MinistryofHealthandFamilyWelfareGovernmentofIndia

NATIONALPOLICYFORTREATMENTOF

RAREDISEASES

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TableofContents

Executivesummary3-7

1 Introduction–Burdenanddefinitionsofrarediseases8-9

1.1 Whatarerarediseases?

1.2 Globalscenario

1.3 Indianscenario

2. Rarediseasesasapublichealthissue10-15

2.1 Lackofepidemiologicaldata

2.2 Varyingdefinitionsandprevalencethresholds

2.3 Diagnosisofrarediseases

2.4 Researchanddevelopment

2.5 UnavailabilityandHighCostoftreatment

2.6 Balancingcompetingprioritiesofpublichealth

3. PolicyDevelopment16-22

3.1 Governmentappointedcommittees

3.1.1 Prof.V.K.PaulCommitteeReport

3.1.1 Prof.I.C.VermaSub-CommitteeReport

3.1.3 Dr.DeepakTempeCommitteeReport

4. PolicyDirection 23-31

4.1 ImplementationMechanism

4.2 ImplementationStrategy

4.3 RoleofMinistries/DepartmentforInter-sectoraland

convergentactiontotacklerarediseases

4.4 ImplementationFramework

4.5 ConstitutionofCommittees

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EXECUTIVESUMMARY

RareDiseases

A rare disease is a health condition of lowprevalence that affects a small number of

people compared with other prevalent diseases in the general population. It is

estimatedthatgloballyaround6000to8000rarediseasesexistwithnewrarediseases

being reported in the medical literature regularly. However, 80% of all rare disease

patientsareaffectedbyapproximately350rarediseases.

Paradoxically, though rare diseases are of low prevalence and individually rare,

collectively they affect a considerable proportion of the population in any country,

whichaccordingtogenerallyacceptedinternationalresearchis–between6%and8%.

Rare diseases include genetic diseases, rare cancers, infectious tropical diseases and

degenerative diseases. 80% of rare diseases are genetic in origin and hence

disproportionatelyimpactchildren.

Thereisnouniversallyaccepteddefinitionofararedisease.Differentcountriesdefine

rare diseases differently. However, the common considerations in the definitions are

primarily, disease prevalence and to varying extent - severity and existence of

alternativetherapeuticoptions.Indiamustarriveatitsowndefinitionsuitedtoitsneed,

basedonacarefulconsiderationofprevalence,diseaseseverityandstudy-ability.

RareDiseasesasapublichealthissueinIndia

Thefieldofrarediseasesiscomplex,heterogeneous,continuouslyevolvingandsuffers

fromadeficitofmedicalandscientificknowledge.Sofarabout450rarediseaseshave

been recorded in India. Globally as well as in India, rare diseases pose a significant

challengetopublichealthsystems in termsof–difficulty incollectingepidemiological

data, which in turn impedes arriving at burden of diseases and cost estimations,

difficulty in research and development,making correct and timely diagnosis, complex

tertiarylevelmanagementinvolvinglongtermcareandrehabilitationandunavailability

andprohibitivecostoftreatment.

Rarediseasesconstituteasignificanteconomicburdenindependentofacountry’ssize

anddemographics,arisingfromincreasedhealthcarespending.Asresourcesarelimited,

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there is amacroeconomicallocationdilemmadue toopportunity costof funding rare

diseasetreatment:ononehand,healthproblemsofamuchlargernumberofpersons

canbeaddressedbyallocatingarelativelysmalleramount,ontheother,muchgreater

resourceswillberequiredforaddressinghealthproblemsofarelativelysmallernumber

ofpersons.

NeedforaPolicy

Rare diseases are, in most cases, serious, chronic, debilitating and life threatening

illnesses, often requiring long-term and specialised treatments/management. In

addition, they often result in some form of handicap, sometimes extremely severe.

Moreover, they disproportionately impact children: 50% of new cases are in children

andareresponsiblefor35%ofdeathsbeforetheageof1year,10%betweentheages

of1and5yearsand12%between5and15years.

The impacton families isoftencatastrophic in termsofemotionalaswell as financial

drain, as the cost of treatment is prohibitively high. As a result, parents of children

sufferingfromrarediseases,whosetreatmentcostwerenotbeingcoveredbyinsurance

orotherwisenotbeingreimbursed,filedwritpetitionsintheDelhiHighCourt,seeking

directionsthatthegovernmentprovidethetreatmentforfree.TheHighCourtofDelhi

in W.P. (C) No. 4444/2016, W.P. (C) No. 7730/2016, and W.P. (C) No. 7729/2013,

directed the Ministry of Health & Family Welfare to frame a “national policy on

treatmentofrarediseases’.

The National Health Policy 2017 also underscores the need for management of

rare/orphan diseases. For the above reasons, a policy is necessary to devise a

multiprongedandmultisectoralapproachtobuildIndia’scapacitytotacklerarediseases

comprehensively,inareasof–epidemiologicaldataforestimatingburden,arrivingata

definition and for cost estimation of treatment; research and development for

treatment and diagnostic modalities, including through international/regional

collaborations; training of health care providers; awareness generation; creating

conduciveenvironment fordrugdevelopmentandmeasures forensuringaffordability

oftreatmentetc.

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PolicyDirection

The GOI appointed high level committees to make recommendations towards

formulation of ‘Policy on treatment of Rare Diseases’. The committeesmade several

recommendations,whichhavebeenincorporatedinthisPolicy.ThePolicyhighlightsthe

measuresandsteps,bothintheshortaswellasinthelongterm,thatneedtobetaken

todealcomprehensivelywithrarediseases.However,recognizingtheexorbitantcostof

treatment for rare diseases, the policy seeks to strike a balance between access to

treatmentwithhealthsystemsustainability.

A. ImmediateMeasures

• ConstitutinganInter-ministerialConsultativeCommitteetocoordinateandsteer

theinitiativesofdifferentministriesanddepartmentsonrarediseasesaslaidout

inthisPolicy

• Constituting a Technical cum Administrative Committee at Central as well as

State levels, for management of corpus funds and developing technical

guideline/criteria for - which rare diseases to fund, towhat extent, reviewof

treatmentetc.

• CreatingacorpusfundatCentralandStateLevelforpartfundingtreatmentof

rarediseaseson thebasisof technicalcriteriadevelopedby theTechnicalcum

AdministrativeCommittee

• Creating aWeb-based application for online application process to access the

corpusfunds

• CreatingapatientregistryforrarediseaseshousedinICMR

• ArrivingatadefinitionofrarediseasessuitedtoIndia

• Developing materials for generating awareness in the general public, patients

andtheirfamiliesandhealthcareproviders.

• Developingandconductingtrainingprogrammesofhealthcareprovidersonrare

diseases

• Constituting aRareDiseasesCellwithinMoHFW, ICMRandDoP inMinistry of

ChemicalsandFertilizersrespectivelytobethe‘nodal’onrarediseasesintheir

respectiveministriesanddepartments

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B. Longtermmeasures:Thebelowmentionedmeasuresareofacontinuingnature

thatoughttobeinitiatednowwithdeliberate,concretestepstowardstheirscale

upandprogressiverealization-

• Putsystemsinplaceforreportinganddatacollection

• Conductepidemiologicalstudiestoestimateprevalenceofrarediseases

• Takemeasurestoimproveresearchanddevelopmentfortreatment,diagnostic

modalities, care and support including assistive devices, drug development for

rarediseasesetc.

• Takemeasures, legislativeorotherwise,forencouraginglocalmanufacturingof

drugsforrarediseases

• Takelegalandothermeasurestocontrolthepricesofdrugsforrarediseasesto

ensureitsaffordabilityandhealthsystemsustainability

• Encourage funding support from Public Sector Undertakings (PSUs) and

corporate sector and exploring other options for sustainable funding for the

corpus

• Ensureinsurancecoverageforraregeneticdisorders

• AllowimportofEnzymeReplacementTherapies(ERTs)andremoveimportduty

onthemaswellasonassistivedevices

• Asastrategyforearlydiagnosisofrarediseases,exploreformulatingaplanfor

piloting,androllingouttestingforraregeneticdiseasesinnewborns,intandem

with development and standardization of diagnosticmodalities and availability

oftreatment

• Develop standardized protocols for diagnosis and treatment/management of

rarediseases,toberevisedinconformitywithevolvingdiagnosisandtreatment

landscape

• Strengthenlaboratorynetworksfordiagnosisofrarediseases

• Accredit centres for diagnosis and treatment of rare diseases which could be

developed as Centres of Excellence (CoE) over a period of time, in a phased

manner

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• As a preventive strategy, explore feasibility of a plan for providing and

progressively scaling up pre-conception and antenatal genetic counseling and

screening in a targetedmanner, or otherwise, to provide option to parents to

preventconceptionorbirthofachildwithararegeneticdiseases

• DrugControllerGeneralofIndia(DCGI)toconsiderfeasibilityofamendingDrugs

andCosmeticsActorotherwisetakingmeasuresunderit,toincludeappropriate

provisions on drugs for rare diseases, including provisions to facilitate clinical

trialsandimportofERTs.

Amulti-sectoralconvergentapproachtotacklingrarediseases

ThePolicydelineatestheroleofseveralministriesinachievingthemeasuresenvisaged.

EachMinistryandconcerneddepartment(s) is requiredtodevelopan implementation

frameworkonmeasurestobetakenbythemontheirsectorwiseresponsetotackling

rarediseases.

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1. Introduction

1.1 WhatareRareDiseases?

Ararediseaseisahealthconditionofaparticularlylowprevalencethataffectsasmall

numberof people comparedwithotherprevalentdiseases in the general population.

There isnouniversallyaccepteddefinitionof rarediseasesand thedefinitionsusually

varyacrossdifferentcountries.However,thecommonconsiderationsinthedefinitions

are primarily, disease prevalence and to varying extent - severity and existence of

alternativetherapeuticoptions(1).

It is estimated that globally around 6000 to 8000 rare diseases exist with new rare

diseasesbeingreportedinthemedicalliteratureregularly(2,3,4,5).However,80%ofall

rarediseasepatientsareaffectedbyapproximately350rarediseases(6).

Paradoxically, though rare diseases are of low prevalence and individually rare,

collectively they affect a considerable proportion of the population in any country,

whichaccordingtogenerallyaccepted international research is–between6%and8%

(7,8).Rarediseasesincludegeneticdiseases,rarecancers,infectioustropicdiseasesand

degenerative diseases (9). 80% of rare diseases are genetic in origin, and thus are

presentthroughoutaperson’slife,evenifsymptomsdonotimmediatelyappear.

1.2 Definitionsofrarediseasesacrossjurisdictions

WHO defines rare disease as often debilitating lifelong disease or disorder condition

withaprevalenceof1orless,per1000population.However,differentcountrieshave

their own definitions to suit their specific requirements and in context of their own

population,healthcaresystemandresources.IntheUS,rarediseasesaredefinedasa

disease or condition that affects fewer than 200,000 patients in the country (6.4 in

10,000people).EUdefinesrarediseasesasalife-threateningorchronicallydebilitating

conditionaffectingnomore than5 in10,000people. Japan identifies rarediseasesas

diseaseswithfewerthan50,000prevalentcases(0.4%)inthecountry.

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Table1:DefinitionsofRareDiseaseindifferentcountries

SNo. Country Per10,000population

1 USA 6.4

2 Europe 5.0

3 Canada 5.0

3 Japan 4.0

4 SouthKorea 4.0

5 Australia 1.0

6 Taiwan 1.0

Source: The I.C. Verma Sub-Committee Report ‘Guidelines for Therapy and

Management’

1.3 TheIndianScenario

India,likemanyotherdevelopingcountries,currentlyhasnostandarddefinitionofrare

diseasesanddataonprevalence. Since there isnoepidemiologicaldata, thereareno

figuresonburdenofrarediseasesandmorbidityandmortalityassociatedwiththem.

Ifweapplytheinternationalestimateof6%to8%ofpopulationbeingaffectedbyrare

diseases,toIndia,wehavebetween72to96millionpeopleaffectedbyrarediseasesin

thecountry,which isasignificantnumber.However, this isatbestageneralestimate

(10) and Indiawill need to arrive at its own estimate and definition of rare diseases,

derivedchieflyfromprevalencedata,whichiscurrentlylacking.

So far only about 450 rare diseases have been recorded in India from tertiary care

hospitals (10). The most common rare diseases include Haemophilia, Thalassemia,

Sickle-cellAnaemiaandPrimaryImmunoDeficiencyinchildren,auto-immunediseases,

Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s

disease,CysticFibrosis,Hemangiomasandcertainformsofmusculardystrophies.

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2. RareDiseasesasapublichealthissue

The fieldof rarediseases is complex andheterogeneous and suffers fromadeficit of

medicalandscientificknowledge.Thelandscapeofrarediseasesisconstantlyevolving

astherearenewrarediseasesandconditionsbeingidentifiedandreportedregularlyin

medicalliterature.Apartfromafewrarediseases,wheresignificantprogresshasbeen

made,thefieldisstillatanascentstage.Foralongtime,doctors,researchersandpolicy

makerswereunawareofrarediseasesanduntilveryrecentlytherewasnorealresearch

or public health policy concerning issues related to the field. This poses formidable

challengesindevelopmentofacomprehensivepolicyonrarediseases.Nevertheless,it

is important to take steps, in the short as well as long term, with the objective of

tacklingrarediseasesinaholisticandcomprehensivemanner.

2.1 Lackofepidemiologicaldata

Data on howmany people suffer from different rare diseases in India is lacking. The

cases identified so far have been diagnosed at tertiary hospitals (11). The lack of

epidemiological data on incidence and prevalence of rare diseases impedes

understanding of the extent of the burden of rare diseases and development of a

definition. It also hampers efforts to arrive at correct estimation of the number of

persons suffering from these diseases and describe their associated morbidity and

mortality. In such a scenario, the economic burdenofmost rarediseases is unknown

andcannotbeadequatelyestimatedfromtheexistingdatasets(12).

Althoughextremelychallenging,consideringthecomplexityofvariousdiseasesandthe

difficulty in diagnosis, there is a clear need to undertake systematic epidemiological

studiestoascertainthenumberofpeoplesufferingfromrarediseasesinIndia.

2.2 Varyingdefinitionsandprevalencethresholds

Theuseofinconsistentdefinitionsanddiverseterminologycanresultinconfusionand

inconsistencies and have implications for access to treatment and for research and

development(1).Accordingtoastudy,whichreviewedandanalyseddefinitionsacross

jurisdictions, most definitions, as discussed above, appear to consider disease

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prevalence,butothercriteriaalsoapplysometimes,suchas-diseaseseverity,whether

the disease is life-threatening, whether there are alternative treatment options

available,andwhetheritisheritable(1).Thestudyfoundthatrelativelyfewdefinitions

(30%) included qualifiers relating to disease severity and/or a lack of existing

treatments, whereas most definitions (58%) included a prevalence threshold. The

average prevalence thresholds used to define rare diseases ranged among different

jurisdictions from 5 to 76 cases/100,000 people, with a global average prevalence

threshold of 40 cases/ 100,000 people. The study concluded that attempts at

harmonising the differing definitions, should focus on standardizing objective criteria

such as prevalence thresholds and avoid qualitative descriptors like severity of the

disease.

However, it has been contested that disease prevalence alone may also not be an

accurate basis for defining rare diseases, as it does not take into account changes in

population over time. Hence, some have suggested that amore reliable approach to

arrivingatadefinitioncouldbebasedonthefactorsof–a)location-adiseasewhichis

uncommoninonecountrymaybequitecommoninotherpartsoftheworld;b)levelsof

rarity - some diseases may be much more rare than other diseases which are also

uncommon; and c) study-ability - whether the prevalence of a disease lends itself to

clinicaltrialsandstudies(13).

Thisunderscorestheneedfor furtherresearchtobetterunderstandtheextentofthe

existingdiversityofdefinitionsforrarediseasesandtoexaminethescopeofarrivingat

adefinitionwhichisbestsuitedtoconditionsinIndia.Itshallbedoneonaprioritybasis

assoonassufficientdataisavailable.

2.3 Diagnosisofrarediseases

Diagnosis of a rare condition may take upto several years, owing to difficulty in

diagnosticmodalitiesandlackofawarenessamongdoctors.Formanyrarediseases,no

diagnosticmethodexists,ordiagnosticfacilitiesareunavailable(14).Traditionalgenetic

testing can only address a few genes at a time. As a result, physicians must often

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provide theirbest guessonwhichgenes to investigate. If the test isnegative, further

testingwillberequired,whichisanexpensiveandtime-consumingprocess.

There is a lack of awareness about rare diseases in general public as well as in the

medicalprofession.Manydoctorslackappropriatetrainingandawarenesstobeableto

correctly and timely diagnose and treat these conditions (15). According to a recent

report, it takes patients inUnited States (US) an averageof 7.6 years andpatients in

UnitedKingdom(UK)anaverageof5.6yearstoreceiveanaccuratediagnosis,typically

involving as many as eight physicians (four primary care and four specialists). In

addition,twotothreemisdiagnosesaretypicalbeforearrivingatafinaldiagnosis(16).

Delay in diagnosis or a wrong diagnosis increases the suffering of the patients

exponentially.There isan immediateneedtocreateawarenessamonggeneralpublic,

patients and their families and doctors, training of doctors for better diagnosis,

standardisation of diagnostic modalities, developing of new diagnostic tools and

investmentingenetherapy.

2.4 Challengesinresearchanddevelopment

Afundamentalchallengeinresearchanddevelopmentforthemajorityofrarediseases

isthatthereisrelativelylittleknownaboutthepathophysiologyorthenaturalhistoryof

thesediseases.Rarediseasesaredifficult to researchuponas thepatientpool isvery

small and it often results in inadequate clinical experience. Therefore, the clinical

explanation of rare diseasesmay be skewed or partial. The challenge becomes even

greaterasrarediseasesarechronicinnature,wherelongtermfollowupisparticularly

important. As a result, rare diseases lack published data on long-term treatment

outcomesandareoftenincompletelycharacterised(17).

This makes it necessary to explore international and regional collaborations for

research, collaborations with the physicians who work on any rare disease and with

patientgroupsandfamiliesdealingwiththeconsequencesofthesedisorders.Thiswill

help gain a better understanding of the pathophysiology of these diseases, and the

therapeuticeffectsthatwouldhaveameaningfulimpactonthelivesofpatients.There

isalsoaneedtoreviewandwherepossiblemodify,clinicaltrialnormskeepinginmind

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the particular challenges in rare diseases, without compromising on the safety and

qualityofthedrugsordiagnostictools.

2.5 Challengesintreatment

2.5.1 Unavailabilityoftreatment

Availability and access tomedicines are important to reducemorbidity andmortality

associated with rare diseases. Despite progress in recent years, effective or safe

treatment is not available formost of the rare diseases. Hence, evenwhen a correct

diagnosisismade,theremaynotbeanavailabletherapytotreattheraredisease.There

arebetween7000 - 8000 rarediseases, but less than300have therapies available to

treatthem(18).About95%rarediseaseshavenoapprovedtreatmentandlessthan1in

10patientsreceivesdiseasespecifictreatment(19).Wheredrugsareavailable,theyare

prohibitivelyexpensive,placingimmensestrainonresourcesoffamilies,healthsystems

anddonoragenciesalike.

2.5.2 Prohibitivecostoftreatment

Asthenumberofpersonssuffering from individual rarediseases is small, theydonot

constituteasignificantmarketfordrugmanufacturerstodevelopandbringtomarket

drugsforthem.Forthisreason,rarediseasesarealsocalled‘orphandiseases’anddrugs

to treat them are called “orphan drugs”. Where, they do make drugs to treat rare

diseases,theysellthematextremelyhighcosts,statedly,torecoupthecostofresearch

and development. At present very few pharmaceutical companies aremanufacturing

drugsforrarediseasesgloballyandtherearenodomesticmanufacturersinIndia.Due

tothehighcost,thegovernmenthasnotbeenabletoprovidethesedrugsforfree.Itis

estimated that forachildweighing10kg, theannualcostof treatment for somerare

diseases, may vary from Rupees 18 Lakhs to 1 crore 70 lakhs (discussed in detail in

Chapter3).Further,themethodologyforevaluatingtreatmentofrarediseasesisoften

still in experimental phase, impeding assessment of clinical relevance and cost

effectiveness.

Several countries have through legislation like Orphan Drug Act (ODA), provided

incentives to drug manufactures to encourage them to manufacture drugs for rare

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diseases. Themost powerful incentive for drugmanufacturers is the grant of 7 to 10

years of exclusive marketing rights including protection from imports, if a drug gets

‘orphandrug’ status.Thismeans thatpharmaceutical companiescanprice theirdrugs

without fear of competition. This has changed the nature of drug development. The

numberofrequestsfororphandrugdesignationhasquadrupledsince2000.Theresult

has been a boom in drug sales and profits. In fact, almost a third of drugs for rare

diseasesnowexceed£1bninannualsales.Theglobalorphandrugsmarketisexpected

toreach£144bnby2020,andaccountfor19%oftotalbrandedprescriptiondrugsales

(20).

The average price of a drug for rare diseases exceeds $100,000 a year. According to

reports,while legislationsonorphandrugdevelopment, like theUSOrphanDrugAct,

have facilitated development of orphan drugs, they have not been able to check the

pricesofthesedrugs,whichhasledtograveconcernsforhealthsystemsustainability.

Forinstance,thecysticfibrosisdrug,Kalydeco(ivacaftor)ispricedat£14,000perpatient

permonth.Infact,eachoneoftheworld's10mostexpensivedrugs,isadrugtotreat

someraredisease,withSoliris(eculizumab)beingthemostexpensiveat£340,000per

patientperyear.Althoughthesedrugsareprescribedtofewerpatients,theirextremely

highpricescanresult inrevenuesequivalenttotraditionalblockbusterdrugs. Publicly

listedpharmaceuticalcompaniesthatareorphandrugmarketauthorisationholdersare

associatedwithhighermarketvalueandgreaterprofitsthancompaniesnotproducing

drugsforrarediseases(21)

Overtheyearsmanydrugshavewon‘orphandrugs’statuseveniftheyarenotnewor

represent a scientific breakthrough. An inexpensive off-patent drug approved by the

FDA for one condition, but widely prescribed as an “off-label” treatment for a rare

disease,canbetransformedintoabigmoneymaker.Theaccompanying7to10yearsof

monopolystatuscanmeanlargepricehikesforadrugthatwasalreadyinwideuse(22,

23, 24). For instance, older medicines for rare diseases like imatinib used to treat

chronicmyloidluekemiacancostoverUSD100,000ayearindevelopedcountries(25).

In India, public health safeguards in thepatent law that restrict evergreeningpatents

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haveallowedIndiangenericmanufacturerstomanufactureandsupplythisdrugatfar

lowerprices.

The exorbitant prices of drugs for rare diseases has led to concerns even in the

developed countries about maintaining sustainability of the rare diseases

funding/reimbursementprogrammes(26,27).Theexorbitantpriceshaveledtocallsfor

transparencyinsettingpricesofdrugsandforpricecontrol(28,29,30)andhaveeven

prompted scrutiny and congressional inquiries (31, 32). This should be a matter of

careful consideration for any legislative or policy measures adopted in India for

promotionof drugdevelopment for rarediseases. There is a pressingneed formulti-

pronged strategies tomakedrugsmoreaffordable–by takingmeasures to lower the

prices of drugs, encourage generic and local manufacturing of drugs and encourage

PSUstomanufacturedrugsforrarediseases.

2.6 Needtobalancecompetingprioritiesofpublichealth inresourceconstrained

settings

Rarediseasesconstituteamajoreconomicburdenindependentofacountry’ssizeand

demographics; these costs arise from increased healthcare spending and lost

productivity (33, 34, 35). The exorbitant prices of medicines, are important

considerationsinpublichealthpolicydevelopmentwithreferencetotreatmentforrare

diseases.Inresourceconstrainedsettings,itispertinenttobalancecompetinginterests

ofpublichealthforachievingoptimaloutcomefortheresourcesallocated.Asresources

are limited, there is a macroeconomic allocation dilemma: on one hand, health

problemsofamuchlargernumberofpersonscanbeaddressedbyallocatingarelatively

smaller amount, on the other, for funding treatment of rare diseases, much greater

resourceswillberequiredforaddressinghealthproblemsofarelativelysmallernumber

of persons. This raises questions of fairness and reasonability. However, it also raises

issues of ethics and equity of opportunity for patients to benefit, in the interest of

patients suffering from rare diseases.Hence anypolicy on treatment of rare diseases

will have to strike a balance between access to treatments with health system

sustainability.

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3. DevelopmentofPolicyforTreatmentofRareDiseases

Rarediseasesare,inmostcases,serious,chronic,debilitatingandlifethreatening,often

requiringlongandspecialisedtreatments.Inaddition,theyoftenresultinsomeformof

handicap, sometimes extremely severe (36). Rare diseases place a huge physical,

psychological,andsocioeconomicburdenonpatientsandtheirfamilies.

At least 80% of rare diseases have an identified genetic origin (37) and hence

disproportionately impact children. 50% of new cases are in children and are

responsiblefor35%ofdeathsbeforetheageof1year,10%betweentheagesof1and5

yearsand12%between5and15years(38).

Rarediseasespresentamajorchallengetopopulationhealthand,untreated,arelikely

to contribute to large social and economic losses, affectingmore than the individual,

sinceoftenfamilymembersmustforegoemploymentoutsidethehomeinordertocare

fortheirsickrelatives. Incontrast,patientsontreatment,areunlikelytorequireother

high cost procedures such as pain management and surgery (39). Further, it is

impossible for most families to fund treatment of rare diseases, without any

governmentalsupport.

The impacton families isoftencatastrophic in termsofemotionalaswell as financial

drain, as the cost of treatment is prohibitively high. This has compelled parents of

childrensufferingfromrarediseases,whosetreatmentcostwerenotbeingcoveredby

insurance or otherwise not being reimbursed, in approaching the courts seeking

directionsthatthegovernmentprovidethedrugsforfree,sothatthetreatmentcould

continue.TheHighCourtofDelhi inW.P. (C)No.4444/2016,W.P. (C)No.7730/2016,

andW.P. (C)No.7729/2013,haddirected theMinistryofHealth&FamilyWelfare to

framea“nationalpolicyontreatmentofrarediseases’.

3.1 Committees appointed by the government to make recommendations for

framingapolicyonrarediseases

PursuanttotheordersoftheHon’bleCourt,theGOIhadconstitutedcommitteeswith

theobjectivetomakesuggestionstowardsframingofa‘nationalpolicyontreatmentof

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rare diseases’. Similarly, the Government of NCT of Delhi had also appointed a high

powered interdisciplinary committee on rare diseases. The various committees that

wereappointedwere:

• Committee under Professor V.K. Paul, Head, Department of Pediatrics,

AIIMS,NewDelhi–‘PrioritisationofTherapyforRareGeneticDisorders’

• Sub-committee on rare diseases in India, under Prof. I.C. Verma, Director,

Institute of Medical Genetics Genomics, Sir Ganga Ram Hospital –

‘GuidelinesforTherapyandManagement’

• A high powered interdisciplinary Committee on rare diseases under the

Chairpersonship of Dr. Deepak K. Tempe, Dean, Maulana Azad Medical

College(MAMC),NewDelhi

3.1.1 TheV.K.PaulCommitteeReport

in their Report ‘prioritisaiton of therapy for rare genetic disorders’, the Committee

attemptstoelaborateontheavailabletherapiesandprioritisaitonforgeneticdisorders

basedonresources,costoftherapy(onetimevs.longterm),outcome(evidence-based),

qualityoflifeandpublishedguidelines.

Table2:TheCommitteecategorisedgeneticdisordersintothreecategoriesandmade

recommendationsforeachcategory

SNo. categories Recommendations

1 Disorders amenable to

one time treatment

(curative)

Prioritisefundingforthiscategoryas:

• One-timetreatmentcostrangesfrom5–20lacs,

which is much less compared to long term

therapy

• Treatmentoutcomeisgood

• Facilities for treatment are available in both

privateandpublicsectorwithgoodexpertiseand

outcome

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• Fundingshouldalsoincludesupportforfollowup

therapy

• Ceilingonexisting funding limit shouldbemade

flexible

2 Disorders requiring

long term/life-long

therapy

• Cost of therapy for most disorders in this

categoryisprohibitiveandfamiliescannotafford

itwithoutsomesupport

• Prioritise disorders in this category based on

documented treatment outcome eg. Gaucher’s

diseasetype1

• Develop objective criteria for inclusion and exit

from therapy and for assessing response to and

progressontherapy

• Identify institutes with sufficient manpower,

facilitiesandexpertisetomanagethesediseases

3 Disorders for which no

known therapy is

currently available but

requires supportive

care

• Supportivetherapyistheonlyavailableoption

• Needtoprovidecareandsupportservices

Asabout80%ofrarediseasesaregeneticinnature,theCommitteerecommendedthat

it is important toprovidegenetic counselingandofferprenatal testing to the families

withgeneticdisorders,as itwillprovidethemanoptionaboutgivingbirthtochildren

withrarediseases.

3.1.2 TheI.C.VermaSub-committeeReport

TheSub-committeereviewedtheburdenanddefinitionsofrarediseasesgloballyaswell

as in India, availability of drugs and treatment options for various rare diseases, cost

estimation and evidence on treatment outcomes. It considered the challenges in

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treatment of rare diseases, reviewed the funding mechanisms for treatment of rare

diseasesinseveralcountriesandmaderecommendationsformechanismsofapproving

andregulatingaccessandotherinputsforformulatinganationalplanonrarediseases.

TheSub-committeespecificallyevaluatedtheavailabilityandefficacyoftreatmentand

cost of rare disorders of immediate relevance in India, namely - Lysosomal Storage

Disorders (LSDs), which is treatable with Enzyme Replacement Therapies (ERTs). The

LSDs include – Gaucher Disease, Mucopolysaccharidosis (MPS) Type 1 (Hurler-Schie),

MPSTypeII(Hunter),MPSIva,MPSVI,PompeDiseaseandFabryDisease.

The sub-committee calculated the annual cost of the available therapies for a 10 kg

child.

Table3.ApproximateAnnualCostofERTs

Disease Enzyme Weightofthepatient

Approximateannual cost(INR)*

Gaucher Cerezyme(Genzyme) 10kg 39,84,768

Gaucher Velaglucerase(Shire) 10kg 71,86,340

Gaucher Taliglucerase - No informationavailable

MPS1 Aldurazyme(Genzyme) 10kg 46,78,464

Pompe Myozyme(Genzyme) 10kg 48,94,368

Fabry Fabrazyme(Genzyme) 10kg 18,29,712

MPSII Elaprase(Shire) 10kg 44,00,000

MPSII

(0.5mg/kg/week)

Vial2mg

Hunterase

(GreenCross-Korea)

3mg/6ml(0.5mg/ml)vial

1,43,520/-pervial

10kg 1,72,22,400

MPSVI

(1mg/kgeveryweek)

Naglazyme

(USD1755pervial)

10kg 1,09,51,200

20

Vial5mg

MPSIV

(2mg/kg/everyweek)

Vial5mg

Vimizim

(USD1068/Vial)

10kg 1,33,28,640

*includingcustomsduty,CVD.Taxes,Octroietc.Source:I.C.VermaSub-committeeReport

TheSub-committeesimilarlyevaluatedthetreatment,efficacyandcostofInbornErrors

ofMetabolism(ELMs).

The Sub-committee notes that various countries have used different approaches for

fundingtreatmentforrarediseases.Astreatmentisoutofreachofmostfamilies,many

countriescoverthecostthroughtheirNationalHealthServices,forinstance,mostofthe

European countries. In USA once a therapy is approved by the FDA, the insurance

companies cover the cost. In many emerging economies, the government funds the

treatment of rare diseases, for instance in Egypt, Thailand, Argentina, Chile, Peru,

Serbia,MalaysiaandPhilippines.

Ittherefore,concludesthatthereisaneedforthegovernmenttoplayaroleinevolving

a fundingmechanism for treatment of rare diseases andmade recommendations for

formulationofanationalpolicyfortreatmentofrarediseases.

3.1.3 TheD.K.TempeCommitteeReport

TheCommitteeconsideredtheprohibitivecostsoftreatmentandcompetingpriorities

of public health and resource allocation; inadequate prevalence data; and still

unravelling diagnostic and treatment landscape. It suggested that the national policy

should cover treatment in a phasedmanner, startingwith rare genetic disorders, for

which treatment with good clinical outcome is available. The policy could be

progressivelyrevisedwithincreasedknowledgeandclarityaboutepidemiologicaldata,

diagnosticandtreatmentoptions,evidenceonclinicaloutcomes,reducedcostofdrugs,

etc.

Expanding on the challenge of economic evaluation and resource allocation for rare

diseases,thecommitteegavetheexampleofcostofERTstotreatLSDs(Gaucher,Pompe,

21

Fabryetc.)andstatedthatEnzymeReplacementTherapy (ERTs) is very costly and life-

longandthepublichealthsystemcannotsupportitinacost-effectivewaywithitsown

funds.Theannual recurring costofonepatientwithERTcould range from1.8–17.0

lakhsperkgofbodyweight.Thismeansthatforachildweighing10kgs,thecostwould

bebetween18 lakhsto1crore70 lakhs.This isahugecost inaresourceconstrained

publichealthsystem.Thisamountcouldtreat400TBor400HIVpatientsinayear(40).

Thiscostcouldalsopotentiallytreat10-100patientswithType1diabetes(annualcost

estimatedatRs.18,000/-basedonastudy inSouthIndia in2011)(41),which isalsoa

lifelong and life threatening disease. This cost could also potentially prevent 10,000

malariacasesayearorpreventalmost600underfivechildrenfrombeinghospitalised

withpneumoniaannually.

Thus, the committee cautioned that when resources in the public health system are

limited,appropriatechoicesneedtobemadetaking intoaccountthe largercanvasof

healthproblemsthataffectthepopulationandtheeconomicconsequencesofeachlife

saved.

Inconclusion,acknowledgingtheseverityand impactof rarediseasesonpatientsand

their families; the directions of the Hon’ble High Court of Delhi and the consequent

recommendation of the government sub-committee, there is a need to chalk out a

roadmapforfacilitatingaccesstotreatmentforrarediseases.However,keepinginmind

the prohibitive cost of treatment and the other formidable challenges as discussed

above,appreciatedfromtheperspectiveofpublichealthprincipleofevidenceinformed

resourceallocationforgarneringoptimaloutcomefortheresourcesallocated,makesit

imperativethatthesamebedoneinaphasedmanner.

3.2 PolicyRecommendations

The committees made several recommendations towards formulation of a ‘national

policy for treatment of rare diseases’. The recommendations go beyond treatment

funding and takes a more holistic approach towards rare diseases, encompassing

suggestions towards - prevention, awareness creation, training, research and

developmentintreatmentanddiagnosis,developmentandmanufacturingofdrugsfor

rarediseasesataffordableprices,provisionofinsurancecoverageetc.

22

Therecommendationsfallinthedomainofseveralministriesanddepartments,whichis

recognition of the fact that an effective response to rare diseases will require a

comprehensive and convergent intersectoral effort. The Policy Directions in the next

section reflect on this recognition and delineates the roles for ministries and

departments,inadditiontotheMinistryofHealthandFamilyWelfare.

23

4. PolicyDirection

Inlightoftheseverityandimpactofrarediseasesonpatientsandtheirfamiliesandthe

directionsof theHighCourtofDelhi, thepolicy for facilitatingaccesstotreatment for

rarediseaseshasbeenprepared.Therecommendationsof thegovernmentappointed

committeeshavebeenconsideredwhilemakingthispolicy.

4.1. ImplementationMechanism

ThePolicyhighlights themeasuresand steps thatought tobe taken immediatelyand

alsothosethatcanbeimplementedprogressivelyinphases.Italsohighlightstheroleof

variousministries anddepartments,which at present is indicative and can be further

extendedbasedonadequateevidenceanddatagatheredfromepidemiologicalstudies

andresearch.

ThePolicyenvisagessettingupaConsultativeCommitteeforimplementingthepolicyin

coordination with variousministries and departments. There will also be a Technical

cumAdministrativeCommitteewithinMoHFW,bothattheCentralandStateLevels,for

handling the corpus fund. The ministries, including MoHFW will design their own

roadmapforimplementationoftheactivitiesindicatedbelow.

4.2 Strategiesforimplementation

A.Immediate/shorttermMeasures

• Constitutinganinter-ministerialConsultativeCommitteeatNationalLeveltobe

ledbyMoHFW,toco-ordinate,overseeandsteertheactivitiesandinitiativesof

theconcernedministriesanddepartmentsonrarediseases.

• Constituting a Technical cum Administrative Committee at Central as well as

State levels for management and release of corpus funds and for developing

technical criteria/guidelines for determining ‘which rare diseases to fund’, ‘to

whatextent’,‘reviewofprogress’etc.

• CreatingacorpusfundatCentralandStateLevelfortreatmentofrarediseases

onthebasisoftechnicalcriteriadevelopedbytheTechnicalcumAdministrative

24

Committeeatthecentre

• Creating a Web-based application for online application process to apply for

fundingsupportfromthecorpusfunds

• CreatingapatientregistryforrarediseaseshousedinICMR

• ArrivingatadefinitionofrarediseasessuitedtoIndia

• On the basis of the current knowledge, developing materials for generating

awareness in the general public, patients and their families and for trainingof

health care providers. This ought to be revised with availability of new

informationandknowledge

• ConstitutingaRareDiseasesCellwithinMoHFW,ICMRandDoPintheMinistry

of Chemicals and Fertilizers to be the nodal for the activities related to rare

diseases

B. Longtermmeasures-Thebelowmentionedmeasuresareofacontinuingnature

thatoughttobeinitiatednowwithdeliberate,concretestepstowardstheirscale

upandprogressiverealization,whererequired-

• Puttingsystemsinplaceforreportinganddatacollection

• Conductingepidemiologicalstudytoestimateprevalenceofrarediseases

• Taking measures to improve research and development for drugs, diagnostic

modalitiesandcareandsupportincludingassistivedevices

• Takingmeasures,legislativeorotherwise,tocreateaconduciveenvironmentfor

localmanufacturingofdrugsforrarediseases

• Takelegalandothermeasurestocontrolthepricesofdrugsforrarediseasesto

ensureitsaffordabilityandforhealthsystemsustainability

• Encouraging funding support from Public Sector Undertakings (PSUs) and

corporate sector and exploring other options for sustainable funding for the

corpus

• Ensuringinsurancecoverageforraregeneticdisorders

• Allowing import of ERTs and removing import duty on them as well as on

assistivedevices

• Asastrategyforearlydiagnosisofrarediseases,exploreformulatingaplanfor

25

piloting and depending on feasibility, rolling out progressively, testing for rare

geneticdiseasesinnewborns,intandemwithdevelopmentandstandardization

ofdiagnosticmodalitiesandavailabilityoftreatment

• Develop standardized protocols for diagnosis and treatment/management of

rarediseases,toberevisedinconformitywithevolvingdiagnosisandtreatment

landscape

• Strengthennetworksoflaboratoriesforaccuratediagnosisofrarediseases

• Accredit centres for diagnosis and treatment of rare diseases which could be

developed as Centres of Excellence (CoE) over a period of time, in a phased

manner

• Asapreventive strategy,explore feasibilityofmakingaplan forprovidingand

progressively scaling up pre-conception and antenatal genetic counseling and

screening in a targetedmanner, or otherwise, to provide option to parents to

preventconceptionorbirthofachildwithararegeneticdisease

4.3 Roleofministriesanddepartments

The activities listed below are indicative and can be expanded depending on

improvement in our knowledge and understanding of rare diseases and the type of

response itwill require,basedonavailabilityofdataanevidencegenerated through

researchandstudies.

4.3.1 MinistryofHealthandFamilyWelfare

a) HealthMinistrytocreateacellonrarediseaseswithinitself,tobeheadedbya

JointSecretary.Itwillactasanodalagencyandcoordinatealltheactivitiesof

theHealthMinistryonrarediseases.

b) IndianCouncil ofMedicalResearch (ICMR) to constitute adivisionor identify

oneofitsexistingdivisions,topromoteresearchanddevelopmentinthefield

ofrarediseasesfordiagnosisandtreatmentofrarediseases,includingthrough

international/regional collaborations. An initiative like open source drug

discoveryandresearchforrarediseasesbasedonprinciplesofpublic funding

of research and access to any treatments arising from this to be available

26

‘IntellectualProperty(IP)–free’andopentocompetitivemanufacturingshould

belookedinto.

c) Constitute an an Inter-Ministerial Consultative Committee to co-ordinate the

activitiesofvariousministriesanddepartmentsonrarediseases

d) Constitute an Administrative and Technical Committee at Central as well as

State levels, for management of corpus funds and developing technical

guideline/criteria for - whichrarediseasestofund,towhatextent,reviewof

treatmentetc.

e) Create a patient registry with information to practitioners and a reporting

system of any patient diagnosed with a rare disease. This will be housed in

ICMR.Patientregistriesmayserveasappropriatetoolstoaidinunderstanding

thenatural history and clinical characteristics of rarediseases and assess the

long-termoutcomesoftreatment.

f) Takemeasurestocollectepidemiologicaldataonrarediseases.

g) Takemeasurestocreateawarenessamongmedicalprofessionals,patientsand

theirfamiliesandgeneralpubliconrarediseases.

h) Develop and conduct training programme for doctors on diagnosis and

managementofrarediseases

i) Asastrategyforearlydiagnosisofrarediseases,exploreformulatingaplanfor

pilotinganddependingonfeasibility,rollingoutprogressively,testingforrare

geneticdiseasesinnewborns,intandemwithdevelopmentandstandardization

ofdiagnosticmodalitiesandavailabilityoftreatment

j) Strengthennetworkoflaboratoriestoeffectivelyandaccuratelydiagnoserare

diseases

k) Drug Controller General of India (DCGI) to consider feasibility of amending

Drugs and Cosmetics Act or otherwise taking measures under it, to include

appropriate provisions on drugs for rare diseases, including provisions to

facilitateclinicaltrialsandimportofERTs.

l) Forpatients in theBPL categorywhogetdiagnosedwith rarediseases,make

available for free, supportive services, whether in private or government

hospital.

27

m) ICD 11 classifies about 5000 rare diseases. The centres identified by the

Central/State government for categorising rare diseases in India, need to

group/put rare diseases under already identified disease classification under

ICD11.Ifanynewrarediseaseisidentified,stepswillbetakenbytheMinistry

for sending requiredevidence toWHO for inclusionof thediseaseunder ICD

classification.

n) Asapreventivestrategy,considerfeasibilityofmakingaplanforprovidingand

progressively scalinguppre-conception and antenatal genetic counseling and

screening inatargetedmanner,orotherwise,toprovideoptiontoparentsto

preventconceptionorbirthofachildwithararegeneticdisease

o) CreatingaNationalandStateLevelCorpus

1. The Government of India (GOI) to set up a corpus fund with an initial

amount of Rs. 100 crore towards funding treatment of rare genetic

diseases. Resources allocated for treatment of rare diseases can be

progressively scaled up with regular improvements in availability of

epidemiological data, cost estimation studies and measures taken to

encouragedevelopmentofdrugsandforreductioninpricesofdrugs.

2. The States to have a similar corpus at the state level and the GOI will

contributefundstowardstheStatecorpustotheratioof60:40.Itwouldbe

open to the states to contribute a larger amount to the corpus. This

fundingarrangementwillbepartofthePIPprocess.

3. Thecorpus fundwillbededicated for raredisorders.However, itwillnot

fundtreatmentforblooddisorders(hemophilia,thalassemiaandsicklecell

anemia) as separate government programs for them exist already.

Dependingonneed,theexistingprogrammesforblooddisordersshallbe

scaledup.

4. Thecorpuswillbeusedforonlypartfundingoftheentiretreatmentcost.

5. To ensure sustainability of the corpus, the Public Sector Undertakings

(PSUs)andcorporatehouses, tobeencouraged tomakecontributionsas

per Section 135 and Schedule VII of the Companies Act as well as the

provisionsoftheCompanies(CorporateSocialResponsibilityPolicy)Rules,

28

2014(CSRRules)

p) Creatingaweb-basedapplicationforonlineapplicationprocess

Toensuretimelydecisionsandreleaseoffunds,aweb-basedapplicationwould

bedevelopedforcreatingonlinemechanismforapplyingtothecorpus.Central

governmentwill create thisweb-basedapplicationwithin6-12monthsof the

releaseofthispolicy.Itwillhavethedetailsofthecorpusandinstructionsand

mechanismforapplyingforfunding.Itwouldbeopentoindividualsinstitutions

aswellasstategovernmentstoapplyforfundsbyenteringdetailsontheweb

applicationasperinstructionsprovided.

4.3.2 MinistryofCommerce,DepartmentofIndustrialPolicyandPromotion(DIPP)

a) ConstituteaCellwithinDIPPtopromotelocaldevelopmentandmanufactureof

drugsforrarediseasesataffordableprices

b) Take legal/legislative measures for creating a conducive environment for

indigenous manufacturing of drugs for rare diseases at affordable prices. For

instance,ensuringstrict scrutinyofpatentapplications related to rarediseases

toensuretheymeetthestrictpatentabilityoftheIndianpatentlawandissuing

compulsorylicensesunderPatentsAct2005,toensureaffordabilityofpatented

drugsforrarediseases.

c) EncouragePSUsforlocalmanufacturingofdrugsforrarediseases

4.3.3 Ministry of Chemicals and Fertilizers, Department of Pharmaceutical (DoP),

NationalPharmaceuticalPricingAuthority(NPPA)

Takemeasures to document andmake publicly available the prices of drugs for rare

diseasesandworktowardsaffordabilityofdrugsforrarediseases,inconsultationwith

theMinistryofHealthandFamilyWelfare

4.3.4 MinistryofCorporateAffairs

Encourage PSUs and corporate houses to contribute to the corpus as per the Section

135andScheduleVIIoftheCompaniesActaswellastheprovisionsoftheCompanies

29

(Corporate Social Responsibility Policy) Rules, 2014 (CRS Rules). Preventive and

promotivehealthcareisincludedinthelistintheScheduleforCSRactivities.

4.3.5 Ministryoffinance

a) DepartmentofRevenuetoconsiderremovingimportdutyonERTsaswellason

assistivedevices.

b) Departmentof Financial Services to consider, on thebasis of actuarial studies,

whetherinsurancesectorcouldcovercostoftreatmentofraregeneticdiseases

and amend the Insurance Act accordingly. It is necessary to bring in health

insurance reforms through IRDA (Insurance Regulatory and Development

AuthorityofIndia)andgovernmentintervention.

4.3.6 MinistryofLabourandEmployment

Employees State Insurance Corporation (ESIC) to consider removing/increasing the

ceilinglimitonfundingtreatmentforrarediseases.

4.4 ImplementationFrameworkonwayforward

Each Ministry and concerned departments should develop an implementation

framework on action points to be taken by them on their sector wise response to

tackling rare diseases. The implementation framework should have a clear targeted

approach,completewithindicatorsandbenchmarks(whereapplicable)andprogressive

realisation,toensuredemonstrabilityofprogress.

4.5SettingUpofCommittees

4.5.1 ConstitutionofConsultativeCommittee

GovernmentofIndia(GOI)toconstituteaConsultativeCommitteeheadedbySecretary

Healthtooversee,coordinateandsteertheinitiativestakenbythedifferentministries

anddepartments.Itsmeetingscouldbeheldatleastonceinayear.

4.5.2 ConstitutionofTechnicalcumAdministrativeCommittee

a) TherewillbeaCentralTechnicalcumAdministrativeCommitteeforthenational

30

corpus. Itwill be chaired by the Additional Secretary/Joint Secretary and have

suchtechnicalexpertsasmembersasconsiderednecessary.

b) TherewillbeTechnicalcumAdministrativeCommitteeattheStatelevelforthe

StateCorpus.ItwillbechairedbytheSecretaryandhavesuchtechnicalexperts

asmembersasconsiderednecessary.

c) TheCommitteeswillmeetonceinthreemonths.

d) TheStatecorpuscouldbeoperatedattheStatelevelundertheoversightofthe

CentralCommittee.

4.5.3 TheBroadRoleoftheTechnicalcumAdministrativeCommittees:

a) Central Committee will come up with a definition of rare diseases for the

purposes of this policy, which could be revised in conformity with increased

informationandknowledgeaboutrarediseases.

b) CentralCommitteewilldevelopaprioritylistofraredisordersforwhichfunding

support will be considered, on the basis of – disease severity, availability of

treatment,reasonablyprovenclinicaloutcome,costdataandcosteffectiveness.

Itwilldevelop thepriority listwithdue regard to the recommendationsof the

governmentappointedcommittees.

c) CentralCommitteewilldevelopobjectiveinclusion/exclusioncriteriaonthebasis

of which individual applications for funding support will be decided and the

extent of funding to be provided will be determined. The inclusion/exclusion

criteriawillincludehouseholdincomeofpatient,curabilityofconditionandcost

effectivenessetc.Thiswillalsobedevelopedaccordingtotherecommendations

ofthegovernmentappointedcommittee.

d) The Central Committee will also develop criteria on the basis of which the

progress of the patients whose treatment is funded from the corpus, will be

reviewed.

e) The Central Committee will develop protocols for diagnosis and

treatment/managementofrarediseases

f) TheCentralandStateCommitteeswillidentifyandaccreditinstitutionsthatwill

carryoutdiagnosisofrarediseases,andinstitutionsthatwillprovidetreatment

forrarediseases,andinstitutionsthatwillbothdiagnoseandtreatrarediseases.

31

g) TheStateCommitteeswill reviewtheapplicationsreceivedonthewebsiteand

decideontheapplications–whether to fundandfundtowhatextent -asper

thedetailsenteredandthecriteriadevelopedbytheCentralCommittee.

h) The State Committees will also review the progress of the case, as per the

criteriadevelopedby theCentralCommitteeandevaluatewhether the clinical

conditionofthepatientisbeingimprovedbythetherapy.

*****

32

ExpertCommitteeMeetingONRareDiseases22ndMarch2017

MinistryofHealth&FamilyWelfareNewDelhi

Participants

33

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