I'm here at the Bridging The Gap @RocheCanada panel, which will explore timely patient access to innovative medicines in the rare conditions, with moderator @billdempster, and panelists Tania Stafinski, @adthoro (AT), @Durhane (DW) and Judith Glennie. #CSPC2019

Thread

So what constitutes a rare disorder?

Bill Dempster (moderator) says that it's defined as a life-threatening or serious chronic condition that only affects a small number of individuals. It's estimated that there are ~7,000 rare disorders, affecting 1 in 12 Canadians. #CSPC2019

First up,Durhane Wong-Rieger! DW is the President & CEO of the Canadian Organization for Rare Disorders ( @raredisorders), which is a network of 102 patient groups who aim to improve lives of all those affected by rare diseases by advancing policy. #CSPC2019

DW says that Canada is still catching up with the rest of the world. We have yet to adopt an orphan drug legislation (orphan drugs = drugs developed for rare conditions). DW says that centering patient engagement is essential for optimal use of therapies. #CSPC2019

So which country provides the best access to rare disease drugs? There is no 'best' country here, says DW, e.g. France has excellent high access but also has higher pricing. #CSPC2019

The answer is that we need to have different developmental processes, says DW. There is a need for structured cooperation between developers, payers and patients. We (as in Canadians) need to have a stake in the 'game' in order to benefit from developing orphan drugs. #CSPC2019

Of note, DW highlights that the 2019 federal budget allocated $1 billion for a Rare Disease Drug Program and Strategy. #CSPC2019

Next up, @adthoro (AT) is a lawyer-academic based at @mcgillu's @genomics_policy centre. AT is coming from a genome sequencing perspective, and begins with talking about the importance of patient engagement across the entire development/regulatory life cycle. #CSPC2019

AT says associated challenges with patient engagement include:
- Negative perceptions, baggage on both sides
- Systemic & structural barriers (e.g. unpaid contributions)
- Tokenism
- Representativeness
- Conflicts of interest #CSPC2019

AT says we've seen a shift from patient-involved to patient-powered research. e.g. a metastatic breast cancer project leveraged patient advocacy groups to recruit over 5,350 men and women from all 50 US states, representing over 1,700 institutions. #CSPC2019

Next, AT discusses consent, where challenges are:
Blurring of research & clinical contexts
Scientific uncertainty and regulatory complexity

Solutions being explored: improve drug labelling have patient centric designs incorporate e-consent. #CSPC2019

AT highlights that rare diseases disproportionately impact minors, so often consent is from the parents. Inclusion is an important principle says AT. Children need to engaged by physicians and parents, and included in the decision-making process. #CSPC2019

To end, AT points out the @GenomeCanada's Canadian Genomics Partnership for Rare Disease (CGP4-RD), where principles also includes access to whole-genome sequencing. #CSPC2019

https://www.genomecanada.ca/en/programs/precision-health-strategy/canadian-genomics-partnership-rare-diseases-cgp4-rd-mission

Next, Judith Glennie is giving us the payer perspective (who will ultimately pay for orphan drugs for rare conditions). There is limited access to these orphan drugs right now because of layers of uncertainty involved. #CSPC2019

Traditionally, drug approval starts at Health Canada. It then undergoes a health technology assessment. Experts will make a decision based on clinical and economic assessment, before negotiating a price. This is a long, step-wise process before (could take two years). #CSPC2019

JG shares that 1) a new supplemental process has been proposed, 2) INESSS Québec has shared a case study. What does this mean for payers? JG says new processes will be used selectively. There's a need for formalization of the provincial/territorial supplemental process. #CSPC2019

Next, Tania Stafinski (TS) is talking about the processes and value of health technology assessments. Who benefits and what costs matter?

Note: TS is a the @UAIberta Director of the Health Technology and Policy Unit in the School of Public Health. #CSPC2019

Traditionally, we see drugs as benefiting a single patient population. But TS shows how many people are involved in working with the patient (such as their family, school, and the industry) -they also benefit. How do we capture these co-benefits in a policy environment? #CSPC2019

Value is seen as beyond individual health gains in countries such as Australia, Catalonia, Italy and the UK. Here international mechanisms to enable appropriate access include managed access programmes, managed entry agreements, etc. #CSPC2019

So where do we go from here? TS says to guide access schemes, we should:
Have everyone at one table at the same time
To agree on priorities for outcomes of new therapies
To leverage patient or managed access schemes to generate ongoing real world evidence. #CSPC2019