EARLY ACCESS IN BELGIUM: STATUS UPDATE. Veerle Kempeneers & Rudy De Cock - Pfizer Kristel De Gauquier pharma.be

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1 EARLY ACCESS IN BELGIUM: STATUS UPDATE Veerle Kempeneers & Rudy De Cock - Pfizer Kristel De Gauquier pharma.be

2 Early Access in Belgium: status update Background Overview New regulatory framework Unmet Medical Need list ETA ETR First experiences Pharma.be proposals to improve the ETR procedure

3 Background ETA-ETR pharma.be objectives = accelerate access to innovative medicines Belgium: one of the longest P&R procedures in the EU National plan for Rare Diseases EU Best Practise : French ATU system (Autorisations temporaires d utilisation) Stringent Belgian Regulatory Framework: 3 steps 1. Unmet Medical Need list (UMN) 2. ETA legislation: a new framework for CUP/MNP (RD 25/04/2014) 3. ETR legislation: a Belgian Early Access Scheme (RD 12/05/2014)

4 Overview ETA - ETR process

5 Unmet Medical Need list Objective = promote demand driven reimbursement Assessment & ranking according to a new algorithm for ranking therapeutic and societal needs Quality of Life 50 Rarity 40 Life threatening 30 Social vulnerability 30 Societal impact 20

6 ETA: A new framework for CUP/MNP Ethics Committee Applicant FAMHP Commission for Medicines FAMHP Applicant fee Validation : 6 working days Evaluation: 40 working days 55 working days (principle of tacit approval) 5 18/09/201 4

7 ETA regulatory framework (RD 25/04/2014 applicable 1 st of July 2014) Current regulatory framework Compassionate use & medical need Submission Single submission to Famhp EC: ethical justification, Informed Consent Commission: quality, (pre)clinical data, justification versus potential alternatives: medicinal products authorised and/or inclusion in clinical trials Timelines Fees Dossier Responsibilities company Publication 55 days + 6 days for validation (max timelines) Fees for initial application, (bi)annual review and substantial amendments Dossier requirements similar to MA applications - execution of the program - designation of responsable physician - central register and safety register Decisions are published by FAMHP on the website (link)

8 New Early Access Scheme ETR Scope: for pharmaceuticals responding to a high medical need severe or life threatening disease no acceptable reimbursed alternative ( ETA: no acceptable authorized alternative) Early: as from 6 months before application for EU MA Temporary: limited in time (4y. max.) does not replace regular reimbursement procedure obligation to ask regular reimbursement 6 months after EU MA Reimbursement: forfeit based financial compensation no price attributed

9 ETR : new CATT CAIT* ctte President Vice-president Chairman College orphan drugs Chairman Commission medicines for human use Effective Prof Westhovens Prof Degaute Commissie voor advies in geval van tijdelijke tegemoetkoming voor het gebruik van een geneesmiddel Commission d avis en cas d intervention temporaire dans l usage d un médicament Replacement Sick fund representatives G. Nelis E. De Baerdemaeker C. Van Hul C. Van Haecht Famhp G. Musch C. Lahorte A. Lhoir W. Penninckx CTG/CRM 1/ chairman or academic K. Denys H. Robays 2/ sick fund F. Sumkay F. Demeulenaere RIZIV/INAMI G. Hans V. Knappenberg M. Van de Casteele V. Millecam Famhp administration T.Bruwier S. Eglem pharma.be Advisory role K. De Gauquier

10 ETR cohort application template pharma.be

11 ETR cohort application company commitments Complete clinical studies successfully Ensure availability Submit MA application within 6 months Reimbursement application within 6 months after MA Support of the cost of pharmaceutical for individual applications Before ETR decision: in line with cohort After ETR decision: Continue patients included that do not match cohort decision After end of cohort: Continue ongoing individual applications between end of cohort and decision about reimbursement

12 ETR individual request Role of the responsible physician Transfers of each individual request to college of physician-directors Documents to join Motivated decision by the responsible physician on Inclusion Duration of inclusion Documents related to the request for inclusion in CUP/MNP Report demonstrating that the patient responds to inclusion and/or exclusion criteria of cohort Therapeutic scheme (dosage, frequency) Attest of deliverance Mentions the number of vials/boxes delivered Copy of the informed consent Statement that patient does not participate in a clinical trial

13 ETR funding for individual requests Funding: double forfeit ETR note on funding - RIZIV/INAMI Forfeit per file Forfeit /patient/year Calculation: pro rata based on number of months of treatment Funding by the special solidarity fund Directly to the company Based on a 3 month declaration by the company List of patients included in the program N Name and social security number Start date Tx End date Tx 1 2

14 ETR first experience 2 cohort applications approved (published on RIZIV/INAMI)

15 Logistic costs for ETA and ETR can be important 1) Logistic costs of the FAMHP Fees and translation in ETA Original application file - Fee for FAMHP ( 4.635,82) Costs for translation of documents Modifcation of application file - Fee for FAMHP ( 4.635,82) Safety report every 6 months - Fee for FAMHP ( 2.882,76) Labeling and shipment in ETA FTE in ETA Medical support: Preparation of protocol, ICF and other required docs; Handling of incoming requests, contact with physicians, follow-up document completeness with physicians, therapy management communications; Internal approvals; Answering specific questions from FAMHP. Project management: Submission, digital filing and archiving, shipment orders, stock management, set-up of the central administrative register requested by the Belgian legislation on ETA. Drug Safety & Regulatory Affairs: Prepare customized safety listings, follow-up of reported adverse events (which is very strict because the medication is not yet commercially available), FAMHP specific request for information on safety/efficacy. Quality Assurance: Product labeling approvals and batch releases. 2) Logistic costs of the RIZIV/INAMI - on top of the ETA specific cost/work Translation of documents FTE in ETR Market Access: Preparation ETR cohort application. Medical Director: Preparation ETR cohort application, Communication (by , fax, scan) with the physicians and the INAMI Special Solidarity Fund for the follow-up on individual patients and payments, Measures to respect the privacy law (with regard to the handling of non-anomyzed patient data). Legal counselor: Measures to respect the privacy law (with regard to the handling of non-anomyzed patient data), File a specific authorization request to the Belgian Privacy Commission regarding the collection of the national register number of the patients, Designation of a specific data security officer.

16 Pharma.be proposals to improve ETR procedure Maximize the re-use of the FAMHP s scientific evaluation that was performed for the CUP /MNP procedure Always involve external experts Company should be able to react to cohort proposition CATT Maintain the same forfeit in 2017 as in 2016 i.e /cohort & 2500/patient/year Re-evaluation of the ETR procedure end 2017 pharma.be will perform an assessment of the logistic costs for ETA and ETR

17 Conclusion: ETR important step forward Unmet Medical Need list : Increase awareness at level of other stakeholders, eg patient associations But difficult to explain to patients ETA: More rigorous and more demanding procedure Legal framework will be improved ETR: Currently still limited enthusiasm within pharma companies Funding is (too) limited to become real incentive Increase of fee of ? Still some uncertainties Limited experience = unpredictability Impact on CTG/CRM procedure? Save the date: pharma.be info session on May 4 th, 2017 (pm) Update about ETA and ETR by FAMHP and RIZIV Further optimisation will help to accelerate access to new innovative medicines for Belgian patients