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Rare Diseases February 2019

Gene therapy success – a revolutionary product requires inspired approach

Sophie Schmitz

Managing Partner, Partners4Access

In order to achieve commercial success, companies have to overcome three main barriers to ensure access to gene therapy products – limited specialised infrastructure, reimbursement and lack of long-term efficacy and tolerability data.

Gene therapies are an investigational technique that offers exciting promise for curing many debilitating and potentially terminal diseases, such as cystic fibrosis, multiple sclerosis, haemophilia, sickle cell disease, and spinal muscular atrophy. 

Just 5% of the 7,000 rare diseases have treatment options. For these patients, gene therapies that represent a curative opportunity could be a potential boon. Significant breakthroughs have been made in the last few years in the development of orphan drugs and cell and gene therapies. Currently over 1,000 late stage clinical trials are registered, and a small number of products have been launched.

But access to these treatments has not been easy. According to Sophie Schmitz, Managing Partner, Partners4Access; “by design, cell and gene therapies are more complex than any traditional drugs in the market so far. So, a new approach is necessary in order to achieve access.”

Innovation does not guarantee access

One challenge is the primary delivery methods required to enable the gene to reach its target. The gene therapy portfolio is broadly split into in-vivo and ex-vivo delivery systems wherein the former delivers the gene directly into the body, and the latter involves extraction of cells outside the body (followed by a modification process), before re-introducing it back into the patient.

The ex-vivo gene therapy approach taken by some recent products has added to access barriers.

Schmitz explains “ex-vivo therapies require a seamless dance of logistics in order to be administered successfully. This means that patients, clinicians and the laboratory conducting the gene modifications are essential to be in a geographical distance that enables this approach.”

However, this is not always possible; “access can be constrained by the fact that there are limited specialised healthcare facilities that have the ability to administer these ex-vivo therapies. This is where the Cross-Border Healthcare directive can be both a barrier and a blessing.” For example, a certain immunostimulant treatment for the inherited disorder ADA-SCID is administered only at a single facility in Milan, meaning that patients need to travel in order to receive the therapy.

Regulatory impasse

A 2011 directive enabled patients in the European Union to access healthcare in any EU member state. The directive known as ‘Cross-Border Healthcare’ sets out conditions under which patients may travel to another EU country and receive medical care while the home country is mandated to provide reimbursement.

In effect, this directive could be the answer to the access barriers related to ex-vivo gene therapies.

However, in November 2018, the European Commission published a progress report on the directive. The report said that patient mobility within the EU shows only a slight increasing trend over 2015-2017 and that patients prefer limited travel primarily to neighbouring countries i.e. closer to home. Following these conclusions, the European Commission is seeking to strengthen this directive by encouraging more pilot programs between member states and improving investment in healthcare delivery.

Long-term efficacy

Gene therapy are designed to develop permanent or sometimes long-term changes in the human body. The unknown long-term impact on patients is an area of concern. The USFDA in 2018 unveiled six new draft guidelines on gene therapy in a bid to support the biotech industry and address some of patient concerns. One of the guidelines relates to clinical trial design of long term follow up observational studies. These studies would collect data on delayed adverse events and their effect on patients following administration of a gene therapy.

Future approaches to access rooted in partnerships

The three main barriers to gene therapy access outlined above, point towards a diverse set of issues that cannot be solved by one stakeholder. Schmitz says, “partnership is essential to achieve successful access to gene therapies for patients and commercial success for biotech companies. At Partners4Access, we take pride in advocating a partnership platform that brings in all stakeholders in healthcare – Patients, Physicians, Pharma, Policy-maker, Payer and Partnership. Our rare disease 6P campaign is aimed at promoting awareness about rare disease and gene therapy challenges and its impact on people and society.”

Some of these partnerships are already becoming evident in the industry. Project Hercules, a wide-ranging stakeholder coalition that brought together patient group Duchene UK and eight biopharma firms to develop disease level economic models and other tools to demonstrate value of new treatments for Duchene Muscular Dystrophy to health technology assessors and support reimbursement decisions.

Indeed, such partnerships could only be the beginning of a long road to achieving gene therapy access.

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