2016 RAPS Convergence - Ruthsatz 1609012

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


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

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)]


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 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


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)

http://www.european-nutrition.org/images/uploads/pub-pdfs/Patient_perspectives_on_nutrition_.pdf

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 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


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 …)


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


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: 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: [email protected]

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)


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 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


Documents

2016 RAPS Convergence - Ruthsatz 1609012