2016 RAPS Convergence - Ruthsatz 1609012

Health-Related Food Session
The Future of Medical Food/FSMP
in the Context of the Global HealthCare Setting
Session Lead & Facilitator
Manfred Ruthsatz
PhD, RPh, DABT, RAC, FRAPS
San Jose, September 19, 2016

The Future of Medical Food/FSMP
 Explore medical food potential in dietary disease management
SPEAKER TOPIC
Manfred RUTHSATZ
Nestlé Health Science
Switzerland
Global Healthcare setting: demographics, disruptive innovations,
nutrition therapies, market access, regulatory convergence.
Medical Food – Demanding a Moon Shot before this Decade is Out?!
Sandy BIGELOW
Vanguard Global Associates
Tucson, AZ
The Future of Medical Food, covering patient setting,
meeting DRIs based on diet and medical food use,
and feasibility of meeting legal requirements.
MEDICAL FOOD - REGULATORY FORUM ROUND TABLE (~4:50-5:30 p.m.)
Invited Expert Speakers from
FSMP Regulations &
Health Related Food Sessions
Facilitator: Manfred RUTHSATZ
Multi-Stakeholder “pre-mortem Scenario” brainstorming:
Can we accept as Patients/Society “Suppose we are in 2025 &
Status Quo of Medical Food legal interpretation & usage have not changed,
i.e. Medical Food contributes to Disease Management as today”?
Objective: to strive for science-based regulatory and policy framework for
Timely, Appropriate, Affordable Healthcare Solutions to Patients/Society,
including Medical Food.
NEED &
URGENCY
LAW, PATIENT
& HCP
NEXT
MULTI-STAKEHOLDER
2Convergence San Jose, Sept. 19, 2016

Medical Food: An opportunity not to miss for the Dietary
Disease Management of the “Patient in the Waiting Room”
– ‘Demanding a Moon Shot before this Decade is Out?!’ 
demographics, disruptive innovations (incl. microbiome),
nutrition therapies, market access, regulatory convergence
– A Multi-Stakeholder ‘show case’: The ENHA Optimal Nutrition
Care for All (ONCA) approach ‘Not because it is easy …’, but
 too big & complex for one (stakeholder) to manage, and
 make ‘…. a giant step for mankind …’ happen
The Future of Medical Food/FSMP in the
context of the Global HealthCare setting
Page 3Convergence San Jose, Sept. 19, 2016

HealthCare Systems
requiring a Moon Shot ?!
Convergence San Jose, Sept. 19, 2016
 Medical Food / FSMP !?

1550 1650 1750 1850 1950 2050
6
3
5
4
2
1
In 2050 = 9.6
Billion
II I I IIII II
2012
Increasing World Population
Changing HealthCare Paradigms …
Address Unmet Need  Opportunities & Challenges
5
«Pharma
Model»
Treatment vs.
Prevention
Blockbuster
Market Access
Precision,
Orphan drugs
«Nutrition
Model»
PharmaFood
Case Law
EFSA et al.
Safety & Claims
«omics»
(R)evolution
Prevention
«Society
Model»
Demographics
NCDs, Lifestyle
& Prevention
HealthCare
Costs
 Timely, Appropriate, Affordable Healthcare Solutions to Patients/Society

RAPS Global Medical Food Journey 2011-2016
– 2016 (San José) - The Future of Medical Food / FSMP. Session & Expert Roundtable
– 2015 (Baltimore) - Medically determined DNRs for Medical Foods. Session & Roundtable
– 2014 (Austin) - Quo Vadis, FSMPs/Medical Foods? Session & Roundtable Panel
– 2013 (Boston) - Personalizing HealthCare: Managing the Regulatory Interfaces with
Nutrition (incl. IVDs). 2 Sessions
– 2012 (Seattle) & 2011 (Indianapolis) - Global Regulatory Environment for Foods
for Dietetic & Medical Purposes. Sessions (1) Americas (2) EU (3) Asia; AU-NZ
High-level Multi-Stakeholder Networking
 Authorities (FDA-CFSAN; Health Canada; EU Commission; CHINA CDC, CFSA; INDIA, FSSAI Scientific Panel;
FSANZ), Associations (Trade, Professional, Patients), Law firms, Medical Science (ILSI, Hospitals), Industry
Key Objectives & Topics: Awareness & Consensus Building
 Medical Foods/FSMPs, FSDUs & Borderlines, Health Claims, Personalization/
Stratification/ IVDs, Globalization-Harmonization-Mutual Recognition (Codex,
ICH, IMDRF), Adherence, Compliance, Access, Health Economics, Reimbursement
RAPS Convergence 2011-2016
Medical Food - Health-Related Food Sessions
Longterm Stewardship - Continous Excellence
Convergence San JosePage 6

Healthy Consumer  Patient
I.V.
Nutrition (Drug)
Medical Food
(Tube Feeds)
Medical Food (ONS)
Intended Use: «Dietary Management…»
Food for Special Dietary Uses
(FSDUs)
General Food
(incl. Dietary Supplements;
«Functional Foods», Health Claims  voluntary)
Diet

Medical Food:
Legal Compliance, Guidance & Challenges
Management
of a disease or
condition
Distinctive
nutritional
requirements
Recognized
scientific
principles
Established by
medical
evaluation
Compliance
Dietary Management
NOT
Treatment/cure/ …
of a patient/disease
Compliance with all requirements viewed together in context
• Nutrient,
not any ingredient
• Specific need
(Patho-mechanism
of Action) …
• Sound science
• Evidence for
clinical benefit
• Sound medical
principles
• Evidence for clinical
benefit …
Nutrition / Science Science / HCP HCP  Patient
Guidance
&
Challenges
ICD, …
Medical Foods - ltd. Guidance / Case-by-Case Approach
FDA Guidance FAQs
(2016); ANPR (1996), …
RCTs taylored to Drugs;
FDA SSA  Claims;
FTC  DS, …
EFSA (2015)
RCTs taylored to drugs;
Medical & Patient
association guidelines..
[EFSA (2015)]

Medical Foods – Key Outstanding Q&As
• Distinctive Nutritional Requirements Evidence:
- Outstanding Questions / requirements more stringent than for drugs?
• Nutrient Requirements & Clinical Advantage
- Patient Needs & Benefit
• Modification of Normal Diet Alone
- HCP role, Safety & Compliance
• Cost-Effectiveness of Enteral Nutrition
Medical Food regulation: A great tool at hand
 Work together to better its
interpretation & enforcement for the patients’ benefit
Convergence - Baltimore - October 27 2015

New Diagnostics require to revisit «Disease Prevention»
& re-define what constitutes the «Patient» in the future
«Disease Prevention = Medicine» !(?)»  Diagnostics / «omics (r)evolution creating new gray zones?
Where does health end (homeostasis), disease start? Implications for early interventions & regulations?
PREVENTION
Level
Definition (US NLM - Medical Subject
Headings (MeSH)): Methods to …
Illness / Disease
Primary
… avoid occurrence of disease.
Most population-based health
promotion efforts (e.g. vaccination)
Absent / absent
Secondary
… diagnose and treat
existent disease in early stages
before it causes significant morbidity
Absent / present
Tertiary
… reduce negative impact of
existent disease by restoring
function/disease-related complication
Present / present
Already Status Quo:
Nutrition &
Disease Prevention
Disease (Symptom) Prevention:
e.g. Cow’s Milk Allergy; PKU &
other I.E.M.s; Crohn's Disease
Disease (Risk Factor) Prevention:
sterols & CVD (US, EU: few claims
approved for foods («DR(F)RCs»))
Prevention (Disease Consequences) -
falls/hip fracture in
osteoporosis
Consequences for
Patients & Society?
Nutrition: to what extent are
• Developers ready to invest into
complex nutrition & disease studies,
i.e. uncertain success with ltd.
incentives/ ROI (incl. development
costs; access)?
• Regulators & Payers ready to
accepting limited evidence & related
predictive «uncertainty»?

http://www.european-nutrition.org/images/uploads/pub-pdfs/Patient_perspectives_on_nutrition_.pdf
De facto nutrition «treatment
(prevention) of disease»: yet
permitted  «For the dietary
management of …»
US FDA: IND Guidance (2013, 2015) -
Section VI, Part D (“Foods”)  AGA
(4/2014) ‘negative consequence … to
human food/nutrition research ... field
of GE & gut microbiome’
ECCO/ESPGHAN (2014) “Consensus
Guidelines on pediatric Crohn‘s
medical management:
exclusive enteral nutrition as
Induction therapy of 1st choice”
Medical Nutrition: Improving Nutritional Status / Clinical Advantage
Can be a de-facto Disease Prevention/Management/Treatment - Complementing Drugs
Enteral Nutrition (EN)
(i.e. tube feeds and/or ONS) &
Parenteral Nutrition (I.V.)
Health Care Professionals’
key role for proper intended
use (compliance, safety)

Increase Flexibility between Food & Drug Frames for
Innovative Solution-Focused Dietary Disease Management
Regulatory Design & Gaps
Food or Drug «Intended use» designed @ very start of development:
‘ Changing horses midstream? ’ 
~Start from scratch to meet compliance requirements
«Disruptive innovations» in dietary disease management:
Difficult to meet all category requirements when switching frames
 Nutrition vs. drug CMC (monographs; analytics; G(X)P; …); clinical (disease) endpoints
 Nutrient «cocktails» not adapted to [mono-]dose-response drug requirements
 Health vs. disease dosage continuum: nutritional  pharmacologic  toxic
 Patho-mechanism of action («DNR») proof for medical food, yet not drugs

Revisit HealthCare Regulatory & Policy Framework requires
Multi-Stakeholder Innovation & Need Based Actions
1. Build Bridges behind common goal
– Foster investment into new/disruptive
science, solutions
2. Refine Food Drug Continuum
– Disease Prevention (primary, secondary, tertiary)
– «Less is More»: Flexibility for nutrients
 remove technical development barriers
(Quality/Safety, not Disease based)
3. Medical Food/FSMP Specifics
– Dietary Disease Management & Therapy
– Strengthen «Certainty»: Phase IV evidence
vs Pre-market registration;
Define acceptable level of «Uncertainty»
– Strive for Global Convergence; Market Access
– Incentivise Use Pathways: expand existing
solutions  DR Malnutrition: EN vs. PN;
HEOR; prevention vs. treatment 14Convergence San Jose, Sept. 19, 2016

ONCA
‘Every patient
who is malnourished or
at risk of undernutrition
is systematically
screened
and
has access to
appropriate, equitable,
high quality
nutritional care’

An Example of a Successful
Multi-Stakeholder Model
in Progress

Conclusion – Actions to Enable Innovation
Demographics & Co(nsequences)
require Medical Food/FSMP
as a key ally for an innovative
disease management
Disruptive innovations:
better understanding
of interconnections
 Genetics, Nutrition,
Medical Treatment
& Lifestyle
Healthcare Regulatory & Policy
Frameworks are largely
sufficient, yet inconsistent or
unprepared in some cases
Simplify  “Phase IV”
Market Access
Nutrition & Disease
Prevention
(primary, secondary, tertiary)
Management/Therapy
Stratification
Microbiome
Dialogue to Accelerate Policy
Making to catalyse incentives
for developing healthcare
solutions, incl. Medical Food
Leverage
Multistakeholder
Expert Venues
Facilitated by
Glocal Platforms
(WHO/Codex, EU, US … -
OECD, RAPS, Mérieux …)

18
1967 - 2004 Treated PKU Patients
FSMP - Making a Difference

ROUNDTABLE: The Future of Medical Food/FSMP
 Explore medical food potential in dietary disease management
SPEAKER TOPIC
Manfred RUTHSATZ
Nestlé Health Science
Switzerland
Global Healthcare setting: demographics, disruptive innovations,
nutrition therapies, market access, regulatory convergence.
Medical Food – Demanding a Moon Shot before this Decade is Out?!
Sandy BIGELOW
Vanguard Global Associates
Tucson, AZ
The Future of Medical Food, covering patient setting,
meeting DRIs based on diet and medical food use,
and feasibility of meeting legal requirements.
MEDICAL FOOD - REGULATORY FORUM ROUND TABLE (~4:50-5:30 p.m.)
Invited Expert Speakers from
FSMP Regulations &
Health Related Food Sessions
Facilitator: Manfred RUTHSATZ
Multi-Stakeholder “pre-mortem Scenario” brainstorming:
Can we accept as Patients/Society “Suppose we are in 2025 &
Objective: to strive for science-based regulatory and policy framework for
Timely, Appropriate, Affordable Healthcare Solutions to Patients/Society,
including Medical Food.
NEXT
MULTI-STAKEHOLDER

Pre-Mortem
Analysis

PRE-MORTEM ANALYSIS  Medical Food –
Contributing to a Global Healthcare Solution!
Requiring a Moonshot ?
• Step 0: The Plan
• Step 1: Preparation
• Step 2: Imagine a Fiasco
• Step 3: Generate
reasons for failure
• Step 4: Consolidate lists
• Step 5: Revisit the plan
• Step 6: Periodically
review the list
 G.Klein, The Power of Intuition (2003)
 RAPS FSMP sessions; your brain; 1 sheet
 Crystal Ball: complete, embarrasing, total
fiasco by 2025 (patient care, costs, …)
 Each of us explore for 3 minutes all
reasons WHY the failure occured?
 YOUR Top Issue 1st; then your #2 …
 WE address top 2-3 of greatest concern to
help avoid or minimize them
 Before RAPS 2017? Join ‘FSMP Think Tank’
 Contact: manfred.ruthsatz@nestle.com
https://en.wikipedia.org/wiki/Fiasco_(bottle)
PRE-MORTEM: can we accept as Patients/Society “Suppose we are in 2025 &

Rising Healthcare spending (US)

Medical Food: Law & Multistakeholder Considerations
«Medically Determined Distinctive Nutritional Requirements»
• “Management of a disease or condition for
which distinctive nutritional requirements,
based on recognized scientific principles, are
established by medical evaluation. …”
[CFR Title 21 §101.9] A food is subject to this exemption only if:
• (i) It is a specially formulated and
processed product (as opposed to a
naturally occurring foodstuff used in its
natural state) for the partial or exclusive
feeding of a patient by means of oral
intake or enteral feeding by tube;
• (iv) It is intended to be used under
medical supervision; and
• (v) It is intended only for a patient
receiving active and ongoing medical
supervision wherein the patient requires
medical care on a recurring basis for,
among other things, instructions on the
use of the medical food.
• (ii) It is intended for the dietary management
of a patient who, because of therapeutic or
chronic medical needs, has limited or impaired
capacity to ingest, digest, absorb, or
metabolize ordinary foodstuffs or certain
nutrients, or who has other special medically
determined nutrient requirements, the dietary
management of which cannot be achieved by
the modification of the normal diet alone;
• (iii) It provides nutritional support specifically
modified for the management of the unique
nutrient needs that result from the specific
disease or condition, as determined by
medical evaluation;

Nutrient Requirements & Clinical Advantage
• Patient’s special dietary requirements may arise as a
result of the disease, disorder or medical condition and/
or from experiencing, or being at risk of malnutrition
– Deficiency of nutrients may result from the inability to ingest,
digest, absorb, metabolise or excrete nutrients.
– Requirements can also be increased (e.g. PKU  Tyrosin).
• Nutritional status focus should not exclude dietary
intervention leading to a clinical advantage from a
Medical Food, e.g.
– Dysphagia; Crohn’s disease; Cow’s Milk Allergy

Modification of Normal Diet Alone («MODA»)
- Role of Healthcare Professional (HCP)
• Medical Foods are not Convenience Foods, yet how figure in
Compliance, Safety, Efficiency, QoL/daily life impact issues?
• The HCP has a key role in deciding when a Medical Food presents
more clinical advantages than modifying the normal diet.
– Taking into account composition, intended use under medical supervision
and the proposed instructions for use (incl. patterns of consumption)
• The HCP is best suited to judge compliance or safety, when a
dietary change is impossible, unrealistic or very difficult for e.g.
– Alter the texture of the diet for a stroke patient with extreme impairments
of chewing/swallowing (dysphagia) to achieve nutritionally adequate intake
– A progressively dement patient unable to adequately modify the diet due
to an inability to remember the nutrients required
– A child with intractable epilepsy to pursue a long-term,
non-palatable ketogenic diet

Cost-Effectiveness of Enteral Nutrition

2016 RAPS Convergence - Ruthsatz 1609012

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