Providing Access to Orphan Drugs: Challenges and opportunities

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Demand for access programs is greater than ever, but misconceptions remain

Guest post provided by Dr Debra Ainge, Head of Project Management at Clinigen Global Access Programs (Clinigen GAP)

The access program space is evolving rapidly, with Orphan diseases a key area of high unmet need that such programs can address. The interest in access programs has never been greater with high profile social media campaigns for access to medicines and changes to regulations regularly reported in the media, but some common misconceptions of the supply of unlicensed medicines in this area remain.

There are many orphan diseases but relatively few approved treatment options which means that early access programs are particularly important. The number of companies investigating orphan diseases is increasing and as a result there are more compounds in clinical development.

However, as Orphan diseases can often be degenerative, patients do not have time to wait for treatment options to go through clinical trials, regulatory approval and commercial launch. As orphan diseases are also by nature very rare, patients with a particular disease are relatively few and located across a wide geographical area so cannot always join a trial. Additionally, some Orphan disease patients may not meet the eligibility criteria for a clinical trial as their disease has already degenerated too far. It may also be the case that the drug that a patient needs simply may not yet be approved or available in their country.

Providing access to investigational products can be a challenging business for Pharma and Biotech companies in general, with confusion about what is and is not possible. The regulatory environment is diverse, changing rapidly and often difficult to navigate.

Although mechanisms do vary widely throughout the world, there are some common factors. Access is usually initiated by the physician and is limited to investigational products for the treatment of serious or rare diseases and where there is an unmet medical need due to the absence of alternative approved and commercially available treatments.

A common area of debate is the interpretation of ‘unmet medical need.’ Although traditionally early access programs have focussed on oncology and infectious disease, regulations do not differentiate between therapy areas, and the decision about what constitutes unmet need lies firstly with the treating physician and secondly with the relevant health authority.

Orphan diseases are a recognised area of high unmet need and this, coupled with increased research and development in this area has resulted in more products reaching later stage clinical development. With this increase in late stage investigational products comes demand for early access from both patient groups and healthcare professionals involved in the treatment of patients suffering from orphan diseases.

For example, Boehringer Ingelheims’ nintedanib for the treatment of idiopathic pulmonary fibrosis, AstraZeneca’s metreleptin for the treatment of familial partial lipodystrophy, and Santhera’s Idebenone for Leber’s hereditary optic neropathy and duchenne muscular dystrophy are three diverse treatments that have received orphan drug designation and where there is a significant demand for early access.

Another common area of concern from the perspective of drug developers and regulators is the impact of an access program on the recruitment for clinical trials, and ultimately the approval of the drug. This is particularly relevant in the area of orphan diseases where patient numbers are small, with a wide geographical spread.

However, with careful consideration in the set-up phase, access programs can be used to:

  • Improve recruitment in ongoing trials by identifying patients that would otherwise not have been included in the trial.
  • Provide access to patients who do not meet the stringent eligibility criteria but whose physicians believe could benefit from treatment.
  • Create a mechanism for longer term access to patients participating in the trial once the treatment within the trial is completed.

Mark Corbett, who heads Clinigen GAP, speaks regularly on these mechanisms and strategies for delivering successful early access programmes. He will present on ‘Early Access to Orphan Drugs: The Rationale, Challenges and Options Available’ at the World Orphan Drug Congress in Brussels, Belgium in November. Global Business Development Director Tom Watson will also chair a roundtable discussion during the conference on ‘Practical Considerations for Establishing an Early Access Program for Orphan Drugs’.

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