I was extremely surprised, thrilled and thankful to be invited to attend the 11th Annual International Experience Exchange with Patient Organisations. 

This conference was my first introduction to personalised healthcare and got to understand its transformative impact on the way healthcare is delivered and why it should matter for patient groups. I learnt also on the shift in patient participation in healthcare, where patients can be true collaborators with pharma, holding the key to unlocking the full potential of healthcare innovation. There were a number of breakout workshops for us to explore the impact of policy, data, and patient partnership on personalised healthcare and drug development.
However, let me instead share more on those activities that offer a more immediate and direct relevance to families with SMA and rare diseases in Malaysia. 

Global SMA Patient Group Workshop 

11 representatives from 9 countries around the world met to explore issues relating to access and support services for people affected by SMA and learn more about the ways some of the organisations are making a difference in the lives of people with SMA, their caregivers and families. 

There is a big divide between countries which have access (to Spinraza) and those that do not. When Malaysia gets access to treatment (let’s be optimistic and determined, “when” not “if”), we can be aware and better prepared to tackle certain challenges e.g. evidence of efficacy in real-world data and different degrees of efficacy in different patients, proving patient reported outcomes etc. There are also common issues e.g. in accurate and early diagnosis, broad awareness of SMA, and doctors’ negative mindsets about treating SMA. 

In more mature care and support environments like USA, there is a shift from broader support services “about SMA” to more targeted interventions for specific patient populations such as teen and adult support packages and local symposia to address state-specific issues. 

I believe that WeCareJourney is on the right track. Although we continue to struggle against significant unmet needs, there is much we can learn from other countries. How I wish to share with relevant stakeholders in the Ministry of Health and doctors on what we can achieve together and it’s time to try new ways to encourage greater interest and cooperation from them. 

[Thank you Fani and team for making this workshop possible!] 

Platform to tackle unmet needs in SMA and rare diseases 

I was given the incredible opportunity ask for feedback and advice from an expert panel of advocates and all the participants of IEEPO. The idea that I presented was a roundtable of stakeholders with the patient/ caregiver voice at the centre.

What could be the keys to success? 

• Need patience with doctors and often they do not know (and are not taught) on how to engage with patient groups
• Media is our advocacy partner and can be very effective to shape support for RD
• Pharma, doctors and patients need to work together
• Create a “shocking” event to grab attention, unlock empathy and support, and exert pressure on stakeholders
• Not everyone in the RD community wants to work together, but to go far we need to try to bring everyone together on common ground
• Accept the culture (of Malaysia) and when dealing with the government don’t go alone and bring the doctors
• Find mutual benefits for all stakeholders and go with an ask but also with part of a solution
• SMA is a “small” disease that needs a big push (such as a high profile medical advisory panel)
Some examples were shared and if anyone wants to know more just reach out to me. 

[Thank you Durhane, KP, Maira and Mary!] 

Q & A with Severin Schwan (CEO of Roche) 

I asked on what can be done to ensure timely access of orphan drugs (e.g. Risdiplam) in Malaysia and other mid-high-income countries, without which critical needs remain unmet and lives lost (which can be saved today). Also what could Roche do to give families in Malaysia hope and confidence that Risdiplam will be available. 

Here’s a summary of Severin’s response: 

• SMA severely impacts patients and families especially the weaker types of the disease
• Just a few years ago there was no option but severe disability then death. But it is different today with an approved drug that really makes a difference (Spinraza from Biogen). And there’s gene therapy on the way from Novartis that is likely to be launched in 2019. Roche also has a medicine in development (Risdiplam).
• So in a short period of time there are real prospects now that an SMA patient can live a normal life.
• The question is that now we have medicines, how to we make them accessible? Pharma needs to talk to the government, show the value of the medicines and make sure that I will be available broadly in a responsible and thoughtful way. We need good data and negotiation with governments to make ODs available.
• He knows how much suffering SMA families go through and hopes for some resolution for everyone with broad access.
• Promising early data for Risdiplam and undergoing discussions with health authorities who need to make sure that it is safe.
• Keep hope alive. 

[Thank you Mary and Severin!] 

It was my pleasure to connect and befriend many patients, advocates and care-givers from around the world working on many disease areas. I believe our desire to ensure we do not get left behind should inspire and encourage each other to stay resilient and optimistic, and learn from each other to do things better and also avoid mistakes.
None of our journeys are easy, neither personally nor professionally. There is so much still we can do to be more effective, and at the same time build a more cohesive and conducive environment for each other to get the results we need. 

It is in our hands to make the difference we desire, and be clear on the purposes and why we strive towards the goals we set to save lives and boost family well-being. 

[Thank you to Roche Switzerland for investing on this study trip for the future of families with SMA]