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 disease progression and manage symptoms, enabling them to go to work or school.  Clinicians have the opportunity to experience the benefits of a new or novel medication in the real world, adding the richness of knowledge and experience that comes from using a long-awaited treatment. 

There are also benefits for pharmaceutical companies. The real-world evidence generated through an early or accelerated access program can provide valuable additional data to: 

  • Support the product pricing strategy
  • Enhance the regulatory submission 
  • Develop your key opinion leaders and formers at an earlier stage.

In addition, engaging in an early access program globally provides a closer understanding of the regulatory complexities in different global markets.

But despite the benefits to all stakeholders, managed access programs for rare disease therapies are in short supply.  In 2019, there was a sum total of 38 clinical trials or papers published on PubMed relating to early or managed access programmes. Most of these were specific only to cancer therapies (n30). When you take out duplications, there were only two left that were truly related to the impact for patients accessing therapy through an early access program.  One was related to treatment for COVID-19 and the other was for a much-needed treatment for very severe asthma.  There were none listed for rare diseases.

Patients living with a rare disease are quite often desperate to find a treatment to help slow down their disease progression, improve their quality of life, or extend their lifespan. Fifty percent of these patients are children whose parents are equally desperate to help them.  Often, they look for opportunities to be included in a clinical trial.  However, clinical trial inclusion criteria exclude so many of those in need, and some patients who meet the criteria opt out due to logistics and costs associated with getting to the trial center.  

By providing an early access program, the pharmaceutical industry can help those patients not able to participate in a trial, while gathering valuable data on the impact of treatment.  An early access program bridges the gap for patients and provides robust evidence of the medicine’s impact in the real world.  The beauty of this type of approach is that patients are treated within the safe conditions that you would expect from a clinical trial, without the constraints of a clinical trial, while generating real world evidence along the way.  Using today’s technology, remote patient monitoring will give clinicians the confidence that patients’ safety is monitored and maintained.

The RareiTi leadership have over 25 years’ experience in managing early access programs. Let us help you shake up the market and provide a brighter future for our patients.  Please get in contact. I would love to discuss how RareiTi can help you.

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