2016 raps convergence - ruthsatz 1609012
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Health-Related Food SessionThe Future of Medical Food/FSMP
in the Context of the Global HealthCare Setting
Session Lead & Facilitator
Manfred RuthsatzPhD, RPh, DABT, RAC, FRAPS
San Jose, September 19, 2016
The Future of Medical Food/FSMP in the Context of the Global HealthCare Setting
Explore medical food potential in dietary disease management
SPEAKER TOPIC
Manfred RUTHSATZNestlé 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 BIGELOWVanguard 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 SessionsFacilitator: 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, andmake ‘…. 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 Systemsrequiring a Moon Shot ?!
Convergence San Jose, Sept. 19, 2016
Medical Food / FSMP !?
1550 1650 1750 1850 1950 2050
6
3
54
21
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»
PharmaFood
Case Law
EFSA et al.
Safety & Claims
«omics» (R)evolutionPrevention
«Society Model»
DemographicsNCDs, Lifestyle& Prevention
HealthCare Costs
Timely, Appropriate, Affordable Healthcare Solutions to Patients/SocietyConvergence San Jose, Sept. 19, 2016
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
7Convergence San Jose, Sept. 19, 2016
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
Convergence San Jose, Sept. 19, 2016
Medical Food: Legal Compliance, Guidance & Challenges
Management of a disease or
condition
Distinctive nutritional
requirements
Recognized scientific principles
Established by medical
evaluation
ComplianceDietary Management
NOT Treatment/cure/ … of a patient/disease
Compliance with all requirements viewed together in context
• Nutrient, not any ingredient
• Specific need(Patho-mechanismof Action) …
• Sound science• Evidence for
clinical benefit
• Sound medicalprinciples
• Evidence for clinicalbenefit …
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)]
9Convergence San Jose, Sept. 19, 2016
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
Convergence San Jose, Sept. 19, 2016
Consequences for Patients & Society?
Nutrition: to what extent are
• Developers ready to invest intocomplex nutrition & disease studies, i.e. uncertain success with ltd. incentives/ ROI (incl. developmentcosts; access)?
• Regulators & Payers ready to accepting limited evidence & relatedpredictive «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 dietarymanagement 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) “ConsensusGuidelines on pediatric Crohn‘s
medical management: exclusive enteral nutrition as
Induction therapy of 1st choice”
Medical Nutrition: Improving Nutritional Status / Clinical AdvantageCan be a de-facto Disease Prevention/Management/Treatment - Complementing Drugs
12Convergence San Jose, Sept. 19, 2016
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
Convergence San Jose, Sept. 19, 2016
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 evidencevs Pre-market registration; Define acceptable level of «Uncertainty»
– Strive for Global Convergence; Market Access
– Incentivise Use Pathways: expand existingsolutions 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 SuccessfulMulti-Stakeholder Model
in Progress
16Convergence San Jose, Sept. 19, 2016
Conclusion – Actions to Enable Innovation
Demographics & Co(nsequences)
require Medical Food/FSMPas 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 healthcaresolutions, incl. Medical Food
Leverage Multistakeholder
Expert Venues
Facilitated by Glocal Platforms (WHO/Codex, EU, US … -OECD, RAPS, Mérieux …)
Convergence San Jose, Sept. 19, 2016
18
1967 - 2004 Treated PKU Patients
FSMP - Making a Difference
ROUNDTABLE: The Future of Medical Food/FSMP in the Context of the Global HealthCare Setting
Explore medical food potential in dietary disease management
SPEAKER TOPIC
Manfred RUTHSATZNestlé 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 BIGELOWVanguard 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 SessionsFacilitator: 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.
NEXT MULTI-STAKEHOLDER
19Convergence San Jose, Sept. 19, 2016
Pre-Mortem Analysis
20Convergence San Jose, Sept. 19, 2016
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: Generatereasons for failure
• Step 4: Consolidate lists
• Step 5: Revisit the plan
• Step 6: Periodicallyreview 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 & Status Quo of Medical Food legal interpretation & usage have not changed,
i.e. Medical Food contributes to Disease Management as today”?
BACKUP
Rising Healthcare spending (US)
23Convergence San Jose, Sept. 19, 2016
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.
Convergence - Baltimore - October 27 2015
• (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 AllergyConvergence - Baltimore - October 27 2015
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
Convergence - Baltimore - October 27 2015
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