Home » Rare diseases » Improve treatment options through early access to medications
Rare diseases

Improve treatment options through early access to medications

rare diseases early access treatment
rare diseases early access treatment

Dr Michel van Harten, Head of Medical Affairs at myTomorrows, believes increasing patient access to developmental medicine needs support and describes how Early Access Programmes could help patients in need.

There are approximately 7,000 known rare diseases that affect 350 million patients worldwide. Only five per cent of these diseases have treatments, with many never receiving potentially life-changing medication. “It’s frustrating to see that there might be a molecule out there that could potentially benefit those patients and they can’t get access to it,” says Dr van Harten. Through improving access to investigational drugs and learning from other countries’ models for Early Access Programmes (EAPs), treatment options can be extended beyond licensed medicines.

Pharmas focus on the illnesses with most patients – rarely rare diseases

“In general, rare disease patients have fewer treatment options. Medicine development often focuses on the areas with largest numbers of patients in need, so the patients with rare diseases quickly run out of options,” says Dr van Harten. With fewer options available, the opportunity to explore all treatment options is important for improving the possibilities of treatment for rare disease patients.

The standard method for providing patients with treatment through developmental medicines is through a clinical trial, in which data on therapies is being gathered. However, this can be largely restricted through eligibility criteria. “There can be very specific inclusion and exclusion criteria, some patients can not be enrolled into a clinical trial ” says Dr van Harten.

Investigational drugs for patients in need

A way for patients with a debilitating or immediately life-threatening disease/condition, where there are no therapeutic alternatives, to gain access to investigational drugs.

Dr van Harten describes EAPs as “a way for patients with a debilitating or an immediately life-threatening disease or condition, where there are no licensed therapeutic alternatives, to gain access to investigational drugs (usually mid- to late-stage development) for treatment use,”.

Next to medicines in development, EAPs can also allow access to medicines that may have approval from regulators internationally, yet are not commercially available in the patient’s country. “Each country has laws in place to allow for treatment with medicines not yet approved and it really depends on the country how this is organised,” he said.  Although there is little literature on the success rate of EAPs, each programme can allow access to otherwise unavailable treatment options.

For Dr van Harten some of the key challenges are the effectiveness of government organisation and the concerns of biotech and pharmaceutical companies. “There should be a lot of work done and a lot of education to encourage companies to make available medicines in an early access setting,” says Dr van Harten.

We could learn from other countries, but all stakeholders must buy in

Similarly, the variety in implementation from country to country contributes to the effectiveness of EAPs in delivering patient treatment. “Every country is organised differently. It’s critical that all stakeholders buy into the treatment plan,” he says.

When looking for EAP implementation for a functional working model, Dr van Harten believes the system in France provides a hopeful framework. “If a physician prescribes a drug, it is assessed by an authority for basic indication of both safety and efficacy, amongst others. If approved then it is also reimbursed. That’s a very good system where they show that early access can make available treatment options for patients with unmet medical needs,” says Dr van Harten.

When all other routes are exhausted, widening access to medication provides greater chance of providing effective treatment. Through funding and support of medicines in development, EAPs can deliver treatments to many patients that otherwise had no more options.

Next article