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RareiTi VIEW

Patient Experience Data: How patients can own the next step.

Patient Experience Data is particularly important for the rare disease community because of the inherent limitations in working with small populations, which can cause delays in approval processes. Accessing life-saving or life-prolonging therapy through unlicensed medicine programs is often the only choice. As a result, Patient Experience Data captured as part of Real-World-Evidence (RWE) can…

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Digital Patient Care Transforms Lives.

The pandemic has transformed the way we manage our lives. Over the past 18 months, we’ve learned to do things differently and not do some things at all, like go to the office! Our ability to adapt and virtually communicate is due largely to the significant enabler that is technology. For those with a chronic…

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Patient Relationships Are Built, Not Bought

Writing a check for a patient advocacy group is not enough. There are no shortcuts when it comes to true patient centricity.  Some companies still think that writing a check to a patient advocacy organization and/or publicly supporting disease days makes them a patient-centric organization. These things are important, but it’s not enough. Being patient-centric means building a genuine working relationship with…

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Compassionate Considerations

The current public debate Biogen finds itself in regarding compassionate use/expanded access to Tofersen is an unfortunate situation for them and other companies who find themselves in a similar dilemma. Unfortunately, this will likely happen again unless handled differently. At RareiTi, we believe alternative steps can be taken to avert a public outcry, providing better…

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Expanded Access – the Right Applies

Prompted by the current high-profile case of Lisa Stockman Mauriello’s devastating rare disease ALS diagnosis and her family’s fight to access an investigational therapy, tofersen, potentially the only option to alter the course of her disease, we comment on the issues surrounding this case and the possible ethical dilemma this presents whilst applying the long-established…

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Opening Pathways to Patient Access

When a patient gets access to a drug that changes the trajectory of their disease, it’s a beautiful thing. As a mother of a child with a rare disease, I’ve dedicated my career to connecting patients with emerging therapies. But there are so many barriers that stand in the way of patient access, especially in…

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What have we learned from the pandemic?

I’ve been thinking recently about the lessons RareiTi needs to absorb from the pandemic and what adjustments we need to make in response to those lessons. RareiTi’s big goal remains focused around access to therapies, but the pandemic is a reminder that diseases are global and that carefully executed, country-specific outreach to patients and providers…

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Becoming a catalyst for change

As International Women’s Day approaches with its theme of “Choose to Challenge”, I wanted to gain inspiration from women who have challenged the status quo to leave the world a different place than they found it.  I found plenty of inspiration at the PharmaVOICE Women of the Week virtual session featuring RareiTi’s own Wendy White.…

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Plan to Succeed – consider all your access options when meeting challenges in rare disease.

Global drug launches always pose unique challenges, but are often even greater when in rare disease. The combination of higher prices, complex therapies, smaller patient populations, unique health systems and public health priorities likely mean you’ll need more data and additional supportive evidence to meet the needs of international markets.   An early access program…

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2021 Rare Disease Day – Leave no one behind!

Managed access programs aim to expand access to medications that are not available to patients through the usual channels around the globe. These programs go by various names, including compassionate use, expanded access, named-patient, and early access programs. While some countries allow costs for these programs to be covered many others do not. The number…

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Rare Disease, Covid 19 and the Need for Mental Health Coaching

Like many of you, I have been watching the sobering developments of the new variant of Covid 19 that seems to be taking a hold across the UK and in some parts of Europe.  Never has the need to support people living with Rare Diseases, Long Term Conditions and Cancer been greater.  Most people in…

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Managed Access: A Solution with Benefits for Everyone

Managed access programs – also known as compassionate use, early access, and named-patient programs- have clear benefits for patients and physicians. These programs provide an alternative regulatory pathway to help patients access potentially life-altering drugs when they are ineligible for clinical trials or the drug has not been approved in their home country.   Historically, the…

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It’s Not Big Data That Matters – it’s Broad Data

Big data will never be an option for rare disease research. By definition, rare diseases affect around 620 people per million.  Ultra-rare diseases may affect as few as 1 in a million. When you have a tiny population, scattered across the globe, how can you hope to find enough clinical trial participants to demonstrate efficacy…

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Rare Shouldn’t Mean Alone

A study published in the Journal of Genetic Counseling provided evidence for something every rare disease patient and caregiver has learned in the school of experience. Alone in a Crowd? Parents of Children with Rare Diseases Experience of Navigating the Healthcare System 1 reported on the challenges rare disease parents face once their children are…

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Managed Access: A Missed Opportunity for Orphan Drug Developers and Patients

Product launch planning has changed since my days of working in big pharma.  Ten years ago, pharmaceutical marketing professionals focused on ensuring patients could access therapy early, even if they didn’t meet the clinical trial criteria.   Early access to therapy, pre-launch and pre-license, offers benefits for all stakeholders.  For patients, access to therapy can slow…

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Rare Disease, COVID-19 and the Need for Mental Health Coaching

In one way or another, the pandemic has impacted nearly everyone on the planet.  But some groups have suffered more than others.  Because COVID-19 preys on those with underlying health conditions, people with rare diseases have had to take extraordinary precautions to avoid contracting the illness. The threat of becoming infected has added tremendous stress…

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Keeping Drug Development Moving – through COVID-19 and Beyond

Along with its direct impact on human health and survival, COVID-19 is indirectly impacting health by causing many companies to hit the pause button on clinical trials.  Hundreds of studies have been suspended, and many more are pushing out their start dates. The reasons are many: Site closures Risk of infection from visiting a medical…

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Real World Evidence as part of a Managed Access Program

The lack of treatment for COVID-19 , which resulted in a decision by the FDA to allow expanded access and compassionate use  for remdesivir  and Emergency Use Authorization  (EUA) for chloroquine, has brought renewed attention to these methods of getting treatment to patients with life-threatening illnesses when there is no approved drug. The FDA’s response…

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Reducing Deaths from COVID-19: What Can We Learn from Rare Disease?

The COVID-19 outbreak has been exposing many of the existing cracks in the foundations of our healthcare system.  But it is not just the lack of equipment, shortage of healthcare workers and hospital beds, and a missing plan for pandemics that are hampering our collective response.   Some patients are dying not because of the virus…

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For the Cystic Fibrosis Community: Self Isolation is NOT the “New” Normal!

RareiTi and CysticFibrosis.com have come together during the COVID-19 pandemic, to find out directly from CF patients and their caregivers what impact the pandemic is having on them.  We wondered what differences they are feeling and what we all can learn from this vulnerable rare disease community, who are already use to self-isolation and physical…

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Stopping the spread of COVID-19 by self-isolation and quarantine is hard. How will home healthcare help?

Since the early days of the COVID-19 outbreak, the focus has been on minimising the risk to the most vulnerable in society; the elderly, immunosuppressed, patients with co-morbidities, whilst preserving the resources of healthcare systems anticipating increased demand for acute services – flattening the curve.  Self- isolation means vulnerable individuals now find themselves being excluded…

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Putting compassion into compassionate use

Compassion can be defined as “ feeling or showing sympathy and concern for others”  RareiTi is founded on the premise that the best way to enable patients to access the medicines that they need, at the earliest opportunity, is to develop our programs from empathy and patient experience, rather than through the prism of the…

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Facing the Future of Rare Together

There are so many reasons to feel hopeful about the future of rare diseases.  Since 1983 when the Orphan Drug Act was signed into law, the number of drugs approved for rare disease has risen dramatically. According to a report released by the Center for Drug Evaluation and Research (CDER) in early January of this year,…

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Questions Around Lottery Access Strategy

“It’s a lottery where we’re leaving children’s lives up to chance. I don’t think it’s a game that any of us want to play.” This is a parent’s response to the news that Novartis has started to receive applications for a lottery-based program to give away 100 doses of its $2.1m gene therapy treatment Zolgensma for the debilitating spinal muscular atrophy (SMA) in countries where it is currently unlicensed.

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COVID-19 Response

The COVID-19 pandemic is having significant impact on clinical trials globally. Many trials face suspension or closure due to clinical priorities and the impact of social distancing and quarantine.

Clinical trial patients with a rare condition face great challenges; being part of a clinical trial when living with a rare condition can often be the only lifeline available, offering the chance to access novel therapies that could improve life enormously. During the pandemic, not being able to access novel therapies could have a major negative impact on health, disease progression and mortality. In addition, progress toward an approved therapy should not have to be completely stalled because of a lack of ability to collect relevant data.

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