World Orphan Drug Congress USA 2019

WeCareJourney’s founder Edmund was invited to participate at the recent World Orphan Drug Congress USA (“WODC USA”) 2019. The WODC USA focuses on the most pressing challenges and opportunities to bring rare disease therapies to patients faster, and brings together a global gathering of 1,200 leaders in orphan drugs from 50 countries, featuring over 135 presentations covering all aspects of orphan drug development and rare disease research.

Here are some of his takeaways from WODC USA.

What an incredible opportunity and I am so grateful to get the chance to participate in a great conference packed full of relevant and important topics which made for non stop morning to evenings. Getting there required 50 hours of travelling and fighting 12 hours jet lag, so not the easiest journey to make. Here are some of my impressions and my thoughts from WODC USA and what it might meansfor SMA and rare diseases (“RD”) and in Malaysia.

  1. The pressing goal of patients needing early access to healthcare and medicines and how manufacturers must focus on getting their products to patients in the most cost effective and most efficient way. However, the increasing pipeline of costly biologics and specialty drugs, unsustainable financing, rising out of pocket costs, and markups, all put tremendous strain on healthcare systems everywhere. This leads to the need to innovate on access and reimbursement - from mortgage financing to subscription models, outcomes or value based care arrangements, binding arbitration, legislative policies that drive transparency, prices and competition, and taking a broader view of value assessment. It would be worthwhile for our Ministry of Health to invest more in assessing all these models of engagements that can help to achieve a more sustainable balance with manufacturers. More importantly, get beyond the years of talking, and please start taking action!
  2. How cooperation and collaboration between health authorities from different countries have proven to be successful in getting access to medicines (and with prices and on terms) which they would not have achieved if going alone, e.g. MoCA and BeNeLuxA. The keys to success are that everyone is clear on their respective and common goals, the mutual benefits to be obtained, and a voluntary and cooperative model of working together to gain leverage in their negotiations. BeNeLuxA stands out for me as it delivered broad access to Spinraza in 5 countries, which individually each were too small and weak negotiating positions. But together they bring to the table a combined population size of 42 million people and enough clout. This should makes us think of the opportunity to cooperate in country, across ASEAN even Asia Pacific or further. It does require leadership from the health authorities, and Malaysian motivation and talent to do what it takes to addresse unmet treatment needs for SMA and other rare diseases.
  3. Germany is such a great place for families with rare diseases, with no access hurdles, inclusive and equitable coverage, and timely access. And it’s really interesting to have discovered (on an early study trip in Europe which I will provide a write up) that there are young adult patients with SMA who decide to decline Spinraza treatment and why. What this indicates is that we must continuously engage within our community on priorities to keep tuning care and support to individual needs. Talking to Ben’s Friends a multi RD patient support group, they have experienced how for the same RD that priorities differ widely between age groups and what stood out for us is that adults with RD need a safe space to be understood and heard, and less on looking for information on treatments which parents of RD kids tend to do a lot more. 
  4. I had the pleasure to sit in on and also help facilitate some round table discussions relating to patient group internal organization and stakeholder engagement. The usual issues surface around the world - bickering between patient organisations, the hypocrisy of manufacturers talking about “unmet needs” and the prohibitive high prices limiting access, the challenge of getting patient voice truly respected etc. There are lessons learnt that within such complex multi-stakeholder environments, there is a great need to be matured to set aside differences in priorities and to cooperate around common goals and combining strengths. Candid and regular dialogue is important. There are opportunities for us to work better, faster, and deliver more results - but how can it be catalyzed and sustained? After meeting and talking with NORD, RDI and numerous patient groups from around the world over the past few weeks, I think some adjustments should be made in Malaysia and I hope the community and leaders will be open minded to focus on outcomes and work on facts and plans with a close eye on results and time. 
  5. There is a trend of patients taking greater control of their data to influence R&D and treatment. I had the privilege to get to know those who have ultra rare diseases, let me share with you about Neena Nizar who has Jansen’s metaphysical chondrodysplasia, where there are only 8 known patients in the world of which 3 are in her family (herself and 2 sons), and their need to travel all over the world to get help taking 32 years to finally get diagnosed. I take my hat off in admiration of her resilience and the family (her bedrock is her father) to keep going and taking ownership to find others, and fund and drive research into the disease and treatment. It’s inspiring she soldiers on with courage and a smile even though it’s so tough (“crazy” in her words).
  6. In the world of RD the often cited top issue are those who remain undiagnosed and the length of time to get an accurate diagnosis. This is where the role of genetic counselors are so important, and networks like Undiagnosed Disease Networks (“UDN”) and UDN International offer access to data, researchers and doctors who can help to lift these families out of crushing uncertainty on what they are dealing with. I think our clinicians and parents/ patients should take more advantage of such networks.
  7. The highlight of my trip is of course around SMA and treatments. Firstly to meet the Chief Scientific Officer and Global Heads of Medical Governance working on gene therapy. The data and outcomes offers so so much hope and we are excited to push on and work hard with them and Malaysian stakeholders on clinical trial opportunities and access to patients if/once the gene therapy is approved. Secondly to meet Biogen’s Heads of Global Access and to get updates on their access (44 markets to-date, with Korea being the latest last week), giving us more ideas to follow up on the work so far that Biogen has done in Malaysia and with us to overcome the treatment barriers in Malaysia. My hope is cautious, the priority is very high. We continue to push on with the initiatives around raising standards of care and getting a registry in place, and the burden of disease study done and published. What remains a big challenge is that no one can get things done alone - so with many stakeholders it is crucial that we can all engage together to get on the same page and coordinate. About 1 baby is born with SMA every week in Malaysia we must do better.
  8. Glad to see many great examples on advocacy done right, innovations in operating patient groups, and insights from patient experiences. 
  9. Concerned with the increasing tensions between unmet needs vs increasing cost and sustainability. I quizzed a few CEOs (on public microphone with everyone as audience!) - what needs to be done better to ensure more timely and equitable access to Malaysia and other mid-high income countries. My thoughts are that on our part (as patient group, patients, caregivers) we need to fight for better local standards of care and promote our well-being “beyond the pill”. And pharma’s part? Shareholders and profits are their king - but in life, there’s also social responsibility and equity, and innocent lives to be saved.

I hope this helps everyone get some insights and that you find it useful.

This will be the start of a series where we aim to help everyone by sharing what we learn, to build capacity and empower others in the SMA and rare disease community.

It is an overview as it is hard to compress 3 days of intense activity across so many presentations, roundtables, and meetups crossing clinical development and regulatory, commercial, rare disease advocacy, global market and patient access, pricing and reimbursement, advanced therapies - clinical, and rare oncology.

If anyone wants to find our more just reach out to me.



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