Canada’s Orphan Drug Regulatory Framework: What Health Canada is doing now and what is yet to come...
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Transcript of Canada’s Orphan Drug Regulatory Framework: What Health Canada is doing now and what is yet to come...
Canada’s Orphan Drug Regulatory Framework: What Health Canada is doing now and what is yet to come Canadian Expert Pa.ents in Health Technology Conference November 7, 2016 Liz Anne Gillham-‐Eisen Health Products and Food Branch
Orphan drugs
Orphan drugs are used to treat rare diseases which: • are life-‐threatening, seriously debilita.ng or both serious and chronic in nature
• affect a rela.vely small number of pa.ents (less than 5 in 10 000, but typically closer to 1 in 100 000)
• are oRen gene.cally based, onset at birth or early childhood, lead to a shortened life-‐span
• reduce quality of life and place significant challenges on pa.ents, caregivers and the healthcare system
• are difficult to study because of the small pa.ent popula.on
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Challenges: Limited pa?ent popula?ons and treatments
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There are more than 7,000 rare diseases
Rare diseases affect 1 in 12, or nearly 3 million Canadians
Diagnosis is challenging and can take years
Once diagnosed, there is likely no treatment available, or limited treatments for the patient
Challenges – Orphan drug development and pa?ent access
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Orphan drugs are challenging to develop:
Small pa.ent popula.ons make it difficult to conduct clinical trials, especially in na.ons without large pa.ent popula.ons
LiXle is oRen known about the rare disease
Researchers and drug sponsors, in the past, did not have incen.ves to invest in developing drugs for rare diseases
Once developed and approved:
Orphan drugs are oRen costly
Pa.ents across Canada may not have equal access to approved orphan drugs
An Orphan Drug Framework for Canada A Regulatory Framework for Drugs for Rare Diseases (Orphan Drugs) proposed to -‐
• channel orphan drugs into a federal regulatory pathway designed to improve market availability in Canada • draw on new post-‐market and transparency powers from Vanessa’s Law • reflect Canada’s legisla.ve and regulatory context, while aligning where possible with other interna.onal regulators’ orphan drug frameworks • encourage the genera.on of knowledge regarding rare diseases in drug development through an open and transparent regulatory environment and the provision of advice to companies developing orphan drugs • allow for accelerated market authoriza.on
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Life-‐cycle Approach What is known about the poten?al/actual benefits, harms and uncertain?es associated with a drug changes over ?me
Current: Limited point-‐in-‐,me oversight based on applica.ons for clinical trials and marke.ng authoriza.ons.
Future: Expanded and con,nuous oversight beginning at early development stage with medical plausibility and encompassing a greater ability to define post-‐approval informa.on gathering.
Transparency throughout the life cycle
More collabora.on with other health system partners, such as HTA Agencies
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Key Components of the Framework Defini?on Orphan drugs are used to treat rare diseases which:
• are life-‐threatening, seriously debilita.ng or both serious and chronic in nature • affect a rela.vely small number of pa.ents (less than 5 in 10 000)
Orphan drug designa?on cer?ficate
• publicly iden.fy medically plausible orphan drugs to enable early considera.on of medically plausible drugs by payers for purposes of improving pa.ent access
Regulator’s Advice • wriXen regulator’s advice for clinical trials, the poten.al balance of pre-‐ and post-‐market evidence requirements, and the development of post-‐market plans • joint advice where possible with interna.onal regulators and HTA bodies
Key Components of the Framework Market Authoriza?on Applica?ons • modernized licensing structure and possibility for abbreviated pathway for ultra-‐rare orphan drugs • ability to issue terms and condi.ons on a market authoriza.on • post-‐authoriza.on plan to characterize, monitor and con.nue to iden.fy, assess and manage the harms and benefits associated with the orphan drug
– includes: enhanced post-‐market surveillance procedures, regular monitoring of terms and condi.ons of market authoriza.on, risk management plan, confirmatory studies, etc.
Transparency
• Transparency throughout the life-‐cycle of the drug to communicate applica.ons under review and regulatory review decisions.
Pa?ent Involvement • Collec.on and considera.on of public input into the review of submissions for orphan drugs.
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Regulatory Openness and Transparency • To help Canadians beXer understand how and why decisions are
made: – Submissions Under Review
• hXp://www.healthycanadians.gc.ca/drugs-‐products-‐medicaments-‐produits/authorizing-‐manufacturing-‐autorisa.on-‐fabrica.on/review-‐approvals-‐evalua.on-‐approba.ons/submissions-‐under-‐review-‐presenta.ons-‐cours-‐examen-‐eng.php
– Drug and Health Products Register • hXps://hpr-‐rps.hres.ca/
– Regulatory Decision Summary (RDS) – purpose and reason • hXp://www.hc-‐sc.gc.ca/dhp-‐mps/prodpharma/rds-‐sdr/drug-‐med/index-‐eng.ph
– Summary Basis of Decision (SBD) – more technical than RDS • hXp://www.hc-‐sc.gc.ca/dhp-‐mps/prodpharma/sbd-‐smd/drug-‐med/index-‐eng.php
– Summary Safety Reviews • hXp://www.hc-‐sc.gc.ca/dhp-‐mps/medeff/reviews-‐examens/ssr-‐rei-‐eng.ph
– Stakeholder Registry • hXp://www.hc-‐sc.gc.ca/ahc-‐asc/public-‐consult/stakeholder-‐intervenants/index-‐
eng.php 9
Pa?ent Involvement: Exploring opportuni?es • Pa.ent input can add value to the evalua.on of human drugs, such as
contribu.ng to benefit/risk assessment • Current approach considered “ad-‐hoc”. Pa.ents as part of expert
commiXees or panels; part of specific issues, and rou.nely as part of policy and regulatory development. Health Canada is currently exploring and examining the most effec.ve ways to involve pa.ents in the review process
• Not a ques.on of “if” but a ques.on of “how” • Consulta.on with pa.ents, pa.ent groups, caregivers and health care
providers cri.cal for the design of any program(s)
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Pa?ent Involvement Pilot Project • Pa.ent Involvement pilot project launched in 2014
• Orphan drugs context was considered a good star.ng point • Pilot was intended to be a simula.on of how input could be obtained and
used to help inform Health Canada’s benefit/risk assessment • Two drugs were involved in the pilot (one biologic and one pharmaceu.cal)
• Input was sought through online ques.onnaires designed to gather the perspec.ves of pa.ents, caregivers, pa.ent groups and health care professionals. Ques.ons related to quality of life, experience with other therapies, unmet need, and risk tolerance.
• Results – more experience is needed; reviewer and pa.ent group training would be useful, .ming of request for, and receipt of input is important, considera.on should be given to establishing criteria for when to request pa.ent input that would be of most value to both reviewers and pa.ents.
Finding the right balance
Who best to provide input?
When most feasible and
useful to collect?
What other information
could be collected?
Most effective and best format/s: summaries?
Other?
How best to use the
input?
Accountability
Routine collection or establish criteria for
when most valuable?
Meaningful data and timeliness of reviews
Vanessa’s Law • Overall policy objec.ves are to:
– Provide a range of pre-‐ and post-‐market regulatory tools that enable the safety oversight of therapeu.c products throughout their lifecycle; and
– Promote greater confidence in the oversight of therapeu.c products by increasing transparency.
• Vanessa’s Law amends the Food and Drugs Act improving Health Canada’s ability to: – collect post-‐market safety informa.on – take appropriate ac.on when a serious risk to health is iden.fied
• These amendments are par.cularly important in the rare disease context where oRen .mes: – many of the pa.ents impacted are vulnerable paediatric popula.ons – informa.on on the drug and the disease is some.mes limited
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Vanessa’s Law • June 18, 2016: No.ce of Intent that Health Canada plans to bring
forward regulatory amendments to implement key authori.es under Vanessa’s Law
Coming soon
• Two regulatory packages are under development: – Authori.es to implement a lifecycle approach to regula.ng drugs
(“Vanessa’s Law Lifecycle Authori.es”) – Recall of drugs and medical devices
Coming later • Analysis con.nues on the other Vanessa’s Law authori.es (terms and
condi.ons for market authoriza.ons, repor.ng of adverse reac.ons, and disclosure of clinical trial informa.on)
Current availability of Orphan Drugs in Canada • Patented Medicine Prices Review Board's (PMPRB) Orphan
Drug Launch Monitor, published in 2016:
– analyzes the interna.onal approval of designated orphan drugs and assesses their availability in Canada
– focuses on a number of select orphan drugs reported at the ac.ve-‐substance level and iden.fied based on the following criteria: 1. drugs that received orphan designa.on and approval in the US and/or EU over the last
decade (2005–2014), and 2. drugs that only have orphan indica.ons
– includes Canada and the seven PMPRB interna.onal comparator countries (PMPRB7): France, Germany, Italy, Sweden, Switzerland, the United Kingdom (UK) and the US
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The PMPRB analysis reveals the following key points:
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Many orphan drugs available in PMPRB7 countries are approved for use in Canada
Health Canada approved 9 of the top 10 select orphan drugs
Orphan drugs not available in Canada represent a small share of the international market: some were accessed through the Special Access Program, while others were approved by Health Canada in 2015
Access to drugs as a priority
Minister of Health mandate leXer (2015) • “…improve access to necessary prescrip.on
medicines”
HPFB priori.es – new Strategic Plan
Research & development
Health Canada review / authorization
Patented Medicine Prices Review Board
(PMPRB)
Health Technology Assessment
Provincial/Territorial Drug Plans
pan-Canadian Pharmaceu?cal Alliance
(pCPA)
Hospitals
Patient Access
Innova.on and access to drugs -‐ What role can the regulator play?
Private Plans
Where do we go from here?
• Improving research, approval, and access • Many of the tools considered for orphan drugs can also
be considered in the context of all drugs • Work is ongoing to explore and examine most effec.ve
ways to involve pa.ents in the review process • Life cycle approach and increasing transparency • Con.nue collabora.ons interna.onally to promote
convergence, and enhance collabora.ons among key Canadian stakeholders across the lifecycle of a drug