Orphan Medication Status in Japan (Orphan Item Approvals) Key offerings: reate !

echni"ue and erti#cation to Ac"uire Orphan Medication Status in Japan

Identi$y regulating path %iterature %oo& $or the Industry (the variety o$ individual and healthcare practice) Identi$y 'nmet Medical (eeds $or Orphan )roducts onsultation *ith KO%s in Japan (egotiate *ith M+%, (Ministry o$ ,ellness- ,or& and ,el$are) Manage Medical !rials .ile (/A $or Orphan )roducts Support Integrating Initiatives $or Orphan )roducts

Apr 01- 2303 )% Appointed as J4!5O 'S Medical 6usiness Advisor )aci#c7%in& onsulting ()% ) 88

Our consultants have particular e9pertise *ith o:taining Orphan /rug /esignations in Japan and the 'nited States $or novel :iologics and drugs *ith a success$ul trac& record o$ approves through the )M/A and ./A8 ,e also speciali;e in Japanese 6ridging Strategies and clinical trial applications in Japan $or Orphan Drugproducts8 )aci#c7%in& is commi<ed to support clients in meeting their :usiness o:=ectives- pro=ect milestones and customi;es our approach to the needs o$ the client company8

Orphan Drug

)aci#c7%in& onsulting can help the clinical improvement needs $or our customer>s starting clinical trials8 Key offerings: 5egulatory Strategy /evelopment and onsultation JapaneseOrphan Drug/esignation and 6ridging Strategies Medical and !echnical *riting $or reg8 su:missions (e8g8 S5s- M Sections- Module 2 Summaries) )reparation o$ electronic ommon !echnical /ossier (e !/) applications $or su:mission to +ealth Authorities Investigational (e* /rug Applications (I(/s) and Investigational Medicinal )roduct /ossiers (IM)/s) in the 'nites States- Japan and 4urope

Our e9pert 5egulatory staff can develop strategic regulatory plans- $acilitate Agency in the 'nited States and Japan8

Key offerings:Orphan Drug? /esignation prepare documents 5e"uest and hold meetings

/r8 !animoto is a pharmaceutical pro$essional *ith over 0@ years o$ e9perience in clinical research and development o$ :iotechnology products8 She is the Managing /irector $or )aci#c7%in& onsulting providing a range o$ development services to :iotechnology and pharmaceutical companies8

Orphan Drug

Ms8 Joyce 5eyes is an e9ecutive *ith over 0@ years o$ 5egulatory Affairs and Auality Assurance e9perience in various roles at :oth small and large :iotechnology or pharmaceutical companies8 Ms8 5eyes has e9tensive e9perience in cell and viral production processes and has :een in :oth manu$acturing and "uality assurance roles prior to =oining regulatory groups at various companies8

Orphan Drug Designation in Japan (Orphan Product Approvals)

B/evelop Strategy and /ocumentation to O:tain Orphan Drug/esignation in Japan BIdenti$y regulatory path B%iterature Search $or the Mar&et (the num:er o$ patient and medical practice) BIdenti$y 'nmet Medical (eeds $or Orphan )roducts B onsultation *ith KO%s in Japan B(egotiate *ith M+%, (Ministry o$ +ealth- %a:or and ,el$are) BManage linical !rials B.ile (/A $or Orphan )roducts

Full Project Management Manage Manu$acturing site and prepare study drug $or clinical trial Manage C%) !o9icology Studies ,or& *ith Academia on )reclinical )rogram ./A Interactions and onsultation )re7)re I(/ onsultation )re7I(/ Meeting Multiple in$ormal discussions- consultations and negotiation 4arly ./A Interactions and )reI(/ Meeting .ile (I+ 5A .ile I(/ lear I(/ )repare and conduct )hase IDII Stud

An overview on Japan Orphan Designation In Japan, drugs and medical devices could be designated asorphan drugs or medical devices based on the Article 77-2 of the Pharmaceutical Affairs Law. his was in the event that the! are e"pected for use in fewer than #$,$$$ patients in Japan. %or which there is a high medical necessit!. he! are designated b! the &inister of 'ealth, Labor and (elfare based on the idea of the Pharmaceutical Affairs and %ood )anitation *ouncil +PA%)*,.

Japan Orphan Designationdoes not immediatel! prompt promoting approbation. he ob-ectives and diagram of the s!stem are described beneath.

OrphanDrug

The basis o the designation s!stem

!heM+%,OrphandrugDmedical apparatus system had esta:lished drugs and medical devices to :e used $or the treatment o$ trou:lesome to7treat diseases and (AI/S) had not :een suEciently created8 !his *as despite the high medical needs :ecause the amount o$ patients *as small8 ,ith the diversi#cation o$ open healthcare needssa$e and "uality medical products *ere o:liged to :e supplied to patients as soon as possi:le8 As needs :e- there is a huge increase in rising open e9pectations and the changing circumstances o$ drug and medical7 mechanism research and improvement8 It had :een chosen to ta&e special measures to support and push research activities $or the advancement o$ orphan drugsDmedical devices8

"riteria or designation

!he Minister o$ +ealth- %a:or and ,el$are may designate drugs and medical devices satis$ying the accompanying criteria as orphan drugs medical devices8 !his *as in the *a&e o$ geFng applications $or OrphanDrug$rom the applicants8 !he amount o$ patients *ho may use the drug or medical mechanism should :e less than @3-333 in Japan8 !he amount o$ patients could :e estimated :ased on the report o$ +ealth and %a:or Science 5esearch or the in$ormation pu:lished :y solid scienti#c societies8 !he amount o$ patients *ith a challenging to7treat disease is sometimes trou:lesome to estimate correctly :ecause o$ a:sence o$ research on the patient populace8

)aci#c7%in& onsulting Services G0H@ 5un o$ the Knolls ourt San /iego- A H202I )hone: G@G7JJ@70J33