Two Organizations Fighting for Access to Today’s and Tomorrow’s Treatments – The Haystack Project and the Rare Cancer Policy Coalition (RCPC)
With a wealth of knowledge and experience in market access and reimbursement, Saira Sultan wanted to do more to support patients and patient groups in navigating this complicated landscape for rare and ultra-rare diseases and the orphan and ultra-orphan drugs needed to treat them. To this end, Saira founded the Haystack Project three years ago with the aim of enabling rare and ultra-rare disease patient advocacy organisations to coordinate and focus efforts that highlight and address systemic reimbursement obstacles to patient access. Their core mission is to influence the evolution of health care payment and delivery systems with an eye toward spurring innovation and quality in care, and ensuring access to effective, accessible treatment options for all Americans living with ultra-rare conditions
Advances in research and the emergence of regenerative medicines, gene therapies and other personalised targeted treatment plans comes high treatment costs and ensuring these treatments will be accepted by payers and reach patients is a pressing concern.
Working at governmental policy level, Haystack Project strives to ensure policy for access and reimbursement is fit for purpose and considers the unique challenges for treating rare and ultra-orphan rare diseases such as how patients define value vs. payers and how payment systems based on averages don't work for ultra-rare numbers.
Haystack Project is now a 501(c)(3) with a Board of Directors seasoned in reimbursement policy, and Saira now serves Haystack as the organisation’s senior policy consultant. Cyndi Goss, Chairman of Haystack's Board noted, "Saira's ability to unify divergent groups around a solution and a message is critical to our efforts, but the real strength of Haystack lies in the patient organisations that come together every month, and several times in between, to share their experiences and learn about the policies they may know little about on paper but see play out in their patients' lives every day"
Time for a dedicated Rare Cancer Policy Coalition
With the National Cancer Institute defining a rare cancer as fewer than 15 out of 100,000 incidence per year, Saira felt that a new initiative was required to tackle the unmet need in rare cancers. Rare cancers are often associated with poorer outcomes, late diagnosis and ineffective treatment options mean higher mortality rates for certain types of rare cancer. A lack of research in this area also results in knowledge gaps and little interest from drug developers, but despite these challenges new therapies do come to market and so it is crucial that these life-saving treatments are accepted by payers and reach patients. Similarly, the metrics and models used to assess “value” of new treatments are a poor fit in evaluating new, and potentially costly, cancer treatments. The advocacy community can and should play a pivotal role in determining what a potentially life-saving treatment is worth.
The Rare Cancer Policy Coalition (RCPC), created under the auspices of Haystack Project, focuses on rare and ultra-rare cancers. RCPC brings together cancer patient organisations to provide them with a powerful and collaborative voice.
RCPC provides participants a platform for focusing specifically on systemic reimbursement barriers and emerging landscape changes that impact new product development and treatment access for rare cancer patients. It is the only rare cancer coalition developed just to focus attention on reimbursement, access and value issues across the rare cancer community. Working within the Haystack Project enables RCPC participants and rare and ultra-rare patient advocates to leverage synergies and common goals to optimise advocacy in disease states where unmet need is high and treatment inadequacies can be catastrophic.
Haystack Project CEO, James Caro, whom worked with Saira in previous roles, said, "Saira has consistently proven that working with the leaders of advocacy organisations and their patients enriches every policy effort. The RCPC is a natural development since Saira has been steeped in oncology policy almost since the beginning of her career."
Working together with researchers, physicians, patients and industry is the most powerful way I know to make the case for rare patients’ needs
“Working together with researchers, physicians, patients and industry is the most powerful way I know to make the case for rare patients’ needs. Systemic barriers to treatment for really rare conditions must be eliminated. Our reimbursement system is the product of a time when we didn’t know if the Orphan Drug Act would work. Well, we now know it has worked and we haven’t adjusted for it on the payer side. Without those changes, we systematically discriminate against ultra-rare patients. This work reminds me why I’ve loved this field for over 25 years.”
Saira is President and CEO of Connect 4 Strategies. She earned her law degree from the University of Viriginia and has worked in the U.S. Congress, Medtronic, Pfizer and Sanofi before starting Connect 4. The board of directors are listed at www.haystackproject.org
To find out more about the Haystack Project or the Rare Cancer Policy Coalition please contact Saira at Saira.Sultan@haystackproject.com
And visit https://haystackproject.org/ and https://haystackproject.org/rare-cancer-policy-coalition
Evaluating the Key Challenges to Advance Commercialization & Patient Access
Conference: 15th - 16th October 2019
Workshop: 17th October 2019
Location: Holiday Inn Kensington Forum, London UK
SAVE £200 AND REGISTER YOUR PLACE ONLINE HERE - EXPIRES SOON
It's imperative for scientific researchers and orphan drugs/rare diseases professionals to stay on top of the latest advancements, technologies and processes related to orphan drugs and rare diseases. For this reason and the success of the last few years conference and workshops, we have gathered experts representing top organisations to share their insights with you at The 9th Annual Orphan Drugs & Rare Diseases Conference taking place on 15th and 16th October 2019 in London, UK.
SMi Pharma had the opportunity to speak with three of our featured speakers and co-chair to discuss some of the challenges they face in the industry and their strategies for overcoming them. The following are excerpts from those Q&A sessions. Visit the download centre to read the full interviews.
We asked Eddie Pease: What do you see as the greatest research challenge to overcome in the field at the moment?
"The main challenge in my opinion is working out the probability of success – will the return of investment be reached? Are the drugs efficient and do we need to take any essential steps/adjustments? It is important that we get a better view of clinical and commercial success in this field and with this information, we can combine it with science and ultimately get drugs to patients quicker and with less cost"
Donatello Crocetta, what is your role within the Rare Diseases field and why are you supporting the Orphan Drugs and Rare Diseases conference for 2019?
"I have been working for many years at Global Medical Affairs in Rare disease field and I believe that it is critical to share best practices in this small community to allow more patients to get access to Diagnosis and advanced treatments."
Rick Thompson can you tell us about the invite letter includes?
"Patient centricity and engagement will also be at the heart of the event. Rare disease patients are now widely recognized as the true experts in their field, and I am heartened to see a patient focus throughout this year’s program. In my role at Findacure, we dedicate our time and resources to helping patient associations form, grow and professionalize, with the aim of delivering a strong patient voice and need into the heart of the orphan drug industry. It is my hope that SMi’s 9th Annual Conference on Orphan Drugs and Rare Diseases will help to inspire more collaborative projects in the rare disease field, which place patients at their heart."
To read the full speaker interviews and co-chairs invite letter please visit the download centre at: www.orphandrugs.co.uk today!
Download the brochure today to see what you could gain from attending and also take a look at our hand-picked expert presentations from the likes of Alexion Pharmaceuticals, Bioconal Emas, Finadcure, Takeda, Minoryx, Rare Life Solutions, Chiesi Farmaceutici and many more.
Reserve your secured place today to benefit from our £200 special early bird saving available when you book by 30th August 2019 online at www.orphandrugs.co.uk
SMi look forward to seeing you in October 2019!
Rare dermatological diseases patient organisations leading the way in community building and skills development
Last month, over 120 leaders of patient groups that support people living with skin diseases came together in Milan for two unique events hosted by the International Alliance of Dermatology Patient Organizations (also known as GlobalSkin): the RareDERM Forum and the GlobalSkin 2019 Conference.
The RareDERM Forum, the first event of its kind, brought together nearly 40 rare and uncommon dermatological disease patient group leaders from 13 countries. Participants benefitted from formal learning sessions and connecting with other patient leaders and dermatology stakeholders.
A primary objective of this two-day Forum was to begin building a cohesive community of rare dermatology disease patient organisations and supporting stakeholders to ensure the lives of these patients are positively and measurably impacted through improved care and treatment. Working in small groups and then as a larger plenary group, participants began developing a strategy that defines challenges and needs; will build a cohesive community to grow knowledge and skills; will help stimulate research, foster connectivity; and deliver advocacy. Several advisory committees were formed within the community and reports on their progress will be shared later this year.
Following this ground-breaking event, most RareDERM patient leaders also attended the three-day GlobalSkin 2019 Conference, which is hosted every second year, and is specifically designed for dermatology patient leaders with a focus on advocacy, science and research, and building organizational capacity resulting in strong, resilient organizations. It attracted over 120 delegates from 35 countries representing a wide cross-section of serious skin diseases. Participants learned from each other and outside experts during stimulating plenaries, engaging workshops, and small group mentorship opportunities through a living library.
To learn more about the RareDERM initiative, please visit the GlobalSkin website. Groups and organisations supporting people living with dermatological diseases are invited to join the global movement for skin by signing up for a no-cost GlobalSkin membership here.
The International Alliance of Dermatology Patient Organizations (also known as GlobalSkin) is a unique global alliance, committed to improving the lives of skin patients worldwide. Together, with our network of over 150 patient organizations, we nurture relationships with members, partners and all involved in healthcare ─ building dialogue with decision-makers around the globe to promote patient-centred healthcare. For more information visit
New research reveals impact of ‘hidden’ health conditions and calls for better care for patients with rare disease, PKU
For the first time, patient groups – supported by funding from BioMarin - from across six countries in Europe have come together to create a coalition with the shared goal of improving health services for PKU patients. The Live Unlimited PKU campaign has launched on the 28th June, International PKU Day and brings together patient organisations from Turkey, Spain, France, Sweden and Italy to tackle public misconceptions of the disease.
The campaign aims to raise awareness of the potential severity of the disease and calls for improved standards of adult care, including consistent provision of metabolic specialists, dieticians and psychological support for every adult with PKU in Europe – aligned with the European Guidelines from 2017.iv
Phenylketonuria (PKU) is a rare, lifelong metabolic condition which affects around 1 in 10,000 people in Europe.ii The condition limits a person’s ability to break down protein, with potentially devastating effects on the brain if left unmanaged.iii In Europe, the condition is screened for during the new born heel-prick test, but just 9 per cent of the public have heard of the condition.iv
There is currently no cure for PKU, so either a heavily restricted diet – with almost no natural protein - and/or medical treatment may be required for life.v Despite the neurological effects of the condition and difficulties following a low-protein diet, just 12% of adults with PKU have access to the multidisciplinary tools and support they need, with many adults having to go to children’s wards to receive any specialist care.vi
PKU can have a serious and long-lasting effect on patients’ mental health and quality of life, with a recent study showing that as many as 52% of adults with PKU lived with anxiety or depression. Other issues reported by patients include ‘brain fog’, sleep issues and lapses in concentration.vii
These findings were in line with polling results conducted as part of the campaign, which demonstrated that PKU patients are more likely to find certain life milestones stressful than people without a hidden health condition. Key findings build a Pan-European picture of the impact PKU has on the lives of patients when compared to those without a hidden health condition, including:
Every patient is entitled to the best possible care regardless of the rarity of their disease. The Live Unlimited PKU message resonates with us as healthcare professionals, with patients and hopefully, with the general public and decision makers. Raising awareness of this condition and the burden it has on patients’ lives on a daily basis is extremely important.
The Live Unlimited PKU campaign has been co-created by many patients, and six patient support organisations - with funding and support from the biopharmaceutical company BioMarin - in order to drive policy change and help put in place the right care for adults living with PKU across Europe.
The campaign includes a suite of visual imagery and videos featuring the personal stories of patients across Europe. There are campaign videos which highlight the lives and challenges of PKU patients in their respective countries and many more case-studies available on the campaign website (www.liveunlimitedPKU.com). The one-year campaign will run until International PKU Day 2020, with many patient groups and individuals helping to raise awareness of PKU until this date. Further events and initiatives will continue throughout the year.
Five patients from across Europe share their experiences of living with PKU in a series of videos. The full video series can be viewed here.
About Live Unlimited PKU
The Live Unlimited PKU campaign launched in June 2019 to raise awareness of the gaps in care for adult patients living with the rare genetic condition, phenylketonuria (PKU). The campaign has been developed alongside the six patient groups and their memberships: AMMeC and Cometa A.S.M.M.E (Padua) (Italy), Les Feux Follets (France), Svenska PKU-föreningen (Sweden), PKU Aile Derneği (Turkey), and FEEMH (Spain), funded and developed by BioMarin Europe Ltd. The campaign seeks to call for consistent provision of adult metabolic specialists, dieticians and psychological support for every adult with PKU in Europe.
About phenylketonuria (PKU)
Phenylketonuria (PKU) is a rare, lifelong metabolic condition which limits a person’s ability to break down protein and can lead to cumulative toxic effects on the brain.iv With PKU, the foods you eat directly impact the way your brain functions.
People with PKU have a problem with the phenylalanine hydroxylase (PAH) enzyme, and so can’t fully break down an amino acid called Phenylalanine (Phe), which is found in all protein-containing foods (e.g. nuts, meat, eggs, dairy) and sweeteners such as aspartame.iv If too much protein is consumed, high levels of Phe build-up in a person’s blood and disrupt the balance of neurotransmitters - or even cause physical damage to the brain itself. This can result in neurological symptoms such as problems with memory and attention, depression and anxiety.[viii] The condition is screened for during the new born heel-prick test, and affects around 1 in 10,000 people in Europe.iv,v PKU is an inherited autosomal recessive disease. This means that if both parents are carriers of the PKU gene, their baby has a 1 in 4 chance of suffering from PKU.[ix] The condition is tested for in almost all European countries during the new born heel prick test, but general awareness of the condition remains low. Until fairly recently, doctors thought that PKU was a condition that was outgrown once the brain fully developed as a teenager. However, we now know that high blood Phe levels continue to result in damage at any age, and so the condition should be managed for life.[x]
Please find the campaign website, including all patient stories, here: www.liveunlimitedPKU.com
[ii] ESPKU. PKU: Closing the Gaps in Care An ESPKU benchmark report on the management of phenylketonuria within EU healthcare economies. Available at: https://www.espku.org/wp-content/uploads/2015/06/PKU_report_FINAL_v2_nomarks.pdf
[iii] Wegberg AMJ, MacDonald A, Ahring K, et al. The complete European guidelines on phenylketonuria: diagnosis and treatment. Orphanet J Rare Dis. 2017;12(1):162. Published 2017 Oct 12. doi:10.1186/s13023-017-0685-2.
[iv] Pitt JJ. Newborn screening. Clin Biochem Rev. 2010;31:57–68. Last accessed April 2019
[v] Al Hafid N, Christodoulou J. Phenylketonuria: a review of current and future treatments. Transl Pediatr. 2015;4(4):304–317. doi:10.3978/j.issn.2224-4336.2015.10.07
[vi] Blau et Al. Management of phenylketonuria in Europe: Survey results from 19 countries. Molecular Genetics and Metabolism 99 (2010) 109–115
[vii] Ford, S. et Al. Living with Phenylketonuria: Lessons from the PKU community. Molecular Genetics and Metabolism Report. 2018
[viii] Bilder DA et Al. Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults With Phenylketonuria. Dev Neuropsychol. 2016 May-Jun;41(4):245-260.
[ix] NORD. 2019. Phenylketonuria. Available at: https://rarediseases.org/rare-diseases/phenylketonuria/. Last accessed April 2019
[x] Berry et Al. Newborn screening 50 years later: access issues faced by adults with PKU. Genetics in Medicine volume 15, pages591–599 (2013)
Rare Revolution Editor