or serious morbidity without treatment
– Often children and babies effected
§ Potential to “cure” or significantly benefit
– Extend life, Improve quality of life
§ Potential good sales revenue but high R&D risk
– Clinical trials difficult (itraconazole severe fungal infection study: 10y to recruit 39pts)
– Significant investment vs uncertain outcomes
§ Difficult to recoup R&D costs without “disproportionate price”
– Especially for smaller companies
* Source: Dear, Lilitkarntakul & Webb;; Are rare diseases still orphans. Br J Clin Pharmacol;; v62(3), 1/9/06

7. Funding Orphan Drugs
7
§ Pricing (can be) very contentious
§ Payers must “fairly” distribute scarce resources to needy cases
– Who is to say which group should be funded and which miss out?
§ Cost justification arguments different to ‘regular pharma’
– QALY > €120,000 not atypical
– Chronic, expensive therapy for decades
– Cost offsets not straightforward
§ Payers devolving funding decisions can create inequities:
– Aldurazyme (MPS I) was funded by some regional UK payers but not others
– Soliris (PNH) was funded by some Spanish Autonomous Regions, not others
§ Only around a third to a half of EUR countries have
concessionary OD reimbursement processes
− e.g. Bel, CH, Fra, Ita, NL, Nor, UK

8. Responsibilities to the OD Patient
Community
8
• Access
• Education
• Champions
• OD / RD
Policy
• Equity
• Appropriate
Price
• Appropriate
HTA
• Responsible
Price
• Relevant Data
• Unmet need
Manufactu-­
ring Industry
Payers
Clinical
Community
Political

9. Price & Value Determination
9
High clinical value,
Unique,
‘High’ priceModerate clinical value,
Unique,
‘Medium’ price
High clinical value,
Moderate unmet need,
‘Referenced’ price
Marginal benefits,
Low unmet need,
‘Low’ price

10. OD Selected Pricing and Market
Access Approaches
10
§ Early engagement
• Data guidance
• Forewarning
§ Pre-­approval use where appropriate
§ Patient access schemes
§ Establishing/accessing ring-­fenced funding
§ Advocacy
§ Patient assistance programmes
§ Launch sequencing
§ Active negotiation / careful lobbying

11. Some Main Takeaways
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§ Growing acceptance by payers that OD/RD require a
different HTA approach but not all countries have special
exemption criteria
§ Not a free ride for pharma – even for OD/RD price:value
is to be justified
§ Audience segmentation not possible
§ Need for transparency and early engagement – all
parties

12. Further Reading
12
Orphan drugs: pricing, reimbursement
and patient access.
IJPHM, Volume 5, Number 4. 2011
Adam Barak & Jyoti Shankar Nandi