Managed Access to Rare Disease Medicines

Expanded access programs provide the opportunity for patients with unmet medical needs, who are unable to participate in a clinical trial, to get access to drugs prior to their approval. Many countries have regulatory programs in place for expanded access-type programs, sometimes referred to as “compassionate use”or “named-patient” programs. 

Not so long ago, these programs weren’t widely understood by the pharmaceutical industry, patients or healthcare professionals. A sea change has happened in recent years, however. The industry has grown increasingly comfortable with offering pre-approval access. The voices of patients and advocacy groups have been amplified in part due to social media channels. And new laws have been enacted; as part of the 21st Century Cures Act which went into effect in the United States in 2017, companies developing new medicines are required to publish their policies for expanded access on their websites. In 2018, the Right to Try Act created a second route to access to investigational drugs. 

Pharmaceutical Executive recently spoke with Natalie Douglas, co-founder of RareiTi, a global managed access platform focused on rare diseases. She is the former CEO of Idis, one of the first companies to provide expanded access services and a leading voice in the field. 

PharmExec: You’ve counseled many pharma and biotech companies on the development and management of expanded access programs. Can you tell us how you first learned about these programs?

Natalie Douglas

Natalie Douglas: I became aware of expanded access and compassionate use through my job at Janssen Pharmaceuticals in the 1990s. I was a sales rep working at many of the large London teaching hospitals and saw some really interesting and innovative new medicines coming through. I was responsible for a medication that was used to treat severe opportunistic infection and many of the patients had late-stage AIDS. Janssen’s parent was J&J and they had a solution formulation of this drug called itraconazole in clinical trials and the doctors were anxious to be able to offer this to patients in need. There was a compelling unmet need and the importance of compassionate use became obvious. 

You joined Idis when expanded access was a relatively unknown term. How did you help advance the understanding and acceptance of these programs?

A: I joined Idis when the company was very small and focused on sourcing and supplying medicines for hospital pharmacies. It was clear to me, however, that we could build a model that supported pharmaceutical companies in making provisions for ethical access to medicines in this way. It was a true greenfield opportunity, a complete unknown. My experience at Janssen/J&J enabled me to go to big pharma and talk about this to chief medical officers and senior members of the regulatory and operations teams. That was in 2000 and there was a lack of awareness and understanding of these programs. Within five years or so and from truly a standing start, however, Idis was working with many of the world’s top pharmaceutical companies in implementing and managing their expanded access programs. 

Apart from persistence, the keys to success were maintaining a patient focus and providing education about something that was, to most of these companies, brand new. 

What’s your perception of where we are today with expanded access? What’s working and what’s not?

If I contrast the early days with what I’m seeing now, the driving force for implementation of these programs has shifted. It used to be that companies with interesting drugs in the pipeline would start to get calls from patients and physicians asking about access. At times, they were inundated with requests. Unfortunately, there wasn’t always someone at the pharma company responsible for taking those calls and most of time, no programs in place to properly handle the requests. I’d hear from companies saying that they were focused on approval or launch – and appropriately so – rather than providing access outside the confines of their ongoing trials. Understandably, patients would use whatever channels of communication they could leverage and sometimes their appeal would wind up on the evening news; this was only further amplified with the advent of social media. 

The Right to Try and 21^st Century Cures Act the US are positive steps. I love the attention these initiatives have brought to the topic; people should remember, however, that the decision to provide this type of access remains with the drug developer. In the UK, there is support for expanded access under a refined mechanism, where there wasn’t a process in place 20 years ago. In 2014, the Early Access to Medicines scheme was introduced and is overseen by the Medicines and Healthcare Products Regulatory Agency (MHRA). I was happy to see this as I’ve always been a major advocate in pushing the MHRA to provide a much clearer protocol-led approach. Initiatives to update and/or establish expanded access regulations are happening around the world. And while these are all important steps, the process can still be perceived as complicated, costly and time-consuming.

I work very closely with the rare disease community and collectively, it has had to push boundaries when there is unmet need. That still continues, which is a good thing. There is still a need for more education on expanded access for all stakeholders – companies, patients and physicians. This will help overcome the fear associated with these programs, how the programs work and for whom. There are still operational, organizational and logistical factors to consider when it comes to expanded access, all happening at the same time the company is focused on successful completion of their clinical trials and commercialization – which ultimately provides access to the greatest number of patients. 

At the end of the day, we need to be looking at how we can make provision possible while ensuring that such provision is the best possible choice for the patient under the circumstances in which they find themselves. I’m happy that the frameworks for these programs are in place, or being put in place, as we enter a whole new realm of therapeutic options with cell and gene therapy. The cost of these drugs, their limited patient population and possibility of a one-dose cure bring a whole different dimension to expanded access. I hope the conversation continues and stakeholders continue to work together to find the best way forward for patients in need and overcome the impediments to access which remain. 

What was your goal in founding RareiTi?

My co-founder Wendy White and I have launched RareiTi, a global managed access and medicines management model, specifically for the rare disease community. Through our combined experiences in healthcare and our work as board members of Global Genes, we saw there was a gap in the market for rare diseases and we had to respond. We help rare patients gain access to medicines and help manage their care and deliver invaluable insights about their disease. Our advisory practice is designed to help the biopharmaceutical industry develop innovative and effective rare disease strategies. This might include conducting gap analyses, identifying opportunities for engagement in the rare disease community, adopting best in class internal practices and policies and improving outcomes in rare disease from clinical trial design through to international commercialization. At the end of the day, our goal is to help extraordinary patients gain access to the care they need.