In Europe, reimbursement approval vary on a country to country basis, conversely to the centralised regulatory approval landscape that faces all drug developers. It can be argued orphan drug developers face an even greater hurdle with premium prices that National Health Systems are all too weary of in a global economic crisis. I posed some questions to Carlo Incerti, who oversees Global Medical Affairs (including Medical Operations, Global Regulatory Affairs and Compliance, Corporate Quality Systems, Global Patient Safety and Risk Management, Evidence Value Development/Health Economics and Outcomes Research and Global Policy) at Genzyme to understand what he felt were the major challenges stifling access of orphan drugs to patients in need and what may be the foreseeable game changers set to influence pricing levels and reimbursement attitudes to orphan drugs.
What are the major challenges impeding or slowing the access of orphan drugs to patients in need?
The obvious major challenge is related to the approval of the reimbursement in the various countries or the restriction of the label at the local level, as compared to the one granted by the regulatory authorities. As to the first point, the timing of the reimbursement processes is extremely variable and in certain countries the products are only available after more than 1 year from the date of regulatory approval. Any effort should be made in order to reduce this lag time, which sometimes forces patients towards a sort of "therapeutic tourism" in order to get the drug and in many cases constitutes a big burden for small/medium companies which are ethically forced to supply free of charge the drugs until the reimbursement has been gained. Secondly, it's now becoming common that local reimbursement authorities are restricting the availability of the drug to sub-categories of the patients for whom the product has been approved by the regulatory authorities. Many times this is done following a sort of efficacy re-assessment at the local level with consequent delays in the process. A big help would be a memorandum of understanding among the reimbursement authorities to utilize a common set of available data to formulate their reimbursement decisions. Efforts in this direction (CAVOD and the so-called "Super EPAR") are on going.
What needs to be displayed by orphan drug developers to demonstrate added value of their product to payers?
As I presented a few times in Orphan Disease Congresses, gone are the times when developers were in a sort of "Franc zone" and the orphan drugs were immune to payer's scrutiny. It is now full responsibility of the biopharma companies to include endpoints in their Drug Development Plans to substantiate and assess the relative clinical efficacy of their products, in order to demonstrate what is the real advantage for the patients and the added value with respect to no therapy or palliative therapy or in some cases standard of care. This attitude has to start early in the development stage and ideally already prior to the Proof Of Concept in man. The use of surrogate markers has to increase with efforts to be made on their clinical validation.
What are the foreseeable game changers set to influence pricing levels and reimbursement attitudes?
It goes without saying that the global economic crisis has created a condition of "perfect storm" in the reimbursement attitudes of governments. Even if it is not publically stated, it is obvious that innovation-inherent costs are quite concerning for the National Health Systems and that is why all of the stakeholders should collaborate to make the system of drug reimbursement sustainable. Appropriately, some governments have started to re-route the savings from generic drugs and the few biosimilars towards innovative specialty therapeutics, including orphan drugs. Furthermore, better ways of diagnosing and categorizing diseases should more and more reinforce the principle of personalized medicine as the more sustainable way forward.
Carlo is confirmed to speak at the World Orphan Drug Congress in Geneva under the session, "Achieving quick and early market access" with a specific focus on how to make your orphan product reimbursable. If you want to find out more about his presentation and the other leading speakers confirmed to present alongside him, DOWNLOAD THE BROCHURE today.
We have plenty more speaker interviews available to read on this blog so take a look at a Payers perspective on the pricing debate for orphan drugs or discover the Must Know Tips to achieve successful clinical development of an orphan drug.
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