Understanding Clinical Aspects: A must for creating a ... · Content shared with grateful...

Content shared with grateful acknowledgment to Stanford India Biodesign: From chapter contributed by Author

Understanding Clinical Aspects:

A must for creating a successful Bioenterprise

Dr. Avijit Bansal Co-founder and CEO,

Windmill Health Technologies

Content shared with grateful acknowledgment to Stanford India Biodesign: From chapter contributed by Author 3

Are we creating enough

perceivable value for enough

number of people for them to

pay sufficiently to enable us to

continue delivering that value

sustainably, scalably and

profitably.


Disease state analysis

Disease state

1

• Why analyze disease states?

• What to consider while analyzing disease states?

• How to analyze disease states?

Learning Objectives


Why study disease states?

Med-tech innovation is essentially the process of seeking to address

GAPS in the TREATMENT OPTIONS for DISEASE STATES

Disease state

Treatment options

GAP



• To find out potential strategies and points for intervention in the disease causation cycle • To choose among different intervention strategies • To be able to understand scientific evidence and communicate more effectively with domain experts • To be able to ask the right questions

It is imperative to understand the causes, mechanisms, effects and risks associated with diseases


•To be exposed to the entire gamut of felt needs associated with the disease •To be able to uncover needs hitherto not identified and addressed •To avoid taking a path – that is soon going to be outdated, due to an upcoming drug/ procedure/ innovation • To further validate a need

Instinct to jump ahead and start inventing may lead to misplaced, unsuccessful solutions and much agony later.


It is imperative to understand the causes, mechanisms, effects and risks associated with diseases:


Hitchhiker’s guide to jargon in the medical galaxy

With inputs from: Biodesign: The Process of Innovating Medical Technologies, Cambridge University Press

Focus Area Description

Anatomy Study of normal structure and organization of the human body

Physiology Study of Normal functioning of the human body

Pathophysiology Describes the disturbance of normal anatomy and physiology caused by disease or other underlying physical, mechanical, electrical, or biochemical abnormality

Clinical Presentation

What the disease leads to Symptoms- what patient perceives and suffers from (Eg: Pain) Signs – What the doctor elicits upon examination (Eg. Tenderness )

Clinical outcomes Profiles most common results of a given disease. Eg: Death, Disability, Hospital admission, Complete Cure

Epidemiology Describes causes, distribution, and control of disease in the population

Economic impact Outlines cost of disease to the patient, family, health care system and the society at large


Before you begin..

• First understand the body’s NORMAL STRUCTURE and FUNCTION (known as ANATOMY and PHYSIOLOGY) • Questions you may want to ask: Which system is affected? What does it do? How does it normally work? Which organ is affected? What does it do? How does it normally work? Which other organs/ systems could this affect? What is their working?

Before you can deal with the abnormal, need to understand what is normal!

• Visual understanding is important- position, size and proximity of affected organs/systems

• May need to have a gross (macroscopic) as well as microscopic understanding

• Explore physiological, biochemical, mechanical and electrical mechanisms


Questions to ask about disease state

is the disease? What

Begin by trying to summarize to yourself in one line (or a few lines): •What organ/ organ system is affected? • What is wrong with the affected system? • What is the immediate impact of the disease on the body? • What is the long term/ overall impact?



is the disease? What

Examples: •Cataract is a disorder of the of the lens in the eye leading to loss of vision •Diabetes affects beta cells in the pancreas, leading to decreased insulin secretion, leading to elevated glucose levels in the body – which puts most other body systems at risk. While short term symptoms include excessive hunger, thirst, urination – in the long term it could cause loss of vision, kidney failure, non-healing ulcers, stroke etc •Inguinal Hernia is a protrusion of an abdominal organ, mostly intestines through an abnormal opening into the scrotal sacs. While in the short term it causes discomfort, it may also get “caught” or strangulated leading to sudden life threatening situation



is affected? (Epidemiology) Who

• Age group • Gender • Socio-economic status • Geographic distribution

• Within the country • Globally

• Ethnic prediliction

All these factors have an important bearing on potential market size, choice of approach to solution, associated risk profile etc


Important terms in epidemiology

• Incidence - number of new cases diagnosed per year

• Prevalence – Total number of people afflicted with a disease at a given point of time

• Epidemiology data serves as background information for market analysis

• Include data for disease as a whole, most prevalent patient sub-segments, and disease dynamics- growth rate

Incidence

Prevalence



What causes the disease?

The term cause is used where there is a single definite cause Eg. The bacterium called MTB (Mycobacterium Tuberculosis) causes tuberculosis The term risk factor is used when multiple factors are associated with causing a disease. Eg. Heridity, Obesity, Sedentary lifestyle, stress are all associated with T2 Diabetes

Example 1: Knowing that default in treatment is the biggest cause for drug resistant tuberculosis will bring treatment compliance enhancing devices into the horizon of solutions for therapy of Tuberculosis – something that may not occur otherwise Example 2: When trying to innovate a device solution for diabetes patients: Understanding that obesity is a risk factor / cause for uncontrolled diabetes will bring obesity reduction devices into the horizon of potential solutions

(Aetiology)


How is the disease caused?

• This is the most important aspect, since it lays the foundation for potential management strategies Example 1: Ardian Example 2: Emphasys

(Pathogenesis or mechanisms)



Excessive fat intake

Excessive absorption

High cholesterol

in blood

Cholesterol deposits and blockade in

heart’s circulation

Ischemia (Angina and heart attack)

Stenting Drug therapy Bypass Surgery Ballooning

Ischemia (Angina and heart attack)

Understanding the pathogenesis broadens innovative possibilities

Conventional choice of management of Ischemic heart disease

Gastric pacemaker

(prevents patient from

eating too much)

Innovative approach to the same : Includes prevention



What is the impact of the disease?

• Knowing the symptoms is essential to understanding the biggest PAIN-POINTS for the patients, caregivers and other stakeholders

• This is key to innovating a

solution that patients (and other stakeholders) will be keen to adopt

• All 4 factors are important

(Symptoms, sequelae and outcomes)

What do they

SAY

What is the disease making them

FEEL

What is the disease making them

THINK

What is the disease

making them

DO

Patients need …



Two important points to consider while assessing impact : Morbidity and Mortality

• Quantitative measures of mortality: • Acute diseases: Case fatality rate, number of deaths • Chronic diseases:

• Reduction in life expectancy • Increased risk of death

• Quantitative measures of morbidity: • Acute diseases : Rate of hospitalization, sick leaves, avg length of hospital stay • Chronic Diseases

• Quality of life score (QOL) • Disease specific indicators eg: SGRQ for QOL in COPD patients • Disability Adjusted Life Years lost (DALYs) • Quality adjusted Life years lost (QUALYs) • Visual analogue scale (for severity of pain)


Points to consider while analyzing impact :

• Physical • Pain • Disability • Activities of daily living • Impact on Routine

• Social and emotional impact

* In a survey of single upper limb amputees, it was found that their most imporant need was “to appear normal” and surprisingly, functionality featured later

• Economic impact • Impact on the family • Impact on the health system

• This forms an important measure of the disease burden and thus need for intervention



Economic Impact

• Cost to patient and family • Cost of treating one episode (Acute illnesses) • Monthly or annual cost of treatment (Chronic illnesses) • Need for hospitalization and avg. length of stay • Analyze different components of the cost: Medication/device

treatment/hospitalization/outpatient setting cost

• Cost to health system • Annual cost of treatment to the health system • Annual cost of hospitalization to the health system

• Cost to society

• Low productivity due to absenteeism from work, • Loss of productivity due to disability of individual • Loss of productivity of caregivers • Loss of productivity due to death • Cost of caring for the disabled • Cost of treatment



More points to consider while analyzing the impact of disease:

A)Associated needs : Analyzing the symptoms may not be enough, disease may pose additional demands Example 1: Diabetes Diabetic patients need blood glucose testing that is less painful and less expensive These patients may also need help with comorbidities sich as heart disease and high blood pressure. Examlple 2: Bronchial Asthma Along with efficacious therapy, patients also need a reliable way to monitor disease and titrate therapy B) Long term sequelae: Example: Diabetic patients may need laser treatment for

retinal disorders, dialysis for renal failure, and specialized foot care to prevent ulcers


How to conduct disease state analysis

1. Talk to a doc Requesting a doctor-friend for a brief overview may be the best starting point for a disease state analysis – provided that such help is available. Junior doctors and medical students may be equally helpful, and easier to find! • Have questions ready (the kind mentioned in this deck) • Keep it short (an hour may be all you need to begin with) • Doctors tend to go on a tangent, be polite but keep the discussion focused on what you need • Ask for a quick summary describing the CRUX of the matter • Ask for a quick primer on anatomy and physiology of the concerned system / organ • Ask for the most important issues surrounding a given disease • Ask for their wish-list (but don’t bias yourself with it) • Ask for key terms to search, read about • Ask for links to reference material

• For basic anatomy and physiology • For specific disease state

• In absence of statistics, try to get estimates based on their individual experience (remember these could have several biases)



2. Need to read Begin with material describing anatomy and physiology Examples: Anatomy Gray’s Anatomy : Students edition; Clinical Anatomy : Moore and Dalley; Textbook of anatomy by BD Chaurasia ; Anatomy Atlas: F. Netter; visiblebody.com (paid

website, similar free services also available) Physiology Guyton’s textbook of Human Physiology (thick, but accessible); Ganong’s review of

Human Physiology; Textbook of Physiology by AK Jain; Principles of Medical Physiology by S. Sircar (thick, but accessible)

While anatomy textbooks may mainly help through illustrations, physiology text may be

more understandable, particularly the ones by Guyton and Sircar. Don’t be intimidated by the jargon – a broad idea is all you need



3. Internet For general info a) google.com: your best friend for all types of searches! b) Medscape.net c) WebMD.com

For epidemiological info India specific data is hard to find a) http://www.rchiips.org/NFHS/index.shtml provides data on certain issues b) http://www.who.int/en/ has some Indian data For evidence on particular diseases, scholarly papers may help a) pubmed.net b) scholar.google.com

http://www.rchiips.org/NFHS/index.shtml

http://www.who.int/en/

Content shared with grateful acknowledgment to Stanford India Biodesign: From chapter contributed by Author Content shared with grateful acknowledgment to Stanford India Biodesign: From chapter contributed by Author

Synthesis and Documentation

• Keep target audience in mind while writing the overview

• Site sources of all statistics, study results, interviews, and clinical outcomes

• Avoid unsubstantiated data from blogs and random news websites

• When plenty of compelling data is available, provide only a few relevant figures . The idea is to convey the point, not to bombard with information.

• In case of conflicting information rely on data from peer- reviewed journals.

• Tell a compelling, empathetic story, not a mere compilation of data

• In the Indian context TRIANGULATION is a CRITICAL SKILL.


Learning Objectives

• Why analyze treatment options?

• What to consider while analyzing treatment options?

• How to analyze treatment options?

Treatment options

2

Learning Objectives


analyze treatment options?

Why

1. To characterize the GAP 2. To define meaningful specifications for the desired solution 3. To understand what may work and what may not 4. To predict patient response to proposed solution

Disease state

Treatment options


Analyzing treatment options..

1. Modern medicine provides treatment options for most of the common diseases

2. These treatment options have some desirable characteristics which often come at the price of certain non-desirable characteristics.

Desirable Characteristics Non-desirable Characteristics

Efficacy Cost

Safety Invasiveness

Painlessness Risk

Speed of action Need for skill

Lasting effect Need for infrastructure

Accessibility Poor availability

Table: Characteristics of treatment options


Analyzing characteristics

Characteristics can be plotted on a graph Often a non-desirable characteristic increases along with a desirable one We seek to find the optima on this plot


Efficacy Cost

Safety Invasiveness

Painlessness Risk




Table: Characteristics of treatment options Non-desirable characteristic

Des

irab

le c

har

acte

rist

ic

Content shared with grateful acknowledgment to Stanford India Biodesign: From chapter contributed by the author


Analyzing characteristics : Cost vs Efficacy

Example


Efficacy Cost

Safety Invasiveness

Painlessness Risk




Fig: Plotting the characteristics of treatment options

Cost

Effi

cacy

High efficacy High Cost

Goal

High efficacy Low cost

Low efficacy Low Cost

Solution needs to have Efficacy better than this

Solution needs to Cost less than this GAP GAP


Analyzing characteristics: Efficacy vs risk


Efficacy Cost

Safety Invasiveness

Painlessness Risk





Invasiveness or Risk

Effi

cacy

High efficacy

High Risk

Low efficacy Low Risk

GAP


Analyzing Characteristics: Efficacy vs skill required


Efficacy Cost

Safety Invasiveness

Painlessness Risk





Skill required

Effi

cacy

High efficacy

High Skill

Low efficacy Low skill

GAP


Analyzing characteristics : Safety vs cost

Example


Efficacy Cost

Safety Invasiveness

Painlessness Risk





Cost

Safe

ty

High safety High Cost

Goal

Poor safety Low Cost

GAP


Factors to consider while analyzing Rx options

Efficacy (therapeutic effect)

• Ability of the treatment option to treat/alleviate/ cure the disease • Most important factor • Measurement of efficacy

• Efficacy is measured through clinical trials • Randomized, controlled, triple blinded prospective trial or

Randomized controlled trial (RCT) is the gold standard • Other types of trials are Double blinded RCT, case control studies , • For an understanding of what an RCT is refer to this video http://www.youtube.com/watch?v=752V173e31o (24-29 minutes)

http://www.youtube.com/watch?v=752V173e31o

http://www.youtube.com/watch?v=752V173e31o


A QUICK DEFINITION OF “TRUTH”


Efficacy may be measured in different ways for different diseases and at times in different ways for the same disease as well Measuring mortality

• Increased life expectancy • Decreased risk of dying due to concerned disease • For diseases like cancer, survival for 5 years following treatment is considered

as a surrogate for cure

Measuring morbidity

• Risk of recurrence • Quality of life • DALYs lost etc • Efficacy in controlling specific symptoms like pain, bleeding, urinary frequency

The claim of a treatment being efficacious must be analyzed to find WHICH ASPECT is it efficacious in?

Efficacy Measurement: Chronic diseases


Measuring mortality • Case fatality rate • Death during hospital stay • Death within 1 month of discharge

Measuring morbidity • Incidence of sickness absenteeism • Incidence of hospital admission • Average length of hospital stay • Need for intensive care • Time to regain full health and functionality • Measurement of specific symptom scores through questionnaires

Efficacy Measurement : acute diseases



Cost of treatment • Indian perspective

• At times the most important factor driving choice treatment

• Even affluent patients are extremely cost conscious regarding healthcare expenses

• Nearly all healthcare expense comes out-of-the patient’s pocket

• The patient makes important therapeutic decisions, based on cost, hence there is greater pressure to make perceivable difference to the patient to justify cost.

(Unlike insurance driven systems where physicians make therapeutic choices)

• Low awareness level and cost consciousness bias patients towards low cost treatments – despite their low efficacy. This is a challenge.

Med-tech innovation in the Indian environment can be challenging



Invasiveness • Invasiveness: Medical< Endoscopic < Surgical • For the same efficacy, a less invasive treatment is always preferred by doctors

and patients alike

Invasiveness or Risk involved in treatments for Prostate enlargement (BPH)

Effi

cacy

TURP High efficacy Less invasive

Lesser risk

Goal

DRUGS Low efficacy

Low Risk

GAP

SURGERY High efficacy

High Risk Very highly

invasive

Neotract, a novel device


Risk profile, side-effects • Risk of side-effects in a current option may form the need for a novel

treatment. In the Prostate enlargement example, a 5% risk of permanent impotency necessitated the search for lesser invasive methods

• Side effects of drug therapy often lead to poor compliance with highly efficacious therapies

• Examples: • Side effects of low-cost iron tablets are a challenge in anemia therapy • Pain and swelling in the forearm – limit the use of continuous non-invasive BP

monitoring, while invasive monitoring has the risk of infection. • Pain and skin dystrophy limit the adoption of subcutaneous Insulin therapy


Efficacy Cost

Safety Invasiveness

Painlessness Risk







Time of action • Time required for relief is an important factor • Time consuming therapies (such as those requiring prolonged

admission) may fall out of favor as the opportunity cost of such procedures may make them unaffordable, particularly for the economically weaker patient population

• Certain therapies (eg. Injectible steroids) –are used rampantly in India because of the rapid symptomatic relief they provide for relatively low cost – despite their positively harmful adverse effects.

• Rapid symptomatic relief is also responsible for widespread abuse of pain-killers.

• Both the above highlight Indian patients’ bias towards ‘QUICK relief’



Home based Nebulization, CPAP, Blood sugar testing Out-patient procedure Banding for hemorrhoids, Fine needle Aspiration for Cytology, Incision and drainage, Pleural fluid tapping Day-Care procedure Arthroscopy, Shock Wave Lithotripsy, Laparoscopic Ligation, Tympanoplasty, Cataract Surgery, Minor breast Surgeries In-patient procedures All major surgeries, Laparoscopic surgeries etc

Setting of intervention: Examples

Incr

easi

ng

infr

astr

uct

ure

req

. an

d c

ost

s.



Setting of intervention

There is a global trend towards downshifting the setting of procedure – with a view to saving infrastructure and manpower cost. Such a shifting of setting is even more important in the context of the Indian healthcare system, which is over-burdened and resource constrained.



Requirement of skill

“There are more doctors of Indian origin, per American in USA, Than there are Indian doctors in India, per Indian” With such acute shortage of skilled manpower, any downshifting of a procedure on the skill spectrum would translate to greater access to the Procedure



Through analysis of treatment options, try to understand: 1. Which are the most common treatments being used? 2. Who is providing them? 3. What profits are they are making? 4. What percentage of their total income does this disease/ treatment

contribute? 5. How will a cheaper/ safer solution affect these stakeholders? 6. There are examples where efforts to empower low-skilled workers to

conduct important procedures have met stiff resitance from influential stakeholders (specialist physicians) seeking to protect “their turf”

Such understanding will later, form the foundation for stakeholder

engagement strategy

Understanding the stakeholders


Flow of care

1. Understanding the flow of care is critical. In an absence of such understanding, meaningful solutions cannot be envisaged 2. Try to find out: - What is the average time before medical advice is sought - Which is the first person/ facility that the patient contacts when symptoms surface - What is the skill and resource level of that facility - What is the next level of care that patient approaches - What investigations are required -What procedures are conducted, who conducts them - What is the average duration of treatment 3. In India, often the first point of contact is often under trained and

under resourced, while the larger facilities are reluctant to try new devices.


Flow of care

Examples: 1. An ultra low cost ultrasound machine for rural areas sounds like a good

idea, until thought is applied to the fact that the machine itself is worthless in the absence of a radiologist to operate it and someone to interpret and act on the results

2. A nationwide medical record storage and retrieval service sounds like a

good idea until one realizes that concerned stakeholders may not have the time nor inclination to use it.

3. A low cost mobile phone based single lead ECG machine alone may not create a great impact until thought is applied to the following: - What conditions can a single lead ECG help diagnose? - What are the symptoms of those conditions? - Are those symptoms usually enough to drive a patient to a hospital? - Can any of these diseases be treated over the phone?


How to conduct treatment option analysis?

1. Understanding of the disease state 2. If possible, a quick chat with a concerned physician may be a good

starting point 3. Material from sources like Medscape.com may provide further search

terms and links 4. Guidelines published by relevant professional and public health bodies

are exceedingly helpful. - There is one for most disease states. - These are based on international consensus and robust evidence. - A guideline document can form the backbone of a treatment option

study - A guideline doc is also a treasure trove of relevant references Examples : GOLD for COPD, IDSA for Hosp. acquired infections, AHA for

resuscitation, FIGO guidelines for Obgyn issues and so on

A: Secondary Research

5. Search further on pubmed.net, Google Scholar and other sources of literature. Market research reports may be helpful too.


How to conduct treatment options analysis?

B: Primary Research

1. OBSERVE patients undergoing treatment. - See what they are DOING - Talk to them to find out what they SAY, - Figure how they FEEL and what they NEED 2. Caregiver interviews 2 Detailed Physician interviews. Don’t hesitate to email known

authorities in the field. You’ll be surprised at how often they respond. 3. Try to be non-judgmental. Refrain from confronting people with

scientific facts when you can guess they may not be aware of them. The idea is to glean information.

4. Collect rate-lists from hospitals and labs


Note about health behaviors in India

1. Not all health related behaviors are driven by considerations of cost, efficacy, safety and comfort.

2. Superstitious and whimsical beliefs are almost universal Examples: a. Male sterilization (vasectomy) is much less invasive, less expensive, and safer than Female sterilization (tubectomy). Yet, majority of couples opt for tubectomy – the males do not accept the

procedure – despite the fact that it has no effect on sexual function. b. Blood donation is perfectly safe, yet voluntarily donated blood is perpetually in short supply – often Leading to maternal deaths c. Inhaled medication for Bronchial Asthma is a thousand times safer than oral medication, yet there is a widespread belief that inhalers are “strong” medicines leading to a “life long addiction”. Patients often prefer to suffer from Asthma than to use inhalers 3. Health often does not form a spending priority, particularly for the female members of the family


Other facts to keep in mind

4. Health-care providers with no formal medical education (colloquially known as RMPs) provide a sizeable fraction of healthcare. No official data estimates or acknowledges their impact. They are

all pervasive – from big cities to small towns. Their primary focus is to maximize profits and are perhaps unlikely to invest in a new technology unless it translates directly to increased profits.

5. There is a common trend of consulting a “family doctor” (who could be an RMP, General physician

or any specialist) as a first point of contact. They then refer the patient to a doctor or facility of their choice

6. Auditing of prescriptions is very rare, and physician prescription habits may be out of sync with

current evidence (in smaller cities). Doctors in larger cities often keep abreast with current science.

7. A variety of factors (medical and non-medical ) decide what all investigations will be ordered.

8. A large, fragmented pvt. Sector delivers 80% of healthcare( 20% share belongs to a public sector).

9. The Laboratory medicine sector (estimated 50,000 labs ) is also disorganized with majority share belonging to small labs in the disorganized sector


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